Rezera Inc. (0002141583) (Filer)

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As filed with the Securities and Exchange Commission on October 21, 2026.​

Registration No. 333-   ​

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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION​

WASHINGTON, D.C. 20549

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FORM S-1

REGISTRATION STATEMENT
UNDER
THE SECURITIES ACT OF 1933​

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Rezera Inc.

(Exact Name of Registrant as Specified in Its Charter)

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265 Franklin Street, Suite 1702
Boston, MA 02110
(617) 694-2481

(Address, including zip code, and telephone number, including area code, of registrant’s principal executive offices)​

Geoffrey McDonough, M.D.
President and Chief Executive Officer
265 Franklin Street, Suite 1702
Boston, MA 02110
(617) 694-2481
(Name, address, including zip code, and telephone number, including area code, of agent for service)​

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Copies to:

Approximate date of commencement of proposed sale to the public: As soon as practicable after the effective date of this registration statement.

If any of the securities being registered on this Form are to be offered on a delayed or continuous basis pursuant to Rule 415 under the Securities Act of 1933, check the following box. ☐

If this Form is filed to register additional securities for an offering pursuant to Rule 462(b) under the Securities Act, please check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

If this Form is a post-effective amendment filed pursuant to Rule 462(c) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

If this Form is a post-effective amendment filed pursuant to Rule 462(d) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 7(a)(2)(B) of the Securities Act. ☐

The registrant hereby amends this Registration Statement on such date or dates as may be necessary to delay its effective date until the registrant shall file a further amendment which specifically states that this Registration Statement shall thereafter become effective in accordance with Section 8(a) of the Securities Act of 1933, as amended, or until the Registration Statement shall become effective on such date as the Securities and Exchange Commission, acting pursuant to said Section 8(a), may determine.

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The information in this preliminary prospectus is not complete and may be changed. We may not sell these securities until the registration statement filed with the Securities and Exchange Commission is effective. This prospectus is not an offer to sell these securities and we are not soliciting offers to buy these securities in any jurisdiction where the offer or sale is not permitted.

SUBJECT TO COMPLETION, DATED               , 2026​

PRELIMINARY PROSPECTUS

          Shares

Common Stock

We are offering               shares of our common stock. This is the initial public offering of our common stock, and prior to this offering, there has been no public market for our common stock. We expect that the initial public offering price will be between $     and $     per share.

Prior to this offering, there has been no public market for our common stock. We intend to apply to list our common stock on the Nasdaq               Market under the symbol “RZRA.” We believe that upon the completion of this offering, we will meet the standards for listing on the Nasdaq               Market, and the completion of this offering is contingent upon such listing.

Investing in our common stock involves a high degree of risk. Please see “Risk Factors” beginning on page 13.

Neither the Securities and Exchange Commission nor any state securities commission has approved or disapproved of these securities or passed upon the accuracy or adequacy of this prospectus. Any representation to the contrary is a criminal offense.

We are an “emerging growth company” and a “smaller reporting company” as defined under the U.S. federal securities laws and, as such, are subject to reduced public company disclosure standards. See “Prospectus Summary — Implications of Being an Emerging Growth Company and a Smaller Reporting Company.”

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See section titled “Underwriting” for a description of the compensation payable to the underwriters.

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We have granted the underwriters an option for a period of 30 days to purchase up to               additional shares of common stock. If the underwriters exercise the option in full, the total underwriting discounts and commissions payable by us will be $     , and the total proceeds to us, before expenses, will be $     .

The underwriters expect to deliver the shares to purchasers on or about               , 2026.

Joint Bookrunners

Lead Manager

Prospectus dated     , 2026

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Through and including               , 2026 (the 25th day after the date of this prospectus), all dealers effecting transactions in our common stock, whether or not participating in this offering, may be required to deliver a prospectus. This delivery requirement is in addition to a dealer’s obligation to deliver a prospectus when acting as an underwriter and with respect to an unsold allotment or subscription.

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Neither we nor the underwriters have authorized anyone to provide you any information or make any representations other than those contained in this prospectus or in any free writing prospectuses prepared by or on behalf of us or to which we have referred you. We and the underwriters take no responsibility for, and can provide no assurance as to the reliability of, any other information that others may give you. We and the underwriters are not making an offer to sell these securities in any jurisdiction where the offer or sale is not permitted. You should assume that the information appearing in this prospectus or in any applicable free writing prospectus is current only as of its date, regardless of its time of delivery or any sale of shares of our common stock. Our business, financial condition, results of operations and prospects may have changed since that date.

Market data and certain other statistical information used throughout this prospectus are based on independent industry publications, governmental publications, reports by market research firms or other independent sources that we believe to be reliable sources. Industry publications and third-party research, surveys and studies generally indicate that their information has been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. We are responsible for all of the disclosures contained in this prospectus, and we believe that these sources are reliable; however, we have not independently verified the information contained in such publications. While we are not aware of any misstatements regarding any third-party information presented in this prospectus, their estimates, in particular, as they relate to projections, involve numerous assumptions, are subject to risks and uncertainties and are subject to change based on various factors, including those discussed under the section titled “Risk Factors” and elsewhere in this prospectus. Some data are also based on our good faith estimates.

For investors outside of the United States: we have not, and the underwriters have not, done anything that would permit this offering or possession or distribution of this prospectus in any jurisdiction where action for that purpose is required, other than the U.S. persons outside of the United States who come into possession of this prospectus must inform themselves about, and observe, any restrictions relating to, the offering of the shares of common stock and the distribution of this prospectus outside of the United States.

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ABOUT THIS PROSPECTUS

Name Change

Effective September 9, 2026, we changed our corporate name from NodThera Inc. to Rezera Inc. Unless otherwise indicated or the context otherwise requires, references in this prospectus to “Rezera,” the “Company,” “we,” “us” and “our” refer to Rezera Inc. and its wholly owned subsidiaries. References to “NodThera Inc.” refer to Rezera Inc. under its former corporate name.

Trademarks

All trademarks or trade names referred to in this prospectus are the property of their respective owners. Solely for convenience, the trademarks and trade names in this prospectus are referred to without the symbols ® and ™, but such references should not be construed as any indicator that their respective owners will not assert, to the fullest extent under applicable law, their rights thereto.

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PROSPECTUS SUMMARY

This summary highlights information contained elsewhere in this prospectus and does not contain all of the information that you should consider in making your investment decision. Before investing in our common stock, you should carefully read this entire prospectus, including our consolidated financial statements and the related notes included elsewhere in this prospectus. You should also consider, among other things, the matters described under sections titled “Risk Factors,” “Business” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” in each case appearing elsewhere in this prospectus. Unless the context otherwise requires, the terms “Rezera,” “the Company,” “we,” “us,” and “our” in this prospectus refer to Rezera Inc. and its wholly owned subsidiaries, or either or both of them as the context may require.

Overview

We are a clinical-stage biotechnology company developing a portfolio of internally invented, proprietary and wholly-owned oral inhibitors of NOD-like receptor protein 3, or NLRP3. NLRP3 is the upstream driver of sterile inflammation – inflammation that is caused by damage signals, such as cholesterol crystals or misfolded proteins, rather than by infection. NLRP3 activation triggers formation of the NLRP3 inflammasome, an intracellular protein complex involved in the body’s inflammatory response. NLRP3 activation directly stimulates the production of interleukin-1 beta, or IL-1β, and interleukin-18, or IL-18, which are potent pro-inflammatory cytokines. In turn, IL-1β stimulates production of another inflammatory molecule, interleukin-6, or IL-6. For this reason, this inflammatory cascade is referred to as the NLRP3/ IL-1β/IL-6/IL-18 pathway.​

When NLRP3 becomes chronically activated, it stimulates this cascade of inflammatory mediators that drive the development and progression of cardiovascular and neuroinflammatory disease. This phenomenon of chronically elevated inflammation that persists and contributes to disease progression even when other known risk factors are well controlled, is known as RIR. The presence of RIR can be assessed using a widely available biomarker called high-sensitivity C-Reactive Protein, or hsCRP, which is a well-validated independent risk factor for future cardiovascular events, and hsCRP can also be used to evaluate whether inflammatory activity of the NLRP3/ IL-1β/IL-6/IL-18 pathway has been reduced by a therapeutic intervention. By directly inhibiting NLRP3 upstream, we aim to broadly reduce the activity of the entire sterile inflammation pathway, thereby reducing RIR and modifying the course of cardiovascular and neurologic diseases.

We have been a leader in the research, discovery and development of NLRP3 inhibitors for over a decade, and we have designed our portfolio to address historical challenges faced by other NLRP3 inhibitors: potency, therapeutic index and tissue penetration. Our lead product candidate, ruvonoflast, is highly potent, has been

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observed to be well tolerated in five completed human clinical trials and in an extensive nonclinical toxicology program, and is designed to achieve broad tissue distribution, including penetration into the central nervous system, or CNS.​

We plan to develop ruvonoflast in peripheral artery disease, or PAD, as our lead indication. PAD is a form of atherosclerotic cardiovascular disease, or ASCVD, in which narrowed or blocked small arteries reduce blood flow to the limbs, most commonly the legs. We subsequently plan to expand development to establish ruvonoflast in ASCVD more broadly. In PAD, our goal is to demonstrate that ruvonoflast is a disease-modifying anti-inflammatory therapy for patients with PAD. We selected PAD because it represents a significant unmet medical need in a large patient population and is associated with a high inflammatory burden and substantial disease morbidity despite broad use of lipid-lowering, antihypertensive and diabetes therapies.

PAD affects approximately 20 million adults in the United States, driving $21 billion in annual direct health care costs. We believe ruvonoflast may address a market opportunity of approximately $5 billion, based on reasonable assumptions for penetration rate, price, reimbursement and competition. In addition, PAD offers a focused commercial opportunity through a concentrated network of treatment centers. We believe PAD is an attractive initial indication because RIR is an important driver of disease progression. More than half of the approximately 20 million adults with diagnosed ASCVD have RIR. We believe PAD provides an opportunity to evaluate the impact of ruvonoflast in a patient population with significant unmet need, and to establish a foundation for the future development of ruvonoflast in broader ASCVD populations.

PAD impairs circulation to the legs and significantly reduces quality of life, walking performance and functional status as the disease progresses. Intermittent claudication (pain on walking), which is the hallmark symptom of symptomatic PAD, can limit daily activities and mobility, reduce exercise capacity and cause progressive functional decline. In early stages of PAD, patients have a two-fold increased risk of cardiovascular death, which rises to approximately four times the risk in later stages. Late-stage disease progression is also characterized by leg ulcerations, ischemia and ultimately amputation, and may result in chronic limb-threatening ischemia, or CLTI. CLTI is the most advanced stage of PAD and affects more than two million patients in the United States and is associated with a high risk of limb loss. PAD is also a leading cause of non-traumatic lower-extremity amputation in the United States, with approximately 150,000 non-traumatic lower-extremity amputations occurring annually.

NLRP3-driven inflammation plays a prominent role in the origin and progression of PAD. NLRP3 expression is elevated in the lower extremity arteries of people with PAD. People with genetically lower IL-6 and IL-18 levels have been observed to have lower rates of PAD, and people with genetically higher rates of NLRP3 activation have been observed to have early onset of PAD in adolescence. Eighty percent of patients with PAD have elevated high-sensitivity C-reactive protein, or hsCRP, a blood-based biomarker of inflammation that is an independent risk factor for cardiovascular risk. Higher hsCRP and IL-6 levels in patients with PAD have been linked to faster loss of walking ability and higher rates of serious limb and cardiovascular events. However, the current standard of care in PAD focuses on improving blood flow, reducing clotting and treating traditional risk factors such as lipids and blood pressure. There are no approved therapies that directly target the underlying inflammatory pathways in PAD.

Ruvonoflast is designed to target inflammatory mechanisms that contribute to PAD progression but are not addressed by current standard-of-care therapies. To date, we have not conducted clinical trials of ruvonoflast in patients with PAD. Our clinical data for ruvonoflast comes from trials conducted in other indications, including cardiometabolic and neuroinflammatory disease populations, where we have observed reductions in hsCRP, IL-18, IL-6 and fibrinogen (an independent risk factor for thrombosis). Taken together, these biomarker changes indicate deep and broad inhibition of the NLRP3 inflammation pathway. In a clinical trial of calorie-restricted patients with high cardiovascular risk, ruvonoflast reduced hsCRP by 82% (versus 37% with placebo), and significantly reduced these additional pro-inflammatory molecules associated with atherogenesis and thrombosis.​

We believe these results are relevant to PAD because hsCRP, IL-6 and fibrinogen are also elevated in patients with PAD and have been linked to faster loss of walking ability and higher rates of limb and cardiovascular events. Based on the observed biomarker reductions in our non-PAD clinical trials, together with the established role of NLRP3-mediated inflammation in PAD progression, we believe ruvonoflast has the potential to improve walking distance and quality of life and may ultimately be able to prevent or delay disease progression and reduce adverse limb and cardiovascular outcomes in patients with PAD.

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We have evaluated ruvonoflast in cardiometabolic disease in our RESOLVE-1 Phase 2 clinical trial. This clinical trial demonstrated geometric LSmean hsCRP reduction of 56.4% at 150 mg QD and 62.6% at 150 mg BID at week 4. Furthermore, hsCRP was reduced below 2 mg/L in 33% and 40% of patients at 150 mg QD and at 150 mg BID doses of ruvonoflast, respectively, and there were reductions in additional biomarkers: median IL-6 by 38.2% and 62.3% and median fibrinogen by 19.0% and 17.8%, at 150 mg QD and at 150 mg BID, respectively.  Additionally, we have discussed our plan to conduct a single well-controlled Phase 3 trial with the U.S. Food and Drug Administration, or the FDA, the European Medicines Agency, or the EMA, and the Medicines and Healthcare products Regulatory Agency, or MHRA, and we plan to initiate our Phase 3 PAD trial in the first half of 2027.​

Approximately 20 million adults in the United States have diagnosed ASCVD, and the disease drives significant annual costs in the United States. Despite advances in the treatment of ASCVD to address the role of lipids, hypertension, thrombosis and diabetes in the origin and progression of the disease, 30% of people with ASCVD have RIR as measured by elevated hsCRP. Similar to PAD, there is no approved therapy that directly targets the inflammatory pathways that contribute to disease progression in this broad population.

The rationale for targeting inflammation in ASCVD is supported by data from multiple third-party clinical trials, including:​

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The Canakinumab Anti-inflammatory Thrombosis Outcome Study, or CANTOS, initiated in 2011 with primary completion in 2017, evaluated more than 10,000 patients and demonstrated a reduction in major adverse cardiovascular events, or MACE, among patients who achieved lower levels of inflammation. In CANTOS, quarterly canakinumab in post-myocardial infarction patients with elevated hsCRP reduced major adverse cardiovascular events by 25% in patients who achieved hsCRP reduction below 2 mg/L. This was achieved without altering lipids, providing strong clinical evidence that lowering IL-1β–mediated inflammation (independent of cholesterol) reduces cardiovascular risk.

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The Justification for the Use of Statins in Primary Prevention: An Intervention Trial Evaluating Rosuvastatin, or the JUPITER trial, demonstrated that in healthy men and women with low-density lipoprotein, or LDL, cholesterol <130 mg/dL but elevated hsCRP ≥2 mg/L, rosuvastatin 20 mg daily produced large reductions in LDL and hsCRP and significantly lowered the incidence of first major cardiovascular events compared with placebo (Hazard Ratio ~0.56). The JUPITER trial was stopped early at a median of 1.9 years because of clear benefit, including a reduction in all-cause mortality. This trial provides evidence that the modest anti-inflammatory effects of rosuvastatin could impact outcomes in patients without elevated lipids.

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In patients with established ASCVD, multiple trials (notably LoDoCo2 and COLCOT) show that adding low-dose colchicine 0.5 mg daily to guideline-directed therapy reduces MACE by roughly 25–30%, supporting inflammation-targeted therapy as an effective strategy to lower residual ASCVD risk.

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We believe ruvonoflast has the potential to meet the significant unmet need in the ASCVD treatment landscape and may offer several advantages over the monoclonal antibodies that have been investigated in the field. Namely, ruvonoflast has an oral route of administration, the potential for combination products, and a low observed and theoretical risk of immune suppression. In addition, ruvonoflast has been designed to achieve high levels of tissue penetration, which may allow it to have increased activity at sites of inflammation such as the vascular wall and other extravascular tissues, as well as for high levels of brain penetration which may allow it to offer therapeutic and/or preventive effects in the brain in patients with cardiovascular disease.​

We also plan to develop ruvonoflast in indications beyond PAD and ASCVD. We believe that ruvonoflast’s high observed tissue and brain penetration in humans could support development in a range of cognitive and neurological diseases that have an association or overlap with ASCVD. We plan to develop and disclose our expanded development strategy for ruvonoflast in the future.​

In addition to ruvonoflast, we are developing trabzanoflast (formerly known as NT-0150), which is an NLRP3 inhibitor designed to be brain-penetrant and achieve CNS exposure and selective upstream inhibition of NLRP3-driven neuroinflammation. We believe that trabzanoflast is differentiated from ruvonoflast by its chemical structure and its optimization specifically for CNS penetration. This design supports NLRP3 inhibition as a potential therapeutic approach in neuroinflammatory diseases. We anticipate that trabzanoflast may benefit from the growing body of clinical and translational evidence generated across our NLRP3 inhibitor portfolio. Trabzanoflast has completed its Phase 1 clinical trial and has demonstrated activity in NLRP3 in vitro models and reduced neuroinflammation in CNS nonclinical models. Emerging evidence implicates activation

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of NLRP3 as a contributor to neuroinflammation and neurodegeneration across multiple disorders, including Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis and other CNS disorders. Our development plan for trabzanoflast will focus on neuroinflammatory indications.

Our Pipeline

We are developing a pipeline of wholly owned, proprietary NLRP3 inhibitors to address inflammatory drivers of cardiovascular and neuroinflammatory disease. We retain exclusive, worldwide development and commercialization rights to all of our product candidates. All of our candidates are potent and selective oral inhibitors of NLRP3 and each has been optimized for activity in physiologically relevant tissue types – ruvonoflast in peripheral tissues and brain and trabzanoflast for brain. In addition, we plan to seek authorization to conduct a Phase 3 clinical trial in PAD, and are engaging with the FDA prior to submitting an Investigational New Drug, or IND, application.

Figure 1: Our clinical pipeline highlights ruvonoflast’s completed Phase 2 clinical trials, as well as trabzanoflast’s completed Phase 1 trial.

Our Strategy

Our goal is to become a leading anti-inflammation company delivering differentiated therapies to reduce the harmful effects of inflammation for patients worldwide. We are preparing to initiate a registrational Phase 3 trial of ruvonoflast, our lead product candidate, in PAD in the first half of 2027, which is designed to enable a Phase 3 readout in 2028 and a planned new drug application, or NDA, submission in early 2029. Assuming our Phase 3 trial progresses as anticipated, and the results of the clinical trial support NDA submission, we believe our current plan would put us on pace for NDA approval in 2029, subject to the FDA’s review and approval of our filing. Ruvonoflast has not been approved by the FDA or any other regulatory authority. Our strategy to develop and, if approved, commercialize, ruvonoflast and any future product candidates has six key elements:​

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Maximize the value of ruvonoflast, our lead NLRP3 inhibitor, by developing it for approval for use in PAD. We plan to secure FDA approval for ruvonoflast in PAD before expanding to other ASCVD indications. Ruvonoflast is designed to directly target inflammatory mechanisms that contribute to PAD progression but are not addressed by current standard-of-care therapies.

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Self-commercialize ruvonoflast for PAD in the United States. If approved, we believe PAD is well suited for a focused commercial launch directed at approximately 50 centers of excellence in the United States, which would allow us to independently establish an initial commercial franchise for ruvonoflast in this large, underserved indication.

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Further develop ruvonoflast in additional ASCVD indications. We plan to explore the use of ruvonoflast in ASCVD broadly, as well as in ASCVD indications similar to PAD, including others such as heart failure, that may be approved by the FDA and other regulatory authorities in the course of a larger ASCVD strategy.

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Advance trabzanoflast, our earlier-stage NLRP3 inhibitor, into a Phase 2 proof-of-concept trial. Trabzanoflast is designed to target neuroinflammation.

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Expand our portfolio of anti-inflammation product candidates via internal discovery and in-licensing or acquisition. We intend to continue to explore opportunities to inhibit NLRP3 and other inflammatory pathways.

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Explore the potential for strategic partnerships to expand and/or accelerate our programs to maximize the worldwide commercial potential of our product candidates. We may seek collaborations with pharmaceutical and biotechnology companies that can provide expertise in areas such as clinical development, regulatory affairs, manufacturing and commercialization.

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Our History and Management Team

Rezera was founded to develop novel inhibitors of NLRP3, which was identified as a compelling drug target in the early 2000s. Mutations in the NLRP3 protein were shown to be responsible for driving autoinflammatory diseases in humans. Furthermore, chronic inflammation associated with common conditions such as cardiovascular, metabolic and neurodegenerative diseases was associated with aberrant NLRP3 activation.​

Despite strong biological validation, NLRP3 remained undruggable for over a decade until 2015 with the publication of MCC950, a potent and selective small molecule inhibitor that directly targets NLRP3. MCC950 was not suitable for further drug development, as clinical development was discontinued due to safety findings, which left a significant opportunity to discover and develop improved NLRP3 inhibitors. From our founding, we have pursued the internal design, discovery and development of molecules that are able to access physiologic compartments, beyond the systemic circulation, where inflammation causes disease such as the tissues and the brain. We have two product candidates in clinical development.

Our leadership team includes executives with more than 100 years of collective experience in senior roles at biotechnology and pharmaceutical companies. Their collective experience covers drug discovery, development and commercialization. Our President and Chief Executive Officer, Geoffrey McDonough, M.D., brings proven experience in company building, business strategy, product development and commercialization. Before joining Rezera, he was the president and chief executive officer of Generation Bio Co., or Generation Bio. He also previously served as the president and chief executive officer of Swedish Orphan Biovitrum AB (publ). Earlier, he held various senior leadership roles at Genzyme Corporation. Our Chief Financial Officer, Chris Guiffre, J.D., M.B.A., also brings substantial senior executive leadership experience in development stage and commercial companies, having served in a variety of C-suite roles at companies including Pear Therapeutics, Inc., Cerulean Pharma Inc. and Cubist Pharmaceuticals, Inc. Our Chief Medical Officer, Jyothis George, MBBS, Ph.D., brings substantial drug development experience, having held senior clinical, medical and regulatory roles at companies including Amgen Inc., Novo Nordisk A/S, or Novo Nordisk, and Boehringer Ingelheim Pharmaceuticals, Inc. Our founder and Chief Scientific Officer, Alan P. Watt, Ph.D., M.B.A., brings substantial research, discovery, nonclinical and translational experience, having held senior scientific positions at companies including Bicycle Therapeutics plc, GSK plc and Merck & Co., Inc. Our Chief Development Officer, Nicholas Clarke, Ph.D., brings substantial nonclinical, translational and drug development experience, having held senior roles at Pfizer Inc.

Since our founding in 2016 (by Epidarex Capital), we have raised approximately $200.0 million in equity financing from investors, including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings A/S, Sofinnova Partners and Aventis Inc. Prospective investors should not rely on the investment decisions of our existing investors. These investors may have different investment objectives and risk tolerances than prospective investors in this offering, and they acquired their shares in prior financings at prices substantially below the public offering price in this offering.​

Risk Factors Summary

Our business is subject to a number of risks of which you should be aware before making an investment decision. These risks include, but are not limited to, the following:

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We are a biotechnology company with a limited operating history and have not generated any revenue to date from drug sales, and may never become profitable.

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We have incurred significant operating losses since our inception and anticipate that we will incur continued losses for the foreseeable future. Our recurring losses from operations raise substantial doubt regarding our ability to continue as a going concern.

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As part of our ongoing business, we will need to raise substantial additional funding beyond our current capital. If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, scale back or discontinue some of our product candidate development programs or future commercialization efforts.

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Our product candidates target NLRP3, a novel therapeutic target, making it difficult to predict the time, cost of development and likelihood of successfully developing any product candidates.

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Our business depends substantially on the success of ruvonoflast.

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The development of pharmaceutical products requires substantial financial, technical and management resources, and because our financial resources are limited, we must prioritize the development of ruvonoflast

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and determine which indications to pursue. We may fail to expend our limited resources on the development of ruvonoflast for the treatment of additional indications or for the development of other potential future product candidates that may have been more profitable or for which there is a greater likelihood of success. 

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We cannot be certain that we will be able to obtain regulatory approval for, or successfully commercialize, any of our current or any future product candidates.

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Even if we receive marketing approval for our current or any future product candidates, they may not achieve broad market acceptance, which would limit the revenue that we generate from the sales of such products, if approved.

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If the FDA or comparable foreign regulatory authorities approve generic versions of any of our product candidates that receive marketing approval, or such authorities do not grant our products appropriate periods of data or market exclusivity before approving generic versions of our products, the sales of our products could be adversely affected.

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We currently rely, and plan to rely in the future, on third parties to conduct our clinical trials for our current and future product candidates. If these third parties do not successfully carry out their contractual duties, comply with regulatory requirements or meet expected deadlines, we may not be able to obtain marketing approval for or commercialize our current and potential future product candidates and our business could be substantially harmed.

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The third parties upon whom we rely on for the supply of drug product and starting materials used in our product candidates are limited in number, and the loss of any of these suppliers, or their noncompliance with regulatory requirements or our quality standards, could significantly harm our business.

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If we are unable to obtain and maintain patent and other intellectual property protection for our technology and product candidates or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired, and we may not be able to compete effectively in our market.

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Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.

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We will need to develop and expand our company, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations.

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The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock in this offering.

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An active trading market for our common stock may not develop, and you may not be able to resell your shares at or above the initial public offering price.

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We have identified material weaknesses in our internal control over financial reporting. If we are unable to successfully remediate these material weaknesses in our internal control over financial reporting, it could have an adverse effect on our company.

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The summary risk factors described above should be read together with the text of the full risk factors in the section titled “Risk Factors” and the other information set forth in this prospectus, including our consolidated financial statements and the related notes, as well as in other documents that we file with the Securities and Exchange Commission, or the SEC. The risks summarized above or described in full elsewhere in this prospectus are not the only risks that we face. Additional risks and uncertainties not presently known to us, or that we currently deem to be immaterial may also materially adversely affect our business, financial condition, results of operations and future growth prospects.

Corporate Information

NodThera Limited was originally formed and registered in Scotland in July 2016 and was the ultimate parent company of a former subsidiary known as NodThera Inc., or OldCo, until NodThera US, Inc., or NewCo, was incorporated in the State of Delaware in December 2021. In January 2022, NodThera Limited sold all of its capital stock in OldCo to NewCo and became a subsidiary of NewCo. Following this transaction, OldCo merged with and into NewCo, with NewCo as the surviving corporation. Upon finalization of the merger, NewCo was renamed NodThera Inc. In September 2026, we changed the name of our company from NodThera Inc. to Rezera Inc. We have two subsidiaries, Rezera Securities Corporation, formed under the laws of the Commonwealth of Massachusetts, and NodThera Limited, formed under the laws of the United Kingdom. Our website address is www.rezera.com. We do not incorporate the information on or accessible through our website into this prospectus, and you should not consider any information on, or that can be accessed through, our website as part of this prospectus. We have included our website address in this prospectus solely as an inactive textual reference.

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Implications of Being an “Emerging Growth Company” and a “Smaller Reporting Company”

We qualify as an “emerging growth company” as defined in the Jumpstart Our Business Startups Act of 2012, as amended, or the JOBS Act. As an emerging growth company, we may take advantage of specified reduced disclosure and other requirements that are otherwise applicable generally to public companies. These provisions include:

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being permitted to present only two years of audited financial statements, in addition to any required unaudited interim financial statements, with correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations” disclosure in our periodic reports and registration statements, including in this prospectus;

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reduced disclosure about our executive compensation arrangements;

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not being required to hold advisory votes on executive compensation or to obtain stockholder approval of any golden parachute arrangements not previously approved;

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an exemption from the auditor attestation requirement in the assessment of our internal control over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act of 2002, or the Sarbanes-Oxley Act; and

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an exemption from compliance with the requirements of the Public Company Accounting Oversight Board regarding the communication of critical audit matters in the auditor’s report on financial statements.

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We may take advantage of these exemptions for up to five years or such earlier time that we are no longer an emerging growth company. We would cease to be an emerging growth company on the date that is the earliest of (i) the last day of the fiscal year in which we have total annual gross revenues of $1.235 billion or more; (ii) the last day of our fiscal year following the fifth anniversary of the date of the completion of this offering; (iii) the date on which we have issued more than $1.0 billion in non-convertible debt during the previous three years; or (iv) the date on which we are deemed to be a “large accelerated filer”, which would occur if the aggregate market value of our equity securities held by non-affiliates exceeds $700.0 million as of the last business day of our most recently completed second fiscal quarter. We may choose to take advantage of some but not all of these exemptions. We have taken advantage of reduced reporting requirements in this prospectus. Accordingly, the information contained herein may be different from the information you receive from other public companies in which you hold stock. In addition, we have elected to use the exemption for the delayed adoption of certain accounting standards until the earlier of the date that we (i) are no longer an emerging growth company or (ii) affirmatively and irrevocably opt out of the extended transition period provided in the JOBS Act.

We are also a “smaller reporting company,” meaning that the market value of our shares held by non-affiliates plus the proposed aggregate amount of gross proceeds to us as a result of this offering is less than $700.0 million and our annual revenue was less than $100.0 million during the most recently completed fiscal year. We may continue to be a smaller reporting company after this offering if either (i) the market value of our shares held by non-affiliates is less than $250.0 million or (ii) our annual revenue was less than $100.0 million during the most recently completed fiscal year and the market value of our shares held by non-affiliates is less than $700.0 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company, we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.

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The Offering

Common stock offered by us

     shares

Option to purchase additional shares

We have granted the underwriters an option for a period of 30 days from the date of this prospectus to purchase up to      additional shares of common stock from us at the public offering price, less underwriting discounts and commissions on the same terms as set forth in this prospectus.

Common stock to be outstanding immediately after this offering

     shares (or      shares if the underwriters exercise their option to purchase additional shares in full)

We will have two series of common stock authorized immediately following this offering: voting common stock and non-voting common stock. We are offering shares of voting common stock in this offering and unless otherwise noted or context otherwise requires, all references in this prospectus to our “common stock” refer to our voting common stock. The non-voting common stock will not be listed for trading on any securities exchange.

Use of proceeds

We estimate that the net proceeds from the sale of shares of our common stock in this offering will be approximately $     million, or $     million if the underwriters exercise their option to purchase additional shares in full, assuming an initial public offering price of $     per share, which is the midpoint of the estimated offering price range set forth on the cover page of this prospectus, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. We currently intend to use the net proceeds from this offering, together with our existing cash and cash equivalents, to advance ruvonoflast through our proposed Phase 3 clinical trial in PAD, invest in commercial readiness for ruvonoflast, if approved, and fund continued development for our earlier stage pipeline; and the remainder for working capital and general corporate purposes. See “Use of Proceeds” for additional information.

Risk factors

You should carefully read the “Risk Factors” section beginning on page 12 of this prospectus for a discussion of factors that you should consider before deciding to invest in our common stock.

Proposed Nasdaq Market symbol

“RZRA”

The number of shares of our common stock to be outstanding after this offering is based on 146,803,003 shares of common stock outstanding as of June 30, 2026, which includes 40,066 shares of restricted stock and after giving effect to (i) the issuance of our Series D4 convertible preferred stock, or the Series D4 Stock, on a pro forma basis subsequent to June 30, 2026, (ii) the automatic conversion of all outstanding shares of our convertible preferred stock as of June 30, 2026 into an aggregate of 144,432,177 shares of common stock immediately prior to the completion of this offering, and (iii) the repurchase of 1,400,998 reverse vesting restricted stock awards, or RSAs, upon completion of this offering, and excludes:

▪

27,245,715 shares of common stock issuable upon the exercise of outstanding stock options as of June 30, 2026 under our 2021 Stock Option and Grant Plan, or the 2021 Plan, with a weighted-average exercise price of $0.21 per share, and 0 shares that are reserved for future issuance under the 2021 Plan as of June 30, 2026;

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▪

1,988,176 shares of common stock issuable upon the exercise of stock options that were granted under our 2021 Plan subsequent to June 30, 2026 at a weighted-average exercise price of $0.99 per share;

​

▪

0 shares of common stock reserved for future issuance as of June 30, 2026, under the 2021 Plan, which will cease to be available for issuance at the time that our 2026 Equity Incentive Plan, or the 2026 Plan, becomes effective;

​

▪

1,639,176 shares of common stock issuable upon the exercise of warrants issued subsequent to June 30, 2026, at an exercise price of $1.00 per share;

​

▪

     shares of our common stock reserved for future issuance under our 2026 Plan, which will become effective on the date immediately prior to the effectiveness of the registration statement of which this prospectus forms a part;

​

▪

     shares of common stock reserved for future issuance under our 2026 Employee Stock Purchase Plan, or the ESPP.

​

Unless otherwise indicated, all information in this prospectus reflects or assumes the following:

▪

the automatic conversion of all outstanding shares of our convertible preferred stock, in the aggregate, into shares of common stock immediately prior to the completion of this offering;

​

▪

the waiver of all accrued and unpaid dividends on our outstanding convertible preferred stock, effective immediately prior to the completion of this offering;

​

▪

no exercise of the outstanding options described above;

​

▪

no exercise by the underwriters of their option to purchase additional shares of common stock in this offering;

​

▪

a one-for-               reverse stock split of our common stock, which was effected on               , 2026 and a corresponding adjustment to the ratio at which our preferred stock will convert into common stock; and

​

▪

the filing and effectiveness of our sixth amended and restated certificate of incorporation to be in effect immediately prior to the completion of this offering and fourth amended and restated bylaws to be in effect upon effectiveness of the registration statement of which this prospectus forms a part.

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Summary Consolidated Financial Data

The summary consolidated statements of operations data for the years ended December 31, 2024 and 2025 have been derived from our audited consolidated financial statements included elsewhere in this prospectus, and the summary consolidated statements of operations data for the six months ended June 30, 2026 and 2025 have been derived from our unaudited condensed consolidated financial statements included elsewhere in this prospectus. Our historical results are not necessarily indicative of the results that should be expected in any future periods. You should read the following summary financial data together with the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” our consolidated financial statements, unaudited interim condensed financial statements and the related notes included elsewhere in this prospectus.

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(1) Pro forma net loss attributable to common stockholders includes stock-based compensation expense from the accelerated vesting of outstanding time-based equity awards granted prior to the completion of this offering pursuant to the proposed amendment to such awards that would provide for accelerated vesting immediately prior to a change in control of the Company and does not include the effect of cumulative dividends on convertible preferred stock, which would not have been earned if the conversion had occurred at the beginning of each respective period.

(2) See Note 2 to our audited consolidated financial statements included elsewhere in this prospectus for details on the calculation of basic and diluted net loss per share attributable to common stockholders.

(3) Pro forma net loss per share of common stock attributable to common stockholders, basic and diluted, has been prepared to give effect to adjustments to our capital structure arising in connection with the completion of this offering and is calculated by dividing the pro forma net loss attributable to common stockholders by the pro forma weighted-average common stock outstanding for the period indicated. Pro forma weighted-average share of common stock outstanding, basic and diluted, is computed by adjusting the weighted-average common stock outstanding to give pro forma effect to the automatic conversion of all shares of our convertible preferred stock outstanding into shares of common stock as if such conversion had occurred on January 1, 2026 or the issuance date of the preferred stock, if later. Pro forma net loss per share of common stock attributable to common stockholders, basic and diluted, does not include the effect of the shares expected to be sold in this offering.

(1) Gives effect to the (i) entry into a Loan and Security Agreement by the Company and NodThera Limited, as co-borrowers, with Silicon Valley Bank, a division of First-Citizens Bank & Trust Company, providing for a $15.0 million term loan facility, or the Term Loan, on July 17, 2026, and the borrowing of $15.0 million thereunder, (ii) issuance and sale of an aggregate of 16,389,452 shares of our Series D4 Stock, on July 15, 2026, for net proceeds of approximately $15.0 million, (iii) automatic conversion of all outstanding shares of our convertible preferred stock into an aggregate of 144,432,177 shares of our common stock immediately prior to the completion of this offering, (iv) filing and effectiveness of our sixth amended and restated certificate of incorporation, which will occur immediately prior to the completion of this offering and (v) stock-based compensation expense arising from the accelerated vesting of outstanding time-based equity awards pursuant to the proposed amendment to such awards that would provide for accelerated vesting immediately prior to a change in control of the Company.

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(2) Gives effect to (i) the pro forma adjustments set forth in footnote (1) above and (ii) the issuance and sale of       shares of our common stock in this offering at an assumed initial public offering price of $     per share, which is the midpoint of the estimated offering price range set forth on the cover page of this prospectus, after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Pro forma as adjusted balance sheet data is illustrative only and will change based on the actual initial public offering price and other terms of this offering determined at pricing. Each $1.00 increase (decrease) in the assumed initial public offering price of $    per share, which is the midpoint of the estimated offering price range set forth on the cover page of this prospectus, would increase (decrease) the pro forma as adjusted amount of each of cash and cash equivalents, working capital, total assets and total stockholders’ deficit by approximately $    million, assuming that the number of shares offered by us, as set forth on the cover page of this prospectus, remains the same, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Similarly, each increase (decrease) of 1.0 million shares in the number of shares offered by us, as set forth on the cover page of this prospectus, would increase (decrease) the pro forma as adjusted amount of each of cash and cash equivalents, working capital, total assets and total stockholders’ deficit by approximately $    million, assuming no change in the assumed initial public offering price per share, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

(3) We define working capital as current assets less current liabilities. See our consolidated financial statements and the related notes included elsewhere in this prospectus for further details regarding our current assets and current liabilities.

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RISK FACTORS

Investing in our common stock involves a high degree of risk. You should carefully consider the risks and uncertainties described below, as well as the other information in this prospectus, including our consolidated financial statements and related notes appearing elsewhere in this prospectus and the sections of this prospectus titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and “Special Note Regarding Forward-Looking Statements,” before you make an investment decision. The risks described below are not the only risks that we face. The occurrence of any of the events or developments described below could harm our business, financial condition, results of operations and prospects. As a result, the market price of our common stock could decline, and you may lose all or part of your investment in our common stock.

Risks Related to Our Financial Position and Capital Needs

We are a biotechnology company with a limited operating history and have not generated any revenue to date from drug sales, and may never become profitable.

Biopharmaceutical drug development is a highly speculative undertaking and involves a substantial degree of risk. Since our formation as NodThera Ltd. in 2016, our operations to date have been limited primarily to organizing and staffing our company, business planning, raising capital, researching and developing our current pipeline of product candidates, including ruvonoflast and trabzanoflast, building our intellectual property portfolio, undertaking nonclinical studies and IND-enabling activities, conducting and completing Phase 1 clinical trials for ruvonoflast and trabzanoflast, conducting and completing our RESOLVE-1 and RESOLVE-2 Phase 2 clinical trials. We have never generated any revenue from drug sales. We have not obtained regulatory approvals for any of our product candidates.

Typically, it takes many years to develop one new pharmaceutical drug from the time it is discovered to when it is available for treating patients. Consequently, any expectations we have about our future success or viability may not be as accurate as they could be if we had a longer operating history. In addition, as a business with a limited operating history, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors, such as market volatility, public health crises or other geopolitical events. We will need to transition from a company focused on research, development and clinical stage activities to a company capable of supporting late-stage development and commercial activities. We may not be successful in such a transition.

We have incurred significant operating losses since our inception and anticipate that we will incur continued losses for the foreseeable future. Our recurring losses from operations raise substantial doubt regarding our ability to continue as a going concern.

Since our inception, we have focused substantially all of our efforts and financial resources on developing our pipeline of product candidates, including ruvonoflast. To date, we have financed our operations primarily through the issuance and sale of convertible promissory notes and our convertible preferred stock to outside investors in private equity financings. From our inception through the date hereof, we raised an aggregate of $201.1 million of gross proceeds from such transactions. As of June 30, 2026, our cash and cash equivalents were $3.9 million. We have incurred net losses in each year since our inception, and we had an accumulated deficit of $(210.7) million as of June 30, 2026. For the years ended December 31, 2025 and 2024, we reported net losses of $42.2 million and $25.2 million, respectively. Substantially all of our operating losses have resulted from costs incurred in connection with our research and initial pipeline programs and from general and administrative costs associated with our operations. We expect to continue to incur significant expenses and increasing operating losses over the next several years and for the foreseeable future. Our prior losses, combined with expected future losses, have had and will continue to have an adverse effect on our stockholders’ (deficit) equity and working capital.

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As discussed in Note 1 to our consolidated financial statements included elsewhere in this prospectus, based on our recurring losses and negative cash flows from operations since inception, expectation of continuing operating losses and negative cash flows from operations for the foreseeable future and our need to raise additional capital to finance our future operations, management has concluded that there is substantial doubt about our ability to continue as a going concern for the one-year period following the issuance of those financial statements.​

We expect our operating losses and negative cash flows from operations to continue into the foreseeable future as we continue to develop and manufacture product candidates and, if and when approved, commence commercialization activities. As such, we plan to generate cash through a combination of equity or debt financings and strategic partnerships or collaboration agreements. If adequate funds are not available to us, we may be required to delay, reduce or eliminate some or all of our research and development programs, obtain funds through arrangements with collaborators on terms unfavorable to us, pursue merger or acquisition strategies, which could adversely affect our business, or we may be unable to continue operations. There is no assurance that we will be successful in obtaining sufficient funding on terms acceptable to us, if at all. As a result, we have concluded that our current anticipated plans do not alleviate the substantial doubt about our ability to continue as a going concern.​

We expect our expenses to significantly increase in connection with our ongoing activities, as we:

▪

initiate our planned Phase 3 clinical trial of ruvonoflast in PAD;

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▪

initiate commercial preparations for ruvonoflast, if and when our planned Phase 3 clinical trial is successful and we receive regulatory approval;

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▪

conduct additional clinical development for trabzanoflast;

​

▪

further develop ruvonoflast for additional ASCVD indications, if supported by data from our planned Phase 3 clinical trial of ruvonoflast in PAD and regulatory feedback;

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▪

prepare and submit IND applications and comparable applications for clinical trial authorization for our current and future product candidates, and prepare NDA and comparable applications for marketing authorization for successful product candidates, as appropriate;

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▪

enter into manufacturing arrangements for our product candidates;

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advance our research and development activities to expand our product pipeline;

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▪

seek regulatory approval for our product candidates that successfully complete clinical development;

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▪

develop and scale up our capabilities to support our future clinical trials for our product candidates and commercialization of any of our product candidates for which we may obtain regulatory approval;

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▪

maintain, expand and protect our intellectual property portfolio;

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▪

hire additional staff, including commercial, clinical, scientific, regulatory and management personnel;

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▪

secure facilities to support continued growth in our research, development and commercialization efforts; and

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▪

incur additional costs of operating as a public company, including increased costs of audit, legal, regulatory and tax-related services associated with maintaining compliance with the listing standards of a national securities exchange and SEC requirements, director and officer insurance premiums and investor and public relations costs.

​

In addition, if we obtain regulatory approval for our current or any future product candidates, we will incur significant commercialization expenses, including for sales, marketing, manufacturing and distribution. Because of the numerous risks and uncertainties associated with developing pharmaceutical drugs, including in light of any economic fluctuations, we are unable to predict the extent of any future losses or when we will become profitable, if at all.

Even if we achieve profitability, we may not be able to sustain or increase our profitability on a quarterly or annual basis. Our failure to achieve, sustain or increase our profitability could depress the value of our company and could impair our ability to raise capital, expand our business, maintain our development efforts, obtain product approvals, diversify our offerings, and could also cause you to lose all or part of your investment.

None of our product candidates has received regulatory approval to date. If we are unable to commercialize our product candidates or experience significant delays in doing so, our business will be materially harmed.

Our ability to become profitable depends upon our ability to generate revenue. To date we have not generated any revenue from our product candidates, and we do not expect to generate any revenue from the sale of drugs in the near future. We do not expect to generate revenue from product sales unless and until we complete

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the development of, obtain marketing approval for, and begin to sell, one or more of our product candidates. We are also unable to predict when, if ever, we will be able to generate revenue from such product candidates due to the numerous risks and uncertainties associated with drug development, including the uncertainty of:

▪

our plans to submit IND applications to the FDA and similar applications for clinical trial authorization to comparable regulatory authorities for our current or any future product candidates;

​

▪

our ability to timely and successfully complete nonclinical studies and clinical trials for our product candidates and any future product candidates, as applicable;

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▪

our successful initiation, enrollment in and completion of clinical trials, including our planned Phase 3 clinical trial of ruvonoflast in PAD, and our ability to generate positive data from such clinical trials;

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▪

our ability to demonstrate, to the satisfaction of the FDA and comparable regulatory authorities, the safety, efficacy, consistent manufacturing quality and acceptable risk-benefit profile of our product candidates for their intended uses;

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▪

our ability to timely receive necessary regulatory approvals from applicable regulatory authorities, including the FDA;

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▪

the costs associated with the development of any additional development programs we identify in-house or via collaborations or other arrangements;

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our ability to establish timely manufacturing capabilities or make arrangements with third-party manufacturers for clinical supply and commercial manufacturing;

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▪

obtaining and maintaining patent and trade secret protection or regulatory exclusivity for our current and future product candidates;

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▪

launching commercial sales of our product candidates, if and when approved, whether alone or in collaboration with others;

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▪

obtaining and maintaining acceptance of our product candidates, if and when approved, by patients, the medical community and third-party payors;

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▪

effectively competing with other therapies and obtaining and maintaining healthcare coverage and adequate reimbursement;

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the costs associated with any future collaboration or license agreements; and

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▪

our ability to maintain a continued acceptable safety profile of our product candidates following approval, if achieved.

​

We expect to incur significant sales and marketing costs as we prepare to commercialize our current or any future product candidates. Even if we initiate and successfully complete pivotal or registration-enabling clinical trials of our current or any future product candidates, and our current or any future product candidates are approved for commercial sale, and despite expending these costs, our current or any future product candidates may not be commercially successful. We may not achieve profitability soon after generating drug sales, if ever. If we are unable to generate revenue, we will not become profitable and may be unable to continue operations without continued funding, which may not be available on terms acceptable to us, or at all.

As part of our ongoing business, we will need to raise substantial additional funding beyond our current capital. If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, scale back or discontinue some of our product candidate development programs or future commercialization efforts.

We plan to initiate our Phase 3 clinical trial of ruvonoflast in PAD following regulatory interactions regarding trial design. We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we continue the research and development of, advance the nonclinical and clinical activities of and seek marketing approval for, our current or any future product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. Furthermore, following the closing of this offering, we expect to incur significant additional costs associated with operating as a public company. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. However, changing circumstances may cause us to consume capital significantly faster than we currently anticipate, and we may need to spend more money than currently expected because of circumstances beyond our control. We cannot be certain that additional funding will be available on acceptable terms, or at all. Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations through a combination of public or private equity offerings, debt financings, governmental funding, collaborations, strategic partnerships and alliances or marketing, distribution or licensing arrangements with

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third parties. If we are unable to raise capital or generate revenue when needed or on attractive terms, we will be forced to delay, reduce or eliminate our nonclinical and clinical development programs or any future commercialization efforts. To the extent that we raise additional capital through the sale of equity or convertible debt securities, our stockholders’ ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

In addition, if we pursue our current strategy of commercializing ruvonoflast ourselves in the United States, our capital requirements could increase significantly as we build commercial, medical affairs, market access and related infrastructure.

Unfavorable movements in foreign currency exchange rates could increase our operating costs and accelerate our cash consumption.

Because a significant portion of our operating costs in the United Kingdom are denominated in British pound sterling while we report our financial results in U.S. dollars, adverse movements in the British pound sterling to U.S. dollar exchange rate could materially increase our reported operating expenses and reduce the effective purchasing power of our U.S. dollar-denominated cash reserves. A strengthening of British pound sterling relative to the U.S. dollar would directly increase the dollar cost of our UK-based employee compensation, facility costs, and payments to UK-based contract research organizations, or CROs, and other vendors, potentially accelerating our cash consumption beyond current projections and requiring us to raise additional capital sooner than anticipated.

Risks Related to Our Business and Industry

Our product candidates target NLRP3, a novel therapeutic target, making it difficult to predict the time, cost of development and likelihood of successfully developing any product candidates.

Our pipeline consists of proprietary NLRP3 inhibitors, and our future success depends on the successful development of our product candidates. Although therapies have been approved to treat the diseases we are targeting, our product candidates are designed to act on a novel therapeutic target. We have concentrated our development efforts on developing NLRP3 inhibitors, and our future success depends on the successful development of this approach. However, NLRP3 is a novel target for which there are currently no approved NLRP3 inhibitors. In addition, while therapies have been approved to treat PAD, there are currently no approved therapies in the United States that directly target the underlying inflammatory pathways associated with PAD. These factors could result in uncertainty regarding our current and future development efforts and our ability to obtain regulatory approval for our current and any future product candidates. We cannot be certain that our product candidates will demonstrate acceptable safety or efficacy or receive regulatory approval.

We cannot predict whether our approaches will result in the development and marketing approval of any product candidates. Any development problems we experience in the future related to our current or any future product candidates may cause significant delays or unanticipated costs or may prevent the development of a commercially viable product. Any of these factors may prevent us from completing our clinical trials, or any nonclinical studies and clinical trials that we may initiate in the future or commercializing any product candidates we may develop on a timely or profitable basis, if at all.

Our business depends substantially on the success of ruvonoflast.

Our business currently depends primarily on our ability to successfully develop, obtain regulatory approval for, and commercialize our lead product candidate, ruvonoflast in a timely manner. We currently intend to develop ruvonoflast first in PAD and, if successful, subsequently pursue development in broader ASCVD indications. Because our development strategy contemplates expansion into broader ASCVD indications following an initial submission of topline data from our planned Phase 3 trial in PAD, any delay, failure or regulatory setback affecting our PAD program could also delay or prevent our ability to pursue those additional indications. We cannot assure you that ruvonoflast will successfully complete clinical development, receive regulatory approval or be successfully commercialized. If ruvonoflast is delayed, fails to receive regulatory approval or is not commercially successful, our business, financial condition, results of operations and prospects would be materially adversely affected.

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The development of pharmaceutical products requires substantial financial, technical and management resources, and because our financial resources are limited, we must prioritize the development of ruvonoflast and determine which indications to pursue. We may fail to expend our limited resources on the development of ruvonoflast for the treatment of additional indications or for the development of other potential future product candidates that may have been more profitable or for which there is a greater likelihood of success.

Due to the significant resources required for the development of our current product candidates, we must decide which indications to pursue and advance and the amount of resources to allocate to each. Our decisions concerning the allocation of research, development, collaboration, management and financial resources toward particular indications may not lead to the development of viable commercial products and may divert resources away from better opportunities. If we make incorrect determinations regarding the viability or market potential of ruvonoflast or other current or any future product candidates or misread trends in the pharmaceutical industry, our business, financial condition, results of operations and growth prospects could be materially and adversely affected. As a result, we may fail to capitalize on viable commercial products or profitable market opportunities, be required to forego or delay pursuit of opportunities to develop ruvonoflast for additional indications outside of PAD, including in other broader ASCVD indications, or any other current or future product candidates with other diseases and disease pathways that may later prove to have greater commercial potential than those we choose to pursue, or relinquish valuable rights to ruvonoflast through collaboration, licensing or royalty arrangements in indications in which it would have been advantageous for us to invest additional resources to retain sole development and commercialization rights.

We may not be successful in our efforts to identify or develop additional potential product candidates.

Our research programs may initially show promise in identifying potential product candidates, yet fail to yield product candidates for clinical development for a number of reasons, including:

▪

the research methodology used may not be successful in identifying potential product candidates;

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▪

potential product candidates may, on further study, be shown to have harmful side effects or other characteristics that indicate that they are unlikely to be drugs that will receive marketing approval and/or achieve market acceptance; and

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▪

potential product candidates may not be safe or effective in treating their targeted diseases.

​

Identifying and advancing additional product candidates requires substantial technical, financial and human resources. If we are unable to identify suitable compounds for nonclinical and clinical development, our business would be harmed.

We cannot be certain that we will be able to obtain regulatory approval for, or successfully commercialize, any of our current or any future product candidates.

The nonclinical studies and current and future clinical trials of our current or any future product candidates and the manufacturing and marketing of our current or any future product candidates are subject to extensive and rigorous review and regulation by numerous government authorities in the United States and in other countries where we intend to test, if any, or, if approved, market any of our current or any future product candidates. Before obtaining regulatory approvals for the commercial sale of any of our current or any future product candidates, we must demonstrate through nonclinical studies and clinical trials that each product candidate is safe and effective for use in each target indication. Drug development is a long, expensive and uncertain process, and delay or failure can occur at any stage of any of our nonclinical studies and clinical trials. This process can take many years and may include post-marketing studies and surveillance, which will require the expenditure of substantial resources beyond the proceeds we expect to raise in this offering. Of the large number of drugs in development in the United States, only a small percentage will successfully complete the FDA regulatory approval process and will be commercialized, with similarly low rates of success for drugs in development in the European Union obtaining regulatory approval from the European Commission. Accordingly, even if we are able to obtain the requisite financing to continue to fund our development and nonclinical studies and clinical trials, we cannot assure you that any of our current or any future product candidates will be successfully developed or commercialized.

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We are not permitted to market our current or any future product candidates in the United States until we receive approval of an NDA from the FDA, or in the European Union (and the EEA, where applicable), until we receive approval of a marketing authorization application, or an MAA, from the European Commission, or in any other foreign countries until we receive the requisite approval from such countries. Obtaining approval of an NDA or MAA is a complex, lengthy, expensive and uncertain process, and the FDA or EMA may delay, limit or deny approval of any of our current or any future product candidates for many reasons, including, among others:

▪

we may not be able to demonstrate that our current or any future product candidates are safe and effective in treating their target indications to the satisfaction of the FDA or applicable foreign regulatory agency;

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▪

the results of our nonclinical studies and clinical trials may not meet the level of statistical or clinical significance required by the FDA or applicable foreign regulatory agency for marketing approval;

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▪

the FDA or applicable foreign regulatory agency may disagree with the number, design, primary endpoints, statistical analysis plan or implementation of our nonclinical studies and clinical trials, including our planned Phase 3 clinical trial of ruvonoflast in PAD, or may require additional trials or studies;

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the FDA or applicable foreign regulatory agency may require that we conduct additional nonclinical studies and clinical trials;

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▪

we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks;

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▪

the FDA or applicable foreign regulatory agency may not approve the formulation, labeling or specifications of any of our current or any future product candidates;

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▪

the CROs that we retain to conduct our nonclinical studies and clinical trials may take actions outside of our control that materially adversely impact our nonclinical studies and clinical trials;

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▪

the FDA or applicable foreign regulatory agency may find the data from nonclinical studies and clinical trials insufficient to demonstrate that our current or any future product candidates’ clinical and other benefits outweigh their safety risks;

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▪

the FDA or applicable foreign regulatory agency may disagree with our interpretation of data from our nonclinical studies and clinical trials;

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the FDA or applicable foreign regulatory agency may not accept data generated at our nonclinical study trial and clinical trial sites;

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if our NDA, if and when submitted, is reviewed by an advisory committee, the FDA may have difficulties scheduling an advisory committee meeting in a timely manner or the advisory committee may recommend against approval of our application or may recommend that the FDA require, as a condition of approval, additional nonclinical studies or clinical trials, limitations on approved labeling or distribution and use restrictions;

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the FDA may require development of a Risk Evaluation and Mitigation Strategy, or REMS, as a condition of approval or post-approval;

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the FDA or the applicable foreign regulatory agency may determine that the manufacturing processes or facilities of third-party manufacturers with which we contract do not conform to applicable requirements, including current Good Manufacturing Practices, or cGMPs;

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the FDA or applicable foreign regulatory agency may be delayed in their review processes due to staffing or other constraints, including constraints arising from public health crises; or

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the FDA or applicable foreign regulatory agency may change its approval policies or adopt new regulations or guidance.

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Any of these factors, many of which are beyond our control, could jeopardize our ability to obtain regulatory approval for and successfully market our current or any future product candidates. In addition, the FDA and other applicable foreign regulatory agencies have substantial discretion in the approval process and determining when or whether regulatory approval will be granted for any product candidate that we develop and may decide that our data are insufficient for approval or that our contract manufacturing facilities are deficient, or require additional nonclinical, clinical or other data.

The U.S. Supreme Court’s June 2024 decision eliminating judicial deference to certain agency interpretations has created additional uncertainty regarding the interpretation and application of FDA regulations and policies. Any setbacks in our pursuit of regulatory approval would have a material adverse effect on our business and prospects.

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If we experience delays or difficulties in the initiation, enrollment and/or retention of patients in clinical trials, our regulatory submissions or receipt of necessary regulatory approvals could be delayed or prevented.

We may not be able to initiate or continue our planned clinical trials on a timely basis or at all if we are unable to recruit and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or similar regulatory authorities outside the United States. Patient enrollment is a significant factor in the timing of clinical trials. Our ability to enroll eligible patients may be limited or may result in slower enrollment than we anticipate.

Moreover, some of our clinical trials will compete with other clinical trials that are in the same therapeutic areas as our current or any future product candidates, and this competition reduces the number and types of patients available to us, as some patients who would otherwise be eligible for our clinical trials may instead enroll in clinical trials of our competitors’ current or future product candidates. Because the number of qualified clinical investigators and clinical trial sites is limited, we expect to conduct some of our clinical trials at the same clinical trial sites that some of our competitors use, which will reduce the number of patients who are available for our clinical trials at such clinical trial sites. There may be limited patient pools from which to draw for clinical studies. In addition, the eligibility criteria of our clinical trials will further limit the pool of available study participants as we will require that patients have specific characteristics that we can measure or to assure their disease is either severe enough or not too advanced to include them in a trial.

Patient enrollment for our current or future clinical trials may be affected by other factors including:

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the size and nature of the patient population;

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competition with other companies for clinical sites or patients;

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competition from clinical trials evaluating investigational therapies for PAD or broader ASCVD indications;

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the willingness of participants to enroll in our clinical trials in our countries of interest;

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the severity of the disease under investigation;

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availability and efficacy of approved drugs or of other investigational drugs for the disease under investigation;

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the eligibility criteria for the clinical trial in question as defined in the protocol;

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the availability of an appropriate screening test for the indications we are pursuing;

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the perceived risks and benefits of the product candidate under study in relation to other available therapies, including any new products that may be approved for the indications we are investigating;

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the efforts to facilitate timely enrollment in and completion of clinical trials;

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delays in or temporary suspension of the enrollment of patients in our future clinical trials due to public health crises;

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ability to obtain and maintain patient consents;

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the patient referral practices of physicians;

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the ability to monitor patients adequately during and after treatment;

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the proximity and availability of clinical trial sites for prospective patients; and

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the risk that patients enrolled in clinical trials may fail to comply with the trial rules or drop out of the trials before completion.

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These factors may make it difficult for us to enroll enough patients to complete our clinical trials in a timely and cost-effective manner. Our inability to enroll a sufficient number of patients for our clinical trials would result in significant delays or may require us to abandon one or more clinical trials altogether. Enrollment delays in our clinical trials may result in increased development costs for our product candidates and jeopardize our ability to obtain marketing approval for the sale of our product candidates. Furthermore, even if we are able to enroll a sufficient number of patients for our clinical trials, we may have difficulty maintaining participation in our clinical trials through the treatment and any follow-up periods.

The incidence and prevalence for target patient populations of our product candidates have not been established with precision, and we are still conducting studies to evaluate the precise target indications and appropriate populations for some of our product candidates. If the market opportunities for our product candidates are smaller than we estimate or if any approval that we obtain is based on a narrower definition of the patient population, our revenue and ability to achieve profitability will be adversely affected, possibly materially.

The precise incidence and prevalence of the indications being pursued by our current and future product candidates is currently unknown. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with our product candidates, are based on estimates. Our product candidates target cardiovascular disease and

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neurology, with our lead product candidate, ruvonoflast, initially targeting PAD followed by broader ASCVD indications, following an initial submission of topline data from our planned Phase 3 trial in PAD. Additionally, following regulatory submission, we plan to extend and/or expand our planned Phase 3 trial to enroll additional patients and collect data on MACE. The total addressable market opportunity for our current and future product candidates will ultimately depend upon, among other things, their proven safety and efficacy, the diagnosis criteria included in the final label for each, whether our product candidates are approved for sale for these indications, acceptance by the medical community and patient access, product pricing and reimbursement. The number of patients for our product candidates in the United States and elsewhere may turn out to be lower than expected, patients may not be otherwise amenable to treatment with our products, or new patients may become increasingly difficult to identify or gain access to, all of which would adversely affect our results of operations and our business.

Nonclinical and clinical drug development is a lengthy and expensive process, with an uncertain outcome. Our nonclinical and clinical programs may experience delays or may never advance, which would adversely affect our ability to obtain regulatory approvals or commercialize our product candidates on a timely basis or at all, which could have an adverse effect on our business.

In order to obtain FDA approval to market a new drug, we must demonstrate the safety and efficacy of our product candidates in humans to the satisfaction of the FDA. To meet these requirements, we will have to conduct adequate and well-controlled clinical trials. Clinical testing is expensive, time-consuming and subject to uncertainty. We have completed three Phase 1 clinical trials and two Phase 2 clinical trials for ruvonoflast, and one Phase 1 clinical trial for trabzanoflast. We also may in the future select additional development candidates or lead development candidates for nonclinical development. We cannot be certain of the timely completion or outcome of our nonclinical studies and cannot predict if the FDA will allow our proposed clinical programs to proceed or if the outcome of our nonclinical studies will ultimately support further development of our programs. We cannot be sure that we will be able to submit INDs or similar applications with respect to our other product candidates on the timelines we expect, if at all, and we cannot be sure that submission of an IND or similar applications will result in the FDA or other regulatory authorities allowing clinical trials to begin. In addition, in many cases the general approach for FDA and other major regulatory agencies for approval of a new drug has been to require dispositive data from two or more adequate and well-controlled clinical trials of the product candidate in the relevant patient population with confirmatory evidence that may be satisfied by other earlier-stage trials or other evidence. In February 2026, the former FDA Commissioner publicly indicated that a single adequate and well-controlled pivotal clinical trial supported by confirmatory evidence would be the FDA’s default standard moving forward for novel products, rather than two such trials; this statement was not a formal agency action, and the scope, implementation and durability of this policy position remain uncertain. In June 2026, FDA issued revised draft guidance clarifying how sponsors can rely on one scientifically rigorous, adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the statutory substantial evidence of effectiveness standard. FDA retains broad discretion to require additional clinical data for any product candidate, including a second adequate and well-controlled clinical trial.

Conducting nonclinical testing and clinical trials represents a lengthy, time-consuming and expensive process. The length of time may vary substantially according to the type, complexity and novelty of the program, and often can be several years or more per program. Delays associated with programs for which we are directly conducting nonclinical studies may cause us to incur additional operating expenses. The commencement and rate of completion of nonclinical studies and clinical trials for a product candidate may be delayed by many factors, including, for example:

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inability to generate sufficient nonclinical or other in vivo or in vitro data to support the initiation of clinical studies;

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timely completion of nonclinical laboratory tests, animal studies and formulation studies in accordance with the FDA’s good laboratory practice requirements and other applicable regulations;

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approval by an independent Institutional Review Board, or IRB, or ethics committee at each clinical site before each trial may be initiated;

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delays in reaching a consensus with regulatory agencies on nonclinical data requirements and study design and obtaining regulatory authorization to commence clinical trials;

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delays in reaching agreement on acceptable terms with prospective CROs and clinical trial sites, the terms of which can be subject to extensive negotiation and may vary significantly among different CROs and clinical trial sites;

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delays in identifying, recruiting and training suitable clinical investigators;

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delays in recruiting suitable patients to participate in our clinical trials;

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delays in manufacturing, testing, releasing, validating or importing/exporting sufficient stable quantities of our product candidates for use in clinical trials or the inability to do any of the foregoing;

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insufficient or inadequate supply or quality of product candidates or other materials necessary for use in clinical trials, or delays in sufficiently developing, characterizing or controlling a manufacturing process suitable for clinical trials;

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imposition of a temporary or permanent partial or full clinical hold by regulatory authorities;

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developments in trials conducted by competitors for related technology that raise FDA or foreign regulatory authority concerns about risk to patients of our product candidate or of the underlying technology broadly, or if the FDA or a foreign regulatory authority finds that the investigational protocol or plan is deficient to meet its stated objectives;

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delays in recruiting, screening and enrolling patients and delays caused by patients withdrawing from clinical trials or failing to return for post-treatment follow-up;

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difficulty collaborating with patient groups and investigators;

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failure by our CROs, other third parties or us to adhere to clinical trial protocols;

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failure to perform clinical trials in accordance with the FDA’s good clinical practice requirements, or GCPs, or applicable regulatory guidelines in other countries;

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occurrence of adverse events associated with the product candidate that are viewed to outweigh its potential benefits, or occurrence of adverse events in a trial of the same class of agents conducted by other companies;

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changes to the clinical trial protocols;

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clinical sites or patients dropping out of a trial;

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changes in regulatory requirements, policies, and guidance that require amending or submitting new clinical protocols;

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changes in the standard of care on which a clinical development plan was based, which may require new or additional trials;

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selection of clinical endpoints that require prolonged periods of observation or analyses of resulting data;

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the cost of clinical trials of our product candidates being greater than we anticipate;

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clinical trials of our product candidates producing negative or inconclusive results, which may result in our deciding, or regulators requiring us, to conduct additional clinical trials or abandon development of such product candidates;

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transfer of manufacturing processes to larger-scale facilities operated by a contract manufacturing organization, or CMO, and delays or failure by our CMOs or us to make any necessary changes to such manufacturing process; and

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third parties being unwilling or unable to satisfy their contractual obligations to us.

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Further, conducting clinical trials in foreign countries, as we have done in the past and may do in the future for our product candidates, presents additional risks that may delay completion of our clinical trials. These risks include the failure of enrolled patients in foreign countries to adhere to clinical protocol as a result of differences in healthcare services or cultural customs, managing additional administrative burdens associated with foreign regulatory schemes, as well as political and economic risks relevant to such foreign countries. In cases where data from clinical trials conducted outside the United States are intended to serve as the sole basis for marketing approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the United States population and United States medical practice; (ii) the trials were performed by clinical investigators of recognized competence and (iii) the data may be considered valid without the need for an on-site inspection by the FDA or, if the FDA considers such an inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means. Additionally, the FDA’s clinical trial requirements, including sufficient size of patient populations and statistical powering, must be met. Many foreign regulatory authorities have similar approval requirements. In addition, such foreign trials would be subject to the applicable local laws of the foreign jurisdictions where the trials are conducted. There can be no assurance that the FDA or any similar foreign regulatory authority will accept data from trials conducted outside of the United States or the applicable jurisdiction.

Additionally, recent policy proposals in the United States may make acceptance by the FDA or inclusion in a marketing application of foreign data more difficult or costly. Delays in the completion of any nonclinical studies or clinical trials of our product candidates will increase our costs, slow down our product candidate

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development and approval process and delay or potentially jeopardize our ability to commence product sales and generate product revenue. In addition, many of the factors that cause, or lead to, a delay in the commencement or completion of clinical trials may also ultimately lead to the denial of regulatory approval of our product candidates. Any delays to our nonclinical studies or clinical trials that occur as a result could shorten any period during which we may have the exclusive right to commercialize our product candidates and our competitors may be able to bring products to market before we do, and the commercial viability of our product candidates could be significantly reduced. Any of these occurrences may harm our business, financial condition and prospects significantly.

The results of our nonclinical testing and clinical trials may not be predictive of the results of later nonclinical studies and clinical trials, and the results of our current and future clinical trials may not satisfy the requirements of the FDA or other comparable regulatory authorities. If we cannot replicate the positive results from our nonclinical studies of our current or any future product candidates in our current or future clinical trials, we may be unable to successfully develop, obtain regulatory approval for and commercialize our current or any future product candidates.

We will be required to demonstrate through adequate and well-controlled clinical trials that our product candidates are safe and effective before we can seek marketing approvals for their commercial sale. Positive results from our nonclinical studies or clinical trials of our current or any future product candidates may not necessarily be predictive of the results from required subsequent nonclinical studies and clinical trials. Similarly, even if we are able to complete our planned clinical trials of our current or any future product candidates according to our current development timeline, the positive results from such clinical trials of our current or any future product candidates may not be replicated in subsequent nonclinical studies or clinical trial results.

Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials after achieving positive results in early-stage development, and we cannot be certain that we will not face similar setbacks. These setbacks have been caused by, among other things, nonclinical findings made while clinical trials were underway or safety or efficacy observations made in nonclinical studies and clinical trials, including previously unreported adverse events. Moreover, nonclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that believed their product candidates performed satisfactorily in nonclinical studies and clinical trials nonetheless failed to obtain approval from the FDA or a comparable foreign regulatory authority. If we fail to produce positive results in our planned clinical trials of any of our current or any future product candidates, the development timeline and regulatory approval and commercialization prospects for our current or any future product candidates, and, correspondingly, our business and financial prospects, would be materially adversely affected. Thus, even if the results from our initial research and nonclinical activities appear positive, we do not know whether subsequent clinical studies we may conduct will demonstrate adequate efficacy and safety to result in regulatory approval to market any product candidates.

Interim, topline and preliminary data from our nonclinical studies and clinical trials that we announce or publish from time to time may change as more data become available and are subject to audit and verification procedures that could result in material changes in the final data.

From time to time, we may publicly disclose interim, topline or preliminary data from our nonclinical studies and clinical trials, which is based on a preliminary analysis of then-available data, and the results and related findings and conclusions are subject to change following a more comprehensive review of the data related to the particular study or trial. We may also make assumptions, estimations, calculations and conclusions as part of our analyses of preliminary or topline data, and we may not have received or had the opportunity to fully and carefully evaluate all data. As a result, the interim, topline or preliminary results that we report may differ from future results of the same studies, or different conclusions or considerations may qualify such results, once additional data have been received and fully evaluated. Interim, topline and preliminary data also remain subject to audit and verification procedures that may result in the final data being materially different from the preliminary data we previously published. Accordingly, such data should be viewed with caution until the final data are available. Adverse differences between preliminary, interim or topline data and final data could significantly harm our business prospects.

Further, others, including regulatory agencies, may not accept or agree with our assumptions, estimates, calculations, conclusions or analyses or may interpret or weigh the importance of data differently, which could impact the value of the particular program, the approvability or commercialization of the particular product

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candidate or product and our company in general. In addition, the information we choose to publicly disclose regarding a particular study or clinical trial is based on what is typically extensive information, and you or others may not agree with what we determine is the material or otherwise appropriate information to include in our disclosure, and any information we determine not to disclose may ultimately be deemed significant with respect to future decisions, conclusions, views, activities or otherwise regarding a particular product, product candidate or our business. If the interim, topline or preliminary data that we report differ from actual results, or if others, including regulatory authorities, disagree with the conclusions reached, our ability to obtain approval for, and commercialize, our product candidates may be harmed, which could harm our business, operating results, prospects or financial condition.

Regulatory standards are subject to change over time, making it difficult to accurately predict the likelihood of marketing approval even when clinical trials meet their endpoints.

Regulatory standards are promulgated by various government entities and are subject to change based on factors such as scientific developments, public perceptions of risk, and political forces. Because clinical trials often take years to complete, it is sometimes possible for standards that exist during the conception and initiation of a clinical trial to change before the clinical trial is completed or reviewed by government regulators. For example, we may initiate clinical trials that are designed to show benefits on relatively short-term endpoints, but ultimately be required to show benefits in longer-term outcome studies. While some government entities have safeguards intended to ensure standards agreed upon by sponsors and regulators at the outset of a clinical trial are applied during regulatory review processes, those safeguards generally permit regulators to apply more rigorous standards where regulators believe doing so is necessary. As such, there can be no assurance that regulatory standards that are appropriate at the outset of a clinical trial program will not become more rigorous during the regulatory approval process and could potentially result in a delayed approval or denial of marketing authorization.

In addition, the FDA, EMA and other regulatory authorities may change their policies, issue additional regulations or revise existing regulations, or take other actions, which may prevent or delay approval of our future products under development on a timely basis. Such policy or regulatory changes could impose additional requirements upon us that could delay our ability to obtain approvals, increase the costs of compliance or restrict our ability to maintain any marketing authorizations we may have obtained. The U.S. Supreme Court’s June 2024 decision to overturn prior established case law giving deference to regulatory agencies’ interpretations of ambiguous statutory language has introduced uncertainty regarding the extent to which FDA’s regulations, policies and decisions may become subject to increasing legal challenges, delays, and/or changes. We cannot predict the full impact of this decision, future judicial challenges brought against the FDA, or the nature or extent of government regulation that may arise from future legislation or administrative action.

Our current or any future product candidates may cause adverse or other undesirable side effects that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.

Undesirable side effects caused by our current or any future product candidates could cause us to interrupt, delay or halt nonclinical studies or could cause us or regulatory authorities to interrupt, delay, halt or require modifications to clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other regulatory authorities. As is the case with many treatments for inflammatory or other diseases, it is likely that there may be adverse side effects associated with the use of our product candidates. Our product candidates may increase susceptibility to infection or produce other immunologic effects that could delay or prevent regulatory approval. There is also the risk of delayed adverse events following treatment using any of our current or any future product candidates.

These side effects could arise due to off-target activity, allergic reactions in trial subjects or unwanted on-target effects in the body. Results of our planned clinical trials could reveal a high and unacceptable severity and prevalence of these or other side effects. In addition, clinical trials by their nature utilize a sample of the potential patient population. With a limited number of patients and limited duration of exposure, rare and severe side effects of our current or any future product candidates may only be uncovered with a significantly larger number of patients exposed to the product candidate for a longer duration. Further, our current or any future product candidates could cause undesirable side effects in clinical trials related to on-target toxicity. If on-target toxicity is observed, or if our current or any future product candidates have characteristics that

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are unexpected, we may need to abandon their development or limit development to more narrow uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe or more acceptable from a risk-benefit perspective. In any of such events, our trials could be suspended or terminated and the FDA or comparable foreign regulatory authorities could order us to cease further development of, or deny approval of, our current or any future product candidates for any or all targeted indications. Drug-related side effects could affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims. Any of these occurrences may harm our business, financial condition and prospects significantly.

If we elect, or are required, not to initiate, or to delay, materially modify, suspend or terminate any future clinical trial of any of our product candidates, the commercial prospects of such product candidates may be harmed and our ability to generate product revenues from any of these product candidates may be delayed or eliminated. Any of these occurrences may harm our ability to develop other product candidates, and may harm our business, financial condition and prospects significantly.

In addition, if our current or any future product candidates receive marketing approval and we or others identify undesirable side effects caused by such current or future product candidates after such approval, a number of potentially significant negative consequences could result, including:

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regulatory authorities may suspend, withdraw or limit approvals of such current or future product candidates, or seek an injunction against their manufacture or distribution;

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regulatory authorities may require the addition of labeling statements or warnings, such as a “boxed” warning or a contraindication;

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we may be required to create a medication guide outlining the risks of such side effects for distribution to patients;

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we may be required to change the way such current or future product candidates are distributed or administered, conduct additional clinical trials or change the labeling of the current or future product candidates;

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we may be required to conduct post-marketing studies or change the way the product is administered;

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regulatory authorities may require a REMS plan to mitigate risks, which could include medication guides, physician communication plans or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools;

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we may be subject to regulatory investigations and government enforcement actions;

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we may decide to remove such current or future product candidates from the market;

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we could be sued and held liable for injury caused to individuals exposed to or taking our current or any future product candidates;

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we may be subject to fines, injunctions or imposition of criminal penalties; and

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our reputation may suffer.

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We believe that any of these events could prevent us from achieving or maintaining market acceptance of the affected product candidates and could substantially increase the costs of commercializing our current or any future product candidates, if approved, and significantly impact our ability to successfully commercialize our current or any future product candidates and generate revenues.

We may seek designations for our product candidates with the FDA and other comparable regulatory authorities that are intended to confer benefits such as a faster development process, an accelerated regulatory pathway, or regulatory exclusivity, there can be no assurance that we will successfully obtain such designations. In addition, even if one or more of our product candidates are granted such designations, we may not be able to realize the intended benefits of such designations.

The FDA and other comparable regulatory authorities offer certain designations for product candidates that are designed to encourage the research and development of product candidates that are intended to address conditions with significant unmet medical need. These designations may confer benefits such as additional interaction with regulatory authorities, a potentially accelerated regulatory pathway and priority review. However, there can be no assurance that we will successfully obtain such designations for our product candidates. In addition, while such designations could expedite the development or approval process, they generally do not change the standards for approval. Even if we obtain such designations for our product candidates, there can be no assurance that we will realize their intended benefits.

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For example, we may seek a Fast Track Designation for current or future product candidates. If a product is intended for the treatment of a serious or life-threatening condition and nonclinical or clinical data demonstrate the potential to address an unmet medical need for this condition, the product sponsor may apply for Fast Track Designation. The FDA has broad discretion whether or not to grant this designation, so even if we believe a particular product candidate is eligible for this designation, we cannot assure you that the FDA would decide to grant it. Even if we do receive Fast Track Designation, we may not experience a faster development process, review or approval compared to conventional FDA procedures. The FDA may rescind the Fast Track Designation if it believes that the designation is no longer supported by data from our clinical development activities.

We may seek Breakthrough Therapy Designation for any product candidate that we develop. A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over currently approved therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For drugs that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Drugs designated as breakthrough therapies by the FDA are also eligible for accelerated approval and priority review.

Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe a product candidate we develop meets the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. In any event, the receipt of Breakthrough Therapy Designation for a product candidate may not result in a faster development process, review, or approval compared to drugs considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA. In addition, even if any product candidate we develop qualifies as a breakthrough therapy, the FDA may later decide that the drug no longer meets the conditions for qualification and rescind the designation.

Even in the absence of obtaining Fast Track and/or Breakthrough Therapy Designations, a sponsor can seek priority review at the time of submitting a marketing application. The FDA may designate a product for priority review if it is a product that treats a serious condition and, if approved, would provide a significant improvement in safety or effectiveness when compared with other available therapies. Significant improvement may be illustrated by evidence of increased effectiveness in the treatment of a condition, elimination, or substantial reduction of a treatment-limiting adverse reaction, documented enhancement of patient compliance that may lead to improvement in serious outcomes, or evidence of safety and effectiveness in a new subpopulation. A priority review designation is intended to direct overall attention and resources to the evaluation of such applications, and to shorten the FDA’s goal for taking action on a marketing application from ten months to six months. Priority review designation may be rescinded if a product no longer meets the qualifying criteria.

Where appropriate, we may seek approval from the FDA or other comparable regulatory authorities through the use of the accelerated approval pathway. If we are unable to obtain such approval, we may be required to conduct additional nonclinical studies or clinical trials beyond those that we contemplate, which could increase the expense of obtaining, and delay the receipt of, necessary marketing approvals. Even if we receive accelerated approval from the FDA or other comparable regulatory authorities, if our confirmatory trials do not verify clinical benefit, or if we do not comply with rigorous post-marketing requirements, the FDA or such other comparable regulatory authorities may seek to withdraw any accelerated approval we have obtained.

We may seek an accelerated approval pathway for one or more of our product candidates from the FDA or other comparable regulatory authorities. Under the accelerated approval provisions in the Federal Food, Drug, and Cosmetic Act, and the FDA’s implementing regulations, the FDA may grant accelerated approval to a therapeutic candidate designed to treat a serious or life-threatening condition that provides meaningful therapeutic benefit over available therapies upon a determination that the therapeutic candidate has an effect on a surrogate endpoint or intermediate clinical endpoint that is reasonably likely to predict clinical benefit. The FDA considers a clinical benefit to be a positive therapeutic effect that is clinically meaningful in the context of a given disease. For the purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. An intermediate clinical endpoint is a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit.

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The accelerated approval pathway may be used in cases in which the advantage of a new drug over available therapy may not be a direct therapeutic advantage but is a clinically important improvement from a patient and public health perspective. If granted, accelerated approval is usually contingent on the sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the drug’s clinical benefit. Under the Food and Drug Omnibus Reform Act of 2022, or FDORA, the FDA is permitted to require, as appropriate, that a post-approval confirmatory study or studies be underway prior to approval or within a specified time period after the date of approval for a product granted accelerated approval. FDORA also gives the FDA increased authority to withdraw approval of a drug or biologic granted accelerated approval on an expedited basis if the sponsor fails to conduct such studies in a timely manner, send status updates on such studies to the FDA every 180 days to be publicly posted by the agency, or if such post-approval studies fail to verify the drug’s predicted clinical benefit. The FDA is empowered to take action, such as issuing fines, against companies that fail to conduct with due diligence any post-approval confirmatory study or submit timely reports to the agency on their progress.

Prior to seeking accelerated approval, we would seek feedback from the FDA or other comparable regulatory authorities and would otherwise evaluate our ability to seek and receive such accelerated approval.

There can be no assurance that after our evaluation of the feedback and other factors we will decide to pursue or submit an NDA for accelerated approval or any other form of expedited development, review or approval. Similarly, there can be no assurance that after subsequent feedback from the FDA or other comparable regulatory authorities, we will continue to pursue or apply for accelerated approval or any other form of expedited development, review, or approval, even if we initially decide to do so. Furthermore, if we decide to submit an application for accelerated approval, there can be no assurance that such application will be accepted or that any approval will be granted on a timely basis, or at all. The FDA or other comparable regulatory authorities could also require us to conduct further studies prior to considering our application or granting approval of any type, including, for example, if other products are approved via the accelerated pathway and subsequently converted by FDA to full approval. A failure to obtain accelerated approval or any other form of expedited development, review, or approval for our therapeutic candidate would result in a longer time period to commercialization of such therapeutic candidate, could increase the cost of development of such therapeutic candidate, and could harm our competitive position in the marketplace. And even if we are able to obtain accelerated approval, it may be withdrawn by the FDA if we are unable to meet statutory and regulatory requirements.​

Even if we receive marketing authorization for our product candidates, we will be subject to extensive ongoing regulatory obligations and continued regulatory review, which may result in significant additional expense and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our product candidates.

If the FDA or a comparable foreign regulatory authority approves any of our current or any future product candidates, the manufacturing processes, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion and recordkeeping for the drug will be subject to extensive and ongoing regulatory requirements. These requirements include submissions of safety and other post-marketing information and reports, registration requirements, continued compliance with cGMPs and GCPs and applicable product tracking and tracing requirements. Any regulatory approvals that we receive for our current or any future product candidates may also be subject to limitations on the approved indicated uses for which the drug may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials, and surveillance to monitor the safety and efficacy of the drug. Later discovery of previously unknown problems with a drug, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things:

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restrictions on the marketing or manufacturing of our product candidates or withdrawal of the product from the market, or voluntary or mandatory product recalls;

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manufacturing delays and supply disruptions where regulatory inspections identify observations of noncompliance during remediation;

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revisions to the labeling, including limitation on approved uses or the addition of warnings, contraindications or other safety information, including boxed warnings;

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imposition of a REMS, which may include distribution or use restrictions;

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requirements to conduct additional post-market studies or clinical trials to assess the safety of the product;

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fines, warning letters or untitled letters or partial or full holds on clinical trials;

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refusal by the FDA to approve pending applications or supplements to approved applications filed by us, or suspension or revocation of approvals;

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voluntary or mandatory publicity requirements;

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product seizure or detention, or refusal to permit the import or export of drugs; and

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injunctions or the imposition of civil or criminal penalties.

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The occurrence of any event or penalty described above may inhibit our ability to commercialize our product candidates and generate revenue and could require us to expend significant time and resources in response and could generate negative publicity. The FDA’s and other regulatory authorities’ policies may change and additional laws and government regulations may be enacted that could prevent, limit or delay regulatory approval of our current or any future product candidates.

We also cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may be subject to administrative or legal action and lose any marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.

Even if we receive marketing approval for our current or any future product candidates in the United States, we may never receive regulatory approval to market our current or any future product candidates outside of the United States.

We plan to seek regulatory approval of our current or any future product candidates outside of the United States. Obtaining and maintaining regulatory approval of our product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain regulatory approval in any other jurisdiction.

For example, even if the FDA grants marketing approval of a product candidate, we may not obtain approvals in other jurisdictions, and comparable regulatory authorities in foreign jurisdictions must also approve the manufacturing, marketing and promotion and reimbursement of the product candidate in those countries. However, a failure or delay in obtaining marketing approval in one jurisdiction may have a negative effect on the regulatory approval process in others. Approval procedures vary among countries and can involve additional product candidate testing and administrative review periods different from those in the United States. The time required to obtain approvals in other countries might differ substantially from that required to obtain FDA approval. The marketing approval processes in other countries generally implicate all of the risks detailed above regarding FDA approval in the United States as well as other risks. In particular, in many countries outside of the United States, products must receive pricing and reimbursement approval before the product can be commercialized. Obtaining this approval can result in substantial delays in bringing products to market in such countries.

Obtaining foreign regulatory approvals and establishing and maintaining compliance with foreign regulatory requirements could result in significant delays, difficulties and costs for us and could delay or prevent the introduction of our products in certain countries. If we or any future collaborator fail to comply with regulatory requirements in international markets or fail to receive applicable marketing approvals, it would reduce the size of our potential market, which could have a material adverse impact on our business, results of operations and prospects.

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Our future growth may depend, in part, on our ability to penetrate foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties that could materially adversely affect our business.

We are not permitted to market or promote any of our current or any future product candidates before we receive regulatory approval from the applicable regulatory authority in that foreign market, and we may never receive such regulatory approval for any of our current or any future product candidates. To obtain separate regulatory approval in many other countries we must comply with numerous and varying regulatory requirements of such countries regarding safety and efficacy. Such requirements govern, among other things, clinical trials and commercial sales, and pricing and distribution of our current or any future product candidates, and we cannot predict success in these jurisdictions. If we obtain approval of our current or any future product candidates and ultimately commercialize our current or any future product candidates in foreign markets, we would be subject to additional risks and uncertainties, including:

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differing regulatory requirements in foreign countries, such that obtaining regulatory approvals outside of the United States may take longer and be more costly than obtaining approval in the United States;

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our customers’ ability to obtain reimbursement for our current or any future product candidates in foreign markets;

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the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements;

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different medical practices and customs in foreign countries affecting acceptance in the marketplace;

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import or export licensing requirements;

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longer accounts receivable collection times;

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longer lead times for shipping;

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language barriers for technical training;

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reduced protection of intellectual property rights in some foreign countries;

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the existence of additional potentially relevant third-party intellectual property rights;

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economic weakness, including inflation or political instability in particular foreign economies and markets;

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compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;

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foreign taxes, including withholding of payroll taxes;

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foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;

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difficulties staffing and managing foreign operations;

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workforce uncertainty in countries where labor unrest is more common than in the United States;

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potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations;

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the interpretation of contractual provisions governed by foreign laws in the event of a contract dispute;

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production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and

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business interruptions resulting from geopolitical actions, including war and terrorism.

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Foreign sales of our current or any future product candidates could also be adversely affected by the imposition of governmental controls, political and economic instability, trade restrictions and changes in tariffs.

Changes in funding or disruptions at the FDA and other government agencies caused by funding shortages or global health concerns, or changes in policy could hinder their ability to hire and retain key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved or commercialized in a timely manner or at all, or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business.

The ability of the FDA to review and approve regulatory submissions and marketing authorizations for new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnel and accept the payment of user fees, and statutory, regulatory and policy changes and other events that may otherwise affect the FDA’s ability to perform routine functions. Average review times at the FDA have fluctuated in recent years as a result. In addition, government funding of other government agencies on which our operations may rely, including those that fund research and development activities is subject to the political process, which is inherently fluid and unpredictable.

We are unable to predict the extent to which the current U.S. government may impose or seek to impose leadership or policy changes at the U.S. federal regulatory agencies responsible for regulating our business or changes to rules and policies impacting our operations. It is unclear how these executive actions or other

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potential actions by the current administration or other parts of the federal government will impact the FDA or other regulatory authorities that oversee our business. Government proposals to reduce or eliminate budgetary deficits or limit federal agency personnel may include reduced allocations to the FDA and other related government agencies. These budgetary pressures may reduce the FDA’s ability to perform its responsibilities, potentially affecting our ability to progress development of our product candidates or obtain regulatory approval for our product candidates and could result in delays in our clinical trial timelines.

Disruptions at the FDA and other agencies may also slow the time necessary for new drugs to be reviewed and/or approved by necessary government agencies, which would adversely affect our business. For example, over the past decade, the U.S. government shut down several times, including for a 43-day period that began on October 1, 2025, and certain regulatory agencies, such as the FDA, had to furlough critical employees and stop critical activities.

If a prolonged government shutdown occurs, or if global health concerns prevent the FDA or other regulatory authorities from conducting their regular inspections, reviews or other regulatory activities, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business. Further, upon completion of this offering and in our operations as a public company, future government shutdowns or delays could impact our ability to access the public markets and obtain necessary capital in order to properly capitalize and continue our operations.

We are subject to certain U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions and other trade laws and regulations. We can face serious consequences for violations.

Among other matters, U.S. and foreign anti-corruption, anti-money laundering, export control, sanctions and other trade laws and regulations, which we collectively refer to as Trade Laws, prohibit companies and their employees, agents, clinical research organizations, legal counsel, accountants, consultants, contractors and other partners from authorizing, promising, offering, providing, soliciting or receiving directly or indirectly, corrupt or improper payments or anything else of value to or from recipients in the public or private sector. Violations of Trade Laws can result in substantial criminal fines and civil penalties, imprisonment, the loss of trade privileges, debarment, tax reassessments, breach of contract and fraud litigation, reputational harm and other consequences. We have direct or indirect interactions with officials and employees of government agencies or government-affiliated hospitals, universities, and other organizations.

Governments outside the United States tend to impose strict price controls, which may adversely affect our revenues, if any.

In some countries, the pricing of prescription pharmaceuticals is subject to governmental control. In these countries, pricing negotiations with governmental authorities can take considerable time after the receipt of marketing approval for a product candidate. In addition, there can be considerable pressure by governments and other stakeholders on prices and reimbursement levels, including as part of cost containment measures. Political, economic and regulatory developments may further complicate pricing negotiations, and pricing negotiations may continue after coverage and reimbursement have been obtained. Reference pricing used by various countries and parallel distribution or arbitrage between low-priced and high-priced countries, can further reduce prices. To obtain reimbursement or pricing approval in some countries, we may be required to conduct a clinical trial that compares the cost-effectiveness of our product candidate to other available therapies, which is time-consuming and costly. If coverage and reimbursement of our product candidates are unavailable or limited in scope or amount, or if pricing is set at unsatisfactory levels, our business could be harmed.

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Risks Related to Our Commercialization

Even if we receive marketing approval for our current or any future product candidates, they may not achieve broad market acceptance, which would limit the revenue that we generate from the sales of such products, if approved.

The commercial success of our current or any future product candidates, if approved by the FDA or other applicable regulatory authorities, will depend upon the awareness and acceptance of our current or any future product candidates among the medical community, including physicians, patients and healthcare payors. If our product candidates do not achieve an adequate level of acceptance, we may not generate significant revenue and we may not become profitable. Market acceptance of our current or any future product candidates, if approved, will depend on a number of factors, including, among others:

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the efficacy of our current or any future product candidates as demonstrated in clinical trials, and, if required by any applicable regulatory authority in connection with the approval for the applicable indications, to provide patients with incremental health benefits, as compared with other available medicines;

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the timing of market introduction of the product candidates and potential advantages to alternative treatments;

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limitations or warnings contained in the labeling approved for our current or any future product candidates by the FDA or other applicable regulatory authorities;

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the clinical indications for which our current or any future product candidates are approved;

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availability of alternative treatments already approved or expected to be commercially launched in the near future;

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the potential and perceived advantages of our current or any future product candidates over current treatment options or alternative treatments, including future alternative treatments;

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the willingness of the target patient population to try new therapies or treatment methods and of physicians to prescribe these therapies or methods;

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the need to dose such product candidates in combination with other therapeutic agents, and related costs;

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the strength of marketing and distribution support and timing of market introduction of competitive products;

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publicity concerning our products or competing products and treatments;

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pricing and cost effectiveness;

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acceptance by physicians and treatment centers;

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the effectiveness of our sales and marketing strategies;

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our ability to increase awareness of our current or any future product candidates;

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our ability to obtain sufficient third-party coverage or reimbursement; or

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the willingness of patients to pay out-of-pocket in the absence of third-party coverage.

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If our current or any future product candidates are approved but do not achieve an adequate level of acceptance by patients, physicians and payors, we may not generate sufficient revenue from our current or any future product candidates to become or remain profitable. Before granting reimbursement approval, healthcare payors may require us to demonstrate that our current or any future product candidates, in addition to treating these target indications, also provide incremental health benefits to patients. Our efforts to educate the medical community, patient organizations and third-party payors about the benefits of our current or any future product candidates may require significant resources and may never be successful.

If the FDA or comparable foreign regulatory authorities approve generic versions of any of our product candidates that receive marketing approval, or such authorities do not grant our products appropriate periods of data or market exclusivity before approving generic versions of our products, the sales of our products could be adversely affected.

Once an NDA is approved, the drug covered thereby becomes a “reference-listed drug” in the FDA’s publication, “Approved Drug Products with Therapeutic Equivalence Evaluations.” Manufacturers may seek marketing approval of generic versions of reference-listed drugs through submission of abbreviated new drug applications, or ANDAs, in the United States. Generic drugs may be significantly less costly to bring to market than the reference-listed drug and companies that produce generic drugs are generally able to offer them at lower prices. Thus, following the introduction of a generic drug, a significant percentage of the sales of any branded product or reference-listed drug is typically lost to the generic drug.

The FDA may not approve an ANDA for a generic drug until any applicable period of non-patent exclusivity for the reference-listed drug has expired, but such exclusivity will not delay the submission or approval of a full NDA. However, an applicant submitting a full NDA would be required to conduct or obtain a right of reference

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to all of the nonclinical studies and adequate and well-controlled clinical trials necessary to demonstrate safety and effectiveness. Manufacturers may seek to launch generic drugs following the expiration of the marketing exclusivity period, even if we still have patent protection for such drugs.

Competition that our product candidates, if approved, may face from generic drugs could materially and adversely impact our future revenue, profitability and cash flows and substantially limit our ability to obtain a return on the investments we have made in those product candidates. Our future revenues, profitability and cash flows could also be materially and adversely affected and our ability to obtain a return on the investments we have made in those product candidates may be substantially limited if our product candidates, if and when approved, are not afforded the appropriate periods of non-patent exclusivity.

If we are unable to establish sales, marketing and distribution capabilities for any product candidate that may receive regulatory approval, we may not be successful in commercializing those product candidates if and when they are approved.

We do not have sales or marketing infrastructure and have no experience in the sale or marketing of pharmaceutical products. To achieve commercial success for any product candidate for which we may obtain marketing approval, we will need to establish a sales and marketing organization. In the future, we expect to build a focused sales and marketing infrastructure to market some of our product candidates in the United States, if and when they are approved. There are risks involved with establishing our own sales, marketing and distribution capabilities. For example, recruiting and training a sales force is expensive and time-consuming and could delay any product launch. If the commercial launch of a product candidate for which we recruit a sales force and establish marketing capabilities is delayed or does not occur for any reason, we would have prematurely or unnecessarily incurred these commercialization expenses. This may be costly, and our investment would be lost if we cannot retain or reposition our sales and marketing personnel.

Factors that may inhibit our efforts to market our products on our own include:

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our inability to recruit, train and retain adequate numbers of effective sales and marketing personnel;

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the inability of sales personnel to obtain access to or educate physicians about our product candidates, once approved;

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the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and

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unforeseen costs and expenses associated with creating an independent sales and marketing organization.

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If we are unable to establish our own sales, marketing and distribution capabilities and are forced to enter into arrangements with, and rely on, third parties to perform these services, our revenue and our profitability, if any, are likely to be lower than if we had developed such capabilities ourselves. In addition, we may not be successful in entering into arrangements with third parties to sell, market and distribute our product candidates or may be unable to do so on terms that are favorable to us. We likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to sell and market our products effectively. If we do not establish sales, marketing and distribution capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our product candidates.

We face competition, which may result in others discovering, developing or commercializing drugs before or more successfully than we do.

The development and commercialization of new drugs is highly competitive. We face and will continue to face competition from third parties that develop NLRP3 inhibitors and other anti-inflammatory therapies, including agents directed at downstream inflammatory cytokines, for the treatment of cardiovascular disease and neurology. Potential competitors also include academic institutions, government agencies and other public and private research organizations that conduct research, seek patent protection and establish collaborative arrangements for research, development, manufacturing and commercialization of new drugs.

We are aware of several biotechnology companies developing oral small-molecule NLRP3 inhibitors for cardiovascular, metabolic, inflammatory and neurodegenerative diseases, including but not limited to, Novartis, (DFV890), AstraZeneca PLC (AZD-4144), Eli Lilly (parunoflast, VTX2735), Roche (selnoflast), Ventus, (VENT-02), BioAge Labs, Inc. (BGE-102), Tenvie Therapeutics, Inc. (TNV262), Brenig (BT-409), InSilico (ISM8969),

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Neumora Therapeutics, Inc. (NMRA-215) and Neurocrine Biosciences, Inc. (TT-02332). Additionally, a NIMA-related kinase 7 degrader is being developed by Monte Rosa Therapeutics, Inc. (MRT-8102). Several large pharmaceutical companies have disclosed investments in companies developing NLRP3 inhibitors.

Many of our current or future competitors have greater financial resources and infrastructure supporting research and development, manufacturing,nonclinical testing, conducting clinical trials, obtaining regulatory approvals and the reimbursement and marketing of approved drugs than we do. Mergers and acquisitions in the pharmaceutical, biotechnology and diagnostic industries may result in even more resources being concentrated among a smaller number of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified scientific, sales, marketing and management personnel and establishing clinical trial sites and patient recruitment for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.

Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize drugs that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any drugs that we or our collaborators may develop. Our competitors also may obtain FDA or other regulatory approval for their drugs more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we or our collaborators are able to enter the market. The key competitive factors affecting the success of all of our current or any future product candidates, if approved, are likely to be their efficacy, safety, convenience, price, the level of generic competition and the availability of reimbursement from government and other third-party payors.

Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any current or future product candidates that we may develop.

We will face an inherent risk of product liability exposure related to the testing of our current or any future product candidates in human clinical trials and will face an even greater risk if we commercially sell any current or future product candidates that we may develop. If we cannot successfully defend ourselves against claims that our current or any future product candidates caused injuries, we could incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:

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decreased demand for any current or future product candidates that we may develop;

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injury to our reputation and significant negative media attention;

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withdrawal of clinical trial participants;

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significant costs to defend the related litigation;

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substantial monetary awards to trial participants or patients;

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loss of revenue; and

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the inability to commercialize any current or future product candidates that we may develop.

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While we maintain product liability insurance, we anticipate that we may need to increase our insurance coverage if we successfully commercialize any product candidate. Insurance coverage is increasingly expensive. We may not be able to maintain product liability insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise.

Even if we are able to commercialize any current or future product candidates, such drugs may become subject to unfavorable pricing regulations or third-party coverage and reimbursement policies, which would harm our business.

Significant uncertainty exists as to the coverage and reimbursement status of any products for which we may obtain regulatory approval. In the United States and in other countries, sales of any products for which we may receive regulatory marketing approval for commercial sale will depend, in part, on the availability of coverage and reimbursement from third-party payors. Third-party payors include government healthcare programs (e.g., Medicare and Medicaid), managed care providers, private health insurers, health maintenance organizations and other organizations. These third-party payors decide which medications they will pay for and establish reimbursement levels. The availability of coverage and extent of reimbursement by governmental and other third-party payors is essential for most patients to be able to afford treatments, such as treatments for inflammatory diseases. Third-party payors may also impose formulary placement requirements, utilization management controls, prior authorization or step therapy requirements, or patient cost-sharing obligations that could limit access to or adoption of our products, even if regulatory approval is obtained. See the section of this prospectus titled, “Business - Government Regulation - Third-party Payor Coverage and Reimbursement.”

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Current and future healthcare legislative reform measures may have a material adverse effect on our business and results of operations.

In the United States and in some foreign jurisdictions, there have been, and likely will continue to be, a number of legislative and regulatory changes and proposed changes intended to broaden access to healthcare, improve the quality of healthcare and contain or lower the cost of healthcare items and services. For example, in March 2010, the Patient Protection and Affordable Care Act, as amended by the Health Care and Education Reconciliation Act, or the ACA, was passed, which substantially changed the way healthcare is financed by both governmental and private insurers, and significantly impacted the U.S. pharmaceutical industry. The ACA, among other things, subjected drug products to potential competition by lower-cost biosimilars, expanded the types of entities eligible for the 340B drug discount program, addressed a new methodology by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected, increased minimum rebates owed by manufacturers under the Medicaid Drug Rebate Program and extended the rebate program to individuals enrolled in Medicaid managed care organizations, established annual fees and taxes on manufacturers of certain branded prescription drugs and created a new Medicare Part D coverage gap discount program - later replaced by a similar program under the Inflation Reduction Act of 2022, or the IRA, under which manufacturers must agree to offer point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during certain coverage periods, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D; and provided incentives to programs that increase the federal government’s comparative effectiveness research. For instance, the IRA also requires companies to pay rebates to Medicare for certain drug prices that increase faster than inflation and allows for the Centers for Medicare & Medicaid Services, or CMS to negotiate prices for certain single-source drugs and biologics reimbursed under Medicare. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors.

In addition, other legislative changes have been proposed and adopted in the United States that may adversely affect the commercial environment for pharmaceutical products. See the section of this prospectus titled, “Business - Government Regulation - Current and Future Healthcare Reform Legislation” for additional information.

We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, affect coverage or reimbursement criteria, or otherwise increase pricing pressure, which could result in reduced demand for our current or any future product candidates. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action in the United States. If we or any third parties we may engage are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we or such third parties are not able to maintain regulatory compliance, our product candidates may lose any regulatory approval that may have been obtained and we may not achieve or sustain profitability.

Our relationships with customers, health care providers, physicians, pharmacies, third-party payors and other entities, and our reporting of information in connection with the Medicaid Drug Rebate Program and other governmental pricing programs, will be subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to criminal sanctions, civil penalties, exclusion from government healthcare programs, contractual damages, reputational harm and diminished future profits and earnings.

Healthcare providers, physicians and third-party payors will play a primary role in the recommendation and prescription of any current or future product candidates for which we obtain marketing approval. Our business operations and any current or future arrangements with third-party payors, customers, healthcare providers, pharmacies and other third-party entities may expose us to broadly applicable federal and state laws relating to fraud and abuse, as well as other healthcare laws and regulations. These laws may impact, among other things, the business or financial arrangements and relationships through which we market, sell and distribute any current or future product candidates for which we obtain marketing approval. In addition, in order for our products to be eligible for coverage under certain government health care programs, we will be required to enter into agreements with the government and provide complex pricing calculations that, in turn, may influence government reimbursement rates. These laws are complex, broadly interpreted, and subject to evolving enforcement priorities. See the section of this prospectus titled, “Business - Government Regulation - Other Healthcare Laws and Regulations.”

It is possible that governmental authorities will conclude that our business practices, including our arrangements with certain physicians, some of whom are compensated in the form of stock or stock options for services provided to us, do not comply with current or future statutes, regulations or case law involving applicable fraud

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and abuse or other healthcare laws and regulations. If we fail to comply with our reporting and payment obligations under the Medicaid Drug Rebate Program or other governmental pricing programs, we could be subject to additional reimbursement requirements, penalties, sanctions and fines. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, disgorgement, contractual damages, reputational harm, diminished profits and future earnings, imprisonment, exclusion from government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations, as well as additional reporting obligations and oversight if we become subject to a corporate integrity agreement or similar settlement to resolve allegations of non-compliance with these laws, any of which could adversely affect our ability to operate our business and our financial results. If any of the physicians or other healthcare providers or entities with whom we expect to do business are found to be not in compliance with applicable laws, they may be subject to similar actions, penalties and sanctions.

The provision of benefits or advantages to physicians to induce or encourage the prescription, recommendation, endorsement, purchase, supply, order or use of medicinal products is also prohibited in the EU and UK. The provision of benefits or advantages to induce improper performance generally is governed by the national anti-bribery laws of EU Member States, and the UK Bribery Act 2010 in the UK. Infringement of these laws could result in substantial fines and imprisonment. Payments made to physicians in certain EU Member States must be publicly disclosed. Moreover, agreements with physicians often must be the subject of prior notification and approval by the physician’s employer, his or her competent professional organization and/or the regulatory authorities of the individual EU Member States. These requirements are provided in the national laws, industry codes or professional codes of conduct, applicable in the EU Member States. Failure to comply with these requirements could result in reputational risk, public reprimands, administrative penalties, fines or imprisonment.

Significant political, trade, regulatory developments and other circumstances beyond our control, including geopolitical events, could have a material adverse effect on our financial condition or results of operations.

We operate beyond the United States and, if approved, we may sell our products in countries throughout the world. Significant political, trade, geopolitical or regulatory developments globally and in the jurisdictions in which we may sell our products are difficult to predict and may have a material adverse effect on us. Similarly, changes in U.S. federal policy that affect the geopolitical landscape could give rise to circumstances outside our control that could have negative impacts on our business operations. For example, in 2025, the United States imposed tariffs on imports on its trading partners, including Canada, Mexico, the EU and China. Historically, tariffs have led to increased trade and political tensions. In response to tariffs, other countries have implemented retaliatory tariffs on U.S. goods. Political tensions as a result of trade policies could reduce trade volume, investment, technological exchange and other economic activities between major international economies, resulting in a material adverse effect on global economic conditions and the stability of global financial markets. Any changes in political, trade, regulatory, geopolitical and economic conditions, including U.S. trade policies or the recent United States and Israeli military action in Iran and effects thereof, could have a material adverse effect on our financial condition or results of operations.

Risks Related to Our Dependence on Third Parties

We currently rely, and plan to rely in the future, on third parties to conduct our clinical trials for our current and future product candidates. If these third parties do not successfully carry out their contractual duties, comply with regulatory requirements or meet expected deadlines, we may not be able to obtain marketing approval for or commercialize our current and potential future product candidates and our business could be substantially harmed.

We utilize and depend upon independent investigators and collaborators, such as medical institutions, CROs, CMOs and strategic partners to help conduct our clinical trials.

We do not have the ability to independently conduct clinical trials. We rely, and plan to continue to rely in the future, on medical institutions, clinical investigators, contract laboratories and other third parties, including collaboration partners, to conduct or otherwise support clinical trials for our current or any future product candidates.

We expect to rely heavily on these parties for execution of clinical trials for our product candidates and control only certain aspects of their activities. Nevertheless, we will be responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs will not relieve us of our regulatory responsibilities.

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We and any third parties that we contract with to conduct clinical trials are required to comply with regulations and requirements, including GCP requirements, which are regulations and guidelines enforced by the FDA and comparable foreign regulatory authorities for product candidates in clinical development, for conducting, monitoring, recording and reporting the results of clinical trials to ensure that the data and results are scientifically credible and accurate, and that the trial patients are adequately informed of the potential risks of participating in clinical trials and their rights are protected. These regulations are enforced by the FDA, the Competent Authorities of the Member States of the EEA and comparable foreign regulatory authorities for any drugs in clinical development. Regulatory authorities enforce these GCP requirements through periodic inspections of clinical trial sponsors, principal investigators and trial sites. If we or the third parties we contract with fail to comply with applicable GCP requirements, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that any of our future clinical trials will comply with GCP requirements.

In addition, our clinical trials must be conducted with current or future product candidates that must be produced under applicable cGMP regulations and will require a large number of study subjects. Our failure or the failure of third parties that we may contract with to comply with these regulations, or to recruit a sufficient number of subjects may require us to repeat some aspects of a specific, or an entire, clinical trial, which would delay the marketing approval process and could also subject us to enforcement action. We also are required to register certain current or future clinical trials and provide certain information, including information relating to the trial’s protocol and results from the trial, on a government-sponsored database, ClinicalTrials.gov, within specific timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions.

Although we intend to design the clinical trials for our current or any future product candidates, or be involved in the design when other parties sponsor the trials, we anticipate that third parties will conduct all of our clinical trials. As a result, many important aspects of our clinical development will be outside of our direct control. Our reliance on third parties to conduct our clinical trials will also result in less direct control over the management of data developed through clinical trials than would be the case if we were relying entirely upon our own staff, and we cannot control whether or not they will devote sufficient time and resources to our product candidates. These third parties may also have relationships with other commercial entities, including our competitors, for whom they may also be conducting clinical trials or other product development activities, which could affect their performance on our behalf. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities. Outside parties may:

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have staffing difficulties;

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fail to comply with contractual obligations;

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experience regulatory compliance issues; and

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form relationships with other entities, some of which may be our competitors.

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These factors may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. If our CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us or fail to comply with regulatory requirements, the development, marketing approval and commercialization of our current or any future product candidates may be delayed, we may not be able to obtain marketing approval and commercialize our current or any future product candidates, or our development programs may be materially and irreversibly harmed. If we are unable to rely on clinical data collected by our CROs, we could be required to repeat, extend the duration of, or increase the size of any clinical trials we conduct and this could significantly delay commercialization and require significantly greater expenditures.

If any of our relationships with these third-party CROs terminate, we may not be able to enter into arrangements with alternative CROs on commercially reasonable terms, or at all. If our CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain are compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any clinical trials such CROs are associated with may be extended, delayed or terminated, and we may not be able to obtain marketing approval for or successfully commercialize our current or any future product candidates. As a result, we believe that our financial results and the commercial prospects for our current or any future product candidates in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.

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The third parties upon whom we rely on for the supply of drug product and starting materials used in our product candidates are limited in number, and the loss of any of these suppliers, or their noncompliance with regulatory requirements or our quality standards, could significantly harm our business.

The drug substance and drug product in our product candidates are supplied to us from a small number of suppliers, and in some cases sole-source suppliers. We currently rely on a sole supplier for ruvonoflast drug substance and our proprietary material. Our ability to successfully develop our current or any future product candidates, and to ultimately supply our commercial drugs in quantities sufficient to meet the market demand, depends in part on our ability to obtain the drug product and drug substance for these drugs in accordance with regulatory requirements and in sufficient quantities for commercialization and clinical testing.

Our reliance on a sole supplier exposes us to a number of risks, including the possibility that the supplier could cease or interrupt production, experience quality or compliance failures, fail to meet our specifications, or terminate or decline to renew its arrangements with us. Our proprietary material used in the manufacture of ruvonoflast is not commercially available from multiple sources, which heightens these risks and could make any transition to an alternative supplier more difficult, time-consuming and costly. Any disruption in the supply of, or any increase in the cost of, ruvonoflast drug substance or our proprietary material could materially and adversely affect our ability to conduct clinical trials and, if approved, to commercialize ruvonoflast.

The facilities used by our contract manufacturers to manufacture our product candidates will be identified in, and subject to inspections that will be conducted after we submit any marketing application to the FDA or other comparable foreign regulatory authorities. We may not control the manufacturing process of, and may be completely dependent on, our contract manufacturing partners for compliance with cGMP requirements and any other regulatory requirements of the FDA or other regulatory authorities for the manufacture of our product candidates. Beyond periodic audits, we have no control over the ability of our contract manufacturers to maintain adequate quality control, quality assurance and qualified personnel. If the FDA or a comparable foreign regulatory authority does not approve our marketing applications identifying these facilities for the manufacture of our product candidates or if it withdraws any approval in the future, we may need to find alternative manufacturing facilities, which would require that we incur significant additional costs and materially adversely affect our ability to develop, obtain regulatory approval for or market our product candidates, if approved. Similarly, if any third-party manufacturers on which we will rely fail to manufacture quantities of our product candidates at quality levels necessary to meet regulatory requirements and at a scale sufficient to meet anticipated demand at a cost that allows us to achieve profitability, our business, financial condition and prospects could be materially and adversely affected.

Further, although we are working to locate and engage second-source suppliers, we do not currently have arrangements in place for a redundant or second-source supply of all drug product or drug substance in the event any of our current suppliers of such drug product and drug substance cease their operations for any reason. Any delays in the delivery of our drug substance, drug product or starting materials could have an adverse effect and potentially harm our business.

For all of our current or any future product candidates, we intend to identify and qualify additional manufacturers to provide drug product and drug substance prior to submission of an NDA, to the FDA and/or an MAA, to the EMA. We are not certain, however, that our single-source and dual source suppliers will be able to meet our demand for their products, either because of the nature of our agreements with those suppliers, our limited experience with those suppliers or our relative importance as a customer to those suppliers. It may be difficult for us to assess their ability to timely meet our demand in the future based on past performance. While our suppliers have generally met our demand for their products on a timely basis in the past, they may subordinate our needs in the future to their other customers.

Establishing additional or replacement suppliers for the drug product and drug substance used in our current or any future product candidates, if required, may not be accomplished quickly. In some cases, the technical skills required to manufacture our products or product candidates may be unique or proprietary to the original supplier and we may have difficulty, or there may be contractual restrictions prohibiting us from, transferring such skills to a back-up or alternate supplier, or we may be unable to transfer such skills at all. If we are able to find a replacement supplier, such replacement supplier would need to be qualified and may require additional regulatory approval, which could result in further delay. In addition, changes in manufacturers often involve changes in manufacturing procedures and processes, which could require that we conduct bridging studies between our prior clinical supply used in our clinical trials and that of any new manufacturer. We may be unsuccessful in demonstrating the comparability of clinical supplies which could require the conduct of additional clinical trials.

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While we seek to maintain adequate inventory of the drug product and drug substance used in our current or any future product candidates, any interruption or delay in the supply of components or materials, or our inability to obtain drug product and drug substance from alternate sources at acceptable prices in a timely manner, could impede, delay, limit or prevent our development efforts, which could harm our business, results of operations, financial condition and prospects.

In addition, some of our suppliers are located outside of the United States. There is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, government regulations and tariffs. It is possible further tariffs may be imposed that could affect imports of product for current or future product candidates, or our business may be adversely impacted by retaliatory trade measures taken by China or other countries, including restricted access to such raw materials used in our product candidate and any future product candidates.

Disruptions in our suppliers’ ability to meet our ongoing demand could have a material adverse effect on our business, financial condition, results of operations or prospects.

Our success is dependent on our executive management team’s ability to successfully pursue business development, strategic partnerships and investment opportunities as our company matures. We may form or seek strategic alliances or acquisitions or enter into collaboration and licensing arrangements in the future, and we may not realize the benefits of such collaborations, alliances, acquisitions or licensing arrangements.

We may in the future form or seek strategic alliances or acquisitions, create joint ventures, or enter into collaboration and licensing arrangements with third parties that we believe will complement or augment our development and commercialization efforts with respect to our current product candidates and any future product candidates that we may develop. Any of these relationships may require us to incur non-recurring and other charges, increase our near and long-term expenditures, or disrupt our management and business.

We face significant competition in seeking appropriate strategic partners and the negotiation process is time-consuming and complex. We may not be successful in our efforts to establish a strategic partnership or acquisition or other alternative arrangements for our current or any future product candidates because they may be deemed to be at too early of a stage of development for collaborative effort and third parties may not view our current or any future product candidates as having the requisite potential to demonstrate safety, potency, purity and efficacy and obtain marketing approval.

Further, collaborations and licensing deals involving our technologies or current or future product candidates are subject to numerous risks, which may include the following:

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collaborators have significant discretion in determining the efforts and resources that they will apply to a collaboration;

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collaborators may not pursue development and commercialization of our current or any future product candidates or may elect not to continue or renew development or commercialization of our current or any future product candidates based on clinical trial results, changes in their strategic focus due to the acquisition of competitive products, availability of funding or other external factors, such as a business combination that diverts resources or creates competing priorities;

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collaborators may delay clinical trials, provide insufficient funding for a clinical trial, stop a clinical trial, abandon a product candidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing;

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collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our current or any future product candidates;

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a collaborator with marketing and distribution rights to one or more products may not commit sufficient resources to their marketing and distribution;

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collaborators may not properly maintain or defend our intellectual property rights or may use our intellectual property or proprietary information in a way that gives rise to actual or threatened litigation that could jeopardize or invalidate our intellectual property or proprietary information or expose us to potential liability;

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disputes may arise between us and a collaborator that cause the delay or termination of the research, development or commercialization of our current or any future product candidates, or that result in costly litigation or arbitration that diverts management attention and resources;

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collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development or commercialization of the applicable current or future product candidates;

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collaborators may own or co-own intellectual property covering our products that results from our collaborating with them, and in such cases, we would not have the exclusive right to commercialize such intellectual property; and

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collaborators may not pay milestones and royalties due to the company in a timely manner.

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As a result, we may not be able to realize the benefits of any future strategic partnerships or acquisitions, collaborations or license arrangements we may enter into if we are unable to successfully integrate them with our existing operations and company culture, which could delay our timelines or otherwise adversely affect our business. We also cannot be certain that, following a strategic transaction, license, collaboration or other business development partnership, we will achieve the revenue or specific net income that justifies such transaction. Any delays in entering into collaborations or strategic partnership agreements related to our current or any future product candidates could delay the development and commercialization of our current or any future product candidates in certain geographies or for certain indications, which would harm our business prospects, financial condition and results of operations.

Manufacturing our current or any future product candidates is complex and we may encounter difficulties in production. If we encounter such difficulties, our ability to provide supply of our current or any future product candidates for our nonclinical studies and clinical trials or for commercial purposes could be delayed or stopped.

The process of manufacturing of our current or any future product candidates is complex and highly regulated. We do not have our own manufacturing facilities or personnel and currently rely, and expect to continue to rely, on third parties for the manufacture of our current or any future product candidates. These third-party manufacturing providers may not be able to provide adequate or timely resources or capacity to meet our needs and may incorporate their own proprietary processes into our product candidate manufacturing processes. We have limited control and oversight of a third party’s proprietary process, and a third party may elect to modify its process without our consent or knowledge. These modifications could negatively impact our manufacturing, including product loss or failure that requires additional manufacturing runs or a change in manufacturer, either of which could significantly increase the cost of and significantly delay the manufacture of our current or any future product candidates.

As our current or any future product candidates progress through nonclinical studies and clinical trials towards potential approval and commercialization, it is expected that various aspects of the manufacturing process will be altered in an effort to optimize processes and results. Such changes may require amendments to be made to regulatory applications which may further delay the timeframes under which modified manufacturing processes can be used for any of our current or any future product candidates and additional bridging studies or trials may be required and may not be successful. We may be unsuccessful in demonstrating the comparability of clinical supplies which could require the conduct of additional clinical trials. Any such delay could have a material adverse impact on our business, results of operations and prospects.

Our manufacturing process needs to comply with FDA regulations relating to the quality and reliability of such processes. Any failure to comply with relevant regulations could result in delays in or termination of our current and future clinical programs and suspension or withdrawal of any regulatory approvals.

We do not own or operate, and currently have no plans to establish, manufacturing facilities. In order to commercially produce our products at a third party’s facility, we and they will need to comply with the FDA’s cGMP regulations and guidelines. We may encounter difficulties in achieving quality control and quality assurance and may experience shortages in qualified personnel. We and our third-party manufacturers are subject to inspections by the FDA and comparable foreign regulatory authorities to confirm compliance with applicable regulatory requirements. Any failure to follow cGMP or other regulatory requirements or delay, interruption or other issues that arise in the manufacture, fill-finish, packaging or storage of our product candidates as a result of a failure of our facilities or the facilities or operations of third parties to comply with regulatory requirements or pass any regulatory authority inspection could significantly impair our ability to develop and commercialize our current or any future product candidates, including leading to significant delays in the availability of our product candidates for our future clinical trials or the termination of or suspension of a future clinical trial, or the delay or prevention of a filing or approval of marketing applications for our current or any future product candidates. Significant non-compliance could also result in the imposition of sanctions, including warning or untitled letters, fines, injunctions, civil penalties, failure of regulatory authorities to grant marketing approvals for our current or any future product candidates, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could damage our reputation and our business.

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If our third-party manufacturers use hazardous and biological materials in a manner that causes injury or violates applicable law, we may be liable for damages.

Our research and development activities involve the controlled use of potentially hazardous substances, including chemical materials, by our third-party manufacturers. Our manufacturers are subject to federal, state and local laws and regulations in the United States, as well as laws and regulations in Europe, governing the use, manufacture, storage, handling and disposal of medical and hazardous materials. Although we believe that our manufacturers’ procedures for using, handling, storing and disposing of these materials comply with legally prescribed standards, we cannot completely eliminate the risk of contamination or injury resulting from medical or hazardous materials. As a result of any such contamination or injury, we may incur liability or local, city, state or federal authorities may curtail the use of these materials and interrupt our business operations. In the event of an accident, we could be held liable for damages or penalized with fines, and the liability could exceed our resources. We do not have any insurance for liabilities arising from medical or hazardous materials. Compliance with applicable environmental laws and regulations is expensive, and current or future environmental regulations may impair our research, development and production efforts, which could harm our business, prospects, financial condition or results of operations.

Risks Related to Our Intellectual Property

If we are unable to obtain and maintain patent and other intellectual property protection for our technology and product candidates or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired, and we may not be able to compete effectively in our market.

We rely upon a combination of patents, trademarks, trade secret protection and confidentiality agreements to protect the intellectual property related to our products and technologies and to prevent third parties from copying and surpassing our achievements, thus eroding our competitive position in our market. Our commercial success depends in part on our ability to obtain and maintain patent or other intellectual property protection in the United States and other countries for our current or any future product candidates and our core technologies, including our clinical stage product candidates, ruvonoflast and trabzanoflast. We seek to protect our proprietary and intellectual property position by, among other methods, filing patent applications in the United States and abroad related to our proprietary technology, inventions and improvements that are important to the development and implementation of our business.

As of December 31, 2025, we owned 8 patent families related to ruvonoflast and trabzanoflast. As of December 31, 2025, we owned one issued U.S. patent. Patent term adjustments, supplementary protection certificate filings, or patent term extensions could result in later expiration dates in various countries, while terminal disclaimers could result in earlier expiration dates in the United States. Further, patent prosecution related to our pending patent applications is ongoing.

The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions and has in recent years been the subject of much litigation. As such, we cannot guarantee that our pending and future patent applications will result in patents being issued or that issued patents will afford sufficient protection of our product candidates or their intended uses against competitors, nor can there be any assurance that the patents issued will not be infringed, designed around, invalidated by third parties or effectively prevent others from commercializing competitive technologies, products or product candidates.

The degree of patent protection we require to successfully commercialize our current or any future product candidates may be unavailable or severely limited in some cases and may not adequately protect our rights or permit us to gain or keep any competitive advantage. We cannot provide any assurances that any of our pending patent applications that mature into issued patents will include claims with a scope sufficient to protect our current or any future product candidates. In addition, if the breadth or strength of protection provided by our patent applications or any patents we may own or in-license is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize our current or any future product candidates.

Other parties have developed technologies that may be related or competitive to our own, and such parties may have filed or may file patent applications, or may have acquired or may acquire patents, claiming inventions that may overlap or conflict with those claimed in our own patent applications or issued patents, with respect

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to either the same compounds, methods, formulations or other subject matter, in either case that we may rely upon to support our patent position in the market. Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until at least 18 months after the earliest priority date of the patent filing, or in some cases not at all. Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in patents we may own or in-license patents or pending patent applications, or that we were the first to file for patent protection of such inventions. In addition, the U.S. Patent and Trademark Office, or USPTO, might require that the term of a patent issuing from a pending patent application be disclaimed and limited to the term of another patent that is commonly owned or names a common inventor. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights cannot be predicted with any certainty. In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States.

In addition, the patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. Further, with respect to certain pending patent applications covering our current or any future product candidates or technologies, prosecution has yet to commence and as such, no patent examiner has scrutinized the merits of such pending patent applications. Patent prosecution is a lengthy process, during which the scope of the claims initially submitted for examination by the relevant patent office(s) may be significantly narrowed by the time they issue, if they ever do. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. Moreover, in some circumstances, we may not have the right to control the preparation, filing and prosecution of patent applications, or to maintain the patents, covering technology that we license from or to third parties. Therefore, these patents and applications may not be prosecuted and enforced in a manner consistent with the best interests of our business.

Even if we acquire patent protection that we expect should enable us to establish and/or maintain a competitive advantage, third parties may challenge the validity, enforceability or scope thereof, which may result in such patents being narrowed, invalidated or held unenforceable. The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our patents may be challenged in the courts or patent offices in the United States and abroad. We may become involved in opposition, derivation, reexamination, inter partes review or post-grant review proceedings challenging our patent rights or the patent rights of others from whom we may in the future obtain licenses to such rights, in the USPTO, the European Patent Office, or EPO, or the relevant patent authorities in other countries. In addition, we may be subject to third-party submissions to the USPTO, the EPO, or elsewhere, that may reduce the scope or preclude the granting of claims from our pending patent applications. Competitors may challenge our issued patents or may file patent applications before we do.

Competitors may also claim that we are infringing their patents and that we therefore cannot practice our technology as claimed under our patents or patent applications. Competitors may also contest our patents by arguing before an administrative patent authority or judge that the invention was not patent-eligible, was not novel, was obvious, and/or lacked inventive steps, and/or that the patent application failed to meet relevant requirements relating to description, basis, enablement, and/or support; in litigation, a competitor could assert that our patents are not valid or are unenforceable for a number of reasons. If a court or administrative patent authority agrees, we would lose our protection of those challenged patents.

An adverse determination in any such submission or proceeding may result in loss of exclusivity or freedom to operate or in patent claims being narrowed, invalidated or held unenforceable, in whole or in part, which could limit our ability to stop others from using or commercializing similar or identical technology and drugs, without payment to us, or could limit the duration of the patent protection covering our technology and current or future product candidates. Such challenges may also result in our inability to manufacture or commercialize our current or any future product candidates without infringing third-party patent rights. In addition, if the breadth or strength of protection provided by our patents and patent applications is threatened, it could dissuade companies from collaborating with us to license, develop or commercialize current or future product candidates.

Even if they are unchallenged, our issued patents and our pending patent applications, if issued, may not provide us with any meaningful protection or prevent competitors from designing around our patent claims to circumvent patents we may own or in-license by developing similar or alternative technologies or drugs in

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a non-infringing manner. For example, a third party may develop a competitive drug or product that provides benefits similar to one or more of our current or any future product candidates but that has a different composition or otherwise falls outside the scope of our patent protection. If the patent protection provided by the patents and patent applications we hold or pursue with respect to our current or any future product candidates is not sufficiently broad to impede such competition, our ability to successfully commercialize our current or any future product candidates could be negatively affected, which would harm our business.

Obtaining and maintaining our patent protection, including patent term, depends on compliance with various procedural, document submission, deadlines, fee payment and other requirements imposed by governmental patent agencies, and our patent protection could be reduced or eliminated if we miss a filing deadline for patent protection on these inventions or otherwise fail to comply with these requirements.

The USPTO and foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other similar provisions during the patent application process and after issuance of any patent. In addition, periodic maintenance fees, renewal fees, annuity fees and/or various other government fees are required to be paid periodically. While an inadvertent lapse can in some cases be cured by payment of a late fee or by other means in accordance with the applicable rules, there are situations in which noncompliance can result in abandonment or lapse of the patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. Noncompliance events that could result in abandonment or lapse of a patent include, but are not limited to, failure to respond to official actions within prescribed time limits, non-payment of fees and failure to properly legalize and submit formal documents. In such an event, our competitors might be able to enter the market with similar or identical products or discovery engines, which could have a material adverse effect on our business prospects and financial condition.

Patent terms may be inadequate to protect our competitive position on our product candidates for an adequate amount of time.

Patents have a limited lifespan. In the United States, and most other jurisdictions in which we have undertaken patent filings, the natural expiration of a patent is generally twenty years after it is filed, assuming all maintenance fees are paid. Various extensions may be available, on a jurisdiction-by-jurisdiction basis; however, the life of a patent, and thus the protection it affords, is limited. Given the amount of time required for the development, testing and regulatory review of new product candidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, patents we may own or in-license may not provide us with adequate and continuing patent protection sufficient to exclude others from commercializing drugs similar or identical to our current or any future product candidates, including generic versions of such drugs.

Depending upon the timing, duration and specifics of FDA marketing approval of our current or any future product candidates, one or more of the U.S. patents we own or license may be eligible for limited patent term restoration under the Drug Price Competition and Patent Term Restoration Act of 1984, or the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent restoration term of up to five years as compensation for patent term lost during product development and the FDA regulatory review process. Different laws govern the extension of patents on approved pharmaceutical products in Europe and other jurisdictions. However, we may not be granted a patent extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. For example, we may not be granted an extension in the United States if all of our patents covering an approved product expire more than fourteen years from the date of NDA approval for a product covered by those patents. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. If we are unable to obtain patent term extension or restoration or the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our ability to generate revenues could be materially adversely affected.

If our trademarks and trade names for our products or company name are not adequately protected in one or more countries where we intend to market our products, we may delay the launch of product brand names, use different trademarks or trade names in different countries or face other potentially adverse consequences to building our product brand recognition.

We use and will continue to use registered and/or unregistered trademarks or trade names to brand and market ourselves and our products. Our trademarks or trade names may be challenged, infringed, diluted, circumvented or declared generic or determined to be infringing on other marks. We intend to rely on both registration and common law protection for our trademarks. We may not be able to protect our rights to these trademarks

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and trade names or may be forced to stop using these names, which we need for name recognition by potential partners or customers in our markets of interest. During the trademark registration process, we may receive Office Actions from the USPTO or from comparable agencies in foreign jurisdictions objecting to the registration of our trademark. In addition, in the USPTO and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and/or to seek the cancellation of registered trademarks. Opposition or cancellation proceedings may be filed against our trademark applications or registrations, and our trademark applications or registrations may not survive such proceedings. In addition, there could be potential trade name or trademark infringement claims brought by owners of other trademarks or trademarks that incorporate variations of our registered or unregistered trademarks or trade names. Over the long run, if we are unable to obtain a registered trademark or establish name recognition based on our trademarks and trade names, we may not be able to compete effectively and our business may be adversely affected.

Additionally, we may license our trademarks and trade names to third parties, such as distributors. Though these license agreements may provide guidelines for how our trademarks and trade names may be used, a breach of these agreements or misuse of our trademarks and trade names by our licensees may jeopardize our rights in or diminish the goodwill associated with our trademarks and trade names.

If we are unable to adequately protect and enforce our trade secrets, our business and competitive position would be harmed.

In addition to the protection afforded by patents we may own or in-license, we seek to rely on trade secret protection, confidentiality agreements and license agreements to protect proprietary know-how that may not be patentable, processes for which patents are difficult to enforce and any other elements of our product discovery and development processes that involve proprietary know-how, information or technology that may not be covered by patents. Although we require all of our employees, consultants, advisors and any third parties who have access to our proprietary know-how, information or technology to enter into non-disclosure and confidentiality agreements, trade secrets can be difficult to protect and we have limited control over the protection of trade secrets used by our collaborators and suppliers. We cannot be certain that we have or will obtain these agreements in all circumstances and we cannot guarantee that we have entered into such agreements with each party that may have or have had access to our trade secrets or proprietary information.

Moreover, any of these parties might breach the agreements and intentionally or inadvertently disclose our trade secret information and we may not be able to obtain adequate remedies for such breaches. In addition, competitors may otherwise gain access (such as through unauthorized access to our information technology systems) to our trade secrets or independently develop substantially equivalent information and techniques. Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. If we choose to go to court to stop a third party from using any of our trade secrets, we may incur substantial costs. These lawsuits may consume our time and other resources even if we are successful. Furthermore, the laws of some foreign countries do not protect proprietary rights and trade secrets to the same extent or in the same manner as the laws of the United States. We may need to share our proprietary information, including trade secrets, with future business partners, collaborators, contractors and others located in countries at heightened risk of theft of trade secrets, including through direct intrusion by private parties or foreign actors, and those affiliated with or controlled by state actors. As a result, we may encounter significant problems in protecting and defending our intellectual property both in the United States and abroad. If we are unable to prevent unauthorized material disclosure of our intellectual property to third parties, we will not be able to establish or maintain a competitive advantage in our market, which could materially adversely affect our business, financial condition, results of operations and future prospects.

We may initiate, become a defendant in, or otherwise become party to lawsuits to protect or enforce our intellectual property rights, which could be expensive, time-consuming and unsuccessful.

Competitors may infringe or otherwise violate any patents or other intellectual property we may own or in-license. In addition, any patents we may own or in-license also may become involved in inventorship, priority, validity or unenforceability disputes. To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming. Any such claims could provoke these parties to assert counterclaims against us, including claims alleging that we infringe their patents or other intellectual property rights. In patent litigation in the United States and in some other jurisdictions, defendant

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counterclaims alleging invalidity and/or unenforceability are commonplace. Grounds for a validity challenge could be an alleged failure to meet any of several statutory requirements, for example, lack of novelty, obviousness or non-enablement. Grounds for an unenforceability assertion could be an allegation that someone connected with prosecution of the patent withheld material information from the USPTO or the applicable foreign counterpart, or made a misleading statement, during prosecution. A litigant or the USPTO itself could challenge our patents on this basis even if we believe that we have conducted our patent prosecution in accordance with the duty of candor and in good faith. The outcome following such a challenge is unpredictable. Moreover, with respect to challenges to the validity of our patents, there might be invalidating prior art, of which we and the patent examiner were unaware during prosecution.

We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. In addition, in an infringement proceeding, a court may decide that one or more of any patents we may own or in-license is not valid or is unenforceable or that the other party’s use of our technology that may be patented falls under the safe harbor to patent infringement under 35 U.S.C. §271(e)(1). There is also the risk that, even if the validity of these patents is upheld, the court may refuse to stop the other party from using the technology at issue on the grounds that any patents we may own or in-license do not cover the technology in question or that such third party’s activities do not infringe our patent applications or any patents we may own or in-license. An adverse result in any litigation or defense proceedings could put one or more of any patents we may own or in-license at risk of being invalidated, held unenforceable, or interpreted narrowly and could put our patent applications at risk of not being issued. Even if resolved in our favor, litigation or other legal proceedings relating to our intellectual property rights may cause us to incur significant expenses, and could distract our technical and management personnel from their normal responsibilities. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, patient support or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources and more mature and developed intellectual property portfolios. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.

We may be required to protect our patents through procedures created to attack the validity of a patent at the USPTO. Post-grant proceedings, including post-grant review, inter partes review and derivation proceedings, provoked by third parties or brought by the USPTO may be necessary to determine the validity or priority of inventions with respect to our patent applications or any patents we may own or in-license. These proceedings are expensive and an unfavorable outcome could result in a loss of our current patent rights and could require us to cease using the related technology or to attempt to license rights to it from the prevailing party. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. In addition to potential USPTO post-grant proceedings, we may become a party to patent opposition proceedings in the EPO, or similar proceedings in other foreign patent offices or courts where our patents may be challenged. The costs of these proceedings could be substantial and may result in a loss of scope of some claims or a loss of the entire patent. An unfavorable result in a post-grant challenge proceeding may result in the loss of our right to exclude others from practicing one or more of our inventions in the relevant country or jurisdiction, which could have a material adverse effect on our business. Litigation or post-grant proceedings within patent offices may result in a decision adverse to our interests and, even if we are successful, may result in substantial costs and distract our management and other employees.

Furthermore, because of the substantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information could be compromised by disclosure during this type of litigation. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments. If securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock.

We may not be able to detect infringement against any patents we may own or in-license. Even if we detect infringement by a third party of any patents we may own or in-license, we may choose not to pursue litigation against or settlement with the third party. If we later sue such third party for patent infringement, the third party may have certain legal defenses available to it, which otherwise would not be available except for the delay between when the infringement was first detected and when the suit was brought. Such legal defenses may make it impossible for us to enforce any patents we may own or in-license against such third party.

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Intellectual property litigation and administrative patent office patent validity challenges in one or more countries could cause us to spend substantial resources and distract our personnel from their normal responsibilities. Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, and could distract our technical and management personnel from their normal responsibilities. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could compromise our ability to compete in the marketplace, including compromising our ability to raise the funds necessary to continue our nonclinical studies and future clinical trials, continue our discovery programs, license necessary technology from third parties, or enter into development collaborations or licensing arrangements that would help us commercialize our current or any future product candidates.

In addition, if our product candidates are found to infringe the intellectual property rights of third parties, these third parties may assert infringement claims against our licensees and other parties with whom we have business relationships, and we may be required to indemnify those parties for any damages they suffer as a result of these claims. The claims may require us to initiate or defend protracted and costly litigation on behalf of licensees and other parties regardless of the merits of these claims. If any of these claims succeed, we may be forced to pay damages on behalf of those parties or may be required to obtain licenses for the products they use.

Any of the foregoing events would harm our business, financial condition, results of operations and prospects.

We may be subject to claims challenging the inventorship of our patents and other intellectual property.

We may be subject to claims that former employees, collaborators or other third parties have an interest in our patents or other intellectual property as an inventor or co-inventor. The failure to name the proper inventors on a patent application can result in the patents issuing thereon being unenforceable. Inventorship disputes may arise from conflicting views regarding the contributions of different individuals named as inventors, the effects of foreign laws where foreign nationals are involved in the development of the subject matter of the patent, conflicting obligations of third parties involved in developing our product candidates or as a result of questions regarding co-ownership of potential joint inventions. Litigation may be necessary to resolve these and other claims challenging inventorship and/or ownership. Alternatively, or additionally, we may enter into agreements to clarify the scope of our rights in such intellectual property. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights, such as exclusive ownership of, or right to use, valuable intellectual property. Such an outcome could have a material adverse effect on our business. Even if we are successful in defending against such claims, litigation could result in substantial costs and be a distraction to management and other employees.

Our current and future licensors may have relied on third-party consultants or collaborators or on funds from third parties, such as the U.S. government, such that our licensors are not the sole and exclusive owners of the patents we in-licensed. If other third parties have ownership rights or other rights to our in-licensed patents, they may be able to license such patents to our competitors, and our competitors could market competing products and technology. This could have a material adverse effect on our competitive position, business, financial conditions, results of operations and prospects.

In the case of employees, we enter into agreements providing that all inventions conceived by the individual, and which are related to our current or planned business or research and development or made during normal working hours, on our premises or using our equipment or proprietary information, are our exclusive property. Although we require all of our employees to assign their inventions to us, we may be unsuccessful in executing such an agreement with each party who, in fact, conceives or develops intellectual property that we regard as our own. The assignment of intellectual property rights may not be self-executing, or the assignment agreements may be breached, and we may be forced to bring claims against third parties, or defend claims that they may bring against us, to determine the ownership of what we regard as our intellectual property. Such claims could have a material adverse effect on our business, financial condition, results of operations and prospects.

Third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain. Defending against such lawsuits will be costly and time-consuming, and an unfavorable outcome in that litigation would have a material adverse effect on our business.

The intellectual property landscape relevant to our products and programs is crowded, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating or otherwise violating their intellectual property rights, the outcome of which would be uncertain and could have a material adverse effect on the success of our business. Our commercial success depends upon our ability to develop,

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manufacture, market and sell our current and future product candidates and use our proprietary technologies without infringing, misappropriating or otherwise violating the valid and enforceable intellectual property rights of third parties. There is a substantial amount of litigation involving patents and other intellectual property rights in the biotechnology and pharmaceutical industries, as well as administrative proceedings for challenging patents, including derivation, interference, reexamination, inter partes review and post grant review proceedings before the USPTO or oppositions and other comparable proceedings in foreign jurisdictions. We or any of our current or future licensors or strategic partners may be party to, exposed to, or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that our current or any future product candidates and/or proprietary technologies infringe, misappropriate or otherwise violate their intellectual property rights. We cannot assure you that our current or any future product candidates and other technologies that we have developed, are developing or may develop in the future do not or will not infringe, misappropriate or otherwise violate existing or future patents or other intellectual property rights owned by third parties.

While certain activities related to development and nonclinical and clinical testing of our current or any future product candidates may be subject to safe harbor of patent infringement under 35 U.S.C. §271(e)(1), upon receiving FDA approval for such candidates we or any of our future licensors or strategic partners may immediately become party to, exposed to or threatened with, future adversarial proceedings or litigation by third parties having patent or other intellectual property rights alleging that such product candidates infringe, misappropriate or otherwise violate their intellectual property rights. Numerous U.S. and foreign issued patents and pending patent applications, which are owned by third parties, exist in the fields in which we are developing our current or any future product candidates. As the biotechnology and pharmaceutical industries expand and more patents are issued, the risk increases that our current or any future product candidates may give rise to claims of infringement of the patent rights of others. Moreover, it is not always clear to industry participants, including us, which patents cover various types of drugs, products or their methods of use or manufacture. Thus, because of the large number of patents issued and patent applications filed in our fields, there may be a risk that third parties may allege they have patent rights encompassing our current or any future product candidates, technologies or methods.

If a third party claims that we infringe, misappropriate or otherwise violate its intellectual property rights, we may face a number of issues, including, but not limited to:

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infringement, misappropriation and other intellectual property claims which, regardless of merit, may be expensive and time-consuming to litigate and may divert our management’s attention from our core business and may impact our reputation;

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substantial damages for infringement, misappropriation or other violations, which we may have to pay if a court decides that the product candidate or technology at issue infringes, misappropriates or violates the third party’s rights, and, if the court finds that the infringement was willful, we could be ordered to pay treble damages and the patent owner’s attorneys’ fees;

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a court prohibiting us from developing, manufacturing, marketing or selling our current or any future product candidates, or from using our proprietary technologies, unless the third party licenses its product rights to us, which it is not required to do on commercially reasonable terms or at all;

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if a license is available from a third party, we may have to pay substantial royalties, upfront fees and other amounts, and/or grant cross-licenses to intellectual property rights for our products, or the license to us may be non-exclusive, which would permit third parties to use the same intellectual property to compete with us; and

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redesigning our current or any future product candidates or processes so they do not infringe, misappropriate or violate third-party intellectual property rights, which may not be possible or may require substantial monetary expenditures and time.

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Some of our competitors may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources. In addition, any uncertainties resulting from the initiation and continuation of any litigation could have a material adverse effect on our ability to raise the funds necessary to continue our operations or could otherwise have a material adverse effect on our business, results of operations, financial condition and prospects. The occurrence of any of the foregoing could have a material adverse effect on our business, financial condition, results of operations or prospects.

Third parties may assert that we are employing their proprietary technology without authorization. Patents issued in the United States by law enjoy a presumption of validity that can be rebutted in U.S. courts only with evidence that is “clear and convincing,” a heightened standard of proof. There may be issued third-party

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patents of which we are currently unaware with claims to compositions, formulations, methods of manufacture or methods for treatment related to the use or manufacture of our current or any future product candidates. Patent applications can take many years to issue. In addition, because some patent applications in the United States may be maintained in secrecy until the patents are issued, patent applications in the United States and many foreign jurisdictions are typically not published until 18 months after their earliest priority filing date, and publications in the scientific literature often lag behind actual discoveries, we cannot be certain that others have not filed patent applications covering our current or any future product candidates or technology. If any such patent applications issue as patents, and if such patents have priority over our patent applications or patents we may own or in-license, we may be required to obtain rights to such patents owned by third parties which may not be available on commercially reasonable terms or at all, or may only be available on a non-exclusive basis. There may be currently pending third-party patent applications which may later result in issued patents that our current or any future product candidates may infringe. It is also possible that patents owned by third parties of which we are aware, but which we do not believe are relevant to our current or any future product candidates or other technologies, could be found to be infringed by our current or any future product candidates or other technologies. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents. Moreover, we may fail to identify relevant patents or incorrectly conclude that a patent is invalid, not enforceable, exhausted, or not infringed by our activities. If any third-party patents were held by a court of competent jurisdiction to cover the manufacturing process of our current or any future product candidates, molecules used in or formed during the manufacturing process, or any final product itself, the holders of any such patents may be able to block our ability to commercialize the product candidate unless we obtained a license under the applicable patents, or until such patents expire or they are finally determined to be held invalid or unenforceable. Similarly, if any third-party patent were held by a court of competent jurisdiction to cover aspects of our formulations, processes for manufacture or methods of use, including combination therapy or patient selection methods, the holders of any such patent may be able to block our ability to develop and commercialize the product candidate unless we obtained a license or until such patent expires or is finally determined to be held invalid or unenforceable. In either case, such a license may not be available on commercially reasonable terms or at all. If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, or at all, our ability to commercialize our current or any future product candidates may be impaired or delayed, which could in turn significantly harm our business. Even if we obtain a license, it may be nonexclusive, thereby giving our competitors access to the same technologies licensed to us.

In addition, parties making claims against us may seek and obtain injunctive or other equitable relief, which could effectively block our ability to further develop and commercialize our current or any future product candidates. Defense of these claims, regardless of their merit, could involve substantial litigation expense and would be a substantial diversion of employee resources from our business. In the event of a successful claim of infringement, misappropriation or other violation against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, obtain one or more licenses from third parties, pay royalties or redesign our infringing products, which may be impossible or require substantial time and monetary expenditure. We cannot predict whether any such license would be available at all or whether it would be available on commercially reasonable terms. Furthermore, even in the absence of litigation, we may need or may choose to obtain licenses from third parties to advance our research or allow commercialization of our current or any future product candidates, which licenses may not be available on commercially reasonable terms, or at all. In that event, we would be unable to further develop and commercialize our current or any future product candidates or technologies, which could harm our business significantly.

We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information, alleged trade secrets of third parties or competitors, or are in breach of non-competition or non-solicitation agreements with our competitors or their former employers.

As is common in the biotechnology and pharmaceutical industries, we employ individuals and engage the services of consultants who previously worked for other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although no claims against us are currently pending and we try to ensure that our employees, consultants and advisors do not use the proprietary information or know-how of others in their work for us, we may be subject to claims that we or these individuals have used or disclosed intellectual property, including trade secrets or other proprietary information, of any such individual’s former employer. We may also be subject to claims that patents and applications we have filed to protect inventions of our

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employees, consultants and advisors, even those related to one or more of our current or any future product candidates or other technologies, are rightfully owned by their former or concurrent employer. Litigation may be necessary to defend against these claims. If we fail in defending any such claims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel. Even if we are successful in defending against such claims, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, and could distract our technical and management personnel from their normal responsibilities.

We will not obtain patent or other intellectual property protection for any current or future product candidates in all jurisdictions throughout the world, and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.

We may not be able to pursue patent coverage of our current or any future product candidates or other technologies in all countries. Filing, prosecuting and defending patents on current or future product candidates and other technologies in all countries throughout the world would be prohibitively expensive, and intellectual property rights in some countries outside the United States can be less extensive than those in the United States. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Consequently, we may not be able to prevent third parties from infringing on our inventions in all countries outside the United States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and further, may export otherwise infringing products to territories where we have patent protection, but where enforcement is not as strong as that in the United States. These products may compete with our current or any future product candidates and in jurisdictions where we do not have any issued patents our patent applications or other intellectual property rights may not be effective or sufficient to prevent them from competing. Much of our patent portfolio is at a very early stage. We will need to decide whether and in which jurisdictions to pursue protection for the various inventions in our portfolio prior to applicable deadlines.

Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to pharmaceutical products, which could make it difficult for us to stop the infringement of any patents we may own or in-license or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce any rights we may have in our patent applications or any patents we may own or in-license in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put any patents we may own or in-license at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing, and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

Many countries have compulsory licensing laws under which a patent owner may be compelled to grant licenses to third parties. In addition, many countries limit the enforceability of patents against government agencies or government contractors. In these countries, the patent owner may have limited remedies, which could materially diminish the value of such patent. If we are forced to grant a license to third parties with respect to any patents we may own or license that are relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations and prospects may be adversely affected.

We may not obtain or grant licenses or sublicenses to intellectual property rights in all markets on equally or sufficiently favorable terms with third parties.

It may be necessary for us to use the patented or proprietary technology of third parties to commercialize our products, in which case we would be required to obtain a license from these third parties. The licensing of third-party intellectual property rights is a competitive area, and more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary. More established companies may have a competitive advantage over us due to their size, capital resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. We also may be unable to license

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or acquire third-party intellectual property rights on terms that would allow us to make an appropriate return on our investment or at all. If we are unable to license such technology, or if we are forced to license such technology on unfavorable terms, our business could be materially harmed. If we are unable to obtain a necessary license, we may be unable to develop or commercialize the affected current or future product candidates, which could materially harm our business, and the third parties owning such intellectual property rights could seek either an injunction prohibiting our sales, or, with respect to our sales, an obligation on our part to pay royalties or other forms of compensation. Even if we are able to obtain a license, it may be non-exclusive, thereby giving our competitors access to the same technologies licensed to us. Any of the foregoing could harm our competitive position, business, financial condition, results of operations and prospects.

Any future collaboration and license agreements that we enter into may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, increase what we believe to be our financial or other obligations under the relevant agreement, or decrease what we believe to be the financial or other obligations of our licensee under the relevant agreement, any of which could materially harm our business, financial condition, results of operations and prospects. Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our current licensing arrangements or obtain additional licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected products or product candidates, which could have a material adverse effect on our business, financial conditions, results of operations and prospects.

Further, our licensors may retain certain rights under their agreements with us, including the right to use the underlying technology for noncommercial academic and research use, to publish general scientific findings from research related to the technology, and to make customary scientific and scholarly disclosures of information relating to the technology. It is difficult to monitor whether our licensors limit their use of the technology to these uses, and we could incur substantial expenses to enforce our rights to our licensed technology in the event of misuse. In addition, the U.S. federal government retains certain rights in inventions produced with its financial assistance under the Patent and Trademark Law Amendments Act, or the Bayh-Dole Act. The federal government retains a “nonexclusive, nontransferable, irrevocable, paid-up license” for its own benefit. The Bayh-Dole Act also provides federal agencies with “march-in rights.” March-in rights allow the government, in specified circumstances, to require the contractor or successors in title to the patent to grant a “nonexclusive, partially exclusive or exclusive license” to a “responsible applicant or applicants.” If the patent owner refuses to do so, the government may grant the license itself. If, in the future, we co-own or license in technology which is critical to our business that is developed in whole or in part with federal funds subject to the Bayh-Dole Act, our ability to enforce or otherwise exploit patents covering such technology may be adversely affected.

Changes in patent law in the United States and in foreign jurisdictions could diminish the value of patents in general, thereby impairing our ability to protect our products.

Changes in either the patent laws or interpretation of the patent laws in the United States could increase the uncertainties and costs surrounding the prosecution of patent applications and the enforcement or defense of issued patents. Assuming that other requirements for patentability are met, prior to March 16, 2013, in the United States, the first to invent the claimed invention was entitled to the patent, while outside the United States, the first to file a patent application was entitled to the patent. On March 16, 2013, under the Leahy-Smith America Invents Act, or the America Invents Act, enacted in September 2011, the United States transitioned to a first inventor to file system in which, assuming that other requirements for patentability are met, the first inventor to file a patent application will be entitled to the patent on an invention regardless of whether a third party was the first to invent the claimed invention. A third party that files a patent application in the USPTO on or after March 16, 2013, but before us could therefore be awarded a patent covering an invention of ours even if we had made the invention before it was made by such third party. This will require us to be cognizant of the time from invention to filing of a patent application. Since patent applications in the United States and most other countries are confidential for a period of time after filing or until issuance, we cannot be certain that we or our licensors were the first to either (i) file any patent application related to our product candidates or (ii) invent any of the inventions claimed in our or our licensor’s patents or patent applications.

The America Invents Act also includes a number of significant changes that affect the way patent applications are prosecuted and also may affect patent litigation. These include allowing third party submission of prior art to the USPTO during patent prosecution and additional procedures to attack the validity of a patent by USPTO

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administered post-grant proceedings, including post-grant review, inter partes review and derivation proceedings. Because of a lower evidentiary standard in USPTO proceedings compared to the evidentiary standard in United States federal courts necessary to invalidate a patent claim, a third party could potentially provide evidence in a USPTO proceeding sufficient for the USPTO to hold a claim invalid even though the same evidence would be insufficient to invalidate the claim if first presented in a district court action. Accordingly, a third party may attempt to use the USPTO procedures to invalidate our patent claims that would not have been invalidated if first challenged by the third party as a defendant in a district court action. Therefore, the America Invents Act and its implementation could increase the uncertainties and costs surrounding the enforcement or defense of our owned or in-licensed issued patents, all of which could have a material adverse effect on our business, financial condition, results of operations and prospects.

In addition, the patent positions of companies in the development and commercialization of biopharmaceuticals are particularly uncertain. Recent U.S. Supreme Court rulings have narrowed the scope of patent protection available in certain circumstances and weakened the rights of patent owners in certain situations. This combination of events has created uncertainty with respect to the validity and enforceability of patents, once obtained. Depending on future actions by the U.S. Congress, the federal courts and the USPTO, the laws and regulations governing patents could change in unpredictable ways that could have a material adverse effect on our existing patent portfolio and our ability to protect and enforce our intellectual property in the future.

We may not identify relevant third-party patents or may incorrectly interpret the relevance, scope or expiration of a third-party patent, which might subject us to infringement claims or adversely affect our ability to develop and market our current or any future product candidates.

We cannot guarantee that any of our or our licensors’ patent searches or analyses, including the identification of relevant patents, the scope of patent claims or the expiration of relevant patents, are complete or thorough, nor can we be certain that we have identified each and every third-party patent and pending patent application in the United States and abroad that is relevant to or necessary for the commercialization of our current or any future product candidates in any jurisdiction. For example, U.S. patent applications filed before November 29, 2000, and certain U.S. patent applications filed after that date that will not be filed outside the United States remain confidential until patents issue. As mentioned above, patent applications in the United States and elsewhere are published approximately 18 months after the earliest filing for which priority is claimed, with such earliest filing date being commonly referred to as the priority date. Therefore, patent applications covering our current or any future product candidates could have been filed by third parties without our knowledge. Additionally, pending patent applications that have been published can, subject to certain limitations, be later amended in a manner that could cover our current or any future product candidates or the use of our current or any future product candidates. The scope of a patent claim is determined by an interpretation of the law, the written disclosure in a patent and the patent’s prosecution history. Our interpretation of the relevance or the scope of a patent or a pending application may be incorrect, which may negatively impact our ability to market our current or any future product candidates. We may incorrectly determine that our current or any future product candidates are not covered by a third-party patent or may incorrectly predict whether a third party’s pending application will be issued with claims of relevant scope. Our determination of the expiration date of any patent in the United States or abroad that we consider relevant may be incorrect, which may negatively impact our ability to develop and market our current or any future product candidates. Our failure to identify and correctly interpret relevant patents may negatively impact our ability to develop and market our current or any future product candidates.

If we fail to identify and correctly interpret relevant patents, we may be subject to infringement claims. We cannot guarantee that we will be able to successfully settle or otherwise resolve such infringement claims. If we fail in any such dispute, in addition to being forced to pay damages, which may be significant, we may be temporarily or permanently prohibited from commercializing any of our current or any future product candidates or technologies that are held to be infringing. We might, if possible, also be forced to redesign current or future product candidates so that we no longer infringe the third-party intellectual property rights. Any of these events, even if we were ultimately to prevail, could require us to divert substantial financial and management resources that we would otherwise be able to devote to our business and could adversely affect our business, financial condition, results of operations and prospects.

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Intellectual property rights do not guarantee commercial success of current or future product candidates or other business activities. Numerous factors may limit any potential competitive advantage provided by our intellectual property rights.

The degree of future protection afforded by our intellectual property rights, whether owned or in-licensed, is uncertain because intellectual property rights have limitations, and may not adequately protect our business, provide a barrier to entry against our competitors or potential competitors, or permit us to maintain our competitive advantage. Moreover, if a third party has intellectual property rights that cover the practice of our technology, we may not be able to fully exercise or extract value from our intellectual property rights. The following examples are illustrative:

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patent applications that we own or may in-license may not lead to issued patents;

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patents, should they issue, that we may own or in-license, may not provide us with any competitive advantages, may be narrowed in scope, or may be challenged and held invalid or unenforceable;

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others may be able to develop and/or practice technology, including compounds that are similar to the chemical compositions of our current or any future product candidates, that is similar to our technology or aspects of our technology but that is not covered by the claims of any patents we may own or in-license, should any patents issue;

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third parties may compete with us in jurisdictions where we do not pursue and obtain patent protection;

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we, or our future licensors or collaborators, might not have been the first to make the inventions covered by a patent application that we own or may in-license;

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we, or our future licensors or collaborators, might not have been the first to file patent applications covering a particular invention;

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others may independently develop similar or alternative technologies without infringing, misappropriating or otherwise violating our intellectual property rights;

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our competitors might conduct research and development activities in the United States and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well as in countries where we do not have patent rights, and may then use the information learned from such activities to develop competitive products for sale in our major commercial markets;

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we may not be able to obtain and/or maintain necessary licenses on reasonable terms or at all;

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third parties may assert an ownership interest in our intellectual property and, if successful, such disputes may preclude us from exercising exclusive rights, or any rights at all, over that intellectual property;

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we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third party may subsequently file a patent covering such trade secrets or know-how;

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we may not be able to maintain the confidentiality of our trade secrets or other proprietary information;

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we may not develop or in-license additional proprietary technologies that are patentable; and

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the patents of others may have an adverse effect on our business.

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Should any of these events occur, they could significantly harm our business, financial condition, results of operations and prospects.

European patents and patent applications could be challenged in the recently created Unified Patent Court, or the UPC, for the European Union.

Our owned European patents and patent applications could be challenged in the recently created UPC for the European Union. We may decide to opt out our European patents and patent applications from the UPC. However, if certain formalities and requirements are not met, our European patents and patent applications could be challenged for non-compliance and brought under the jurisdiction of the UPC. We cannot be certain that our or our licensors’ European patents and patent applications will avoid falling under the jurisdiction of the UPC, if we decide to opt out of the UPC. Under the UPC, a granted European patent would be valid and enforceable in numerous European countries. A successful invalidity challenge to a European patent under the UPC would result in loss of patent protection in those European countries. Accordingly, a single proceeding under the UPC could result in the partial or complete loss of patent protection in numerous European countries, rather than in each validated European country separately as such patents always have been adjudicated. Such a loss of patent protection could have a material adverse impact on our business and our ability to commercialize our technology and products and, resultantly, on our business, financial condition, prospects and results of operations.

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The use of new and evolving technologies, such as artificial intelligence, or AI, in our business may result in spending material resources and presents risks and challenges that can impact our business including by posing security and other risks to our confidential information, proprietary information and personal information.

Like most other businesses, we are increasingly integrating AI into our business, primarily through the adoption of third-party tools. These activities present cybersecurity, data privacy, intellectual property, regulatory, operational, competitive and reputational risks. In particular, AI technologies may produce inaccurate, biased or harmful outputs, and may be vulnerable to targeted attacks such as model or data poisoning. AI use also gives rise to intellectual property risks, including the potential disclosure or compromise of our confidential or proprietary information, intellectual property infringement and challenges to our ability to assert or defend ownership rights in AI-assisted work product.

A growing number of legislators and regulators globally are adopting laws and regulations, and focusing enforcement efforts, on the development and deployment of AI. These developments may increase our compliance burden and costs and expose us to legal liability if we fail to meet evolving legal standards or if our use of such technologies results in unanticipated harms. For example, the FDA has issued draft guidance on the use of AI in regulatory decision-making for drug and biological products, establishing a credibility assessment framework for evaluating AI model outputs intended to support regulatory submissions.

Our vendors may incorporate AI tools into their own offerings, and the providers of these AI tools may not meet existing or rapidly evolving regulatory or industry standards, including with respect to privacy and data security. In addition, the use of generative AI models in our internal or third-party systems may create new attack surfaces or methods for adversaries. Bad actors around the world use increasingly sophisticated methods, including AI, to engage in illegal activities involving the theft and misuse of personal information, confidential information and intellectual property. Any of these developments could damage our reputation, result in the loss of valuable property and information, cause us to breach applicable laws and regulations, and adversely impact our business.

The cost of maintaining separate intellectual property protection across multiple jurisdictions could be significant and could divert capital from our development programs.

Prosecuting, maintaining, and enforcing our intellectual property portfolio across the United States, the United Kingdom and other jurisdictions requires substantial and ongoing expenditure, and following the withdrawal of the United Kingdom from the European Union, European patents must be validated and maintained separately in the United Kingdom, increasing the cost of our global IP strategy. If we are unable or choose not to maintain patent protection in any key jurisdiction due to cost constraints, the value of our product candidates and our ability to compete effectively in those markets could be materially impaired, which in turn could reduce our ability to attract partnership or licensing revenues and raise capital on favorable terms. The costs of our intellectual property maintenance program are difficult to predict and could increase materially as our pipeline expands.

Risks Related to Employee Matters and Managing Growth

Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.

We are highly dependent on the research and development, clinical and business development expertise of Geoffrey McDonough, M.D., our President and Chief Executive Officer, Chris Guiffre, J.D., M.B.A., our Chief Financial Officer, Jyothis George, MBBS, Ph.D., our Chief Medical Officer, Alan P. Watt, Ph.D., M.B.A., our founder and Chief Scientific Officer, and Nicholas Clarke, Ph.D., our Chief Development Officer, as well as the other principal members of our management and scientific teams. Although we have entered into employment letter agreements with our executive officers, each of them may terminate their employment with us at any time. We do not maintain “key person” insurance for any of our executives or other employees. In addition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.

Recruiting and retaining qualified scientific, clinical, manufacturing and sales and marketing personnel will also be critical to our success. The loss of the services of our executive officers or other key employees could impede the achievement of our research, development and commercialization objectives and seriously harm

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our ability to successfully implement our business strategy. Furthermore, replacing executive officers and key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to successfully develop, gain regulatory approval of and commercialize drugs. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel. We also experience competition for the hiring of scientific and clinical personnel from universities and research institutions. Failure to succeed in clinical trials may make it more challenging to recruit and retain qualified scientific personnel. In addition, in order to induce employees to continue their employment with us, we have provided equity awards that vest over time and the value to our employees of such equity awards may be significantly affected by movements in our stock price that are beyond our control and may be at any time insufficient to counteract more lucrative offers from other companies. If we are unable to continue to attract and retain high quality personnel, the rate and success at which we can develop and commercialize product candidates will be limited.

We will need to develop and expand our company, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations.

As of June 30, 2026, we had 21 full-time employees and six part-time consultants. In connection with becoming a public company, we expect to increase our number of employees and the scope of our operations, including the areas of sales, marketing and market access, medical affairs, clinical development, regulatory affairs, quality assurance, quality control, manufacturing, finance, accounting, legal, human resources, information technology and information security. To manage our anticipated development and expansion, we must continue to implement and improve our managerial, operational and financial systems, expand our facilities and continue to recruit and train additional qualified personnel. Our management may need to divert a disproportionate amount of its attention away from its day-to-day activities and devote a substantial amount of time to managing these development activities. Due to our limited resources, we may not be able to effectively manage the expansion of our operations or recruit and train additional qualified personnel. This may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. The expansion of our operations may lead to significant costs and may divert financial resources from other projects, such as the development of our current or any future product candidates. If our management is unable to effectively manage our expected development and expansion, our expenses may increase more than expected, our ability to generate or increase our revenue could be reduced and we may not be able to implement our business strategy. Our future financial performance and our ability to commercialize our current or any future product candidates, if approved, and compete effectively will depend, in part, on our ability to effectively manage the future development and expansion of our company.

We have personnel in multiple countries and we may further expand in the future, which presents challenges in managing our business operations.

We are headquartered in Boston, Massachusetts and also have an office in Cambridge, United Kingdom. Additionally, we have personnel in multiple states within the United States. Conducting our business in multiple jurisdictions subjects us to a variety of risks and complexities that may materially and adversely affect our business, results of operations, financial condition and growth prospects, including, among other things:

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the increased complexity and costs inherent in managing multi-jurisdictional operations;

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diverse regulatory, financial and legal requirements, and any future changes to such requirements, in one or more countries where we are located or do business;

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country- and/or state-specific tax, labor and employment laws and regulations;

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challenges inherent in efficiently managing employees in diverse geographies, including the need to adapt systems, policies, benefits and compliance programs to differing labor and other regulations;

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liabilities for activities of, or related to, our international operations or product candidates;

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costs associated with tariffs;

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changes in currency rates; and

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regulations relating to data security and the unauthorized use of, or access to, commercial and personal information.

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We continue to expand our operations, and our corporate structure and tax structure is complex. In connection with our current and future potential partnerships, we are actively engaged in developing and applying technologies and intellectual property with a view toward commercialization of products globally, often with

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commercialization partners. In connection with those activities, we will likely engage in complex cross-border and global transactions involving our technology, intellectual property and other assets, between us and other entities such as partners and licensees, and between us and our subsidiaries. Such cross-border and global arrangements are both difficult to manage and can potentially give rise to complexities in areas such as tax treatment, particularly since we are subject to multiple tax regimes and different tax authorities can also take different views from each other, even as regards the same cross-border transaction or arrangement. There can be no assurance that we will effectively manage this increased complexity without experiencing operating inefficiencies, control deficiencies or tax liabilities. Significant management time and effort is required to effectively manage the increased complexity of our company, and our failure to successfully do so could have a material adverse effect on our business, financial condition, results of operations and growth prospects.

We may be unable to adequately protect our information systems from cyberattacks, data breaches and cybersecurity incidents, which could result in the disclosure of confidential or proprietary information, including personal data, damage our reputation and subject us to significant financial and legal exposure.

We rely on information technology systems that we or our third-party providers operate to process, transmit and store electronic information in our day-to-day operations. Our management team, including personnel responsible for information technology and information security, is responsible for assessing and managing cybersecurity risks and reporting material cybersecurity matters to our board of directors or the appropriate board committee. In connection with our product discovery efforts, we may collect and use a variety of personal data, such as name, mailing address, email addresses, phone number and clinical trial information. We, like other organizations in our industry, have experienced threats and cybersecurity incidents relating to our information technology systems and infrastructure. Specifically in August 2025, we experienced a cybersecurity incident involving an unauthorized transfer of funds. As a result of the incident, we recognized an approximately $2.0 million loss during the year ended December 31, 2025. We promptly engaged external cybersecurity experts, implemented remediation measures, enhanced our internal controls and security procedures and notified affected individuals and regulators as appropriate. Although the incident has been contained, we cannot assure you that our remediation efforts will prevent future cybersecurity incidents. A successful data breach, cybersecurity incident or other cyberattack could result in the theft or destruction of intellectual property, data or other misappropriation of assets, or otherwise compromise our confidential or proprietary information and disrupt our operations. Cyberattacks generally are increasing in their frequency, sophistication and intensity, and have become increasingly difficult to detect. Cyberattacks could include wrongful conduct by hostile foreign governments, wrongful conduct or error by vendors or employees, industrial espionage, wire fraud and other forms of cyber fraud, the deployment of malware, denial-of-service, ransomware, business email compromises, data breaches, viruses, social engineering (including phishing attacks) fraud or other means to threaten or compromise the security, confidentiality, integrity and availability of systems and information. A successful cyberattack, data breach or cybersecurity incident could cause serious negative consequences for us, including, without limitation, the disruption of operations, the misappropriation of protected information or confidential business information, including financial information, trade secrets, financial loss and the disclosure of corporate strategic plans.

Although we devote resources to protect our information systems, we realize that cyberattacks are a threat, and there can be no assurance that our efforts will prevent cybersecurity incidents or data breaches that would result in business, legal, financial or reputational harm to us, or would have a material adverse effect on our results of operations and financial condition. Attempts to disrupt or gain unauthorized access to our and our third-party vendors’ information systems from malicious third parties or insider threats may incorporate widely varying and frequently changing tactics, which may be enhanced or facilitated by evolving technology, including artificial intelligence. In addition, although we carry cyber insurance, in the event of a cybersecurity incident or breach, such coverage may not be sufficient to cover all losses. Any failure to prevent or mitigate or adequately address any cybersecurity incidents, data breaches, or other improper access to, use of or disclosure of our clinical data or patients’ personal data could require us to notify impacted stakeholders (including affected individuals, regulators and investors) and result in significant liability through litigation and regulatory investigations and enforcement actions, including under U.K. data protection laws, U.S. state (e.g., state breach notification laws), and/or federal laws and laws of other foreign jurisdictions (e.g., the EU General Data Protection Regulation, or GDPR) and may cause a material adverse impact to our reputation, affect our ability to use collected data, conduct new studies and potentially disrupt our business.

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For example, the loss of data from nonclinical studies or clinical trials for our current or any future product candidates could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or cybersecurity incident results in a loss of or damage to our data or applications, other data or applications relating to our technology or current or future product candidates, or inappropriate disclosure of confidential or proprietary information, we could incur liabilities and the further development of our current or any future product candidates could be delayed.

We rely on our third-party providers to implement effective security measures and identify and correct for any such failures, deficiencies, cybersecurity incidents or data breaches. We also rely on our employees and consultants to safeguard their security credentials and follow our policies and procedures regarding use and access of computers and other devices that may contain our sensitive information. If we or our third-party providers fail to maintain or protect our information technology systems and data integrity effectively or fail to anticipate, plan for or manage significant disruptions to our information technology systems, we or our third-party providers could have difficulty preventing, detecting and controlling such cyber-attacks and any such attacks could result in losses described above, as well as disputes with physicians, patients and our partners, litigation, regulatory sanctions or penalties, increases in operating expenses, expenses or lost revenues or other adverse consequences, any of which could have a material adverse effect on our business, results of operations, financial condition, prospects and cash flows. Any failure by such third parties to prevent or mitigate or adequately address any cybersecurity incidents, data breaches, or other improper access to or disclosure of such information could have similarly adverse consequences for us. If we are unable to prevent or mitigate the impact of such cybersecurity incidents or data breaches, we could be exposed to litigation and governmental investigations, which could lead to a potential disruption to our business, or fines and penalties. Our contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages or claims related to our privacy and data security obligations.

We, our collaborators and our service providers may be subject to a variety of privacy and data security laws, regulations and contractual obligations, and our failure to comply with them could harm our business.

We maintain a large quantity of sensitive information, including confidential business and patient health information including in connection with our clinical trials and nonclinical studies, and are subject to laws and regulations governing the privacy and security of such information. In the United States, there are numerous federal and state privacy and data security laws and regulations governing the collection, use, disclosure and protection of personal information, including federal and state health information privacy laws, federal and state security breach notification laws, and federal and state consumer protection laws. Each of these laws is subject to varying interpretations and new laws continue to be proposed. At the state level, numerous states have or are in the process of enacting or considering comprehensive state-level data privacy and security laws, rules and regulations while other states have focused on more narrow aspects of privacy. In the state of Washington, for example, the My Health My Data Act requires regulated entities to obtain consent to collect health information, grant consumers certain rights, including to request deletion of their information, and provide for robust enforcement mechanisms, including enforcement by the Washington state attorney-general and a private right of action for consumer claims.

Outside of the United States, many jurisdictions have enacted stringent privacy and data protection laws. The collection, use, disclosure, transfer or other processing of personal data originating from the European Economic Area, or EEA, and United Kingdom, or UK, is governed by the General Data Protection Regulation, or EU GDPR, and the UK General Data Protection Regulation, or UK GDPR, which, together with the EU GDPR, is referred to as the GDPR. The GDPR imposes stringent requirements for controllers and processors of personal data of persons in the EU and UK, including, for example, ensuring an appropriate legal basis or condition applies to the processing of personal data, more robust disclosures to individuals and a strengthened individual data rights regime, shortened timelines for data breach notifications, limitations on retention of information, increased requirements pertaining to special categories of data, such as health data, and additional obligations when we contract with third-party processors in connection with the processing of the personal data. The GDPR also imposes strict rules on the transfer of personal data out of the EU and UK to the United States and other third countries. We rely on the UK International Data Transfer Agreement, or IDTA, as the principal mechanism for transferring personal data from the United Kingdom to the United States and other third countries. These transfer mechanisms remain subject to legal challenge and to evolving regulatory and judicial

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interpretation, and the IDTA or other mechanisms on which we rely could be invalidated, suspended or made subject to additional conditions, which could require us to implement alternative safeguards, restrict or suspend data transfers or incur additional compliance costs. In addition, the GDPR provides that EU member states may make their own further laws and regulations limiting the processing of personal data, including genetic, biometric or health data. Failure to comply with the requirements of the GDPR may result in fines of up to €20,000,000 (or £17.5 million in the UK) or up to 4% of the total worldwide annual turnover of the preceding financial year, whichever is higher, and other administrative penalties. GDPR regulations may impose additional responsibility and liability in relation to the personal data that we process and we may be required to put in place additional mechanisms to ensure compliance with the new data protection rules. For additional information on these regimes, see “Business - Government Regulation - Data Privacy and Security”. Compliance with these and any other applicable privacy and data security laws and regulations is a rigorous and time-intensive process, and we may be required to put in place additional mechanisms to ensure compliance, and despite those efforts, if we fail, or are perceived to fail, to comply with any such laws or regulations, we may face significant fines and penalties that could adversely affect our reputation, business, financial condition and results of operations.

Our employees, principal investigators, CROs and consultants may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading laws.

We are exposed to the risk that our employees, principal investigators, CROs and consultants may engage in fraudulent conduct or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violate the regulations of the FDA and other regulatory authorities, including those laws requiring the reporting of true, complete and accurate information to such authorities; healthcare fraud and abuse laws and regulations in the United States and abroad; or laws that require the reporting of financial information or data accurately. In particular, sales, marketing, patient support and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs, patient support and other business arrangements. Other activities subject to these laws include the improper use of information obtained in the course of clinical trials or creating fraudulent data in our nonclinical studies or clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of conduct applicable to all of our employees, but it is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. Additionally, we are subject to the risk that a person could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal healthcare programs, contractual damages, reputational harm, diminished profits and future earnings and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations.

Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more of such laws. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant criminal, civil and administrative sanctions including monetary penalties, damages, fines, disgorgement, individual imprisonment, reputational harm, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws and the curtailment or restructuring of our operations, any of which could adversely affect our ability to operate our business and our results of operations.

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The risk of our being found in violation of these laws is increased by the fact that many of them have not been fully interpreted by the regulatory authorities or the courts, and their provisions are open to a variety of interpretations. Any action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business. The shifting compliance environment and the need to build and maintain robust and expandable systems to comply with multiple jurisdictions with different compliance and/or reporting requirements increases the possibility that a healthcare company may run afoul of one or more of the requirements.

Risks Related to Our Common Stock and This Offering

The price of our common stock may be volatile and fluctuate substantially, which could result in substantial losses for purchasers of our common stock in this offering.

Our stock price is likely to be volatile. The stock market in general and the market for biopharmaceutical companies in particular have experienced extreme volatility that has often been unrelated to the operating performance of particular companies. As a result of this volatility, you may not be able to sell your common stock at or above the initial public offering price. The market price for our common stock may be influenced by many factors, including:

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the success of competitive drugs or technologies;

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results of nonclinical studies and clinical trials of our current or any future product candidates or those of our competitors;

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unanticipated safety concerns related to the use of any of our product candidates;

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regulatory or legal developments in the United States and other countries;

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developments or disputes concerning patent applications, issued patents or other proprietary rights;

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the recruitment or departure of key personnel;

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the level of expenses related to any of our current or any future product candidates or clinical development programs;

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the results of our efforts to discover, develop, acquire or in-license additional current or future product candidates or drugs;

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actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts;

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variations in our financial results or those of companies that are perceived to be similar to us;

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product liability claims or other litigation;

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changes in the structure of healthcare payment systems;

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market conditions in the pharmaceutical and biotechnology sectors;

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general economic, industry and market conditions, including inflation and potential tariffs; and

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the other factors described in this “Risk Factors” section.

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The stock market in general, including The Nasdaq Stock Market LLC, or Nasdaq, and biopharmaceutical companies in particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies, which has resulted in decreased stock prices for many companies notwithstanding the lack of a fundamental change in their underlying business models or prospects. Broad market and industry factors, including potentially worsening economic conditions and other adverse effects or developments relating to geopolitical events or public health crises, may negatively affect the market price of our common stock, regardless of our actual operating performance. The realization of any of the above risks or any of a broad range of other risks, including those described in this section, could have a significant and material adverse impact on the market price of our common stock.

An active trading market for our common stock may not develop, and you may not be able to resell your shares at or above the initial public offering price.

Prior to this offering, there has been no public market for shares of our common stock. Although we intend to apply to list our common stock on Nasdaq, an active trading market for our common stock may never develop or be sustained following this offering. The initial public offering price of our common stock was determined through negotiations between us and the underwriters. This initial public offering price may not be indicative

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of the market price of our common stock after this offering. In the absence of an active trading market for our common stock, investors may not be able to sell their common stock at or above the initial public offering price or at the time that they would like to sell.

We may not be able to maintain a listing of our common stock on Nasdaq.

We must meet certain financial and liquidity criteria to maintain our listing on Nasdaq. If we fail to meet any of Nasdaq’s listing rules, our common stock may be delisted. The delisting of our common stock from Nasdaq may materially impair our stockholders’ ability to buy and sell our common stock and could have an adverse effect on the market price of, and the efficiency of the trading market for, our common stock. The delisting of our common stock could significantly impair our ability to raise capital and the value of your investment.

If you purchase our common stock in this offering, you will incur immediate and substantial dilution in the book value of your shares.

You will suffer immediate and substantial dilution in the net tangible book value of the common stock you purchase in this offering. Based on the assumed initial public offering price of $       per share, the midpoint of the range set forth on the cover of this prospectus, purchasers of common stock in this offering will experience immediate dilution of $       per share in net tangible book value of the common stock. In addition, investors purchasing common stock in this offering will contribute % of the total amount invested by stockholders since inception but will only own % of the shares of common stock outstanding. In the past, we issued options and other securities to acquire common stock at prices significantly below the initial public offering price. To the extent these outstanding securities are ultimately exercised, investors purchasing common stock in this offering will sustain further dilution. See the section of this prospectus titled “Dilution” for a more detailed description of the dilution to new investors in the offering.​

Certain of our management have anti-dilution rights, which may cause further dilution to our stockholders, including purchasers in this offering.

We have granted anti-dilution rights to Geoffrey McDonough, M.D., our President and Chief Executive Officer. On September 3, 2026, we issued Dr. McDonough a warrant to purchase up to 862,426 shares of our common stock at an exercise price of $1.00 per share in consideration of services under his service agreements with us. The exercise of these awards will dilute the ownership interests of our existing stockholders and purchasers in this offering, and the issuance or perceived issuance of the underlying shares could adversely affect the market price of our common stock. We may also agree to similar arrangements in the future in order to attract or retain executive officers or key employees, which could result in further dilution. See the sections titled “Executive Compensation” and “Certain Relationships and Related Party Transactions” for additional information.

Raising additional capital may cause dilution to our stockholders, including purchasers of common stock in this offering, restrict our operations or require us to relinquish rights to our current or any future product candidates.

Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of private and public equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. We do not currently have any committed external source of funds. To the extent that we raise additional capital through the sale of common stock or securities convertible or exchangeable into common stock, your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that materially adversely affect your rights as a common stockholder. Debt financing, if available, would increase our fixed payment obligations and may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

If we raise funds through additional collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our intellectual property, future revenue streams, discovery programs or current or future product candidates or to grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, scale back or discontinue the development and commercialization of one or more of our product candidates, delay our pursuit of potential in-licenses or acquisitions or grant rights to develop and market current or future product candidates that we would otherwise prefer to develop and market ourselves.

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If securities analysts do not publish research or reports about our business or if they publish negative evaluations of our stock, the price of our stock could decline.

The trading market for our common stock will rely in part on the research and reports that industry or financial analysts publish about us or our business. We do not currently have and may never obtain research coverage by industry or financial analysts. If no or few analysts commence coverage of us, the trading price of our stock would likely decrease. In the event we do have research analyst coverage, we will not have any control over the analysts or the content and opinions included in their reports. Additionally, if one or more of the analysts covering our business downgrade their evaluations of our stock, the price of our stock could decline. If one or more of these analysts cease to cover our stock, we could lose visibility in the market for our stock, which in turn could cause our stock price to decline.​

We have identified material weaknesses in our internal control over financial reporting. If we are unable to successfully remediate these material weaknesses in our internal control over financial reporting, it could have an adverse effect on our company.

We have identified certain material weaknesses in our internal control over financial reporting. These material weaknesses include: (1) ineffective control activities, including those related to recognition in the appropriate period for certain transactions; (2) an ineffective control environment, including a lack of sufficient accounting personnel and oversight processes; (3) ineffective risk assessment controls, including those policies and practices that would identify changes in our business practices and information technology, which could significantly impact our consolidated financial statements and system of internal controls; and (4) ineffective monitoring of controls related to the financial close and reporting process. Had we performed an evaluation of our internal control over financial reporting in accordance with Section 404 of the Sarbanes-Oxley Act, or Section 404, additional control deficiencies may have been identified by management, and those control deficiencies could have also represented one or more material weaknesses.

In an effort to remediate these material weaknesses, we have retained an accounting consulting firm to provide additional depth and breadth in our technical accounting and financial reporting capabilities. We intend to continue this arrangement until permanent technical accounting resources are identified and hired. We intend to formalize our policies and procedures surrounding our financial close, financial reporting and other accounting processes. We intend to further develop and document necessary policies and procedures regarding our internal control over financial reporting, such that we are able to perform a Section 404 analysis of our internal control over financial reporting when and as required following the completion of this offering. We are in the process of recruiting additional qualified accounting and finance personnel to provide needed levels of expertise in our internal accounting function. We cannot assure you that these measures will significantly improve or remediate the material weaknesses described above. We also cannot assure you that we have identified all or that we will not have additional material weaknesses in the future. Accordingly, material weaknesses may still exist when we report on the effectiveness of our internal control over financial reporting for purposes of our attestation when required by reporting requirements under the Exchange Act of 1934, as amended, or the Exchange Act or Section 404 after this offering. Further, while we remain an emerging growth company, we will not be required to include an attestation report on internal control over financial reporting issued by our independent registered public accounting firm.

We expect to incur additional costs to remediate these control deficiencies, though there can be no assurance that our efforts will be successful or avoid potential future material weaknesses. If we are unable to successfully remediate our existing or any future material weaknesses in our internal control over financial reporting, or if we identify any additional material weaknesses, the accuracy and timing of our financial reporting may be adversely affected, we may be unable to maintain compliance with securities law requirements regarding timely filing of periodic reports in addition to applicable stock exchange listing requirements, investors may lose confidence in our financial reporting, and our stock price may decline as a result. We also could become subject to investigations by Nasdaq, the SEC or other regulatory authorities.

If we fail to establish and maintain proper and effective internal controls, our ability to produce accurate and timely financial results could be impaired, which could result in sanctions or other penalties that would harm our business, as well as lost confidence from investors, and the trading price of our common stock may decline.

Ensuring that we have adequate internal financial and accounting controls and procedures in place so that we can produce accurate financial statements on a timely basis is a costly and time-consuming effort that needs to be reevaluated frequently. Our internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements in accordance with generally accepted accounting principles, or GAAP, in the United States.

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After the completion of this offering, we will be subject to the reporting requirements of the Exchange Act, the Sarbanes-Oxley Act and the rules and regulations of Nasdaq. The Sarbanes-Oxley Act requires, among other things, that we maintain effective disclosure controls and procedures and internal controls over financial reporting. Commencing with our fiscal year ending the year after this offering is completed, we must perform system and process design evaluation and testing of the effectiveness of our internal controls over financial reporting to allow management to report on the effectiveness of our internal controls over financial reporting in our Annual Report on Form 10-K for that year, as required by Section 404. To achieve compliance with Section 404 within the prescribed period, in connection with this offering, we intend to begin the process of documenting, reviewing and improving our internal controls and procedures, which is both costly and challenging. In this regard, we will need to continue to dedicate internal resources, including through hiring additional financial and accounting personnel, potentially engage outside consultants and adopt a detailed work plan to assess and document the adequacy of internal control over financial reporting, continue steps to improve control processes as appropriate, validate through testing that controls are functioning as documented and implement a continuous reporting and improvement process for internal control over financial reporting. This will require that we incur substantial additional professional fees and internal costs to expand our accounting and finance functions and that we expend significant management efforts. Prior to this offering, we have never been required to test our internal controls within a specified period and, as a result, we may experience difficulty in meeting these reporting requirements in a timely manner.

If we are not able to comply with the requirements of Section 404 in a timely manner, or if we are unable to maintain proper and effective internal controls over financial reporting, we may not be able to produce timely and accurate financial statements. In addition, any testing by us conducted in connection with Section 404, or any subsequent testing by our independent registered public accounting firm, may reveal deficiencies in our internal controls over financial reporting that are deemed to be material weaknesses or that may require prospective or retroactive changes to our financial statements or identify other areas for further attention or improvement. If that were to happen, our investors could lose confidence in our reported financial information, the market price of our stock could decline and we could be subject to sanctions or investigations by the SEC or other regulatory authorities, including equivalent foreign authorities.

We will be required to disclose changes made in our internal controls and procedures on a quarterly basis and our management will be required to assess the effectiveness of these controls annually. However, for as long as we are an emerging growth company or a non-accelerated filer, our independent registered public accounting firm will not be required to attest to the effectiveness of our internal controls over financial reporting pursuant to Section 404(b) of the Sarbanes-Oxley Act. We could be an emerging growth company for up to five years following completion of this initial public offering. An independent assessment of the effectiveness of our internal controls over financial reporting could detect problems that our management’s assessment might not. Undetected material weaknesses in our internal controls over financial reporting could lead to restatements of our financial statements and require us to incur the expense of remediation.

Our executive officers, directors, principal stockholders and their affiliates will continue to exercise significant influence over our company after this offering, which will limit your ability to influence corporate matters and could delay or prevent a change in corporate control.

Immediately following the completion of this offering, and disregarding any shares of common stock that they purchase in this offering, the existing holdings of our executive officers, directors, principal stockholders and their affiliates will represent beneficial ownership, in the aggregate, of approximately % of our outstanding common stock, assuming no exercise of the underwriters’ option to acquire additional common stock in this offering and assuming we issue the number of shares of common stock as set forth on the cover page of this prospectus. In addition, five of our directors are affiliated with our principal stockholders. As a result, these stockholders, if they act together, will be able to influence our management and affairs and the outcome of matters submitted to our stockholders for approval, including the election of directors and any merger, consolidation or sale of all or substantially all of our assets. These stockholders acquired their shares of common stock for substantially less than the price of the shares of common stock being acquired in this offering, and these stockholders may have interests with respect to their common stock that are different from those of investors in this offering. The concentration of voting power among these stockholders may have an adverse effect on the price of our common stock. In addition, this concentration of ownership might adversely affect the market price of our common stock by:

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delaying, deferring or preventing a change of control of us;

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impeding a merger, consolidation, takeover or other business combination involving us; or

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discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of us.

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See the section of this prospectus titled “Principal Stockholders” for more information regarding the ownership of our outstanding common stock by our executive officers, directors, principal stockholders and their affiliates.

We will incur increased costs as a result of operating as a public company, and our management will be required to devote substantial time to new compliance initiatives.

As a public company, we will incur significant legal, accounting and other expenses that we did not incur as a private company. We will be subject to the reporting requirements of the Exchange Act, which will require, among other things, that we file with the SEC annual, quarterly and current reports with respect to our business and financial condition. In addition, the Sarbanes-Oxley Act, as well as rules subsequently adopted by the SEC and Nasdaq to implement provisions of the Sarbanes-Oxley Act, imposes significant requirements on public companies, including requiring establishment and maintenance of effective disclosure and financial controls and changes in corporate governance practices. Further, in July 2010, the Dodd-Frank Wall Street Reform and Consumer Protection Act, or the Dodd-Frank Act, was enacted. There are significant corporate governance and executive compensation related provisions in the Dodd-Frank Act that require the SEC to adopt additional rules and regulations in these areas, such as “say on pay” and proxy access. Emerging growth companies may implement many of these requirements over a longer period and up to five years from the pricing of this offering. We intend to take advantage of these extended transition periods, but cannot guarantee that we will not be required to implement these requirements sooner than budgeted or planned and thereby incur unexpected expenses. Stockholder activism, the current political environment and the current high level of government intervention and regulatory reform may lead to substantial new regulations and disclosure obligations, which may lead to additional compliance costs and impact the manner in which we operate our business in ways we cannot currently anticipate.

We expect the rules and regulations applicable to public companies to substantially increase our legal and financial compliance costs and to make some activities more time-consuming and costly. If these requirements divert the attention of our management and personnel from other business concerns, they could have a material adverse effect on our business, financial condition and results of operations. The increased costs will decrease our net income or increase our net loss and may require us to reduce costs in other areas of our business or increase the prices of our products or services. For example, we expect these rules and regulations to make it more difficult and more expensive for us to obtain director and officer liability insurance and we may be required to incur substantial costs to maintain the same or similar coverage. We cannot predict or estimate the amount or timing of additional costs we may incur to respond to these requirements. The impact of these requirements could also make it more difficult for us to attract and retain qualified persons to serve on our board of directors, our board committees or as executive officers.

Anti-takeover provisions under our charter documents and Delaware law could delay or prevent a change of control, which could limit the market price of our common stock and may prevent or frustrate attempts by our stockholders to replace or remove our current management.

Our sixth amended and restated certificate of incorporation, which will become effective immediately prior to the closing of this offering, and the effectiveness of our fourth amended and restated bylaws, which will become effective upon the effectiveness of the registration statement of which this prospectus is a part, will contain provisions that could delay or prevent a change of control of our company or changes in our board of directors that our stockholders might consider favorable. Some of these provisions include:

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a board of directors divided into three classes serving staggered three-year terms, such that not all members of the board will be elected at one time;

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a prohibition on stockholder action through written consent, which requires that all stockholder actions be taken at a meeting of our stockholders;

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a requirement that special meetings of stockholders be called only by the board of directors acting pursuant to a resolution approved by the affirmative vote of a majority of the directors then in office;

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advance notice requirements for stockholder proposals and nominations for election to our board of directors;

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a requirement that no member of our board of directors may be removed from office by our stockholders except for cause and, in addition to any other vote required by law, upon the approval of not less than two-thirds of all outstanding shares of our voting stock then entitled to vote in the election of directors;

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a requirement of approval of not less than two-thirds of all outstanding shares of our voting stock to amend any bylaws by stockholder action or to amend specific provisions of our certificate of incorporation; and

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the authority of the board of directors to issue preferred stock on terms determined by the board of directors without stockholder approval and which preferred stock may include rights superior to the rights of the holders of common stock.

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In addition, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law, or DGCL, which may prohibit certain business combinations with stockholders owning 15% or more of our outstanding voting stock. These antitakeover provisions and other provisions in our fifth amended and restated certificate of incorporation and amended and restated bylaws could make it more difficult for stockholders or potential acquirers to obtain control of our board of directors or initiate actions that are opposed by the then-current board of directors and could also delay or impede a merger, tender offer or proxy contest involving our company. These provisions could also discourage proxy contests and make it more difficult for you and other stockholders to elect directors of your choosing or cause us to take other corporate actions you desire. Any delay or prevention of a change of control transaction or changes in our board of directors could cause the market price of our common stock to decline.

Our amended and restated bylaws that will become effective upon the effectiveness of our registration statement designate specific courts as the exclusive forum for certain litigation that may be initiated by our stockholders, which could limit stockholders’ ability to obtain a favorable judicial forum for disputes with us.

Pursuant to our amended and restated bylaws that will become effective upon the effectiveness of our registration statement, unless we consent in writing to the selection of an alternative forum, the Court of Chancery of the State of Delaware is the sole and exclusive forum for any state law claims for (i) any derivative action or proceeding brought on our behalf; (ii) any action asserting a claim of or based on a breach of a fiduciary duty owed by any director, officer or other employee of ours to us or our stockholders; (iii) any action asserting a claim pursuant to any provision of the DGCL, our amended and restated certificate of incorporation or our amended and restated bylaws; or (iv) any action asserting a claim governed by the internal affairs doctrine, or the Delaware Forum Provision. The Delaware Forum Provision will not apply to any causes of action arising under the Securities Act or the Exchange Act. Our amended and restated bylaws further provide that unless we consent in writing to the selection of an alternative forum, the United States District Court for the District of Massachusetts shall be the sole and exclusive forum for resolving any complaint asserting a cause of action arising under the Securities Act, or the Federal Forum Provision. In addition, our amended and restated bylaws provide that any person or entity purchasing or otherwise acquiring any interest in shares of our capital stock is deemed to have notice of and consented to the Delaware Forum Provision and the Federal Forum Provision; provided, however, that stockholders cannot and will not be deemed to have waived our compliance with the federal securities laws and the rules and regulations thereunder.

We recognize that the Delaware Forum Provision and the Federal Forum Provision in our amended and restated bylaws may impose additional litigation costs on stockholders in pursuing any such claims, particularly if the stockholders do not reside in or near the State of Delaware or the Commonwealth of Massachusetts, as applicable. Additionally, the forum selection clauses in our amended and restated bylaws may limit our stockholders’ ability to bring a claim in a judicial forum that they find favorable for disputes with us or our directors, officers or employees, which may discourage the filing of lawsuits against us and our directors, officers and employees, even though an action, if successful, might benefit our stockholders. In addition, while the Delaware Supreme Court ruled in March 2020 that federal forum selection provisions purporting to require claims under the Securities Act be brought in federal court are “facially valid” under Delaware law, there is uncertainty as to whether other courts will enforce our Federal Forum Provision. If the Federal Forum Provision is found to be unenforceable, we may incur additional costs associated with resolving such matters. The Federal Forum Provision may also impose additional litigation costs on stockholders who assert that the provision is not enforceable or invalid. The Court of Chancery of the State of Delaware and the United States District Court for the District of Massachusetts may also reach different judgments or results than would other courts, including courts where a stockholder considering an action may be located or would otherwise choose to bring the action, and such judgments may be more or less favorable to us than our stockholders.

Sales of a substantial number of shares of our common stock in the public market could cause our stock price to fall.

If our existing stockholders sell, or indicate an intention to sell, substantial amounts of our common stock in the public market after the lock-up and other legal restrictions on resale discussed in this prospectus lapse, the market price of our common stock could decline. Based upon the number of shares of common stock, on

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an as-converted basis, outstanding as of June 30, 2026, upon the completion of this offering, we will have outstanding a total of shares of common stock, assuming no exercise of the underwriters’ option to purchase an additional shares. Of these shares, as of the date of this prospectus, approximately shares of our common stock, plus any shares sold upon exercise of the underwriters’ option to purchase additional shares, will be freely tradable, without restriction, in the public market immediately following this offering, assuming that current stockholders do not purchase shares in this offering. The representatives of the underwriters, however, may, in their sole discretion, permit our officers, directors and other stockholders who are subject to these lock-up agreements to sell shares prior to the expiration of the lock-up agreements.

The lock-up agreements pertaining to this offering will expire 180 days from the date of this prospectus. After the lock-up agreements expire, based upon the number of shares of common stock, on an as-converted basis, outstanding as of June 30, 2026, up to an additional shares of common stock will be eligible for sale in the public market, approximately   % of which shares are held by directors, executive officers and other affiliates and will be subject to certain limitations of Rule 144 under the Securities Act.

Upon completion of this offering, shares of common stock that are either subject to outstanding options, reserved for future issuance under our equity incentive plans will become eligible for sale in the public market to the extent permitted by the provisions of various vesting schedules, the lock-up agreements and Rule 144 and Rule 701 under the Securities Act. If these additional shares of common stock are sold, or if it is perceived that they will be sold, in the public market, the market price of our common stock could decline.

After this offering, the holders of approximately shares of our common stock will be entitled to rights with respect to the registration of their shares under the Securities Act, subject to the lock-up agreements described above. Registration of these shares under the Securities Act would result in the shares becoming freely tradable without restriction under the Securities Act, except for shares purchased by affiliates. Any sales of securities by these stockholders could have a material adverse effect on the market for our common stock.

We have broad discretion in how we use the proceeds of this offering and may not use these proceeds effectively, which could affect our results of operations and cause our stock price to decline.

We will have considerable discretion in the application of the net proceeds of this offering, including for any of the purposes described in the section of this prospectus titled “Use of Proceeds,” and you will not have the opportunity as part of your investment decision to assess whether the net proceeds are being used appropriately. As a result, investors will be relying upon management’s judgment with only limited information about our specific intentions for the use of the balance of the net proceeds of this offering. We may use the net proceeds for purposes that do not yield a significant return or any return at all for our stockholders. In addition, pending their use, we may invest the net proceeds from this offering in a manner that does not produce income or that loses value.

Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud.

Upon completion of this offering, we will become subject to certain reporting requirements of the Exchange Act. Our disclosure controls and procedures are designed to reasonably assure that information required to be disclosed by us in reports we file or submit under the Exchange Act is accumulated and communicated to management, recorded, processed, summarized and reported within the time periods specified in the rules and forms of the SEC. We believe that any disclosure controls and procedures or internal controls and procedures, no matter how well conceived and operated, can provide only reasonable, not absolute, assurance that the objectives of the control system are met. These inherent limitations include the realities that judgments in decision-making can be faulty, and that breakdowns can occur because of simple error or mistake. Additionally, controls can be circumvented by the individual acts of some persons, by collusion of two or more people or by an unauthorized override of the controls. Accordingly, because of the inherent limitations in our control system, misstatements or insufficient disclosures due to error or fraud may occur and not be detected.

We are an emerging growth company and a smaller reporting company, and we cannot be certain if the reduced reporting requirements applicable to emerging growth companies and smaller reporting companies will make our common stock less attractive to investors.

We are an emerging growth company, as defined in the JOBS Act, enacted in April 2012. For as long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies, including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in this prospectus and our periodic

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reports and proxy statements, and exemptions from the requirements of holding nonbinding advisory votes on executive compensation and stockholder approval of any golden parachute payments not previously approved. We could be an emerging growth company for up to five years following the year in which we complete this offering, although circumstances could cause us to lose that status earlier. We will remain an emerging growth company until the earlier of (i) the last day of the fiscal year (a) following the fifth anniversary of the closing of this offering, (b) in which we have total annual gross revenue of at least $1.235 billion, or (c) in which we are deemed to be a large accelerated filer, which requires the market value of our common stock that is held by non-affiliates to exceed $700 million as of the prior June 30th, and (ii) the date on which we have issued more than $1 billion in non-convertible debt during the prior three-year period.

Under the JOBS Act, emerging growth companies can also delay adopting new or revised accounting standards until such time as those standards apply to private companies. We have elected to not “opt out” of this exemption from complying with new or revised accounting standards and, therefore, we will adopt new or revised accounting standards at the time private companies adopt the new or revised accounting standard and will do so until such time that we either (i) irrevocably elect to “opt out” of such extended transition period or (ii) no longer qualify as an emerging growth company.

Even after we no longer qualify as an emerging growth company, we may still qualify as a “smaller reporting company,” which would allow us to continue to take advantage of many of the same exemptions from disclosure requirements, including not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act and reduced disclosure obligations regarding executive compensation in this prospectus and our periodic reports and proxy statements. We cannot predict if investors will find our common stock less attractive because we may rely on these exemptions. If some investors find our common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be more volatile.

Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will be your sole source of gain.

We have never declared or paid cash dividends on our capital stock. We currently intend to retain all of our future earnings, if any, to finance the growth and development of our business. As a result, capital appreciation, if any, of our common stock will be your sole source of gain for the foreseeable future.

After the completion of this offering, we may be at an increased risk of securities class action litigation.

Historically, securities class action litigation has often been brought against a company following a decline in the market price of its securities. This risk is especially relevant for us because biotechnology and pharmaceutical companies have experienced significant stock price volatility in recent years. If we were to be sued, it could result in substantial costs and a diversion of management’s attention and resources, which could harm our business.

If our estimates or judgments relating to our critical accounting policies are based on assumptions that change or prove to be incorrect, our operating results could fall below our publicly announced guidance or the expectations of securities analysts and investors, resulting in a decline in the market price of our common stock.

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in our consolidated financial statements and accompanying notes. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets, liabilities, equity, revenue and expenses that are not readily apparent from other sources. If our assumptions change or if actual circumstances differ from our assumptions, our operating results may be adversely affected and could fall below our publicly announced guidance or the expectations of securities analysts and investors, resulting in a decline in the market price of our common stock.

General Risk Factors

Adverse developments affecting the financial services industry, such as actual events or concerns involving liquidity, defaults or non-performance by financial institutions or transactional counterparties, could adversely affect our current and projected business operations and our financial condition and results of operations.

Adverse developments that affect financial institutions, such as events involving liquidity that are rumored or actual, have in the past and may in the future lead to market-wide liquidity problems. Although we assess our banking relationships as we believe necessary or appropriate, our access to funding sources and other credit arrangements in amounts adequate to finance or capitalize our current and projected future business operations

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could be significantly impaired by factors that affect us, the financial institutions with which we have credit agreements or arrangements directly, or the financial services industry or economy in general. These factors could include, among others, events such as liquidity constraints or failures, the ability to perform obligations under various types of financial, credit or liquidity agreements or arrangements, disruptions or instability in the financial services industry or financial markets or concerns or negative expectations about the prospects for companies in the financial services industry. While it is not possible at this time to predict the extent of the impact that high market volatility and instability of the financial services industry could have on economic activity and our business in particular, the failure of other banks and financial institutions and the measures taken by governments, businesses and other organizations in response to these events could adversely impact our business, financial condition and results of operations.

Public health crises, such as a pandemic, epidemic or outbreak of other highly infectious or contagious diseases, could seriously harm our research, development and potential future commercialization efforts, increase our costs and expenses and have a material adverse effect on our business, financial condition and results of operations.

Public health crises, such as a pandemic, epidemic or outbreak of other highly infectious or contagious diseases, could adversely impact our business, the business operations of third parties on whom we rely and our ongoing or planned research and development activities. Additionally, timely enrollment in our planned clinical trials is dependent upon clinical trial sites which may be adversely affected by global health concerns. Public health crises could result in increased adverse events and deaths in our clinical trials. Some factors from public health crises that could delay or otherwise adversely affect enrollment in the clinical trials of our product candidates, as well as our business generally, include:

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the potential diversion of healthcare resources away from the conduct of clinical trials to focus on public health crises, including the attention of physicians serving as our clinical trial investigators, hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our prospective clinical trials and the need for drugs and other supplies that clinical trial sites must have on hand to conduct our clinical trials to be used to address such public health crises;

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limitations on travel that could interrupt key trial and business activities, such as clinical trial site initiations and monitoring, domestic and international travel by employees, contractors or patients to clinical trial sites, including any government-imposed travel restrictions or quarantines that will impact the ability or willingness of patients, employees or contractors to travel to our clinical trial sites or secure visas or entry permissions, a loss of face-to-face meetings and other interactions with potential partners, any of which could delay or adversely impact the conduct or progress of our prospective clinical trials;

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interruption in global shipping affecting the transport of clinical trial materials, such as patient samples, investigational drug product and conditioning drugs and other supplies used in our prospective clinical trials;

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interruptions in operations at our third-party manufacturers, which could result in delays or disruptions in the supply of our current product candidates and any future product candidates; and

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business disruptions caused by potential workplace, laboratory and office closures and an increased reliance on employees working from home, disruptions to or delays in ongoing laboratory experiments and operations, product manufacturing and supply, staffing shortages, travel limitations or mass transit disruptions, any of which could adversely impact our business operations or delay necessary interactions with local regulators, ethics committees and other important agencies and contractors.

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Any of these factors, and other factors related to any such disruptions that are unforeseen, could have a material adverse effect on our business and our results of operations and financial condition. Further, uncertainty around these and related issues could lead to adverse effects on the economy of the United States and other economies, which could impact our ability to raise the necessary capital needed to develop and commercialize our product candidates.

We or the third parties upon whom we depend may be adversely affected by natural disasters and our business continuity and disaster recovery plans may not adequately protect us from a serious disaster.

Natural disasters could severely disrupt our operations and have a material adverse effect on our business, results of operations, financial condition and prospects. If a natural disaster, power outage or other event occurred that prevented us from using all or a significant portion of our headquarters, that damaged critical infrastructure, such as the manufacturing facilities on which we rely, or that otherwise disrupted operations, it may be difficult or, in certain cases, impossible for us to continue our business for a substantial period of time. The disaster recovery and business continuity plans we have in place may prove inadequate in the event

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of a serious disaster or similar event. We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have a material adverse effect on our business. For example, following Hurricane Maria, shortages in production and delays in a number of medical supplies produced in Puerto Rico resulted, and any similar interruption due to a natural disaster affecting us or any of our third-party manufacturers could materially delay our operations.

Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price.

As widely reported, global credit and financial markets have experienced extreme volatility and disruptions in the past several years, declines in consumer confidence, concerns about declines in economic growth, increases in the rate of inflation and uncertainty about economic stability, including in connection with actions undertaken by the U.S. Federal Reserve Board to address inflation and geopolitical conflicts. There can be no assurance that further volatility in credit and financial markets and confidence in economic conditions will not occur.

Regional or single-source dependencies may in some cases accentuate these risks. For example, the pharmaceutical industry in general, and in some instances we or our collaborators or other third parties on which we or they rely, depend on China-based suppliers or service providers for certain raw materials, products and services or other activities. Our ability or the ability of our collaborators or such other third parties to continue to engage these China-based suppliers or service providers for certain nonclinical research programs and clinical development programs could be restricted due to geopolitical developments between the United States and China, including as a result of the escalation of tariffs.

Our general business strategy may be adversely affected by any such economic downturn, volatile business environment or continued unpredictable and unstable market conditions. If the current equity and credit markets continue to be volatile it may make any necessary debt or equity financing more difficult, more costly, and more dilutive. Furthermore, our stock price may decline due in part to the volatility of the stock market and a general economic downturn.

Failure to secure any necessary financing in a timely manner and on favorable terms could have a material adverse effect on our growth strategy, financial performance and stock price and could require us to delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions. In addition, there is a risk that one or more of our current service providers, manufacturers and other partners may not survive these difficult economic times, which could directly affect our ability to attain our operating goals on schedule and on budget.

Our ability to utilize our net operating loss carryforwards and certain other tax attributes to offset future taxable income may be subject to certain limitations.

As of December 31, 2025, we had federal net operating loss, or NOL, carryforwards of $17.9 million and no federal tax credits. The Company’s federal net operating loss carryforwards have an indefinite life. The Company also had gross state NOLs of $13.7 million that begin to expire in 2042. As of December 31, 2025, we had gross foreign net operating loss carryforwards of $73.1 million that have an indefinite life. These net operating loss and tax credit carryforwards could expire unused and be unavailable to offset future income tax liabilities. In addition, in general, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, or the Code, a corporation that undergoes an “ownership change” is subject to limitations on its ability to utilize its pre-change NOLs or tax credits to offset future taxable income or taxes. For these purposes, an ownership change generally occurs where the aggregate stock ownership of one or more stockholders or groups of stockholders who owns at least 5% of a corporation’s stock increases its ownership by more than 50 percentage points over its lowest ownership percentage within a specified testing period.

Our existing NOLs or credits may be subject to limitations arising from previous ownership changes, and our ability to utilize NOLs or credits could be further limited by Sections 382 and 383 of the Code. In addition, this offering and future changes in our stock ownership, some of which may be outside of our control, could result in an ownership change under Sections 382 and 383 of the Code. Our use of NOLs or credits may also be limited under state law. Accordingly, we may not be able to utilize a material portion of our NOLs or credits.

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Furthermore, our ability to utilize our NOLs or credits is conditioned upon our attaining profitability and generating U.S. federal and state taxable income. As described above under “Risk Factors-Risks Related to Our Financial Position and Capital Needs,” we have incurred significant net losses since our inception and anticipate that we will continue to incur significant losses for the foreseeable future. Therefore, we do not know whether or when we will generate the U.S. federal or state taxable income necessary to utilize our NOL or credit carryforwards.

Changes in tax law may adversely affect us or our investors.

The rules dealing with U.S. federal, state and local income taxation are constantly under review by persons involved in the legislative process and by the Internal Revenue Service, or IRS, and the U.S. Treasury Department. Changes to tax laws (which changes may have retroactive application) could adversely affect us or holders of our common stock. In recent years, many changes have been made and changes are likely to continue to occur in the future.

For example, the One Big Beautiful Bill Act, or the OBBBA, was signed into law on July 4, 2025 and made significant changes to U.S. federal tax law. Under Section 174 of the Code, for instance, in taxable years beginning after December 31, 2021, expenses that are incurred for research and development performed outside the United States will be capitalized and amortized, which may have had an adverse effect on our cash flow. The OBBBA provides that for taxable years beginning after December 31, 2024, expenses that are incurred for research and development performed in the United States may, at the taxpayer’s election, be immediately deducted or capitalized and amortized under Section 174 of the Code. In addition, the OBBBA provides that, for taxable years beginning after December 31, 2021 and before January 1, 2025, certain eligible taxpayers generally may elect to retroactively deduct expenses for research and development performed in the United States in such taxable years by filing amended tax returns for such taxable years and all other taxpayers that are not eligible to make such an election and that amortized expenses for research and development performed in the United States in such taxable years generally may elect to accelerate and deduct the remaining unamortized amounts of such research and development expenses (i) in the first taxable year beginning after December 31, 2024, or (ii) ratably over the two-taxable year period beginning with the first taxable year beginning after December 31, 2024.

It cannot be predicted whether, when, in what form, or with what effective dates, new tax laws may be enacted, or regulations and rulings may be enacted, promulgated or issued under existing or new tax laws, which could result in an increase in our or our stockholders’ tax liability or require changes in the manner in which we operate in order to minimize or mitigate any adverse effects of changes in tax law or in the interpretation thereof.

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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS

This prospectus, including the sections titled “Prospectus Summary,” “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations,” and “Business,” contains express or implied forward-looking statements that are based on our management’s belief and assumptions and on information currently available to our management. These statements relate to future events or our future operational or financial performance, and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. Forward-looking statements in this prospectus include, but are not limited to, statements about:

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the initiation, timing, progress and results of our ongoing and future clinical trials;

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our ability to successfully complete our clinical trials at the pace we project, and to replicate positive results from earlier nonclinical studies;

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our ability to advance ruvonoflast, trabzanoflast and any future product candidates through applicable regulatory approval processes;

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our ability to demonstrate that our current and any future product candidates are safe and effective for their proposed indications and our expectations around their beneficial characteristics and therapeutic effects;

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our ability to establish ruvonoflast and trabzanoflast as a new standard of care for inhibiting the activation of NLRP3;

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our ability to maximize the potential of our product candidates by advancing them through a planned registrational Phase 3 trial as a treatment for addressing NLRP3-driven diseases;

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the prevalence of the unmet needs in cardiometabolic and neuroinflammatory disease and the size of the market opportunity;

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our estimates of the number of patients that we will enroll in our clinical trials;

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the timing of announcement of interim and final results from clinical trials;

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our ability to meet future regulatory standards with respect to ruvonoflast and trabzanoflast or any future product candidates, if approved;

​

▪

the implementation of our business model and strategic plans, including our ability to build a preeminent independent biopharmaceutical company;

​

▪

our ability to identify, acquire and advance additional development candidates targeting unmet and under-met medical needs in cardiometabolic and neuroinflammatory diseases;

​

▪

our ability to rely on third party manufacturers, and successfully manufacture our current and any future product candidates for clinical trials and on a larger scale for commercial use, if approved;

​

▪

our ability to realize the benefits of business development activities to expand our pipeline;

​

▪

our ability to maintain, expand and protect our intellectual property;

​

▪

developments relating to our competitors and our industry;

​

▪

existing regulations and regulatory developments in the United States and other jurisdictions;

​

▪

general economic, industry, and market conditions, including rising interest rates and inflation;

​

▪

our ability to attract, hire, and retain our key personnel and additional qualified personnel;

​

▪

our expectations regarding the period during which we will qualify as an emerging growth company under the JOBS Act or a smaller reporting company;

​

▪

our anticipated use of our existing cash and cash equivalents and the net proceeds from this offering;

​

▪

our estimates regarding expenses, future revenue, capital requirements and needs for additional financing and our ability to continue as a going concern; and

​

▪

other risks and uncertainties, including those listed under the caption “Risk Factors.”

​

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In some cases, forward-looking statements can be identified by terminology such as “may,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue,” or the negative of these terms or other comparable terminology. These statements are only predictions. You should not place undue reliance on forward-looking statements because they involve known and unknown risks, uncertainties and other factors, which are, in some cases, beyond our control and which could materially affect results. Factors that may cause actual results to differ materially from those implied or projected by forward-looking statements include, among other things, those listed under the section titled “Risk Factors” and elsewhere in this prospectus. If one or more of these risks or uncertainties occur, or if our underlying assumptions prove to be incorrect, actual events or results may vary significantly from those implied or projected by the forward-looking statements. No forward-looking statement is a guarantee of future performance. You should read this prospectus and the documents that we reference in this prospectus and have filed with the SEC as exhibits to the registration statement, of which this prospectus is a part, completely and with the understanding that our actual future results may be materially different from any future results expressed or implied by these forward-looking statements.

While we may elect to update these forward-looking statements at some point in the future, we have no current intention of doing so except to the extent required by applicable law. You should therefore not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this prospectus. You should, however, review the factors and risks and other information we describe in the reports we will file from time to time with the SEC after the date of this prospectus.

In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this prospectus, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to unduly rely upon these statements.

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MARKET AND INDUSTRY AND OTHER DATA

Unless otherwise indicated, market and industry data contained in this prospectus, including potential market opportunities, are based on our management’s estimates and research, as well as information obtained from industry and general publications, scientific literature, surveys and reports, as well as research and studies conducted by third parties. Although we believe that the information from these third-party publications, research and studies included in this prospectus is reliable, and we are responsible for the accuracy of such information, neither we nor the underwriters have independently verified the accuracy or completeness of this information. Management’s estimates are derived from publicly available information, their knowledge of our industry and their assumptions based on such information and knowledge, which we believe to be reasonable. This data involves a number of assumptions and limitations and the industry in which we operate is subject to a high degree of uncertainty and risk due to a variety of factors, including those described in the sections titled “Risk Factors” and “Special Note Regarding Forward-Looking Statements.” These and other factors could cause our future performance to differ materially from our assumptions and estimates.

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USE OF PROCEEDS

We estimate that our net proceeds from the sale of shares of our common stock in this offering will be approximately $     million, or $     million if the underwriters exercise their option to purchase additional shares in full, assuming an initial public offering price of $     per share, the midpoint of the estimated offering price range set forth on the cover page of this prospectus, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

Each $1.00 increase (decrease) in the assumed initial public offering price of $     per share, the midpoint of the estimated offering price range set forth on the cover page of this prospectus, would increase (decrease) our net proceeds from this offering by $     million, assuming the number of shares offered by us, as set forth on the cover page of this prospectus, remains the same, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Similarly, each 1.0 million share increase (decrease) in the number of shares offered by us, as set forth on the cover page of this prospectus, would increase (decrease) our net proceeds from this offering by $     million, assuming no change in the assumed initial public offering price of $     per share, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

As of June 30, 2026, we had cash and cash equivalents of $3.9 million. We currently intend to use the net proceeds from this offering, together with our existing cash and cash equivalents, plus the proceeds from the issuance of the Series D4 Stock and the Term Loan, for the following:

▪

approximately $     million to advance ruvonoflast through our proposed Phase 3 clinical trial in PAD;

​

▪

approximately $     million to invest in commercial readiness for ruvonoflast, if approved;

​

▪

approximately $     million to fund continued development for our earlier stage pipeline; and

​

▪

the remainder for working capital and general corporate purposes.

​

Based on our current plans, we believe that our existing cash and cash equivalents, together with the net proceeds from this offering, will be sufficient to enable us to fund our operating expenses and capital expenditure requirements at least through                . We have based this estimate on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect. We do not expect that the anticipated net proceeds from this offering, together with our existing cash and cash equivalents, will be sufficient for us to fund ruvonoflast, our earlier stage pipeline or any future product candidates through regulatory approval, and we will need to raise substantial additional capital to complete the development and commercialization. See “Risk Factors — Risks Related to Our Financial Position and Capital Needs — As part of our ongoing business, we will need to raise substantial additional funding beyond our current capital. If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, scale back or discontinue some of our product candidate development programs or future commercialization efforts.” We plan to continue to fund our operations through proceeds from public or private equity offerings, or other strategic transactions. We do not have any committed external source of funds.

Our expected use of the net proceeds from this offering represents our intentions based upon our current plans and business conditions. As of the date of this prospectus, we cannot predict with certainty all of the particular uses for the net proceeds to be received upon the completion of this offering or the amounts that we will actually spend on the uses set forth above and we expect that we will require additional funds in order to fully accomplish the specified uses of the proceeds of this offering.​

Pending our use of the net proceeds from this offering, we intend to invest the net proceeds in a variety of capital preservation instruments, including direct or guaranteed obligations of the U.S. government, money market fund shares, and bank deposits that are insured by the Federal Deposit Insurance Corporation. We cannot predict whether the proceeds invested will yield a favorable return. Our management will retain broad discretion in the application of the net proceeds we receive from our initial public offering, and investors will be relying on the judgment of our management regarding the application of the net proceeds.

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DIVIDEND POLICY

We have never declared or paid cash dividends on our capital stock. We do not intend to pay cash dividends to our stockholders in the foreseeable future. We currently intend to retain all available funds and any future earnings to fund the growth and development of our business. Any future determination to declare dividends will be made at the discretion of our board of directors and will depend on then-existing conditions, including our financial condition, operating results, contractual restrictions, capital requirements, general business conditions and other factors that our board of directors may deem relevant. Investors should not purchase our common stock with the expectation of receiving cash dividends.

In addition, our ability to pay cash dividends on our capital stock in the future may be limited by the terms of any future debt or preferred securities we issue or any credit facilities we enter into.

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CAPITALIZATION

The following table sets forth our cash and cash equivalents and our capitalization as of June 30, 2026:

▪

on an actual basis;

​

▪

on a pro forma basis gives effect to the (i) entry into the Term Loan in July 2026 and the borrowing of $15.0 million thereunder, (ii) issuance and sale of an aggregate of 16,389,452 shares of our Series D4 Stock on July 15, 2026, for net proceeds of approximately $15.0 million, (iii) automatic conversion of all outstanding shares of our convertible preferred stock, in the aggregate, into 144,432,177 shares of common stock prior to the completion of this offering, (iv) repurchase of 1,400,998 reverse vesting RSAs, (v) stock-based compensation expense from the accelerated vesting of outstanding time-based equity awards pursuant to the proposed amendment to such awards that would provide for accelerated vesting immediately prior to our change in control and (vi) filing and effectiveness of our sixth amended and restated certificate of incorporation, which will occur immediately prior to the completion of this offering; and

​

▪

on a pro forma as adjusted basis to give further effect to (i) the pro forma adjustments set forth above and (ii) the issuance and sale of     shares of common stock in this offering at an assumed initial public offering price of $     per share, which is the midpoint of the estimated offering price range set forth on the cover page of this prospectus, after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.​

​

The pro forma and pro forma as adjusted information below is illustrative only, and our capitalization following the completion of this offering will be adjusted based on the actual initial public offering price and other terms of this offering determined at pricing. You should read the information in this table together with our consolidated financial statements and the related notes, “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and other financial information included elsewhere in this prospectus.

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​

(1)

Each $1.00 increase (decrease) in the assumed initial public offering price of $     per share, which is the midpoint of the estimated offering price range set forth on the cover page of this prospectus, would increase (decrease) the pro forma as adjusted amount of each of cash and cash equivalents, total stockholders’ equity and total capitalization by $     million, assuming that the number of shares offered by us, as set forth on the cover page of this prospectus, remains the same, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Each increase (decrease) of 1.0 million shares in the number of shares offered by us, as set forth on the cover page of this prospectus, would increase (decrease) the pro forma as adjusted amount of each of cash and cash equivalents, total stockholders’ equity and total capitalization by $     million, assuming no change in the assumed initial public offering price per share and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

​

The number of shares of our common stock to be outstanding after this offering is based on 146,803,003 shares of common stock outstanding as of June 30, 2026, which includes 40,066 shares of restricted stock and after giving effect to (i) the issuance of our Series D4 convertible preferred stock, or the Series D4 Stock, on a pro forma basis subsequent to June 30, 2026, (ii) the automatic conversion of all outstanding shares of our convertible preferred stock as of June 30, 2026 into an aggregate of 144,432,177 shares of common stock immediately prior to the completion of this offering, and (iii) the repurchase of 1,400,998 reverse vesting RSAs upon completion of this offering, and excludes:

▪

27,245,715 shares of common stock issuable upon the exercise of outstanding stock options as of June 30, 2026 under our 2021 Plan, with a weighted-average exercise price of $0.21 per share, and 0 shares that are reserved for future issuance under the 2021 Plan as of June 30, 2026;

​

▪

1,988,176 shares of common stock issuable upon the exercise of stock options that were granted under our 2021 Plan subsequent to June 30, 2026 at a weighted-average exercise price of $0.99 per share;

​

▪

0 shares of common stock reserved for future issuance as of June 30, 2026 under the 2021 Plan, which was subsequently amended on September 3, 2026 to reserve an additional 2,000,000 shares for issuance, and which will cease to be available for issuance at the time that our 2026 Plan becomes effective;

​

▪

1,639,176 shares of common stock issuable upon the exercise of warrants issued subsequent to June 30, 2026, at an exercise price of $1.00 per share;

​

▪

     shares of our common stock reserved for future issuance under our 2026 Plan, which will become effective on the date immediately prior to the effectiveness of the registration statement of which this prospectus forms a part;

​

▪

     shares of common stock reserved for future issuance under our ESPP; and

​

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DILUTION

If you invest in our common stock in this offering, your ownership interest will be immediately diluted to the extent of the difference between the initial public offering price per share of our common stock and the pro forma as adjusted net tangible book value per share of our common stock immediately after this offering.

Our historical net tangible book value as of June 30, 2026 was a deficit of $201.2 million, or $(53.33) per share of our common stock. Our historical net tangible book value (deficit) is the amount of our total tangible assets less our total liabilities and the carrying values of our convertible preferred stock, which is not included within stockholders’ deficit. Our historical net tangible book value per share represents historical net tangible book value (deficit) divided by the number of shares of our common stock outstanding as of June 30, 2026.

Our pro forma net tangible book value as of June 30, 2026 was $15.1 million, or $0.10 per share of our common stock. Pro forma net tangible book value represents the amount of our total tangible assets less our total liabilities, assuming the (i) entry into the Term Loan in July 2026 and the borrowing of $15.0 million thereunder, (ii) issuance and sale of an aggregate of 16,389,452 shares of our Series D4 Stock on July 15, 2026, for net proceeds of approximately $15.0 million, (iii) automatic conversion of all outstanding shares of our convertible preferred stock, in the aggregate, into 144,432,177 shares of common stock prior to the completion of this offering, (iv) repurchase of 1,400,998 reverse vesting RSAs, and (v) filing and effectiveness of our sixth amended and restated certificate of incorporation, which will occur immediately prior to the completion of this offering. Pro forma net tangible book value per share represents pro forma net tangible book value divided by the total number of shares outstanding as of June 30, 2026, after giving effect to the pro forma adjustments described above.

After giving further effect to our issuance and sale of shares of our common stock in this offering at an assumed initial public offering price of $     per share, the midpoint of the price range set forth on the cover page of this prospectus, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us, our pro forma as adjusted net tangible book value as of June 30, 2026 would have been $     million, or $     per share. This amount represents an immediate increase in pro forma as adjusted net tangible book value per share of $     to our existing stockholders and immediate dilution of $     in pro forma as adjusted net tangible book value per share to new investors purchasing common stock in this offering.

Dilution per share to new investors is determined by subtracting pro forma as adjusted net tangible book value per share after this offering from the assumed initial public offering price per share paid by new investors. The following table illustrates this dilution on a per share basis (without giving effect to any exercise by the underwriters of their option to purchase additional shares):

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The dilution information discussed above is illustrative only and will change based on the actual initial public offering price and other terms of this offering determined at pricing.

Each $1.00 increase (decrease) in the assumed initial public offering price of $     per share, which is the midpoint of the price range set forth on the cover page of this prospectus, would increase (decrease) our pro forma as adjusted net tangible book value per share after this offering by $     and dilution per share to new investors purchasing common stock in this offering by $     , assuming that the number of shares offered by us, as set forth on the cover page of this prospectus, remains the same and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Similarly, each increase of 1.0 million shares in the number of shares offered by us, as set forth on the cover page of this prospectus, would increase our pro forma as adjusted net tangible book value per share after this offering by $     and decrease dilution per share to new investors purchasing common stock in this offering by $     , assuming no change in the assumed initial public offering price per share and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. Each decrease of 1.0 million shares in the number of shares offered by us, as set forth on the cover page of this prospectus, would decrease our pro forma as adjusted net tangible book value per share after this offering by $     and increase dilution per share to new investors purchasing common stock in this offering by $     , assuming no change in the assumed initial public offering price and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

If the underwriters exercise their option to purchase up to               additional shares of our common stock in full, our pro forma as adjusted net tangible book value per share after this offering would be $    per share, representing an immediate increase in pro forma as adjusted net tangible book value per share of $     per share to existing stockholders and immediate dilution in pro forma as adjusted net tangible book value per share of $     per share to new investors purchasing common stock in this offering, based on the assumed initial public offering price of $     per share, which is the midpoint of the estimated price range set forth on the cover page of this prospectus, and after deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us.

The following table summarizes on the pro forma as adjusted basis described above, as of June 30, 2026, the total number of shares of common stock purchased from us on an as converted basis to common stock, the total consideration paid or to be paid, and the average price per share paid or to be paid by existing stockholders and by new investors in this offering at an assumed initial public offering price of $     per share, which is the midpoint of the estimated price range set forth on the cover of this prospectus, before deducting estimated underwriting discounts and commissions and estimated offering expenses payable by us. New investors purchasing shares of our common stock in this offering will pay an average price per share substantially higher than our existing stockholders paid.

The table above assumes no exercise of the underwriters’ option to purchase additional shares in this offering. If the underwriters’ option to purchase additional shares of our common stock is exercised in full, the number of shares of our common stock held by existing stockholders would be reduced to     % of the total number of shares of our common stock outstanding after this offering, and the number of shares of common stock held by new investors purchasing common stock in this offering would be increased to     % of the total number of shares of our common stock outstanding after this offering.

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The number of shares of our common stock to be outstanding after this offering is based on 146,803,003 shares of common stock outstanding as of June 30, 2026, which includes 40,066 shares of restricted stock and after giving effect to (i) the issuance of our Series D4 convertible preferred stock, or the Series D4 Stock, on a pro forma basis subsequent to June 30, 2026, (ii) the automatic conversion of all outstanding shares of our convertible preferred stock as of June 30, 2026 into an aggregate of 144,432,177 shares of common stock immediately prior to the completion of this offering, and (iii) the repurchase of 1,400,998 reverse vesting RSAs upon completion of this offering, and excludes:

▪

27,245,715 shares of common stock issuable upon the exercise of outstanding stock options as of June 30, 2026 under our 2021 Plan, with a weighted-average exercise price of $0.21 per share, and 0 shares that are reserved for future issuance under the 2021 Plan as of June 30, 2026;

​

▪

1,988,176 shares of common stock issuable upon the exercise of stock options that were granted under our 2021 Plan subsequent to June 30, 2026 at a weighted-average exercise price of $0.99 per share;

​

▪

0 shares of common stock reserved for future issuance as of June 30, 2026 under the 2021 Plan, which was subsequently amended on September 3, 2026 to reserve an additional 2,000,000 shares for issuance, and which will cease to be available for issuance at the time that our 2026 Plan becomes effective;

​

▪

1,639,176 shares of common stock issuable upon the exercise of warrants issued subsequent to June 30, 2026, at an exercise price of $1.00 per share;

​

▪

     shares of our common stock reserved for future issuance under our 2026 Plan, which will become effective on the date immediately prior to the effectiveness of the registration statement of which this prospectus forms a part; and

​

▪

     shares of common stock reserved for future issuance under our ESPP.

​

To the extent that new stock options are issued or any outstanding options are exercised, or we issue additional shares of common stock in the future, there will be further dilution to new investors. In addition, we may choose to raise additional capital because of market conditions or strategic considerations, even if we believe that we have sufficient funds for our current or future operating plans. If we raise additional capital through the sale of equity or convertible debt securities, the issuance of these securities could result in further dilution to our stockholders.

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MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS

You should read the following discussion of our financial condition and results of operations in conjunction with our audited consolidated financial statements as of December 31, 2025 and 2024 and for the years then ended, our unaudited condensed consolidated financial statements as of June 30, 2026 and December 31, 2025 and for the six months ended June 30, 2026 and 2025, and, in each case, the related notes thereto and other financial information included elsewhere in this prospectus. The following discussion contains forward-looking statements that reflect our current plans, estimates and beliefs. Our historical results are not necessarily indicative of the results that may be expected for any period in the future. Our actual results and the timing of events could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to these differences include those discussed below and elsewhere in this prospectus, particularly in the section titled “Risk Factors.” Please also see the section titled “Special Note Regarding Forward-Looking Statements.”

Overview

We are a clinical-stage biotechnology company developing a portfolio of internally invented, proprietary and wholly owned oral inhibitors of NLRP3. NLRP3 is the upstream driver of sterile inflammation – inflammation that is caused by damage signals, such as cholesterol crystals or misfolded proteins, rather than by infection. NLRP3 activation triggers formation of the NLRP3 inflammasome, an intracellular protein complex involved in the body’s inflammatory response. NLRP3 activation directly stimulates the production of IL-1β and IL-18, which are potent pro-inflammatory cytokines. In turn, IL-1β stimulates production of another inflammatory molecule, IL-6. For this reason, this inflammatory cascade is referred to as the NLRP3/ IL-1β/IL-6/IL-18 pathway.​

Since inception, we have devoted substantially all of our resources to conducting and completing Phase 1 and Phase 2 clinical trials for ruvonoflast and a Phase 1 clinical trial for trabzanoflast, undertaking nonclinical studies and IND enabling activities of our product candidates, researching and developing our pipeline of product candidates, including ruvonoflast and trabzanoflast, building our intellectual property portfolio, raising capital, business planning, and organizing and staffing our company. We have never generated any revenue from drug sales. We have not obtained regulatory approvals for any of our product candidates. To date, we have funded our operations primarily with proceeds from the issuance and sale of convertible preferred stock across multiple financing rounds since 2016 and the issuance of our convertible promissory notes, issued pursuant to the Note and Warrant Purchase Agreement in November 2023, or the Convertible Notes.

We have incurred operating losses since inception as a result of our research and development activities and expenditures to advance our product candidates. We generated net losses of $25.5 million and $13.7 million for the six months ended June 30, 2026 and 2025, respectively, and $42.2 million and $25.2 million for the years ended December 31, 2025 and 2024, respectively. As of June 30, 2026, we had an accumulated deficit of $210.7 million. We expect to continue to incur significantly increased expenses for the foreseeable future if, and as, we:

▪

initiate our planned Phase 3 clinical trial of ruvonoflast in PAD;

​

▪

initiate commercial preparations for ruvonoflast, if and when our planned Phase 3 clinical trial is successful and we receive regulatory approval;

​

▪

conduct additional clinical development for trabzanoflast;

​

▪

further develop ruvonoflast for additional ASCVD, indications, if supported by data from our planned Phase 3 clinical trial of ruvonoflast in PAD and regulatory feedback;

​

▪

prepare and submit IND applications and comparable applications for clinical trial authorization for our current and future product candidates, and prepare NDAs, and comparable applications for marketing authorization for successful product candidates, as appropriate;

​

▪

enter into manufacturing arrangements for our product candidates;

​

▪

advance our research and development related activities to expand our product pipeline;

​

▪

seek regulatory approval for our product candidates that successfully complete clinical development;

​

▪

develop and scale up our capabilities to support our future clinical trials for our product candidates and commercialization of any of our product candidates for which we may obtain regulatory approval;

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▪

maintain, expand and protect our intellectual property portfolio;

​

▪

hire additional staff, including commercial, clinical, scientific, regulatory and management personnel;

​

▪

secure facilities to support continued growth in our research, development and commercialization efforts; and

​

▪

incur additional costs of operating as a public company, including increased costs of audit, legal, regulatory and tax-related services associated with maintaining compliance with the listing standards of a national securities exchange and Securities and Exchange Commission, or SEC, requirements, director and officer insurance premiums and investor and public relations costs.

​

In addition, if we obtain regulatory approval for our current or any future product candidates, we will incur significant commercialization expenses, including for sales, marketing, manufacturing and distribution. Because of the numerous risks and uncertainties associated with developing pharmaceutical drugs, including in light of any economic fluctuations, we are unable to predict the extent of any future losses or when we will become profitable, if at all.

Even if we achieve profitability, we may not be able to sustain or increase our profitability on a quarterly or annual basis. Our failure to achieve, sustain or increase our profitability could depress the value of our company and could impair our ability to raise capital, expand our business, maintain our development efforts, obtain product approvals, diversify our offerings and could also cause you to lose all or part of your investment.

As of June 30, 2026, we had cash and cash equivalents of $3.9 million. In July 2026, we received aggregate gross proceeds of $15.2 million from the issuance and sale of shares of the Series D4 Stock, or the Series D4 Financing, and borrowed $15.0 million under the Term Loan, all of which had been drawn as of the date of this prospectus. Based on the proceeds received, we expect the funding to support operations into January 2027. To finance our operations beyond that point, we will need to raise additional capital, which cannot be assured. See “—Liquidity and Capital Resources” below and “Risk Factors—Risks Related to Our Financial Position and Capital Needs” elsewhere in this prospectus.​

Impact of General Economic Risk Factors on Operations

Uncertainty in the global economy presents significant risks to our business. We are subject to continuing risks and uncertainties in connection with the current macroeconomic environment. While we are closely monitoring the impact of the current macroeconomic and geopolitical conditions on all aspects of our business, the ultimate extent of the impact remains highly uncertain and will depend on future developments and factors that continue to evolve. Most of these developments and factors are outside our control and could exist for an extended period of time. We will continue to evaluate the nature and extent of the potential impacts to our business, results of operations, liquidity and capital resources. For additional information, see the section titled “Risk Factors — Risks Related to Our Business and Industry.”

Components of Results of Operations

The following discussion describes the principal components of our consolidated statements of operations and comprehensive loss as well as the factors affecting our results of operations.

Revenue

To date, we have not generated revenue from any sources, including product sales, and do not expect to generate any revenue from the sale of products in the foreseeable future. If our development efforts for our product candidates are successful and result in regulatory approval, we may generate revenue in the future from product sales or payments from future collaboration or license agreements with third parties. We cannot predict if, when, or to what extent we will generate revenue from the commercialization and sale of our product candidates.

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Operating Expenses

Our operating expenses consist of research and development expenses and general and administrative expenses.

Research and Development

Research and development expenses consist primarily of costs incurred in connection with the research and development of our programs. These expenses include:

▪

fees paid to CROs, in connection with clinical trials as well as nonclinical and toxicology studies;

​

▪

fees paid to CMOs, in connection with the production of research products and clinical trial materials;

​

▪

salaries, benefits, and other employee-related costs, including stock-based compensation expense, for personnel engaged in research and development functions;

​

▪

fees paid to vendors that conduct certain research and development activities on our behalf;

​

▪

other expenses associated with laboratory supplies and other materials;

​

▪

professional service fees for consulting and related services; and

​

▪

facility costs, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities and other supplies.

​

We expect our research and development expenses to increase substantially in connection with our ongoing activities, particularly as we continue the research and development of, advance the nonclinical and clinical activities of, and seek marketing approval for, our current or any future product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution.

Additionally, as a company that carries out extensive research and development activities, we seek to benefit from United Kingdom research and development incentive programs. Qualifying expenditures primarily consist of employment costs for research staff, consumables and certain internal overhead costs incurred as part of qualifying research projects. Such incentives are accounted for as a benefit within research and development expense in the period in which the applicable recognition criteria are met. For the six months ended June 30, 2026 and 2025, we did not record a benefit because the amount was not reasonably estimable. We recorded a benefit of $4.3 million for the year ended December 31, 2024. For the year ended December 31, 2025, we did not record a benefit because the amount was not reasonably estimable as of the date the annual consolidated financial statements were issued.

General and Administrative

Our general and administrative expenses consist primarily of personnel-related expenses, including salaries, bonuses, stock-based compensation, benefits and recruiting costs, for individuals in our executive, finance, operations, human resources, business development and other administrative functions. Our other significant general and administrative expenses include legal fees relating to corporate matters and patent-related activities, insurance costs, information technology and professional and consulting fees associated with accounting, audit, tax and investor and public relations. For the year ended December 31, 2025, general and administrative expenses additionally include a loss related to the August 2025 cybersecurity incident totaling $2.0 million.

We expect that our general and administrative expenses will increase substantially for the foreseeable future as we increase our headcount and incur increased expenses associated with becoming a public company, including increased costs of accounting, audit, legal, regulatory and tax-related services, additional director and officer insurance costs and investor and public relations costs.

Other Income (Expense), Net

Our other income (expense), net primarily relates to changes in the fair value of our Convertible Notes, for which we elected the fair value option under ASC Topic 825, Financial Instruments, and interest accrued on the Convertible Notes. Changes in fair value attributable to base market risk are recorded in other income (expense), net. Following the conversion of the Convertible Notes into Series D3 convertible preferred stock, or the Series D3 Stock, in April 2025, no further fair value remeasurement was required. Accordingly, the six months ended June 30, 2025 include fair value remeasurement through the conversion date, while the six months ended June 30, 2026 do not include any such remeasurement.

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Income Taxes

No provision for income taxes was recorded for the six months ended June 30, 2026 and 2025, or for the years ended December 31, 2025 and 2024. We have recorded a full valuation allowance against our net deferred tax assets at each balance sheet date, as we believe it is not more likely than not that the benefit will be realized due to our cumulative losses generated to date and our expectation of future losses.

Foreign Currency Transactions

NodThera Limited transacts with CROs, CMOs and other vendors in U.S. dollars and Euros and holds cash in those currencies. Its monetary assets and liabilities denominated in currencies other than the pound sterling are remeasured at each balance sheet date and upon settlement, the resulting gains and losses are recognized in foreign currency transaction gain (loss), net, within other income (expense), net. We recognized net foreign currency transaction (losses) gains of $(1.5) million and $2.0 million for the six months ended June 30, 2026 and 2025, respectively, and $0.3 million and $(0.3) million for the years ended December 31, 2025 and 2024, respectively, driven primarily by movements in the U.S. dollar and the Euro against the pound sterling.

Foreign Currency Translation Adjustment

Our reporting currency is the U.S. dollar. The functional currency of NodThera Limited, our wholly owned UK subsidiary, is British pound sterling. Assets and liabilities of NodThera Limited are translated into U.S. dollars at the exchange rate in effect at each balance sheet date, while expenses are translated using average exchange rates prevailing during the respective reporting period. The resulting cumulative foreign currency translation adjustments are recorded as a component of accumulated other comprehensive loss within stockholders’ deficit. For the six months ended June 30, 2026 and 2025, we recorded foreign currency translation adjustments of $0.9 million and $(0.5) million, respectively. For the years ended December 31, 2025 and 2024, we recorded foreign currency translation adjustments of $(0.2) million and $0.1 million, respectively.

Results of Operations

Comparison of the Six Months Ended June 30, 2026 and 2025 (unaudited)​

The following table summarizes our unaudited condensed consolidated statements of operations and comprehensive loss for the periods presented (in thousands):​

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Research and Development Expenses

The following table summarizes our research and development expenses for the periods presented (in thousands):

Research and development expenses increased by $9.5 million to $19.9 million for the six months ended June 30, 2026, compared with $10.3 million for the six months ended June 30, 2025. The change was primarily due to an increase in services received under our CRO and CMO arrangements as our clinical trials progressed into later and more cost-intensive stages of activity, mainly reflecting the focus on completing the Phase 2 trial of ruvonoflast. There was a $9.2 million increase in ruvonoflast costs driven by a $7.9 million increase in CRO services fees and passthrough costs to complete Phase 2 trials. Additionally, there was $1.4 million of chemistry, manufacturing, and controls, or CMC work for ruvonoflast as we prepare for Phase 3 trials. The change also reflected a $0.4 million increase in other research and development related costs driven by an increase in research and development employees including the Chief Medical Officer who joined in October 2025. This was offset by a decrease of $0.1 million in safety and toxicology studies that were performed in 2025 with no additional work required in 2026.​

General and Administrative Expenses

General and administrative expenses increased by $1.1 million to $4.7 million for the six months ended June 30, 2026, compared with $3.6 million for the six months ended June 30, 2025. The change was primarily attributable to $0.6 million of higher personnel-related costs, largely driven by $0.4 million of stock-based compensation expense and $0.6 million of higher legal, accounting and consulting costs associated with our preparation for becoming a public company. This was offset by a decrease of $0.1 million in travel and entertainment driven by an effort to reduce travel costs.

Other Income (Expense), Net

The following table summarizes our other income (expense), net for the periods presented (in thousands):

Other income (expense), net was $(1.0) million for the six months ended June 30, 2026, compared with $0.2 million for the six months ended June 30, 2025. The $1.2 million decrease was driven primarily by a $3.5 million decrease in net foreign currency transaction results driven by intercompany funding of NodThera Limited. This was partially offset by a $2.2 million increase from the change in fair value of the Notes through their conversion in April 2025 and no remeasurement was required in 2026. Interest income decreased $0.3 million, primarily due to lower average cash balances, and other income, net increased $0.4 million related to additional funds recovered from the cybersecurity incident.​

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Comparison of the Years Ended December 31, 2025 and 2024

The following table summarizes our consolidated statements of operations and comprehensive loss for the periods presented (in thousands):

Research and Development Expenses

The following table summarizes our research and development expenses for the periods presented (in thousands):

Research and development expenses increased by $14.6 million to $31.4 million for the year ended December 31, 2025, compared to $16.8 million for the year ended December 31, 2024. The increase was primarily driven by higher spending on ruvonoflast due to changes in estimates of services rendered under the Company’s CRO and CMO arrangements, driven mainly by the ramp up of Phase 2 trials, which were completed in June 2026 with data readouts in July 2026. Additionally, there was an increase in trabzanoflast costs, and other research and development costs, partially offset by a decrease in NT-0249 related expenses.​

Ruvonoflast costs increased by $7.3 million, driven by a $10.6 million increase in CRO service fees and pass-through costs associated with advancing Phase 2 trials. This increase was offset partially by a decrease of $1.9 million of CMC work as the Company completed the majority of its CMC work for Phase 2 trials during the year ended December 31, 2024. Additionally, there was a decrease of $1.2 million in safety and toxicology study work, which was substantially completed in the year ended December 31, 2024 with a decreased amount of work needed in the year ended December 31, 2025.​

Trabzanoflast costs increased by $3.3 million, driven by a $1.8 million increase in CRO service fees and pass-through costs associated with advancing Phase 1 trials, as well as a $2.4 million increase in safety and toxicology study work, which ramped up for Phase 1 studies during the year ended December 31, 2025 and

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was in the initial phase as of the end of the year ended December 31, 2024 with minimal expense compared to the year ended December 31, 2025. These increases were offset partially by a decrease of $1.0 million of CMC work as the Company completed the majority of its CMC work for Phase 1 trials during the year ended December 31, 2024.​

NT-0249 costs decreased by $0.6 million, driven by a $0.1 million decrease in CRO service fees and pass-through costs associated with Phase 1 trials as the Company decided to realign resources away from NT-0249 to focus on trabzanoflast and ruvonoflast. There was also a decrease of $0.3 million in CMC as the Company had the majority of the drug developed in the year ended December 31, 2024 that was needed during Phase 1 trials. Additionally, there was a $0.2 million decrease in safety and toxicology study work, as that work was completed during the year ended December 31, 2024 and no further studies were engaged due to the decision to move away from the development and focus of NT-0249.​

General and Administrative Expenses

General and administrative expenses increased by $3.0 million to $9.6 million for the year ended December 31, 2025, compared to $6.6 million for the year ended December 31, 2024. The increase was primarily attributable to a $2.0 million loss related to a cybersecurity incident, higher legal fees reflecting increased corporate and transactional activities associated with our preparation for becoming a public company, and increased consulting fees to support our accounting and financial reporting readiness. The increase was partially offset by lower legal fees related to patent filings and lower personnel-related costs. The prior year also included a $0.4 million income tax credit that did not recur in the current year, which also contributed to the year-over-year increase.

Other Income (Expense), Net

Other income (expense), net was an expense of $1.2 million for the year ended December 31, 2025, compared with an expense of $1.8 million for the year ended December 31, 2024. The 2025 amount was primarily attributable to the $2.2 million loss in fair value of our Convertible Notes resulting from their remeasurement prior to their conversion in 2025. The loss was partially offset by realized foreign currency transaction gains and interest income. Other income (expense), net for the year ended December 31, 2024 was primarily attributable to the $2.0 million loss resulting from the change in fair value of the Convertible Notes resulting from their remeasurement as of December 31, 2024.

Liquidity and Capital Resources

Sources of Liquidity

Since our inception, we have incurred significant operating losses. We expect to incur significant expenses and operating losses for the foreseeable future as we continue the clinical development of our programs, including ruvonoflast. We have not yet commercialized any products and do not expect to generate revenue from sales of products for several years, if at all. To date, we have funded our operations primarily with proceeds from the sale of convertible preferred stock and Convertible Notes, as further described under the subsection titled “Prior Financings” below. As of June 30, 2026 and December 31, 2025, we had cash and cash equivalents of $3.9 million and $28.6 million, respectively. The change in cash and cash equivalents during the six months ended June 30, 2026 primarily reflected net cash used in operating activities and $6.4 million repayment of our Convertible Note.

We believe that the anticipated net proceeds from this offering, together with our existing cash and cash equivalents, including proceeds from the Series D4 Financing and Term Loan, will enable us to fund our operating expenses and capital expenditure requirements through . We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. Our future viability beyond that point is dependent on our ability to raise additional capital to finance our operations. Without giving effect to the net proceeds from this offering, we believe that our existing cash and cash equivalents as of June 30, 2026 and proceeds from our Series D4 Financing and Term Loan will enable us to fund our operating expenses and capital expenditure requirements into January 2027. To finance our operations beyond that point, we will need to raise additional capital, which cannot be assured. We have concluded that there is substantial doubt about our ability to continue as a going concern for at least one year from the date the unaudited condensed consolidated financial statements included elsewhere in this prospectus are issued. See Note 1 to our unaudited condensed consolidated financial statements included elsewhere in this prospectus for additional information on our assessment. For more information, see “Risk Factors – Risks Related to Our Financial Position and Capital Needs” elsewhere in this prospectus.

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Cash Flows for the Six Months Ended June 30, 2026 and 2025 (unaudited)

The following table summarizes our unaudited condensed consolidated cash flows for the periods presented (in thousands):

Net Cash Used in Operating Activities

Net cash used in operating activities was $17.9 million and $14.6 million for the six months ended June 30, 2026 and 2025, respectively. Net cash used in operating activities for the six months ended June 30, 2026 was primarily due to our net loss of $25.5 million, a net change in operating assets and liabilities of $7.0 million and $0.6 million of non-cash charges, including stock-based compensation. The $3.3 million increase in net cash used in operating activities compared with the prior-year period was primarily driven by higher spending on our clinical programs and changes in prepaid CRO and CMO costs, accrued research and development costs, UK research and development incentive receivables, insurance recoveries, foreign currency translation, accounts payable and accrued expenses.​

Net Cash Used in Investing Activities

We had no investing activities for the six months ended June 30, 2026 and 2025.​

Net Cash (Used in) Provided by Financing Activities​

Net cash used in financing activities was $6.8 million for the six months ended June 30, 2026, compared with net cash provided by financing activities of $49.5 million for the six months ended June 30, 2025. The 2026 activity primarily consisted of the $6.4 million repayment of the promissory note in connection with the cybersecurity incident. The 2025 activity primarily consisted of approximately $49.5 million of net cash proceeds from the issuance of Series D1 convertible preferred stock, or the Series D1 Stock and Series D2 convertible preferred stock, or the Series D2 Stock, net of issuance costs. The conversion of the Convertible Notes into Series D3 Stock was a non-cash transaction and did not affect financing cash flows.

Cash Flows for the Years Ended December 31, 2025 and 2024

The following table summarizes our cash flows for the periods presented (in thousands):

Net Cash Used in Operating Activities

Net cash used in operating activities was $39.1 million and $22.1 million for the years ended December 31, 2025 and 2024, respectively. Net cash used in operating activities for the year ended December 31, 2025 was primarily due to our net loss of $42.2 million and a net change in our operating assets and liabilities of $0.6

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million, partially offset by $3.6 million in non-cash charges, including stock-based compensation, foreign currency transaction adjustments and the change in fair value of our Convertible Notes. Net cash used in operating activities for the year ended December 31, 2024 was primarily due to our net loss of $25.2 million and a net change in our operating assets and liabilities of $0.1 million, partially offset by $3.2 million in non-cash charges, including stock-based compensation and the change in fair value of our Convertible Notes. The $17.0 million increase in net cash used in operating activities was primarily driven by a higher net loss reflecting the continued advancement of our clinical programs. Working capital changes included a $4.2 million collection of the UK research and development tax incentive receivable, a $3.0 million increase in accounts payable, cash recovered in connection with the cybersecurity incident and a $6.6 million increase in prepaid expenses reflecting advance payments made to CROs in support of our expanding clinical programs.

Net Cash Used in Investing Activities

We had no investing activities for the years ended December 31, 2025 or 2024.

Net Cash Provided by Financing Activities

Net cash provided by financing activities was $55.8 million for the year ended December 31, 2025, compared with $19,000 for the year ended December 31, 2024. The 2025 activity was primarily attributable to proceeds of $49.8 million from the issuance of Series D1 Stock, net of issuance costs of $0.2 million, and $6.4 million from the issuance of a promissory note in connection with our cybersecurity incident. In 2025, following the unauthorized transfer of company funds to Hong Kong, we received a promissory note in the amount of $6.4 million, representing funds in the process of being recovered by Hong Kong authorities. The promissory note was repaid in February 2026. The 2024 activity consisted solely of proceeds from the exercise of stock options.

Future Funding Requirements

To date, we have not generated any revenue from product sales. We do not expect to generate revenue from product sales unless and until we successfully complete nonclinical and clinical development of, receive regulatory approval for, and commercialize a product candidate. We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we advance our clinical activities and clinical trials. Our material cash requirements for the 12 months following June 30, 2026 are expected to consist primarily of costs associated with our planned clinical trials, manufacturing and clinical supply, personnel, public-company readiness, intellectual property and lease obligations. Beyond the next 12 months, we expect material cash requirements related to Phase 3 development, additional clinical and regulatory activities, manufacturing scale-up and, if approved, commercialization. We expect to fund these requirements through our existing cash and cash equivalents, including the proceeds from the issuance of the Series D4 Stock and the Term Loan, and the anticipated net proceeds from this offering and additional equity or debt financing, collaborations or other arrangements. If we obtain regulatory approval for any programs, we expect to incur significant expenses related to product sales, marketing and distribution. Further, upon the completion of this offering, we expect to incur additional costs associated with operating as a public company. For more information, see the section titled “Risk Factors — Risks Related to Our Financial Position and Capital Needs” found elsewhere in this prospectus.

Our funding requirements and the timing and amount of our operating expenses will depend on many factors, including, but not limited to:

▪

the rate of progress in the development of our existing and future research programs, including ruvonoflast and trabzanoflast;

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the scope, progress, results and costs of additional research programs, product candidates and discovery activities and nonclinical studies for those programs;

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▪

our ability to file IND applications or comparable foreign applications and obtain authorization to commence future clinical trials;

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the costs of enrollment and successful completion of clinical trials;

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the costs necessary to obtain regulatory approvals, if any, and the costs of post-marketing studies that could be required by regulatory authorities;

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the costs of developing, maintaining and enhancing sustainable, scalable, reproducible and transferable clinical and commercial-scale current good manufacturing capabilities;

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the costs and timing of future commercialization activities, including establishing sales, marketing and distribution infrastructure;

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▪

the costs and timing of maintaining, expanding, enforcing, defending and protecting our intellectual property rights;

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the timing and payment of milestone, royalty or other payments pursuant to existing and potential future collaborations;

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the costs associated with being a public company, including audit, legal, regulatory and tax-related services, director and officer insurance premiums and investor and public relations costs;

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the effect of competing technological and market developments; and

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the extent to which we acquire or invest in businesses, products and technologies, including entering into licensing or collaboration arrangements.

​

Adequate additional funds may not be available to us on acceptable terms, or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders and may include covenants limiting or restricting our ability to take specific actions, such as incurring debt, making capital expenditures or declaring dividends. If we are unable to raise additional funds when needed, we may be required to delay, limit or terminate our product development programs or grant rights to develop and market product candidates to third parties that we would otherwise prefer to develop and market ourselves.

Contractual Obligations and Other Commitments

Our principal contractual commitments consist of operating lease obligations and obligations under agreements with CROs, CMOs and other third-party service providers in connection with our research and development activities. As of June 30, 2026, operating lease liabilities totaled approximately $0.1 million, of which all was classified as current, related primarily to our operating lease of office space at The Mansion, Chesterford Park, Little Chesterford, UK, which was extended through April 2027. Payments due upon cancellation of contracts with CROs, CMOs, and other research and development commitments consist only of payments for services provided or expenses incurred, including noncancelable obligations of our service providers, up to the date of cancellation.​

In December 2025, in connection with our cybersecurity incident, we were issued a promissory note in the amount of $6.4 million, representing funds that were subject to an unauthorized transfer to Hong Kong and were in the process of being recovered by Hong Kong authorities. The promissory note was repaid in February 2026 and was no longer outstanding as of June 30, 2026.

Prior Financings

Since inception, we have financed our operations through the following principal financing transactions:

▪

Series A Preferred Stock: Beginning in July 2016, we raised proceeds of $2.7 million from the initial Series A1 convertible preferred stock, or the Series A1 Stock, closing. Additional Series A1 Stock and Series A2 convertible preferred stock, or the Series A2 Stock, closings occurred through September 2019, resulting in aggregate net proceeds of approximately $45.5 million from the Series A Preferred Stock financing. The Series A2 Stock, together with the Series A1 Stock, is collectively referred to as the Series A Preferred Stock.

​

▪

Series Junior Preferred Stock: Beginning in July 2016 and in connection with certain Series A Preferred Stock financings, we issued shares of Series Junior convertible preferred stock, or the Series Junior Preferred Stock, including (i) 1,910,000 shares issued in July 2016 as consideration for the acquisition of certain intellectual property and other assets under an asset contribution arrangement and (ii) an aggregate of 648,030 shares issued in exchange for the termination of a purchase option previously held by Eli Lilly and Company in March and June of 2019.

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Series B Preferred Stock: Beginning in May 2020, we raised aggregate net proceeds of approximately $59.3 million through the issuance of Series B1 convertible preferred stock, or the Series B1 Stock, and Series B2 convertible preferred stock, or the Series B2 Stock, across multiple closings through February 2022. The Series B2 Stock, together with the Series B1 Stock, is collectively referred to as the Series B Preferred Stock.

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Series C Preferred Stock: In September 2022, we issued and sold 8,698,376 shares of Series C convertible preferred stock, or the Series C Preferred Stock, at a price of $2.8741 per share, resulting in total net proceeds of approximately $24.5 million.

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Convertible Notes: In November 2023, we issued $20.0 million in aggregate principal amount of the Convertible Notes, together with warrants to purchase 3,986,941 shares of our common stock. The Convertible Notes converted into shares of Series D3 Stock in April 2025.

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▪

Series D Preferred Stock: In April 2025, we raised approximately $49.5 million in net cash proceeds through the issuance of the Series D1 Stock, and Series D2 Stock and converted the outstanding Convertible Notes, with aggregate face value of approximately $22.3 million, into Series D3 Stock. In July 2026 we raised approximately $15.0 million in net cash proceeds through the issuance of Series D4 Stock. The Series D4 Stock, together with the Series D1 Stock, the Series D2 Stock and the Series D3 Stock, is collectively referred to as the Series D Preferred Stock.

​

Critical Accounting Estimates

Our management’s discussion and analysis of our financial condition and results of operations is based on our audited consolidated financial statements and unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States of America. The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported expenses incurred during the reporting periods. Our estimates are based on our historical experience and on various other factors that we believe are reasonable under the circumstances. Actual results may differ from these estimates under different assumptions or conditions.

While our significant accounting policies are described in more detail in the notes to our audited consolidated financial statements and unaudited condensed consolidated financial statements included elsewhere in this prospectus, we believe the following estimates involve a significant level of estimation uncertainty and have had or are reasonably likely to have a material impact on our financial condition or results of operations. The estimates and assumptions discussed below reflect the information available to management as of June 30, 2026 and are updated as facts and circumstances change.

Research and Development Contract Costs and Accruals

We record the costs associated with research studies and manufacturing development as incurred. We accrue for expenses resulting from obligations under agreements with CROs, CMOs and other outside service providers for which payment flows do not match the periods over which materials or services are provided. Accruals are recorded based on estimates of services received and efforts expended pursuant to agreements established with such outside service providers, typically based on contracted amounts applied to the proportion of work performed and determined through analysis with internal personnel and external service providers as to the progress or stage of completion of services. In the event advance payments are made to a CRO, CMO or outside service provider, the payments will be recorded as a prepaid asset that will be expensed as the contracted services are performed. Changes in these estimates that result in material changes to our accruals could materially affect our results of operations.

For clinical trial accruals, the most significant judgments generally relate to the number of patients enrolled, the timing and extent of services performed by clinical sites and CROs, and changes in trial protocols or scope of work. If actual timing of patient enrollment, site activity, or vendor performance differs from our estimates, the amount of accrued research and development expense recorded in any period could differ materially from the amounts ultimately paid.

Stock-Based Compensation

We measure stock-based awards granted to employees, directors and non-employees based on their fair value on the date of grant using the Black-Scholes option-pricing model. Compensation expense for employee and director awards is recognized using the straight-line method over the requisite service period, which is generally the vesting period. We account for forfeitures as they occur. We measure restricted common stock awards using the difference, if any, between the purchase price per share of the award and the fair value of our common stock at the date of grant.

The Black-Scholes model requires inputs including the expected volatility of our common stock, the expected term, the risk-free interest rate and the expected dividend yield. We have historically been a private company and lack company-specific historical and implied volatility information. Therefore, we estimate our expected stock volatility based on the historical volatility of a representative group of public companies in the biotechnology industry. The expected term of our stock options has been determined using the simplified method for awards that qualify as plain-vanilla stock options. The risk-free interest rate is determined by reference

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to the U.S. Treasury yield curve. We have estimated a 0% dividend yield. The weighted-average grant-date fair value of stock options granted was $0.14 for the six months ended June 30, 2026, and $0.24 and $0.59 per share for the years ended December 31, 2025 and 2024, respectively. There were no grants for the six months ended June 30, 2025. As of June 30, 2026, there was $2.8 million of total unrecognized compensation cost related to unvested stock-based awards, which we expect to recognize over a remaining weighted-average period of 3.3 years.

Determination of Fair Value of Common Stock

As there has been no public market for our common stock, the estimated fair value of stock-based awards has been determined by our board of directors as of the date of grant, with input from management, considering various objective and subjective factors, including: the prices at which we sold shares of our convertible preferred stock; our common stock valuations prepared by third-party valuation specialists; the progress of our research and development programs; our stage of development and business strategy; external market conditions affecting the biotechnology industry; our financial position and its historical and forecasted performance; and the lack of an active public market for our common stock and our convertible preferred stock at the grant dates.

Once a public trading market for our common stock has been established in connection with the completion of this offering, it will no longer be necessary for our board of directors to estimate the fair value of our stock-based awards, as the fair value will be determined based on the quoted market price of our common stock.

Fair Value of Convertible Promissory Notes

We elected to account for the Convertible Notes under the fair value option under ASC Topic 825. The estimated fair value was determined using Level 3 inputs based on a probability-weighted payoff scenario approach that considered the stated interest rate, potential conversion scenarios, applicable discounts and premiums, and our risk profile. Key inputs used to value the Convertible Notes for the year ended December 31, 2024 included a discount rate of 38.2%, an 80% discount on conversion, a 90% probability of next-round financing, a 5% probability of maturity and a 5% probability of an event of default. The Convertible Notes were carried at a fair value of $20.1 million as of December 31, 2024 and were remeasured to a fair value of $22.3 million immediately before their conversion into Series D3 Stock in April 2025. This estimate affected change in fair value of convertible promissory notes for the six months ended June 30, 2025 and the year ended December 31, 2025, but it did not affect change in fair value of convertible promissory notes for the six months ended June 30, 2026 because no further remeasurement was required after the conversion.

Off-Balance Sheet Arrangements

During the periods presented, we did not have, nor do we currently have, any off-balance sheet arrangements as defined in the rules and regulations of the SEC.

Recently Issued Accounting Pronouncements

A description of recently issued accounting pronouncements that may potentially affect our financial position, results of operations or cash flows is disclosed in the notes to our audited consolidated financial statements and unaudited condensed consolidated financial statements included elsewhere in this prospectus. We do not believe that any recently issued but not yet adopted accounting pronouncements will have a material effect on our consolidated financial statements, except as otherwise disclosed in those notes.

Emerging Growth Company and Smaller Reporting Company Status

We are an “emerging growth company,” as defined in Section 2(a) of the Securities Act of 1933, as amended, or the Securities Act, as modified by the JOBS Act. As such, we are eligible to take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies including:

▪

being permitted to provide only two years of audited financial statements in addition to any required unaudited interim financial statements and a correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations” disclosure in this prospectus;

​

▪

not being required to comply with the auditor attestation requirements in the assessment of our internal control over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act;

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▪

reduced disclosure obligations regarding executive compensation;

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▪

exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and stockholder approval of any golden parachute payments not previously approved; and

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▪

exemptions from compliance with the requirements of the Public Company Accounting Oversight Board regarding the communication of critical audit matters in the auditor’s report on the financial statements.

​

Further, Section 102(b)(1) of the JOBS Act exempts emerging growth companies from being required to comply with new or revised financial accounting standards until private companies (that is, those that have not had a Securities Act registration statement declared effective or do not have a class of securities registered under the Exchange Act) are required to comply with the new or revised financial accounting standards. The JOBS Act provides that an emerging growth company can elect to opt out of the extended transition period and comply with the requirements that apply to non-emerging growth companies but any such election to opt out is irrevocable. We have elected not to opt out of such extended transition period which means that when a standard is issued or revised and it has different application dates for public or private companies, we, as an emerging growth company, will adopt the new or revised standard whenever such adoption is required for private companies. We may also choose to early adopt any new or revised accounting standards whenever such early adoption is permitted. This may make comparison of our financial statements with another public company, which is neither an emerging growth company nor an emerging growth company that has opted out of using the extended transition period, difficult or impossible because of the potential differences in accounting standards used.

We will remain an emerging growth company until the earliest of (i) the last day of the fiscal year following the fifth anniversary of the completion of this initial public offering, (ii) the last day of the fiscal year in which we have total annual gross revenue of at least $1.235 billion, (iii) the last day of the fiscal year in which we are deemed to be a “large accelerated filer” as defined in Rule 12b-2 under the Exchange Act, which would occur if the market value of the common stock held by non-affiliates exceeded $700.0 million as of the last business day of the second fiscal quarter of such year, or (iv) the date on which we have issued more than $1.0 billion in non-convertible debt securities during the prior three-year period.

We are also a “smaller reporting company” as defined in the Exchange Act. We may continue to be a smaller reporting company even after we are no longer an emerging growth company. We may take advantage of certain of the scaled disclosures available to smaller reporting companies and will be able to take advantage of these scaled disclosures for so long as (i) the market value of our voting and non-voting common stock held by non-affiliates is less than $250.0 million measured on the last business day of our second fiscal quarter, or (ii)(a) our annual revenue is less than $100.0 million during the most recently completed fiscal year, and (b) the market value of our voting and non-voting common stock held by non-affiliates is less than $700.0 million measured on the last business day of our second fiscal quarter.

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BUSINESS

Overview

We are a clinical-stage biotechnology company developing a portfolio of internally invented, proprietary and wholly owned oral inhibitors of NLRP3. NLRP3 is the upstream driver of sterile inflammation – inflammation that is caused by damage signals, such as cholesterol crystals or misfolded proteins, rather than by infection. NLRP3 activation triggers formation of the NLRP3 inflammasome, an intracellular protein complex involved in the body’s inflammatory response. NLRP3 activation directly stimulates the production of IL-1β and IL-18, which are potent pro-inflammatory cytokines. In turn, IL-1β stimulates production of another inflammatory molecule, IL-6. For this reason, this inflammatory cascade is referred to as the NLRP3/ IL-1β/IL-6/IL-18 pathway.​

When NLRP3 becomes chronically activated, it stimulates this cascade of inflammatory mediators that drive the development and progression of cardiovascular and neuroinflammatory disease. This phenomenon of chronically elevated inflammation that persists and contributes to disease progression even when other known risk factors are well controlled, is known as RIR. The presence of RIR can be assessed using a widely available biomarker called hsCRP, which is a well-validated independent risk factor for future cardiovascular events, and hsCRP can also be used to evaluate whether inflammatory activity of the NLRP3/ IL-1β/IL-6/IL-18 pathway has been reduced by a therapeutic intervention. By directly inhibiting NLRP3 upstream, we aim to broadly reduce the activity of the entire sterile inflammation pathway, thereby reducing RIR and modifying the course of cardiovascular and neurologic diseases.

We have been a leader in the research, discovery and development of NLRP3 inhibitors for over a decade, and we have designed our portfolio to address historical challenges faced by other NLRP3 inhibitors: potency, therapeutic index and tissue penetration. Our lead product candidate, ruvonoflast, is highly potent, has been observed to be well tolerated in five completed human clinical trials and in an extensive nonclinical toxicology program, and is designed to achieve broad tissue distribution, including penetration into the CNS.

We plan to develop ruvonoflast in PAD as our lead indication. PAD is a form of ASCVD in which narrowed or blocked small arteries reduce blood flow to the limbs, most commonly the legs. We subsequently plan to expand development to establish ruvonoflast in ASCVD more broadly. In PAD, our goal is to demonstrate that ruvonoflast is a disease-modifying anti-inflammatory therapy for patients with PAD. We selected PAD because it represents a significant unmet medical need in a large patient population and is associated with a high inflammatory burden and substantial disease morbidity despite broad use of lipid-lowering, antihypertensive and diabetes therapies.

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PAD affects approximately 20 million adults in the United States, driving $21 billion in annual direct health care costs. We believe ruvonoflast may address a market opportunity of approximately $5 billion, based on reasonable assumptions for penetration rate, price, reimbursement and competition. In addition, PAD offers a focused commercial opportunity through a concentrated network of treatment centers. We believe PAD is an attractive initial indication because RIR is an important driver of disease progression. More than half of the approximately 20 million adults with diagnosed ASCVD have RIR. We believe PAD provides an opportunity to evaluate the impact of ruvonoflast in a patient population with significant unmet need, and to establish a foundation for the future development of ruvonoflast in broader ASCVD populations.

PAD impairs circulation to the legs and significantly reduces quality of life, walking performance and functional status as the disease progresses. Intermittent claudication (pain on walking), which is the hallmark symptom of symptomatic PAD, can limit daily activities and mobility, reduce exercise capacity and cause progressive functional decline. In early stages of PAD, patients have a two-fold increased risk of cardiovascular death, which rises to approximately four times the risk in later stages. Late-stage disease progression is also characterized by leg ulcerations, ischemia and ultimately amputation, and may result in CLTI. CLTI is the most advanced stage of PAD and affects more than two million patients in the United States and is associated with a high risk of limb loss. PAD is also a leading cause of non-traumatic lower-extremity amputation in the United States, with approximately 150,000 non-traumatic lower-extremity amputations occurring annually.

NLRP3-driven inflammation plays a prominent role in the origin and progression of PAD. NLRP3 expression is elevated in the lower extremity arteries of people with PAD. People with genetically lower IL-6 and IL-18 levels have been observed to have lower rates of PAD, and people with genetically higher rates of NLRP3 activation have been observed to have early onset of PAD in adolescence. Eighty percent of patients with PAD have elevated hsCRP, a blood-based biomarker of inflammation that is an independent risk factor for cardiovascular risk. Higher hsCRP and IL-6 levels in patients with PAD have been linked to faster loss of walking ability and higher rates of serious limb and cardiovascular events. However, the current standard of care in PAD focuses on improving blood flow, reducing clotting and treating traditional risk factors such as lipids and blood pressure. There are no approved therapies that directly target the underlying inflammatory pathways in PAD.

Ruvonoflast is designed to target inflammatory mechanisms that contribute to PAD progression but are not addressed by current standard-of-care therapies. To date, we have not conducted clinical trials of ruvonoflast in patients with PAD. Our clinical data for ruvonoflast comes from trials conducted in other indications, including cardiometabolic and neuroinflammatory disease populations, where we have observed reductions in hsCRP, IL-18, IL-6, and fibrinogen (an independent risk factor for thrombosis). Taken together, these biomarker changes indicate deep and broad inhibition of the NLRP3 inflammation pathway. In a clinical trial of calorie-restricted patients with high cardiovascular risk, ruvonoflast reduced hsCRP by 82% (versus 37% with placebo), and significantly reduced these additional pro-inflammatory molecules associated with atherogenesis and thrombosis.​

We believe these results are relevant to PAD because hsCRP, IL-6 and fibrinogen are also elevated in patients with PAD and have been linked to faster loss of walking ability and higher rates of limb and cardiovascular events. Based on the observed biomarker reductions in our non-PAD clinical trials, together with the established role of NLRP3-mediated inflammation in PAD progression, we believe ruvonoflast has the potential to improve walking distance and quality of life and may ultimately be able to prevent or delay disease progression and reduce adverse limb and cardiovascular outcomes in patients with PAD.​

We have evaluated ruvonoflast in cardiometabolic disease in our RESOLVE-1 Phase 2 clinical trial. This clinical trial demonstrated geometric LSmean hsCRP reduction of 56.4% at 150 mg QD and 62.6% at 150 mg BID at week 4. Furthermore, hsCRP was reduced below 2mg/L in 33% and 40% of patients at 150 mg QD and 150 mg BID doses of ruvonoflast, respectively, and there were reductions in additional biomarkers: median IL-6 by 38.2% and 62.3% and median fibrinogen by 19.0% and 17.8%, at 150 mg QD and 150 mg BID, respectively.  Additionally, we have discussed our plan to conduct a single well-controlled Phase 3 trial with the FDA, EMA, and MHRA and we plan to initiate our Phase 3 PAD trial in the first half of 2027.

Approximately 20 million adults in the United States have diagnosed ASCVD, and the disease drives significant annual costs in the United States. Despite advances in the treatment of ASCVD to address the role of lipids,

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hypertension, thrombosis and diabetes in the origin and progression of the disease, 30% of people with ASCVD have RIR as measured by elevated hsCRP. Similar to PAD, there is no approved therapy that directly targets the inflammatory pathways that contribute to disease progression in this broad population.

The rationale for targeting inflammation in ASCVD is supported by data from multiple third-party clinical trials, including:

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CANTOS, initiated in 2011 with primary completion in 2017, evaluated more than 10,000 patients and demonstrated a reduction in MACE among patients who achieved lower levels of inflammation. In CANTOS, quarterly canakinumab in post-myocardial infarction patients with elevated hsCRP reduced major adverse cardiovascular events by 25% in patients who achieved hsCRP reduction below 2 mg/L. This was achieved without altering lipids, providing strong clinical evidence that lowering IL-1β–mediated inflammation (independent of cholesterol) reduces cardiovascular risk.

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The JUPITER trial, demonstrated that in healthy men and women with low-density lipoprotein, or LDL, cholesterol <130 mg/dL but elevated hsCRP ≥2 mg/L, rosuvastatin 20 mg daily produced large reductions in LDL and hsCRP and significantly lowered the incidence of first major cardiovascular events compared with placebo (Hazard Ratio ~0.56). The JUPITER trial was stopped early at a median of 1.9 years because of clear benefit, including a reduction in all-cause mortality. This trial provides evidence that the modest anti-inflammatory effects of rosuvastatin could impact outcomes in patients without elevated lipids.

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In patients with established ASCVD, multiple trials (notably LoDoCo2 and COLCOT) show that adding low-dose colchicine 0.5 mg daily to guideline-directed therapy reduces MACE by roughly 25–30%, supporting inflammation-targeted therapy as an effective strategy to lower residual ASCVD risk.

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We believe ruvonoflast has the potential to meet the significant unmet need in the ASCVD treatment landscape and may offer several advantages over the monoclonal antibodies that have been investigated in the field. Namely, ruvonoflast has an oral route of administration, the potential for combination products, and a low observed and theoretical risk of immune suppression. In addition, ruvonoflast has been designed to achieve high levels of tissue penetration, which may allow it to have increased activity at sites of inflammation such as the vascular wall and other extravascular tissues, as well as for high levels of brain penetration which may allow it to offer therapeutic and/or preventive effects in the brain in patients with cardiovascular disease.​

We also plan to develop ruvonoflast in indications beyond PAD and ASCVD. We believe that ruvonoflast’s high observed tissue and brain penetration in humans could support development in a range of cognitive and neurological diseases that have an association or overlap with ASCVD. We plan to develop and disclose our expanded development strategy for ruvonoflast in the future.​

In addition to ruvonoflast, we are developing trabzanoflast, which is an NLRP3 inhibitor designed to be brain-penetrant and achieve CNS exposure and selective upstream inhibition of NLRP3-driven neuroinflammation. We believe that trabzanoflast is differentiated from ruvonoflast by its chemical structure and its optimization specifically for CNS penetration. This design supports NLRP3 inhibition as a potential therapeutic approach in neuroinflammatory diseases. We anticipate that trabzanoflast may benefit from the growing body of clinical and translational evidence generated across our NLRP3 inhibitor portfolio. Trabzanoflast has completed its Phase 1 clinical trial and has demonstrated activity in NLRP3 in vitro models and reduced neuroinflammation in CNS nonclinical models. Emerging evidence implicates activation of NLRP3 as a contributor to neuroinflammation and neurodegeneration across multiple disorders, including Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis and other CNS disorders. Our development plan for trabzanoflast will focus on neuroinflammatory indications.

Our Pipeline

We are developing a pipeline of wholly owned, proprietary NLRP3 inhibitors to address inflammatory drivers of cardiovascular and neuroinflammatory disease. We retain exclusive, worldwide development and commercialization rights to all of our product candidates. All of our candidates are potent and selective oral inhibitors of NLRP3 and each has been optimized for activity in physiologically relevant tissue types –

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ruvonoflast in peripheral tissues and brain and trabzanoflast for brain. In addition, we plan to seek authorization to conduct a Phase 3 clinical trial in PAD, and are engaging with the FDA prior to submitting an IND application.

Figure 1: Our clinical pipeline highlights ruvonoflast’s completed Phase 2 clinical trials, as well as trabzanoflast’s completed Phase 1 trial.

Our Strategy

Our goal is to become a leading anti-inflammation company delivering differentiated therapies to reduce the harmful effects of inflammation for patients worldwide. We are preparing to initiate a registrational Phase 3 trial of ruvonoflast, our lead product candidate, in PAD in the first half of 2027, which is designed to enable a Phase 3 readout in 2028 and a planned NDA submission in early 2029. Assuming our Phase 3 trial progresses as anticipated, and the results of the clinical trial support NDA submission, we believe our current plan would put us on pace for NDA approval in 2029, subject to the FDA’s review and approval of our filing. Ruvonoflast has not been approved by the FDA or any other regulatory authority. Our strategy to develop and, if approved, commercialize, ruvonoflast and any future product candidates has six key elements:​

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Maximize the value of ruvonoflast, our lead NLRP3 inhibitor, by developing it for approval for use in PAD. We plan to secure FDA approval for ruvonoflast in PAD before expanding to other ASCVD indications. Ruvonoflast is designed to directly target inflammatory mechanisms that contribute to PAD progression but are not addressed by current standard-of-care therapies.

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Self-commercialize ruvonoflast for PAD in the United States. If approved, we believe PAD is well suited for a focused commercial launch directed at approximately 50 centers of excellence in the United States, which would allow us to independently establish an initial commercial franchise for ruvonoflast in this large, underserved indication.

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Further develop ruvonoflast in additional ASCVD indications. We plan to explore the use of ruvonoflast in ASCVD broadly, as well as in ASCVD indications similar to PAD, including others such as heart failure, that may be approved by the FDA and other regulatory authorities in the course of a larger ASCVD strategy.

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Advance trabzanoflast, our earlier-stage NLRP3 inhibitor, into a Phase 2 proof-of-concept trial. Trabzanoflast is designed to target neuroinflammation.

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Expand our portfolio of anti-inflammation product candidates via internal discovery and in-licensing or acquisition. We intend to continue to explore opportunities to inhibit NLRP3 and other inflammatory pathways.

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Explore the potential for strategic partnerships to expand and/or accelerate our programs to maximize the worldwide commercial potential of our product candidates. We may seek collaborations with pharmaceutical and biotechnology companies that can provide expertise in areas such as clinical development, regulatory affairs, manufacturing and commercialization.

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Our History and Management Team

Rezera was founded to develop novel inhibitors of NLRP3, which was identified as a compelling drug target in the early 2000s. Mutations in the NLRP3 protein were shown to be responsible for driving autoinflammatory diseases in humans. Furthermore, chronic inflammation associated with common conditions such as cardiovascular, metabolic and neurodegenerative diseases was associated with aberrant NLRP3 activation.

Despite strong biological validation, NLRP3 remained undruggable for over a decade until 2015 with the publication of MCC950, a potent and selective small molecule inhibitor that directly targets NLRP3. MCC950 was not suitable for further drug development, as clinical development was discontinued due to safety findings, which left a significant opportunity to discover and develop improved NLRP3 inhibitors. From our founding, we have pursued the internal design, discovery and development of molecules that are able to access physiologic

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compartments, beyond the systemic circulation, where inflammation causes disease such as the tissues and the brain. We have two product candidates in clinical development.

Our leadership team includes executives with more than 100 years of collective experience in senior roles at biotechnology and pharmaceutical companies. Their collective experience covers drug discovery, development and commercialization. Our President and Chief Executive Officer, Geoffrey McDonough, M.D., brings proven experience in company building, business strategy, product development and commercialization. Before joining Rezera, he was the president and chief executive officer of Generation Bio. He also previously served as the president and chief executive officer of Swedish Orphan Biovitrum AB (publ). Earlier, he held various senior leadership roles at Genzyme Corporation. Our Chief Financial Officer, Chris Guiffre, J.D., M.B.A., also brings substantial senior executive leadership experience in development stage and commercial companies, having served in a variety of C-suite roles at companies including Pear Therapeutics, Inc., Cerulean Pharma Inc. and Cubist Pharmaceuticals, Inc. Our Chief Medical Officer, Jyothis George, MBBS, Ph.D., brings substantial drug development experience, having held senior clinical, medical and regulatory roles at companies including Amgen Inc., Novo Nordisk and Boehringer Ingelheim Pharmaceuticals, Inc. Our founder and Chief Scientific Officer, Alan P. Watt, Ph.D., M.B.A., brings substantial research, discovery, nonclinical and translational experience, having held senior scientific positions at companies including Bicycle Therapeutics plc, GSK plc and Merck & Co., Inc. Our Chief Development Officer, Nicholas Clarke, Ph.D., brings substantial nonclinical, translational and drug development experience, having held senior roles at Pfizer Inc.

Financing

Since our founding in 2016 (by Epidarex Capital), we have raised approximately $200.0 million in equity financing from investors, including 5AM Ventures, Blue Owl Capital, Epidarex Capital, F-Prime Capital, Novo Holdings A/S, Sofinnova Partners and Aventis Inc. Prospective investors should not rely on the investment decisions of our existing investors. These investors may have different investment objectives and risk tolerances than prospective investors in this offering, and they acquired their shares in prior financings at prices substantially below the public offering price in this offering.​

In addition to these equity financings, on July 17, 2026, we entered into the Term Loan, which has been fully drawn. The Term Loan bears interest at a floating annual rate equal to the greater of 6.00% and the prime rate less 0.75%. Interest is payable monthly, with principal amortization beginning on July 1, 2027, subject to extension to July 1, 2028 upon satisfaction of a specified condition, and the Term Loan matures on June 1, 2030.​

The Term Loan may be voluntarily prepaid only in full and is subject to a final payment equal to 2.50% of the aggregate original principal amount advanced. We are also required to pay a $275,000 success fee upon the occurrence of certain specified transactions, including the effectiveness of the registration statement of which this prospectus forms a part. Our obligations under the Term Loan are secured by a first-priority security interest, subject to permitted liens, in substantially all of our assets other than intellectual property, and we have agreed not to encumber our intellectual property without the lender’s prior written consent. The Term Loan also requires us to maintain specified percentages of our cash with the lender and contains customary affirmative and negative covenants and events of default.

The Cardiovascular Disease Landscape: Burden of Disease

PAD

Prevalence and Morbidity

PAD affects an estimated 20 million adults in the United States. PAD is the third leading cause of morbidity, attributable to ASCVD, following ischemic heart disease and stroke. PAD progression is characterized by reduced blood flow to the lower extremities that leads to worsening functional impairment, including progressively limited walking ability, and may ultimately result in CLTI. Affecting more than two million patients in the United States, CLTI represents the most advanced stage of PAD and is characterized by ischemic rest pain, non-healing wounds and high risk for limb loss. PAD is also a leading cause of non-traumatic lower-extremity amputation in the United States, with approximately 150,000 non-traumatic lower-extremity

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amputations occurring annually. Approximately one to three percent of all patients with PAD undergo major amputation annually, and among those with CLTI, 25% to 30% undergo limb amputation within one year of diagnosis.

Mortality

Patients with PAD suffer from an increased risk for cardiovascular mortality in all stages of the disease: there is a 1.9 times higher risk of cardiovascular death in early stage, 2.6 times higher risk in middle stage and 3.5 times higher risk in late stage. CLTI carries an estimated one-year mortality rate of 24% and a five-year mortality rate of approximately 60%. Among patients aged 65 and older who underwent limb amputation, nearly 50% died within one year after surgery.​

The Clinical and Economic Burden of PAD

The majority of PAD’s economic burden stems from frequent and recurring hospitalizations and repeat revascularizations, a procedure to improve blood flow. Total direct costs attributable to PAD in the United States have been estimated at approximately $21 billion.

Amputation frequently results in job loss, disability and heightened risk of subsequent complications, including osteomyelitis, further burdening healthcare resources, reducing economic productivity and generating costs that extend well beyond the acute procedural episode.

Quality of Life and Productivity

PAD impairs quality of life across physical, functional and psychological domains. The 2024 American College of Cardiology/American Heart Association, or ACC/AHA, multisociety guideline identified impaired quality of life, walking performance and functional status as central features of the disease. Intermittent claudication, the hallmark symptom of symptomatic PAD, can limit daily activities and mobility, reduce exercise capacity and cause progressive functional decline.

Current Standard of Care and Limitations

Current pharmacological management of PAD is organized around several complementary drug classes with distinct pathophysiological mechanisms. Despite meaningful advances, significant residual cardiovascular and limb event risk persists across treated populations, demonstrating a significant unmet need.

Lipid-Lowering Agents

High-intensity statin therapy is a cornerstone of PAD pharmacological management, recommended as first-line treatment to achieve at least a 50% reduction in LDL cholesterol.

Antiplatelet and Antithrombotic Agents

Antiplatelet therapy is a Class I recommendation for patients with symptomatic PAD and established ASCVD. Single antiplatelet therapy with aspirin or clopidogrel is a standard treatment for PAD.

Antihypertensive Agents

Blood pressure control to a target of less than 130/80 mmHg is a Class I recommendation in both ASCVD and PAD. Angiotensin-converting enzyme inhibitors and angiotensin receptor blockers carry specific guideline endorsement for patients with PAD, with selective use recommended to reduce MACE, independent of blood pressure lowering.

Glucose-Lowering Agents with Cardiovascular Benefit

In patients with type 2 diabetes and established PAD, glucagon-like peptide-1 receptor agonists, or GLP-1 RAs, including liraglutide, semaglutide and dulaglutide, and sodium-glucose cotransporter-2 inhibitors, including empagliflozin, canagliflozin and dapagliflozin, carry Class I recommendations to reduce MACE.

Anti-Inflammatory Agents

Low-dose colchicine has been incorporated into the 2024 European Society of Cardiology guidelines as adjunctive therapy for patients with elevated RIR despite optimized lipid-lowering treatment, reflecting a growing recognition of the inflammatory pathway as a therapeutic target in chronic ASCVD. Additionally, in a small

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exploratory clinical trial, canakinumab reduced inflammatory biomarkers and improved mean walking distance, or MWD, in patients with PAD.​

Claudication-Specific Therapy

Cilostazol, a phosphodiesterase-3 inhibitor, is the only pharmacological agent with a Class I recommendation specifically for the improvement of claudication symptoms and walking distance in patients with PAD.

ASCVD

Atherosclerosis is a multi-factorial disease characterized by the accumulation of cholesterol-containing lipoproteins and the resultant inflammatory response in the arterial wall. Significant residual risk persists despite management of traditional cardiovascular risk factors. Among patients with well-managed traditional risk factors such as elevated lipids, hypertension, diabetes and smoking, approximately 33% have elevated markers of chronic systemic inflammation as measured by hsCRP, which has been shown to identify individuals at high residual risk of cardiovascular events in multiple large cohort studies.

The 2025 ACC Scientific Statement on Inflammation and Cardiovascular Disease identified targeted anti-inflammatory interventions as a strategic priority in ASCVD care. Despite this unmet need, approved anti-inflammatory therapies for ASCVD remain scarce; low-dose colchicine is currently the only anti-inflammatory agent with a specific indication for secondary CVD prevention, but it is not widely used.

Prevalence and Morbidity

ASCVD is one of the most prevalent chronic diseases in the United States. The diagnosed prevalence of ASCVD is estimated to be approximately 8%, representing approximately 20 million adults in the United States with angina, stroke, heart attack or coronary heart disease. This estimate excludes PAD, transient ischemic attack and undiagnosed ASCVD, which suggests that the actual prevalence of ASCVD may be higher.

Mortality

CVD was the underlying cause of approximately 900,000 deaths in the United States in 2022. Heart disease and stroke together claim more lives than all forms of cancer and chronic lower respiratory disease combined.

The Clinical and Economic Burden of Cardiovascular Disease

The aggregate economic burden of CVD in the United States is substantial and growing, and drives significant annual costs.

Quality of Life and Productivity

ASCVD adversely affects patients across physical, functional and psychological domains. Globally, ASCVD accounts for approximately 261 million disability-adjusted life years annually. Financial toxicity, defined as healthcare spending that consumes a disproportionate share of household income, has been associated with reduced use of preventive medications and worse cardiovascular outcomes. Among individuals under age 65, productivity losses attributable to CVD exceed direct healthcare spending, underscoring the broader economic impact of premature and undertreated disease.

Our Solution

Ruvonoflast

Our lead asset, ruvonoflast, is a small molecule inhibitor of NLRP3. NLRP3 activation triggers formation of the NLRP3 inflammasome, an intracellular protein complex involved in the body’s inflammatory response. When NLRP3 becomes chronically activated, it stimulates a cascade of inflammatory mediators that drive the development and progression of cardiovascular and neuroinflammatory disease. This phenomenon, known as RIR, refers to the ongoing inflammatory burden that contributes to disease progression even when risk factors are well controlled. Ruvonoflast is a tissue- and brain-penetrant prodrug that is designed to release its active metabolite intracellularly. The active metabolite binds to the NLRP3 protein and inhibits formation of the active NLRP3 complex. This strategy utilizes the physiochemical properties of ruvonoflast to achieve high levels of tissue and brain penetration, where the intracellular conversion to the active metabolite further increases the concentration within cells.

Nonclinical studies have shown that NLRP3 primarily responds to damage signals. As a result, while upstream inhibition of NLRP3 significantly reduces the production of IL-1β, IL-18, and IL-6, each of these cytokines,

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which are important for mounting an immune response to infection, may be activated through non-NLRP3-mediated infection signals. We believe this may contribute to our observation of no increased rate of infections or serious infections in clinical trials for patients treated with ruvonoflast compared to placebo.​

Therefore, we believe the mechanism of selective upstream NLRP3 inhibition with ruvonoflast may offer advantages over monoclonal antibody blockade of downstream cytokines by more broadly inhibiting pathogenic inflammation while preserving broader immune function.  In addition, ruvonoflast is an oral small molecule engineered for high levels of penetration beyond the blood vessels where inflammation causes disease, such as in the peripheral tissues and in the brain. Ruvonoflast is also suitable for formulation in fixed dose combinations.

Key Nonclinical and Clinical Data

Ruvonoflast has been extensively studied in five completed trials and has a nonclinical foundation evaluating target engagement, efficacy, safety and toxicology. To date, none of our clinical trials have been conducted in patients with PAD. The clinical evidence supporting our PAD development program consists of data generated in cardiometabolic and neuroinflammatory populations showing reductions in hsCRP and other relevant biomarkers, together with the biological rationale derived from the established role of NLRP3-mediated inflammation in PAD progression.

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Nonclinical Data

In nonclinical studies, ruvonoflast produced improvements in NLRP3-sensitive cardiometabolic biomarkers and neuroinflammatory endpoints in diet-induced obese mice, both as monotherapy and in combination with the GLP-1 RA, semaglutide. Across studies, anti-inflammatory effects were observed both centrally and peripherally, alongside favorable changes in inflammation-related cardiovascular biomarkers, highlighting the potentially broad biological impact of ruvonoflast. Additionally, the comprehensive nonclinical safety data supported chronic clinical development with exposure margins that support all planned clinical doses.

Phase 1 - Ruvonoflast - 001 – SAD/MAD in Healthy Volunteers

We conducted a Phase 1 trial evaluating ruvonoflast in 78 healthy volunteers in a SAD and MAD study. The data from that trial indicated that ruvonoflast was generally well tolerated. The Phase 1 data demonstrated that levels of inflammation returned to baseline within seven days after treatment discontinuation. Similar numbers of infections were observed in participants treated with ruvonoflast as compared with placebo. One participant who had a lower respiratory infection on ruvonoflast demonstrated a normal ability to mount an appropriate infection response to clear the infection while remaining on treatment.

At dose levels ranging from 1 mg to 300 mg, single doses of ruvonoflast were rapidly absorbed as evidenced by peak levels of ruvonoflast detected at 30 minutes post dosing, and efficiently converted to its active metabolite. In this trial, participants received ruvonoflast twice daily for 14 days at dose levels of 30 mg, 100 mg and 300 mg. Ruvonoflast was generally well tolerated, with the majority of treatment-emergent adverse events, or TEAEs, being mild and unrelated to study drug. Inflammatory markers, hsCRP and fibrinogen, were reduced from baseline in the 100 mg and 300 mg dose groups and ruvonoflast was rapidly absorbed and showed no evidence of accumulation. Further, concentrations of the active metabolite were detected in the cerebrospinal fluid, or CSF, suggesting that ruvonoflast may be suitable for the treatment of inflammatory disorders of the CNS. No serious adverse events were observed.​

Phase 1b – Ruvonoflast - 002 - Neuroinflammation in Patients with Parkinson’s Disease

We conducted a Phase 1b clinical trial evaluating ruvonoflast in two cohorts at 150 mg twice daily. The primary endpoints were safety (as measured by TEAEs) and tolerability endpoints (changes in clinical laboratory tests, vital signs, electrocardiogram parameters and physical examination findings), and the secondary endpoints were pharmacokinetics and pharmacodynamics. The first cohort consisted of four healthy volunteers who were dosed for seven days and the second cohort consisted of 10 patients with Parkinson’s disease who were dosed for 28 days. Ruvonoflast was generally well tolerated in both cohorts. The most common observed TEAEs in ruvonoflast-treated patients were headache (related to lumbar puncture), nausea, back or muscle pain, pre-syncope, dizziness, myalgia and constipation. The majority of TEAEs were mild, self-limited and resolved without intervention by the end of the trial, and deemed unrelated to ruvonoflast. A favorable pharmacokinetic profile was observed with consistent absorption and a gradual decline in ruvonoflast concentrations over time. The half-life was nine hours for ruvonoflast and 14 hours for the active metabolite. We observed no difference in exposure between healthy volunteers and patients with Parkinson’s disease. Exposure of the active metabolite was confirmed in the CSF of all patients with Parkinson’s disease. Pharmacodynamic data were suggestive of an anti-neuroinflammatory effect, as demonstrated by reductions in CSF biomarkers such as IL-1β, IL-6, CCL2, IL-8 and CXCL-1, and of a peripheral anti-inflammatory effect, as demonstrated by reductions in hsCRP, fibrinogen, plasma IL-6 and free IL-18. Many patients did not have elevated baseline inflammatory markers; however, among patients with elevated baseline hsCRP, reductions

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exceeded 75%. Responses were observed at all time points up to 28 days, which we believe may support studying dosing with ruvonoflast over longer periods in future trials. No serious adverse events were observed.​

Figure 2: CSF biomarker data showing reduced IL-1β and IL-6 levels after treatment with ruvonoflast 150 mg twice daily, or BID, in patients with Parkinson’s disease, highlighting the compound’s anti-neuroinflammatory and anti-inflammatory activity (n=10 patients with Parkinson’s disease; n=4 healthy volunteers). Clarke et al., Movement Disorders, 2025.

Phase 1b – Ruvonoflast - 003 – Patients with Cardiovascular Risk

We conducted a double-blind, placebo-controlled Phase 1b clinical trial in 63 patients with elevated cardiovascular risk. At baseline, inclusion criteria for patients included hsCRP at least 2.5 mg/L and body mass indexes between 30 and 40 kg/m², plus dyslipidemia, hypertension or type 2 diabetes. Patients received oral ruvonoflast at 225 mg twice daily. Active and placebo groups were maintained on a 2,000 kcal/day diet. The primary endpoint was change in hsCRP at day 28, with changes from baseline in inflammatory markers and body weight serving as secondary endpoints. Median hsCRP at baseline was 5.7 mg/L across all participants, which was reduced by 82% with ruvonoflast. In the comparator arm, where identical weight loss was induced with calorie restriction to 2,000 kcal/day, there was a 37% reduction in hsCRP, consistent with prior literature on hsCRP reduction with weight loss. As weight loss and maintenance of a healthy weight is central to the management of ASCVD, significant additive hsCRP reduction observed with ruvonoflast on top of calorie restriction suggests the potential for use in combination with the current standard of care. HsCRP returned to baseline seven days after treatment discontinuation.​

Additionally, there were statistically significant reductions versus placebo in the key biomarkers: fibrinogen IL-18 and IL-6 at days 7 and 28. Because both groups were calorie-restricted, similar mean body weight reductions were observed for ruvonoflast and placebo. Exploratory analyses of percentage change in calculated free IL-18 also showed statistically significant reductions versus placebo at days 7 and 28 using post-hoc testing. Four ruvonoflast-treated patients discontinued treatment due to TEAEs, including transient asymptomatic transaminitis, urticaria and an allergic reaction, although overall adverse events were similar between the ruvonoflast-treated and placebo groups. No serious adverse events were observed among the ruvonoflast-treated patients.​

Phase 2 – RESOLVE-1 - Patients with Cardiometabolic Disease​

RESOLVE-1 is a randomized, multi-center, double-blind placebo-controlled Phase 2 trial that enrolled 176 cardiometabolic patients for six months. Approximately half of the patients had a diagnosis of type 2 diabetes. Patients received ruvonoflast 150 mg daily or twice daily, or placebo in three equal groups. All patients were allowed to continue on concomitant medications. The primary endpoints were hsCRP reduction and weight loss and the secondary endpoints were a series of biomarkers. The patients in the groups were well balanced between the diabetic cohort and the non-diabetic cohort, and they were well balanced in both cohorts across age, gender, race, ethnicity, weight and median hsCRP at baseline. Participants were originally consented with weight loss as the only primary endpoint, but before the study was completed, the study protocol was amended to include the hsCRP primary endpoint, and a hierarchical statistical testing strategy was implemented to test the inflammatory endpoint first.

Reduction in hsCRP up to week 24 was statistically significant at both doses in participants with and without diabetes when tested in all randomized participants using an intent-to-treat (ITT) analysis. Reduction in hsCRP up to week 24 was statistically significant at 150 mg BID in both diabetic and non-diabetic participants

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and at 150 mg QD in diabetic participants, when tested using a pre-defined modified intention (mITT) to treat analysis. The mITT excluded some participants from the analysis, for example those with clinical symptoms of and/or a diagnosis of an infection, and those with substantial missing data. The primary endpoint of weight loss was not met.

Elevated hsCRP was not an inclusion criterion in RESOLVE-1, and median hsCRP at baseline was 2.85 and 3.70 mg/L in the populations dosed with 150 mg QD and 150 mg BID ruvonoflast, respectively. In contrast, elevated baseline hsCRP was an inclusion criterion for the previous Phase 1b clinical trial, defined as hsCRP >2.5 mg/L, and median baseline hsCRP was 6.42 mg/L in ruvonoflast-treated participants. Therefore, to enable cross-trial comparison, we conducted a post-hoc analysis of a subgroup of patients in the ruvonoflast group within the RESOLVE-1 trial who had baseline hsCRP >3 mg/L, and who also demonstrated adherence using predefined trough study drug concentrations which were measured throughout the trial. Median hsCRP at baseline in this “inflamed and adherent” subgroup was 5.08 and 4.75 mg/L at 150 mg QD and 150 mg BID ruvonoflast, respectively. Geometric LS mean hsCRP was reduced by 56.4% at 150 mg QD and by 62.6% at 150mg BID ruvonoflast at week 4. Furthermore, hsCRP was reduced below 2 mg/L in 33% and 40% of patients at 150 mg QD and 150 mg BID ruvonoflast, respectively. There were reductions in additional biomarkers in the “inflamed and adherent” population. Median IL-6 was reduced by 38.2% and 62.3%, and median fibrinogen was reduced by 19.0% and 17.8% at 150 mg QD and 150 mg BID, respectively. These reductions show consistent dose-dependent reductions when compared with those measured in the ruvonoflast-003 Phase 1b study at 450 mg daily of ruvonoflast.​

Importantly, the safety profile of ruvonoflast was confirmed and strengthened by the six-month duration of dosing data obtained in RESOLVE-1. There was no imbalance in the adverse event profile across the active and placebo dosed groups and similarly, no imbalance was observed in the incidence of infections. There were no serious or severe infections. Furthermore, when looking at liver function tests there were two patients with elevations outside the normal range – one on placebo (with diverticulitis), and one on 150 mg QD ruvonoflast that occurred after strenuous exercise and correlated with creatine kinase elevation, supporting the muscular origin of the enzyme. Three ruvonoflast-treated patients experienced serious adverse events that were determined not to be related to treatment: hyperglycemia related to an underlying medical condition of type 2 diabetes; a preexisting medical condition that was subsequently identified as B-cell lymphoma; and subdural hematoma following an injury-related concussion. The most common reasons for premature study discontinuation were not related to adverse events. ​

We believe RESOLVE-1 data in concert with the data from our 003 Phase 1b trial demonstrate a dose-dependent effect of ruvonoflast and provide supporting information for our planned Phase 3 doses for evaluation of ruvonoflast in PAD at 225 mg BID.​

Phase 2 – RESOLVE-2 – Patients with Cardiometabolic Disease​

RESOLVE-2 was a randomized placebo-controlled Phase 2 trial that enrolled 82 cardiometabolic patients for eight months to explore the effect of ruvonoflast in addition to GLP-1 therapy. Patients naïve to GLP-1 RA therapy were started on semaglutide and dose titrated over 20 weeks in accordance with the FDA-approved regimen for weight loss, and patients were randomized to receive either ruvonoflast or placebo from study start. Patients received oral ruvonoflast at 150 mg twice daily. The primary objective of the trial was assessment of the safety and tolerability of ruvonoflast as an adjunct to semaglutide. The safety profile was consistent with prior ruvonoflast clinical trial experience and comparable to semaglutide monotherapy. There was no imbalance in the adverse event profile between the groups and similarly, no imbalance in the incidence of infections or serious infections. Two ruvonoflast-treated patients experienced serious adverse events that were determined not to be treatment related: a urinary tract infection and a viral hepatitis infection causing acute liver injury, transaminitis, and hyperbilirubinemia. Comparable weight loss was observed with ruvonoflast as an adjunct to semaglutide, compared to semaglutide monotherapy.​

The trial demonstrated statistically significant reductions in hsCRP with ruvonoflast as an adjunct to semaglutide compared to semaglutide monotherapy. Further, among participants with baseline hsCRP ≥2 mg/L, ruvonoflast plus semaglutide more than doubled the likelihood of achieving hsCRP<2 mg/L, below the threshold for elevated RIR, versus semaglutide alone. The effect was observed in a study population without a baseline hsCRP entry criterion, providing early data on the potential benefit of NLRP3 inhibition as an adjunctive strategy for patients receiving GLP-1 RA therapy.​

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Clinical Development Plan

We have recently begun recruitment for sites and participants for an observational study, REVEAL-PAD, which is planned to enroll PAD in the first half of 2027 patients to characterize key aspects of the disease such as baseline inflammation markers, functional capacity, quality of life and other features of the disease. The study is also intended to create a pool of patients who may also be eligible to be screened for our planned Phase 3 trial. Over time, REVEAL-PAD may also contribute background information on rates of limb and cardiovascular events in PAD, for comparison with rates observed in the active and extension phases of our planned Phase 3 PAD trial.

We intend to conduct a single randomized, placebo-controlled Phase 3 clinical trial of ruvonoflast in patients with PAD, RELIEVE-PAD. Following our discussions with regulatory authorities on the design of this trial, we currently plan to enroll approximately 800 patients who have pain with walking, are able to walk 200 meters and who have an ankle-brachial index <0.9 and inflammation as measured by >2 mg/L hsCRP. These patients will be randomized to either ruvonoflast at 450 mg/day or placebo and followed and evaluated for approximately 12 months. The primary endpoint of RELIEVE-PAD will be MWD, a functional endpoint. Subsequently, we plan to explore the impact of ruvonoflast on the timing and severity of limb events and cardiovascular events. We believe this Phase 3 trial design, if successful, could support approval in PAD, with the opportunity to expand into other broader indications in the ASCVD cardiometabolic space. After initial discussions with the FDA, EMA and MHRA, we have aligned on core elements of the Phase 3 trial design. It is important to note that some elements of this design may change following further discussions with regulatory authorities, and we may conduct additional nonclinical studies or clinical trials in advance of, or concurrently with, the planned Phase 3 trial. We currently plan to read out data from our planned Phase 3 trial in 2028.

We are also planning to enroll PAD patients in a separate imaging study, REIMAGINE-PAD, that will run concurrently with the Phase 3 trial. REIMAGINE-PAD will visualize and quantify the impact of ruvonoflast on tissue inflammation in the legs as well as the rest of the body using a whole-body PET imaging using tracers that can detect inflammation in tissues. REIMAGINE-PAD is designed to examine the level of inflammation at baseline, then explore the effect of ruvonoflast or placebo over six months in a 90-day treatment phase crossover design.

Trabzanoflast (neurology)

We intend to build on the observed brain-penetrant features of trabzanoflast by developing it for neurologic diseases where inflammation is a key driver in the etiology and progression of the disease. Trabzanoflast is a novel pyridazone-based NLRP3 inhibitor designed to achieve high levels of brain penetration. In nonclinical studies, trabzanoflast demonstrated activity in both in vitro and in vivo NLRP3 models and reduced neuroinflammation in CNS animal models.​

Phase 1 - SAD/MAD in Healthy Volunteers

We conducted a Phase 1 trial evaluating trabzanoflast in 55 healthy volunteers in a SAD and MAD study. The primary objective of the trial was assessment of safety and tolerability. The trial also evaluated pharmacokinetics and effects on inflammatory biomarkers. The data from the trial indicated that trabzanoflast was generally well tolerated. Pharmacokinetic analysis showed dose-proportional exposure across all doses tested in both the single and multiple ascending dose phases of the trial. The majority of TEAEs were mild and unrelated to study drug. No serious adverse events were observed and there were no clinically significant changes in

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laboratory values relevant to safety monitoring following treatment with trabzanoflast. The study also showed target engagement in both CSF and blood. Continuous CSF sampling at steady state confirmed high CNS exposure, with a Kp,uu of 1.9. Based on nonclinical data, brain exposure to trabzanoflast is expected to remain above the IC₉₀ at trough. We believe these data suggest that trabzanoflast may be suitable for the treatment of inflammatory disorders of the CNS.  

Competition

Ruvonoflast

The market for therapies targeting inflammation is highly competitive and includes approved products as well as investigational product candidates in development that target downstream inflammatory mediators. We discuss our competitive landscape in three categories– target, pathway and indication.

Target (NLRP3)

We face target competition from companies developing oral small-molecule NLRP3 inhibitors for cardiovascular, metabolic, inflammatory and neurodegenerative diseases. These companies include but are not limited to, Novartis AG, or Novartis, (DFV890), AstraZeneca PLC (AZD-4144), Eli Lilly & Company, or Eli Lilly, (parunoflast, VTX2735), F. Hoffmann-La Roche Ltd., or Roche, (selnoflast), Ventus Therapeutics U.S., Inc., or Ventus, (VENT-02), BioAge Labs, Inc. (BGE-102), Tenvie Therapeutics, Inc. (TNV262), Brenig Therapeutics, Inc., or Brenig, (BT-409), InSilico Medicine, Inc., or InSilico, (ISM8969), Neumora Therapeutics, Inc. (NMRA-215) and Neurocrine Biosciences, Inc. (TT-02332). Additionally, a NIMA-related kinase 7 degrader is being developed by Monte Rosa Therapeutics, Inc. (MRT-8102). All of these programs are at an earlier stage of development than ruvonoflast, and we believe ruvonoflast is further differentiated by its high levels of tissue and brain penetration, which are designed to facilitate penetration into the local sites of inflammation that lie outside systemic circulation.

Pathway

We face pathway competition from companies targeting individual cytokines downstream from NLRP3 such as IL-6 and IL-1β. These companies include but are not limited to, Novartis (pacibekitug), CSL Limited, or CSL, (clazakizumab) and Novo Nordisk (ziltivekimab). Evidence from clinical studies of IL-1β and IL-6 blocking antibodies has validated inflammation as a therapeutic target in CVD. However, these approaches target individual cytokines downstream of NLRP3 activation. Because NLRP3 activation drives production of IL-1β, IL-18 and downstream IL-6 signaling, the upstream inhibition of NLRP3 may provide broader suppression of inflammatory pathways implicated in CVD than inhibition of any single downstream mediator.

Indication

There are no approved products in the United States that specifically modify the underlying inflammatory and atherosclerotic disease processes that drive progression of PAD, but we also face indication competition from approved products for ASCVD that include lipid-lowering agents, antihypertensive therapies, antiplatelet agents, anticoagulants and claudication therapies. We also face competition in PAD from companies with development candidates. Novo Nordisk is developing semaglutide and Eli Lilly is developing orforglipron, both with dedicated clinical trials in PAD. While these approved products have demonstrated benefits in reducing cardiovascular and limb-related events or improving walking performance, none directly targets the inflammatory mechanisms that drive PAD. We believe that ruvonoflast has the potential to provide a more favorable product profile than treatments whose anti-inflammatory effects are off-mechanism.

In ASCVD more broadly, approved anti-inflammatory products include colchicine marketed by Agepha, which has demonstrated reductions in cardiovascular events in secondary prevention populations. Additionally, approved products targeting lipid metabolism like statins and proprotein convertase subtilisin/kexin type 9 inhibitors and incretins such as semaglutide and tirzepatide provide indirect anti-inflammatory benefits. There are three monoclonal antibodies in development for ASCVD, ziltivekimab developed by Novo Nordisk, clazakizumab developed by CSL and pacibekitug developed by Novartis. We believe these agents may be associated with an increased risk of infection and scale and adoption may be further limited by their injectable route of administration.

Trabzanoflast (neurology)

There are currently no NLRP3 inhibitors approved specifically for neuroinflammatory indications. Multiple companies are pursuing approaches targeting neuroinflammatory pathways, including NLRP3 inhibition, microglial modulation, alpha-synuclein biology and other mechanisms. Direct competitors in NLRP3 inhibition for neurology include Eli Lilly, AC Immune SA, Roche, Ventus, Brenig Therapeutics, InSilico/Hygtia Therapeutics Co., Ltd. and Zydus Lifesciences Ltd.

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Commercialization Strategy

Target Market

PAD represents a significant unmet medical need and a substantial healthcare burden. PAD affects an estimated 20 million adults in the United States. Patients with PAD face an increased risk of cardiovascular events, disease progression and limb-related complications, particularly in more advanced stages of disease.

We selected PAD for several compelling reasons: it represents a large patient population in the United States; inflammation plays a central role in both the onset and progression of the disease; there remains significant disease severity and unmet medical need; and the competitive landscape is relatively limited. In addition, patients with PAD are typically managed within a concentrated network of centers of excellence, making the indication well suited for a focused and efficient commercialization strategy.

Disease progression and PAD can be segmented according to the Fontaine functional classification as follows:

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Fontaine I – early stage, no pain on walking, approximately 10 million U.S. patients, mostly undiagnosed;

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Fontaine II – middle stage, pain on walking, approximately 8.5 million diagnosed U.S. patients; and

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Fontaine III – late-stage disease, restricted walking, risk of limb events, approximately 1.4 million diagnosed U.S. patients.

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Fontaine IV - late-stage disease, tissue loss, ulcers, approximately 600,000 diagnosed U.S. patients.

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Our initial focus for the development and launch of ruvonoflast is to demonstrate functional improvement in MWD and in quality of life in patients with Fontaine Class II PAD who retain the ability to walk approximately 200 meters without claudication. We believe this population is at a stage of disease where treatment may have the potential to slow disease progression while improving functional outcomes, thereby delaying or reducing the severity of downstream CLTI. We aim to generate data on the frequency and severity of limb events in this population, and to follow the impact of ruvonoflast in patients who progress to Class III/IV. In addition, we believe that demonstrating disease-specific activity for ruvonoflast in Fontaine Class II patients could contribute to earlier diagnosis among the large group of U.S. patients in Class I, because the availability of an approved therapy to address RIR may motivate earlier identification of patients and initiation of treatment. We discussed the design of our planned PAD study at a meeting with FDA in July 2026 and achieved alignment on several areas of our study design.

PAD exhibits a high degree of regional concentration based on demographics and relevant health habits, such as smoking. As a result, care is largely delivered in large reference centers across the United States and elsewhere. In our target segment of Fontaine II patients, care is delivered by cardiologists, preventive cardiologists and high-volume internists associated with centers of excellence.

Go-to-Market Approach

PAD is concentrated in the United States, tracking the prevalence of associated risk factors such as smoking and diabetes. As a result, care is highly concentrated in about 50 centers of excellence in the United States. We plan to execute a center of excellence based approach. We intend to recruit, train and deploy a specialized commercial organization comprised of key account managers, key opinion leader liaisons and reimbursement specialists to support our ability to independently launch ruvonoflast in PAD, if approved.

We believe that strongly differentiated functional improvement, MWD and quality of life, supported by the potential to delay and/or reduce the severity of critical limb ischemia, could support sustainable, differentiated pricing in PAD.

Manufacturing and Supply Chain

Manufacturing Overview

We do not own or operate, and currently have no plans to establish, manufacturing facilities. We collaborate with well-established third-party suppliers to handle GMP, drug substance production of our lead asset, ruvonoflast, and its conversion to drug product. We anticipate scaling the manufacture of our ruvonoflast drug substance and drug product through third-party suppliers. We plan to have a phased approach to commercial production that will ensure we can meet the projected commercial demand.

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Supply Chain and Raw Materials

We currently rely on a single supplier of ruvonoflast drug substance and our proprietary material, but intend to enter into agreements with additional suppliers for commercial supply. As our product candidates advance through development, we expect to enter into longer-term commercial supply agreements with key suppliers and manufacturers to fulfill and secure our production needs.

Quality and Compliance

The quality and compliance function is currently outsourced to an established provider to the industry, QRC Consultants Limited. Our Quality Management System is designed to support a virtual operating model in which all GMP activities are performed by third-party contract development and manufacturing organizations, or CDMOs, and testing laboratories.

We select CDMOs based on technical capability, regulatory history, capacity and quality performance. Drug substance manufacturing is fully outsourced to GMP-certified suppliers with expertise in the relevant synthetic and analytical technologies. Drug product manufacturing, packaging and testing are performed by specialized CDMOs with capabilities appropriate for our products.

We maintain written quality agreements with each CDMO that define roles, responsibilities and expectations for GMP compliance. We ensure alignment with applicable International Council for Harmonisation and GMP expectations through our supplier qualification program, routine audits and ongoing oversight of our CDMOs.

We have not been subject to FDA inspections relating to cGMP or bioresearch monitoring. Our CDMOs are subject to routine inspections by the FDA and/or other regulatory authorities. We review inspection outcomes and incorporate this information into our vendor oversight and risk-management processes.

Intellectual Property

We seek to protect the intellectual property and proprietary technology that we consider important to our business, including by pursuing patent applications that cover ruvonoflast (formerly known as NT-0796) and trabzanoflast (formerly known as NT-0150), combination therapies that include these compounds, and future product candidates or combination therapies we may develop, and methods of using the same, as well as any other relevant inventions and improvements that we believe to be commercially important for the development of our business. We strive to protect and enhance the proprietary technology, inventions and improvements that are commercially important for the development of our business, including seeking, maintaining and defending patent rights in the United States and elsewhere directed to our proprietary technology, inventions, improvements and product candidates (including compositions, methods of use, dosing and formulations). We may also pursue patent protection for our manufacturing and drug development processes and technologies. We also rely on know-how and continuing technological innovation to develop and maintain our proprietary and intellectual property position as well as trade secrets, data exclusivity, market exclusivity and patent term extensions, where available.

Our commercial success will depend, in part, on our ability to obtain, maintain, enforce and protect our intellectual property and other proprietary rights for the technology, inventions and improvements we consider important to our business, to defend any patents we may own or in-license in the future, to prevent others from infringing any patents we may own or in-license in the future, to preserve the confidentiality of our trade secrets, and to operate without infringing, misappropriating or otherwise violating the valid and enforceable patents and proprietary rights of third parties.

Issued patents can provide protection for varying periods of time, depending upon the date of filing of the patent application, the date of patent issuance and the legal term of patents in the countries in which they are obtained. In the United States, if all maintenance fees are timely paid, the natural expiration of a patent is generally 20 years from its earliest U.S. non-provisional filing date. In addition, in certain instances, the term of an issued U.S. patent that covers or claims an FDA- approved product can be extended to recapture a portion of the term effectively lost as a result of the FDA regulatory review period, which is called patent term extension. The restoration period cannot be longer than five years and the total patent term, including the restoration period, must not exceed 14 years following FDA approval. In Europe (including Great Britain), if annual renewal fees are timely paid, the natural expiration of a patent is generally 20 years from the date of

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filing of the application. As in the United States, patent term extensions are available in Europe in the form of a Supplementary Protection Certificate, or SPC, which allows a maximum period of extension of five years (the formal calculation is the date of the first marketing authorization (MA) in the EEA minus the basic patent filing date, minus five years). The term of patents outside of the United States and Europe varies in accordance with the laws of the foreign jurisdiction, but is typically also 20 years from the earliest effective non-provisional filing date. However, the actual protection afforded by a patent varies on a product-by-product basis, from country-to-country and depends upon many factors, including the type of patent, the scope of its coverage, the availability of regulatory-related extensions, the availability of legal remedies in a particular country and the validity and enforceability of the patent.

The patent position of pharmaceutical and biotechnology companies is generally highly uncertain, involves complex legal and factual questions, and has been the subject of much litigation in recent years. As a result, the issuance, scope, validity, enforceability and commercial value of our patent rights are highly uncertain. Our pending and future patent applications and those of our collaborators may not result in patents being issued that protect our product candidates or that effectively prevent others from commercializing competitive product candidates. Moreover, obtaining and enforcing patents in the pharmaceutical and biotechnology industries is inherently uncertain, due in part to ongoing changes in the patent laws. Changes in either the patent laws or the interpretations of patent laws in the United States and other countries may diminish the value of our intellectual property and may increase the uncertainties and costs surrounding the prosecution of patent applications and the enforcement or defense of issued patents. Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses and could distract our technical and management personnel from their normal responsibilities.

Patent expiration dates noted in the following paragraphs refer to statutory expiration dates and do not take into account any potential patent term adjustment or extension that may be available.

Patent Rights Relating to Ruvonoflast and Trabzanoflast

As of December 31, 2025, we own a number of patent families directed to each of our ruvonoflast and trabzanoflast product candidates, which are described in the following section.

Ruvonoflast

We own a patent family that generically and specifically covers ruvonoflast with patents granted in Australia, China, Hong Kong, Eurasia, Europe (validated in over 35 jurisdictions), Israel, India, Japan, Korea, Macau, Mexico, New Zealand, Singapore, Taiwan, the United States and South Africa and pending applications in Brazil, Canada and the United States. The patents in this family are expected to expire in July 2038, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees. These patents could extend to July 2043 pending patent term extension.

We also own a second patent family directed to crystalline forms of ruvonoflast that includes applications pending in Australia, Brazil, Canada, China, Eurasia, Europe, Hong Kong, India, Israel, Japan, Mexico, New Zealand, Singapore, South Africa, South Korea and the United States. Any patents in this family, if issued, are expected to expire in October 2043, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

We also own a third patent family directed to a process of preparing ruvonoflast that includes applications pending in Australia, Brazil, Canada, China, Eurasia, Europe, Hong Kong, India, Israel, Japan, Mexico, New Zealand, Singapore, South Africa, South Korea and the United States. Any patents in this family, if issued, are expected to expire in October 2043, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

We also own a fourth patent family directed to an additional process of preparing ruvonoflast that is pending as a PCT application. Any patents in this family, if issued, are expected to expire in March 2045, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

We also own a fifth patent family directed to a ruvonoflast formulation that is pending as a PCT application. Any patents in this family, if issued, are expected to expire in March 2046, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

We also own a sixth patent family directed to use of ruvonoflast for the treatment of peripheral arterial disease that is pending as a priority application in U.K. Any patents in this family, if issued, are expected to expire in April 2047, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

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Trabzanoflast

We own a patent family that generically and specifically covers trabzanoflast and includes applications pending in Australia, Brazil, Canada, China, Egypt, Eurasia, Europe, Hong Kong, India, Indonesia, Israel, Japan, Korea, Malaysia, Mexico, New Zealand, Philippines, Saudi Arabia, Singapore, South Africa, and the United States. Any patents in this family, if issued, are expected to expire in January 2043, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees. These patents could extend to January 2048 pending patent term extension.

Combination Therapies

In addition to the patents and patent applications described above, we also own a patent family directed to a combination of an NLRP3 inhibitor (including ruvonoflast or trabzanoflast) and a GLP-1 receptor agonist that is pending as a PCT application and non-provisional patent applications pending in Argentina and Taiwan. Any issued patents from this family are expected to expire in January 2045, assuming payment of all appropriate maintenance, renewal, annuity or other governmental fees.

Government Regulation

The FDA and other regulatory authorities at the federal, state and local level, as well as in foreign countries and local jurisdictions, extensively regulate among other things, the research, development, testing, manufacture, quality control, sampling, import, export, safety, effectiveness, labeling, packaging, storage, distribution, record-keeping, approval, advertising, promotion, marketing, post-approval monitoring and post-approval reporting of drugs. We, along with our vendors, CROs and contract manufacturers, will be required to navigate the various nonclinical, clinical, manufacturing and commercial approval requirements of the governing regulatory agencies of the countries in which we wish to conduct studies or seek approval of our product candidates. The process of obtaining regulatory approvals of drugs and ensuring subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of substantial time and financial resources. Even after obtaining initial marketing approval, a product and its manufacturer remain subject to extensive, continuing regulatory requirements, including with respect to manufacturing, quality control, adverse event reporting, advertising and promotion and periodic inspections by regulatory authorities.

In the United States, the FDA regulates drug products under the Federal Food, Drug, and Cosmetic Act, or the FD&C Act, as amended, its implementing regulations and other laws. If we fail to comply with applicable FDA or other requirements at any time with respect to product development, clinical testing, approval or any other legal requirements relating to product manufacture, processing, handling, storage, quality control, safety, marketing, advertising, promotion, packaging, labeling, export, import, distribution or sale, we may become subject to administrative or judicial sanctions or other legal consequences. These sanctions or consequences could include, among other things, the FDA’s refusal to approve pending applications, issuance of clinical holds for ongoing studies, withdrawal of approvals, warning or untitled letters, product withdrawals or recalls, product seizures, relabeling or repackaging, total or partial suspensions of manufacturing or distribution, injunctions, fines, civil penalties or criminal prosecution.

The process required by the FDA before a drug may be marketed in the United States generally involves the following:

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completion of extensive nonclinical studies in accordance with applicable regulations, including studies conducted in accordance with good laboratory practice, or GLP, requirements;

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submission to the FDA of an IND application, which must become effective before clinical trials may begin;

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approval by an IRB or independent ethics committee at each clinical trial site before each trial may be initiated;

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manufacture of the proposed drug candidate in accordance with applicable cGMPs;

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performance of adequate and well-controlled clinical trials in accordance with applicable IND regulations, GCP requirements and other clinical trial-related regulations, to establish the safety and efficacy of the investigational product for each proposed indication;

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submission to the FDA of a new drug application after completion of all pivotal clinical trials;

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a determination by the FDA within 60 days of its receipt of an NDA to accept the filing for review;

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satisfactory completion of one or more FDA pre-approval inspections of the manufacturing facility or facilities where the drug will be produced to assess compliance with cGMP requirements to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity;

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potential FDA audit of the clinical trial sites that generated the data in support of the NDA;

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payment of user fees for FDA review of the NDA; and

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FDA review and approval of the NDA, including consideration of the views of any FDA advisory committee, prior to any commercial marketing or sale of the drug in the United States.

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Nonclinical Studies and Clinical Trials for Drugs

Before testing any drug in humans, the product candidate must undergo rigorous nonclinical testing. Nonclinical studies may include laboratory evaluations of drug chemistry, formulation and stability, as well as in vitro and animal studies to assess safety and in some cases to establish the rationale for therapeutic use. In April 2025, the FDA published a roadmap to reduce animal testing in nonclinical safety studies, including those required in INDs, with scientifically validated new approach methodologies, or NAMs, and in March 2026, the agency published draft guidance on using NAMs in drug development. The conduct of nonclinical studies is subject to federal and state regulations and requirements, including applicable GLP requirements for safety/​toxicology studies. The results of the nonclinical studies, together with manufacturing information and analytical data must be submitted to the FDA as part of an IND. An IND is a request for authorization from the FDA to administer an investigational product to humans and must become effective before clinical trials may begin. Some long-term nonclinical testing may continue after the IND is submitted. The IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA, within the 30-day time period, raises concerns or questions about the conduct of the clinical trial, including concerns that human research patients will be exposed to unreasonable health risks, and imposes a partial or full clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin. Submission of an IND may result in the FDA not allowing clinical trials to commence or not allowing clinical trials to commence on the terms originally specified in the IND.

The clinical stage of development involves the administration of the product candidate to healthy volunteers or patients under the supervision of qualified investigators, generally physicians not employed by or under the trial sponsor’s control, in accordance with GCP requirements, which include the requirements that all research subjects provide their informed consent for their participation in any clinical trial. Clinical trials are conducted under protocols detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria and the parameters and criteria to be used in monitoring safety and evaluating effectiveness. Each protocol, and any subsequent amendments to the protocol must be submitted to the FDA as part of the IND. Furthermore, each clinical trial must be reviewed and approved by an IRB for each institution at which the clinical trial will be conducted to ensure that the risks to individuals participating in the clinical trials are minimized and are reasonable related to the anticipated benefits. The IRB also approves the informed consent form that must be provided to each clinical trial subject or his or her legal representative, and must monitor the clinical trial until completed. The FDA, the IRB or the sponsor may suspend or discontinue a clinical trial at any time on various grounds, including a finding that the patients are being exposed to an unacceptable health risk or that the trial is unlikely to meet its stated objectives. Some studies also include oversight by an independent group of qualified experts organized by the clinical study sponsor, known as a data safety monitoring board, which provides authorization for whether or not a study may move forward at designated check points based on access to certain data from the study and may halt the clinical trial if it determines that there is an unacceptable safety risk for subjects or other grounds, such as no demonstration of efficacy. There also are requirements governing the reporting of ongoing clinical trials and completed clinical trials to public registries. Information about applicable clinical trials, including clinical trial results, must be submitted within specific timeframes for publication on the www.clinicaltrials.gov website.

A sponsor who wishes to conduct a clinical trial outside of the United States may, but need not, obtain FDA authorization to conduct the clinical trial under an IND. When a foreign clinical study is conducted under an IND, all IND requirements must be met unless waived. If a foreign clinical trial is not conducted under an IND, the sponsor must submit data from the clinical trial to the FDA in support of an NDA. The FDA will accept a well-designed and well-conducted foreign clinical trial not conducted under an IND if the trial was conducted in accordance with GCP requirements, including review and approval by an independent ethics committee and use of proper procedures for obtaining informed consent from subjects, and the FDA is able to validate the data through an onsite inspection if deemed necessary.

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Clinical trials to evaluate therapeutic indications to support NDAs for marketing approval are typically conducted in three sequential phases, which may overlap or be combined.

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Phase 1 clinical trials involve initial introduction of the investigational product into healthy human volunteers or patients with the target disease or condition. These studies are typically designed to test the safety, dosage tolerance, absorption, metabolism and distribution of the investigational product in humans, evaluate the side effects associated with increasing doses, and, if possible, to gain early evidence of effectiveness. In the case of some products for severe or life-threatening diseases, such as cancer, especially when the product may be too inherently toxic to ethically administer to healthy volunteers, the initial human testing is often conducted in patients.

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Phase 2 clinical trials typically involve administration of the investigational product to a limited patient population with a specified disease or condition to evaluate the preliminary efficacy, optimal dosages and dosing schedule and to identify possible adverse side effects and safety risks.

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Phase 3 clinical trials typically involve administration of the investigational product to an expanded patient population to further evaluate dosage, to provide statistically significant evidence of clinical efficacy and to further test for safety, generally at multiple geographically dispersed clinical trial sites. These clinical trials are intended to establish the overall risk/benefit ratio of the investigational product and to provide an adequate basis for product approval and physician labeling. Generally, two adequate and well-controlled clinical trials have been required by the FDA for approval of an NDA, although there are known exceptions, particularly for rare diseases. Prior FDA leadership announced in February 2026 that the FDA would, going forward, adopt the default position that one adequate and well-controlled trial, combined with confirmatory evidence, can serve as the basis of approval for novel products, with discretion to require a second pivotal study if confirmatory evidence is insufficient.

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Post-approval trials, sometimes referred to as Phase 4 clinical trials, may be conducted after initial marketing approval. These trials are used to gain additional experience from the treatment of patients in the intended therapeutic indication and are commonly intended to generate additional safety data regarding use of the product in a clinical setting. In certain instances, the FDA may mandate the performance of Phase 4 clinical trials as a condition of approval of an NDA.

Progress reports detailing the results of the clinical trials, among other information, must be submitted at least annually to the FDA. Written IND safety reports must be submitted to the FDA and the investigators fifteen days after the trial sponsor determines the information qualifies for reporting for serious and unexpected suspected adverse event, or AEs, findings from other studies or animal or in vitro testing that suggest a significant risk for human volunteers and any clinically important increase in the rate of a serious suspected adverse reaction over that listed in the protocol or investigator brochure. The sponsor must also notify the FDA of any unexpected fatal or life-threatening suspected adverse reaction as soon as possible but in no case later than seven calendar days after the sponsor’s initial receipt of the information.

Concurrent with clinical trials, companies usually complete additional animal studies and must also develop additional information about the chemistry and physical characteristics of the product candidate and finalize a process for manufacturing the drug product in commercial quantities in accordance with cGMP requirements. The manufacturing process must be capable of consistently producing quality batches of the product candidate and manufacturers must develop, among other things, methods for testing the identity, strength, quality and purity of the final drug product. Additionally, appropriate packaging must be selected and tested, and stability studies must be conducted to demonstrate that the product candidate does not undergo unacceptable deterioration over its shelf life.

U.S. Marketing Approval for Drugs

Assuming successful completion of the required clinical testing, the results of the nonclinical studies and clinical trials, together with detailed information relating to the product’s chemistry, manufacture, controls and proposed labeling, among other things are submitted to the FDA as part of an NDA requesting approval to market the product for one or more indications. An NDA must contain proof of the drug’s safety and efficacy in order to be approved. The marketing application may include both negative and ambiguous results of nonclinical studies and clinical trials, as well as positive findings. Data may come from company-sponsored clinical trials intended to test the safety and efficacy of a product’s use or from a number of alternative sources, including studies initiated by investigators. To support marketing approval, the data submitted must be sufficient in quality and quantity to establish the safety and efficacy of the investigational product to the satisfaction of the FDA. FDA approval of an NDA must be obtained before a drug may be marketed in the United States.

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The FDA reviews all submitted NDAs to determine if they are sufficiently complete to permit substantial review before it accepts them for filing and may request additional information rather than accepting the NDA for filing. The FDA must make a decision on accepting an NDA for filing within 60 days of receipt, and such decision could include a refusal to file by the FDA. Once the submission is accepted for filing, the FDA begins an in-depth substantive review of the NDA. The FDA reviews an NDA to determine, among other things, whether the drug is safe and effective and whether the facility in which it is manufactured, processed, packaged or held meets standards designed to assure the product’s continued safety, quality and purity. Under the goals and policies agreed to by the FDA under the Prescription Drug User Fee Act, or PDUFA, the FDA targets ten months, from the filing date, in which to complete its initial review of a new molecular entity NDA and respond to the applicant, and six months from the filing date of a new molecular entity NDA for priority review. The FDA does not always meet its PDUFA goal dates for standard or priority NDAs, and the review process is often extended by FDA requests for additional information or clarification.

Further, under PDUFA, as amended, each NDA must be accompanied by a user fee. The FDA adjusts the PDUFA user fees on an annual basis. Fee waivers or reductions are available in certain circumstances, including a waiver of the application fee for the first application filed by a small business. Additionally, no user fees are assessed on NDAs for products designated as orphan drugs, unless the product also includes a non-orphan indication.

The FDA also may require submission of a Risk Evaluation and Mitigation Strategy, or REMS, program to ensure that the benefits of the drug outweigh its risks. A REMS is a safety strategy to manage a known or potential serious risk associated with a product and to enable patients to have continued access to such medicines by managing their safe use. The REMS program could include medication guides, physician communication plans, assessment plans and/or elements to assure safe use, such as restricted distribution methods, patient registries or other risk-minimization tools.

The FDA may refer an application for a novel drug to an advisory committee. An advisory committee is a panel of independent experts, including clinicians and other scientific experts, which reviews, evaluates and provides a recommendation as to whether the application should be approved and under what conditions. The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when making decisions.

Before approving an NDA, the FDA typically will inspect the facility or facilities where the product is manufactured. The FDA will not approve an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to ensure consistent production of the product within required specifications. Additionally, before approving an NDA, the FDA may inspect one or more clinical trial sites to assure compliance with GCP and other requirements and the integrity of the clinical data submitted to the FDA.

After evaluating the NDA and all related information, including the advisory committee recommendation, if any, and inspection reports regarding the manufacturing facilities and clinical trial sites, the FDA may issue an approval letter, or, in some cases, a complete response letter. A complete response letter will describe all of the deficiencies that the FDA has identified in the NDA, except that where the FDA determines that the data supporting the application are inadequate to support approval, the FDA may issue the complete response letter without first conducting required inspections, testing submitted product lots and/or reviewing proposed labeling. A complete response letter generally contains a statement of specific conditions that must be met in order to secure final approval of the NDA and may require additional clinical or nonclinical testing in order for the FDA to reconsider the application. Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria for approval. If and when those conditions have been met to the FDA’s satisfaction, the FDA will typically issue an approval letter. An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications.

Even if the FDA approves a product, depending on the specific risk(s) to be addressed, it may limit the approved indications for use of the product, require that contraindications, warnings or precautions be included in the product labeling, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess a drug’s safety after approval, require testing and surveillance programs to monitor the product after commercialization or impose other conditions, including distribution and use restrictions or other risk

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management mechanisms under a REMS, which can materially affect the potential market and profitability of the product. The FDA may prevent or limit further marketing of a product based on the results of post-marketing studies or surveillance programs. After approval, some types of changes to the approved product, such as adding new indications, manufacturing changes and additional labeling claims, are subject to further testing requirements and FDA review and approval.

Expedited Development and Review Programs for Drugs

The FDA maintains several programs intended to facilitate and expedite development and review of new drugs to address unmet medical needs in the treatment of serious or life-threatening diseases or conditions. These programs include Fast Track Designation, Breakthrough Therapy Designation, Priority Review and Accelerated Approval, and the purpose of these programs is to either expedite the development or review of important new drugs to get them to patients earlier than under standard FDA development and review procedures.

A new drug is eligible for Fast Track Designation if it is intended to treat a serious or life-threatening disease or condition and demonstrates the potential to address unmet medical needs for such disease or condition. Fast Track Designation provides increased opportunities for sponsor interactions with the FDA during nonclinical and clinical development, in addition to the potential for rolling review once a marketing application is filed, meaning that the agency may review portions of the marketing application before the sponsor submits the complete application, as well as Priority Review, discussed below.

In addition, a new drug may be eligible for Breakthrough Therapy Designation if it is intended to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug, alone or in combination with one or more other drugs, may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. Breakthrough Therapy Designation provides all the features of Fast Track Designation in addition to intensive guidance on an efficient drug development program beginning as early as Phase 1, and FDA organizational commitment to expedited development, including involvement of senior managers and experienced review staff in a cross-disciplinary review, where appropriate.

Any product submitted to the FDA for approval, including a product with Fast Track or Breakthrough Therapy Designation, may also be eligible for additional FDA programs intended to expedite the review and approval process including Priority Review designation and Accelerated Approval. A product is eligible for Priority Review if there is evidence that it has the potential to provide a significant improvement in safety or effectiveness in the treatment, diagnosis or prevention of a serious disease or condition. Under priority review, the FDA targets reviewing an application in six months after filing compared to ten months after filing for a standard review.​

Additionally, products are eligible for Accelerated Approval if they are intended to treat a serious or life-threatening disease or condition and can be shown to have an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or an effect on a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality which is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity or prevalence of the condition and the availability or lack of alternative treatments. Accelerated Approval is usually contingent on a sponsor’s agreement to conduct additional post-approval studies to verify and describe the product’s clinical benefit and, under the Food and Drug Omnibus Reform Act of 2022, or FDORA, the FDA may require, as appropriate, that such trials be underway prior to approval or within a specific time period after the date of approval for a product granted Accelerated Approval. Under FDORA, the FDA has increased authority for expedited procedures to withdraw approval of a drug or indication approved under Accelerated Approval if, for example, the confirmatory trial fails to verify the predicted clinical benefit of the product. In addition, for products being considered for accelerated approval, the FDA generally requires, unless otherwise informed by the agency, that all advertising and promotional materials that are intended for dissemination or publication within 120 days following marketing approval be submitted to the agency for review during the pre-approval review period, and that after 120 days following marketing approval, all advertising and promotional materials must be submitted at least 30 days prior to the intended time of initial dissemination or publication.

Even if a product qualifies for one or more of these programs, the FDA may later decide that the product no longer meets the conditions for qualification or the time period for FDA review or approval may not be shortened. Furthermore, Fast Track Designation, Breakthrough Therapy Designation, Priority Review and Accelerated Approval do not change the scientific or medical standards for approval or the quality of evidence necessary to support approval but may expedite the development or review process.

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Pediatric Information and Pediatric Exclusivity

Under the Pediatric Research Equity Act, or PREA, as amended, certain NDAs and certain supplements to an NDA must contain data to assess the safety and efficacy of the drug for the claimed indications in all relevant pediatric subpopulations and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective. The FDA may grant deferrals for submission of pediatric data or full or partial waivers. The FD&C Act requires that a sponsor who is planning to submit a marketing application for a drug that includes a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration submit an initial Pediatric Study Plan, or PSP, within 60 days of an end-of-Phase 2 meeting or, if there is no such meeting, as early as practicable before the initiation of the Phase 3 or Phase 2/3 trial or as agreed upon by the sponsor or FDA. The FDA and the sponsor must reach an agreement on the PSP. A sponsor can submit amendments to an agreed-upon initial PSP at any time if changes to the pediatric plan need to be considered based on data collected from nonclinical studies, early phase clinical trials and/or other clinical development programs.

A drug can also obtain pediatric market exclusivity in the U.S. Pediatric exclusivity, if granted, adds six months to existing exclusivity periods and patent terms. This six-month exclusivity, which runs from the end of other exclusivity protection or patent term, may be granted based on the voluntary completion of a pediatric trial or of multiple pediatric trials in accordance with an FDA-issued “Written Request” for such trials, provided that at the time pediatric exclusivity is granted there is not less than nine months of existing exclusivity or patent term remaining.

U.S. Post-Approval Requirements for Drugs

Drugs manufactured or distributed pursuant to FDA approvals are subject to pervasive and continuing regulation by the FDA, including, among other things, requirements relating to record-keeping, periodic reporting, product sampling and distribution, reporting of adverse experiences with the product, complying with promotion and advertising requirements, which include restrictions on promoting products for unapproved uses or patient populations (known as “off-label use”) and limitations on industry-sponsored scientific and educational activities. Although physicians may prescribe legally available products for off-label uses, manufacturers and individuals working on behalf of manufacturers may not market or promote such uses. The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability, including investigation by federal and state authorities. Prescription drug promotional materials must be submitted to the FDA in conjunction with their first use or first publication. Further, if there are any modifications to the drug, including changes in indications, labeling or manufacturing processes or facilities, the applicant may be required to submit and obtain FDA approval of a new NDA or NDA supplement, which may require the development of additional data or nonclinical studies and clinical trials. The FDA may impose a number of post-approval requirements as a condition of approval of an NDA. For example, the FDA may require post-market testing, including Phase 4 clinical trials, and surveillance to further assess and monitor the product’s safety and effectiveness after commercialization.

Drug manufacturers and their subcontractors involved in the manufacture and distribution of approved drugs, and those supplying products, ingredients and components of them, are required to register their establishments with the FDA and certain state agencies and are subject to periodic unannounced inspections by the FDA and certain state agencies for compliance with ongoing regulatory requirements, including cGMP, which impose certain procedural and documentation requirements upon us and our contract manufacturers. Manufacturers and other parties involved in the drug supply chain for prescription drug products must also comply with product tracking and tracing requirements and for notifying the FDA of counterfeit, diverted, stolen and intentionally adulterated products or products that are otherwise unfit for distribution in the United States. Failure to comply with statutory and regulatory requirements can subject a manufacturer to possible legal or regulatory action, such as warning letters, suspension of manufacturing, product seizures, injunctions, civil penalties or criminal prosecution.

Later discovery of previously unknown problems with a product, including adverse events of unanticipated severity or frequency, or with manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to the approved labeling to add new safety information, requirements for post-market studies or clinical trials to assess new safety risks, or imposition of distribution or other restrictions under a REMS. Other potential consequences include, among other things:

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restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls;

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safety alerts, Dear Healthcare Provider letters, press releases or other communications containing warnings or other safety information about the product;

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fines, warning letters or untitled letters or holds on post-approval clinical trials;

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refusal of the FDA to approve applications or supplements to approved applications, or suspension or revocation of product approvals;

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product seizure or detention, or refusal to permit the import or export of products;

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consent decrees, corporate integrity agreements, debarment or exclusion from federal healthcare programs or mandated modification of promotional materials and labeling and issuance of corrective information.

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Abbreviated New Drug Applications for Generic Drugs

In 1984, with passage of the Hatch-Waxman Amendments amending the Federal Food, Drug, and Cosmetic Act, or the FDCA, Congress authorized the FDA to approve generic drugs that are the same as drugs previously approved by the FDA under the NDA provisions of the statute. To obtain approval of a generic drug, an applicant must submit an ANDA to the agency. Upon approval of an ANDA, the FDA indicates that the generic product is “therapeutically equivalent” to the drug product previously approved under an NDA, known as the reference listed drug or the RLD, and it assigns a therapeutic equivalence rating to the approved generic drug in its publication “Approved Drug Products with Therapeutic Equivalence Evaluations,” also referred to as the “Orange Book”. Physicians and pharmacists consider the therapeutic equivalence rating to mean that a generic drug is fully substitutable for the RLD. In addition, by operation of certain state laws and numerous health insurance programs, the FDA’s designation of a therapeutic equivalence rating often results in substitution of the generic drug without the knowledge or consent of either the prescribing physician or patient.

Hatch-Waxman Patent Certification and the 30 Month Stay

Upon approval of an NDA or a supplement thereto, NDA sponsors are required to list with the FDA each patent with claims that cover the applicant’s product or a method of using the product. When an ANDA applicant files its application with the FDA, the applicant is required to certify to the FDA concerning any patents listed for the reference product in the Orange Book, except for patents covering methods of use for which the ANDA applicant is not seeking approval.

A certification that the new product will not infringe the already approved product’s listed patents or that such patents are invalid or unenforceable is called a Paragraph IV certification. If the applicant does not challenge the listed patents or indicate that it is not seeking approval of a patented method of use, the ANDA application will not be approved until all the listed patents claiming the referenced product have expired. If the ANDA applicant has provided a Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certification to the NDA and patent holders once the ANDA has been accepted for filing by the FDA. The NDA and patent holders may then initiate a patent infringement lawsuit in response to the notice of the Paragraph IV certification. The filing of a patent infringement lawsuit within 45 days after the receipt of a Paragraph IV certification automatically prevents the FDA from approving the ANDA until the earlier of 30 months, expiration of the patent, settlement of the lawsuit or a decision in the infringement case that is favorable to the ANDA applicant.

505(b)(2) New Drug Applications​

As an alternative path to FDA approval for modifications to formulations or uses of products previously approved by the FDA pursuant to an NDA, an applicant may submit an NDA under Section 505(b)(2) of the FDCA. Section 505(b)(2) was enacted as part of the Hatch-Waxman Amendments and permits the filing of an NDA where at least some of the information required for approval comes from studies not conducted by, or for, the applicant, and for which the applicant has not obtained a right of reference. If the 505(b)(2) applicant can establish that reliance on the FDA’s previous findings of safety and effectiveness is scientifically and legally appropriate, it may eliminate the need to conduct certain nonclinical studies or clinical trials of the new product. The FDA may also require companies to perform additional bridging studies or measurements, including clinical trials, to support the change from the previously approved reference drug. The FDA may then approve the new drug candidate for all, or some, of the label indications for which the reference drug has been approved, as well as for any new indication sought by the 505(b)(2) applicant.

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Marketing Exclusivity

Market exclusivity provisions under the FD&C Act can delay the submission or the approval of certain marketing applications. The FD&C Act provides a five-year period of non-patent exclusivity within the United States to the first applicant to obtain approval of an NDA for a new chemical entity. A drug is a new chemical entity if the FDA has not previously approved any other new drug containing the same active moiety, which is the molecule or ion responsible for the action of the drug substance. During the exclusivity period, the FDA may not approve or even accept for review an ANDA or an NDA submitted under Section 505(b)(2), or 505(b)(2) NDA, submitted by another company for another drug based on the same active moiety, regardless of whether the drug is intended for the same indication as the original innovative drug or for another indication. However, such an application may be submitted after four years if it contains a certification of patent invalidity or non-infringement to one of the patents listed with the FDA by the innovator NDA holder.

The FD&C Act alternatively provides three years of marketing exclusivity for an NDA, or supplement to an existing NDA, if new clinical investigations, other than bioavailability studies, that were conducted or sponsored by the applicant are deemed by the FDA to be essential to the approval of the application, for example new indications, dosages or strengths of an existing drug. This three-year exclusivity covers only the modification for which the drug received approval on the basis of the new clinical investigations and does not prohibit the FDA from approving ANDAs or 505(b)(2) NDAs for drugs containing the active agent for the original indication or condition of use. Five-year and three-year exclusivity will not delay the submission or approval of a full NDA. However, an applicant submitting a full NDA would be required to conduct or obtain a right of reference to any nonclinical studies and adequate and well-controlled clinical trials necessary to demonstrate safety and effectiveness.

Patent Term Extension

In the United States, after an NDA is approved, owners of relevant drug patents may apply for up to a five-year patent extension, which permits patent term restoration as compensation for the patent term lost during the FDA regulatory process. The allowable patent term extension is typically calculated as one-half the time between, the latter of the effective date of an IND and issue date of the patent for which extension is sought, and the submission date of an NDA, plus the time between NDA submission date and the NDA approval date up to a maximum of five years. The time can be shortened if the FDA determines that the applicant did not pursue licensure with due diligence. The total patent term after the extension may not exceed 14 years from the date of product approval. Only one patent applicable to an approved drug product is eligible for extension and only those claims covering the product, a method for using it, or a method for manufacturing it may be extended and the application for the extension must be submitted prior to the expiration of the patent in question. However, we may not be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents or otherwise failing to satisfy applicable requirements. Some, but not all, foreign jurisdictions possess patent term extension or other additional patent exclusivity mechanisms that may be more or less stringent and comprehensive than those of the United States.

Other Regulatory Matters

From time to time, legislation is drafted, introduced, passed in Congress and signed into law that could significantly change the statutory provisions governing the approval, manufacturing and marketing of products regulated by the FDA. In addition to new legislation, FDA regulations, guidance and policies are often revised or reinterpreted by the agency in ways that may significantly affect the manner in which pharmaceutical products are regulated and marketed.

Manufacturing, sales, promotion and other activities of product candidates following product approval, where applicable, or commercialization are also subject to regulation by numerous regulatory authorities in the United States in addition to the FDA, which may include the CMS, other divisions of the Department of Health and Human Services, the Department of Justice, the Drug Enforcement Administration, the Consumer Product Safety Commission, the Federal Trade Commission, the Occupational Safety & Health Administration, the Environmental Protection Agency and state and local governments and governmental agencies.

Current and Future Healthcare Reform Legislation

In the U.S. and in some foreign jurisdictions, there have been, and likely will continue to be, a number of legislative and regulatory changes and proposed changes intended to broaden access to healthcare, improve the quality of healthcare, and contain or lower the cost of healthcare items and services. For example, in the U.S., the ACA, among other things, subjected products to potential competition by lower-cost products,

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expanded the types of entities eligible for the 340B drug discount program, increased minimum rebates owed by manufacturers under the Medicaid Drug Rebate Program and extended the rebate program to individuals enrolled in Medicaid managed care organizations, established annual fees and taxes on manufacturers of certain branded prescription drugs, and created a Medicare Part D coverage gap discount program, in which manufacturers must agree to offer 50% (increased effective January 2019 to 70%) point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D. The coverage gap discount program was replaced effective January 1, 2025 by the Medicare Part D Manufacturer Discount Program established under the Inflation Reduction Act of 2022.

Other legislative changes have been proposed since the ACA was enacted and, while proposed changes may require authorization through additional legislation to become effective, and future administrations or Congress may reverse or otherwise change these measures, both the Trump administration and Congress have indicated that they will continue to seek new legislative measures to control drug costs.

Other federal health reform measures have been proposed and adopted in the United States since the ACA was enacted. The Budget Control Act of 2011, for example, included aggregate reductions to Medicare payments to providers of up to 2% per fiscal year that will remain in effect through fiscal year 2032. In addition, the American Taxpayer Relief Act of 2012 was signed into law which, among other things, reduced Medicare payments to several providers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years.

Furthermore, there has been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several congressional inquiries and proposed legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient assistance programs and reform government program reimbursement methodologies for drug products.

In addition, the Inflation Reduction Act of 2022, or the IRA, included several provisions that could impact our business to varying degrees. The IRA, among other things, allows for CMS to negotiate prices for certain single-source drugs and biologics reimbursed under Medicare Part B and Part D, with negotiated maximum fair prices for the first ten Medicare Part D drugs having taken effect January 1, 2026, and with Medicare Part B drugs becoming subject to negotiation beginning January 1, 2028. The legislation subjects drug manufacturers to civil monetary penalties and a potential excise tax for offering a price that is not equal to or less than the price negotiated under the law or for taking price increases that exceed inflation. The legislation also requires manufacturers to pay rebates for drugs in Medicare Part D and Part B for certain prices that increase faster than the rate of inflation. Further, the legislation caps Medicare beneficiaries’ annual out-of-pocket drug expenses at $2,000. The One Big Beautiful Bill Act of 2025, or OBBBA, amended the IRA negotiation program, effective for initial price applicability year 2028 and subsequent years, to expand the orphan drug exclusion (so that drugs designated for one or more rare diseases or conditions are excluded from the definition of qualifying single source drug) and to delay the start of the negotiation eligibility period of orphan-designated products until the date of first approval for a non-orphan indication. The effect of IRA, as amended, on our business and the healthcare industry in general is not yet known.

The OBBBA imposed significant reductions in Medicaid funding, additional work requirements for Medicaid recipients, and more frequent reenrollment requirements. These changes are expected to place substantial pressure on state Medicaid budgets, reduce enrollment, and limit covered services, which could decrease utilization of, and reimbursement for, our products, if approved.

We cannot predict the initiatives that may be adopted in the future. The continuing efforts of the government, insurance companies, managed care organizations and other payors of healthcare services to contain or reduce costs of healthcare and/or impose price controls may adversely affect:

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Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payors, which may adversely affect our future profitability.

Prescription drug pricing in the U.S. is subject to significant political, legislative, and regulatory scrutiny. In recent years, Congress and federal and state agencies have considered and adopted measures aimed at increasing pricing transparency, reducing drug costs under government programs, reforming reimbursement methodologies, and examining manufacturer patient assistance programs. In 2025, the Trump Administration issued executive orders and supported regulatory initiatives focused on implementing most-favored-nation, or MFN, pricing concepts and expanding direct-to-consumer sales models, and CMS proposed multiple reimbursement models that would incorporate international reference pricing benchmarks into Medicare and Medicaid payment structures. These initiatives, if implemented, could adversely affect the prices that manufacturers are able to obtain for prescription drugs in the U.S. Although certain proposals are not finalized and, even if finalized, remain subject to rulemaking, legal challenge or voluntary participation, continued efforts to regulate or constrain drug pricing could materially impact our future revenue, pricing flexibility and commercial strategy if our product candidates are approved.

Individual states in the United States have also become increasingly active in passing legislation and implementing regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. In addition, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs. It is difficult to predict the future legislative landscape in healthcare and the effect on our business, results of operations, financial condition and prospects. However, we expect that additional state and federal healthcare reform measures will be adopted in the future.

Third-Party Payor Coverage and Reimbursement

Significant uncertainty exists as to the coverage and reimbursement status of any products for which we may obtain regulatory approval. In the U.S. and markets in other countries, sales of any products for which we may receive regulatory marketing approval for commercial sale will depend, in part, on the availability of coverage and reimbursement from third-party payors. Third-party payors include government healthcare programs (e.g., Medicare, Medicaid), managed care providers, private health insurers, health maintenance organizations and other organizations. These third-party payors often decide which medications they will cover and establish reimbursement levels. The availability of coverage and extent of reimbursement by governmental and other third-party payors is essential for most patients to be able to afford treatments such as targeted protein degradation therapies.

In the U.S., no uniform policy exists for coverage and reimbursement for products across all third-party payors. Therefore, decisions regarding the extent of coverage and amount of reimbursement to be provided can differ significantly from payor to payor. Third-party payors often follow Medicare coverage policy and payment limitations in setting their own reimbursement rates, but also have their own methods and approval process apart from Medicare determinations. Factors payors consider in determining reimbursement are based on whether the product is:

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a covered benefit under its health plan;

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safe, effective and medically necessary;

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appropriate for the specific patient;

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cost-effective; and

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neither experimental nor investigational.

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One third-party payor’s decision to cover a particular product or service does not ensure that other payors will also provide coverage for the medical product or service. Third-party payors may limit coverage to specific products on an approved list or formulary, which may not include all FDA-approved products for a particular indication. Also, third-party payors may refuse to include a particular branded product on their formularies or otherwise restrict patient access to a branded drug when a less costly generic equivalent or other alternative is available. Our ability to successfully commercialize our product candidates will depend in part on the extent to which coverage and adequate reimbursement for these products and related treatments will be available from third-party payors.

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Moreover, the process for determining whether a payor will provide coverage for a product may be separate from the process for setting the reimbursement rate a payor will pay for the product. A payor’s decision to provide coverage for a product does not imply that an adequate reimbursement rate will be approved. Further, third-party payors are increasingly challenging the price and examining the medical necessity and cost-effectiveness of medical products and services, in addition to their safety and efficacy. In order to secure coverage and reimbursement for any product that might be approved for sale, we may need to conduct expensive pharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of our products, in addition to the costs required to obtain FDA or comparable regulatory approvals. Additionally, we may also need to provide discounts or rebates to purchasers, private health plans or government healthcare programs. Despite our best efforts, our product candidates may not be considered medically necessary or cost-effective. If third-party payors do not consider a product to be cost-effective compared to other available therapies, they may not cover an approved product as a benefit under their plans or, if they do, the level of payment may not be sufficient to allow us to sell our products at a profit. A decision by a third-party payor not to cover a product could reduce physician utilization once the product is approved and have a material adverse effect on sales, our operations and financial condition.

Finally, in some foreign countries, the proposed pricing for a product candidate must be approved before it may be lawfully marketed. The requirements governing product pricing vary widely from country to country. For example, in the European Union, or EU, pricing and reimbursement of pharmaceutical products are regulated at a national level under the individual EU Member States’ social security systems. Some foreign countries provide options to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and can control the prices of medicinal products for human use. To obtain reimbursement or pricing approval, some of these countries may require the completion of clinical trials that compare the cost effectiveness of a particular product candidate to currently available therapies. A country may approve a specific price for the medicinal product or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market. There can be no assurance that any country that has price controls or reimbursement limitations for products will allow favorable reimbursement and pricing arrangements for any of our product candidates. Even if approved for reimbursement, historically, product candidates launched in some foreign countries, such as some countries in the EU, do not follow price structures of the United States and prices generally tend to be significantly lower.

Other Healthcare Laws and Regulations

Healthcare providers, physicians, and third-party payors will play a primary role in the recommendation and prescription of any products for which we obtain marketing approval. Our business operations and any current or future arrangements with third-party payors may expose us to broadly applicable federal and state fraud and abuse laws, as well as other healthcare laws and regulations. These laws may impact, among other things, our proposed sales, marketing and distribution strategies. In the United States, these laws include, among others:

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The federal Anti-Kickback Statute, or AKS, which prohibits, among other things, any person or entity from knowingly and willfully offering, soliciting, receiving or paying remuneration (a term interpreted broadly to include anything of value, including, for example, gifts, discounts and credits), directly or indirectly, overtly or covertly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase, lease, order or recommendation of, or arranging for, an item, good, facility or service for which payment may be made under a federal healthcare program such as Medicare and Medicaid. The AKS has been interpreted to apply to arrangements between manufacturers on one hand and prescribers, purchasers, formulary managers, and pharmacies, among other entities, on the other. A person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation. Violations can result in significant civil monetary and criminal penalties for each violation, plus imprisonment, and exclusion from government healthcare programs.

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Additionally, the civil False Claims Act, or FCA, prohibits, among other acts, knowingly presenting or causing the presentation of a false, fictitious or fraudulent claim for payment to the U.S. government. Actions under the FCA may be brought by the Attorney General or as a qui tam action by a private individual in the name of the government. Violations of the FCA can result in very significant monetary damages (up to three times the amount of the government’s damages) and civil penalties for each false claim, as well as potential exclusion from government healthcare programs or ongoing integrity obligations. Manufacturers can be held liable under the FCA even when they do not submit claims directly to government payors if they are deemed to “cause” the submission of false or fraudulent claims. Further, violations of other statutes such as the AKS and FDCA may, in certain instances, form the basis for FCA liability.

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The U.S. federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, imposes additional criminal and civil liability for knowingly and willfully executing, or attempting to execute, a scheme to defraud any healthcare benefit program or obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any healthcare benefit program, regardless of the payor (e.g., public or private); and knowingly and willfully falsifying, concealing or covering up by any trick or device a material fact or making any materially false statement in connection with the delivery of, or payment for, healthcare benefits, items or services. Similar to the AKS, a person or entity does not need to have actual knowledge of the statute or specific intent to violate it in order to have committed a violation.

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HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, or HITECH, and its implementing regulations, including the final omnibus rule published on January 25, 2013, imposes, among other things, certain requirements relating to the privacy, security and transmission of individually identifiable health information. Among other things, HITECH makes HIPAA’s privacy and security standards directly applicable to “business associates,” defined as independent contractors or agents of covered entities that create, receive, maintain, transmit or obtain, protected health information in connection with providing a service for or on behalf of a covered entity. HITECH also increased the civil and criminal penalties that may be imposed against covered entities, business associates and possibly other persons, and gave state attorneys general new authority to file civil actions for damages or injunctions in federal courts to enforce the federal HIPAA laws and seek attorney’s fees and costs associated with pursuing federal civil actions.

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Federal transparency laws, including the federal Physician Payments Sunshine Act created under the ACA, and its implementing regulations, which requires manufacturers of certain drugs, devices, medical supplies and biologics, among others, to track and disclose payments under Medicare, Medicaid or the Children’s Health Insurance Program (with certain exceptions) and other transfers of value they make to U.S. physicians (defined to include doctors, dentists, optometrists, podiatrists and chiropractors), other non-physician practitioners (like physician assistants and nurse practitioners) and teaching hospitals, as well as ownership and investment interests held by physicians and their immediate family members. This information is subsequently made publicly available in a searchable format on a CMS website.

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Federal government price reporting laws, which require us accurately and timely to calculate and report complex pricing metrics to government programs, where such reported prices may be used in the calculation of reimbursement and/or discounts on approved products. Violations of these laws may, in certain circumstances, result in civil monetary penalties and/or violations of the FCA.

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Federal consumer protection and unfair competition laws, which broadly regulate marketplace activities and activities that potentially harm consumers.

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Analogous state law equivalents of each of the above U.S. federal laws and similar healthcare laws and regulations in the EU and other jurisdictions, such as: anti-kickback and false claims laws, which may apply to items or services reimbursed by any third-party payor, including commercial insurers or patients; state and local marketing and/or transparency laws applicable to manufacturers that may be broader in scope than the federal requirements; state laws that require the reporting of information related to drug pricing; state laws that require drug manufacturers to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures and pricing information; state and local laws that require the licensure and/or registration of pharmaceutical sales representatives; state laws that require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government; and state laws governing the privacy and security of health information and/or other health information in certain circumstances, many of which differ from each other in significant ways and often are not pre-empted by HIPAA, thus complicating compliance efforts. There are ambiguities as to what is required to comply with these state requirements and if we fail to comply with an applicable state law requirement we could be subject to penalties. Finally, there are state and foreign laws governing the privacy and security of health information, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts.

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The scope and enforcement of each of these laws is uncertain and subject to rapid change in the current environment of healthcare reform, especially in light of the lack of applicable precedent and regulations. Federal, state and foreign enforcement bodies have recently increased their scrutiny of interactions between healthcare companies and healthcare providers, which has led to a number of investigations, prosecutions, convictions and settlements in the healthcare industry. It is possible that governmental authorities will conclude that our

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business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrative penalties, damages, fines, disgorgement, contractual damages, reputational harm, diminished profits and future earnings, individual imprisonment, exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuring of our operations, as well as additional reporting obligations and oversight if we become subject to a corporate integrity agreement or similar settlement to resolve allegations of non-compliance with these laws, any of which could adversely affect our ability to operate our business and our financial results. If any of the physicians or other healthcare providers or entities with whom we expect to do business is found to be not in compliance with applicable laws, they may be subject to similar actions, penalties and sanctions. Ensuring business arrangements comply with applicable healthcare laws, as well as responding to possible investigations by government authorities, can be time- and resource consuming and can divert a company’s attention from the business.

Compliance With Other Federal And State Laws or Requirements; Changing Legal Requirements

If any products that we may develop are made available to authorized users of the Federal Supply Schedule of the General Services Administration, additional laws and requirements apply. Products must meet applicable child-resistant packaging requirements under the U.S. Poison Prevention Packaging Act. Manufacturing, labeling, packaging, distribution, sales, promotion and other activities also are potentially subject to federal and state consumer protection and unfair competition laws, among other requirements to which we may be subject.

The distribution of pharmaceutical products is subject to additional requirements and regulations, including extensive record-keeping, licensing, storage and security requirements intended to prevent the unauthorized sale of pharmaceutical products.

The failure to comply with any of these laws or regulatory requirements subjects firms to possible legal or regulatory action. Depending on the circumstances, failure to meet applicable regulatory requirements can result in criminal prosecution, fines or other penalties, injunctions, exclusion from federal healthcare programs, requests for recall, seizure of products, total or partial suspension of production, denial or withdrawal of product approvals, relabeling or repackaging or refusal to allow a firm to enter into supply contracts, including government contracts. Any claim or action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business. Prohibitions or restrictions on marketing, sales or withdrawal of future products marketed by us could materially affect our business in an adverse way.

Changes in regulations, statutes or the interpretation of existing regulations could impact our business in the future by requiring, for example: (i) changes to our manufacturing arrangements; (ii) additions or modifications to product labeling or packaging; (iii) the recall or discontinuation of our products; or (iv) additional record-keeping requirements. If any such changes were to be imposed, they could adversely affect the operation of our business.

European Union Drug Development

Similar to the United States, the various phases of nonclinical and clinical research in the European Union (EU) are subject to significant regulatory controls. In April 2014, the EU adopted the new Clinical Trials Regulation No. 536/2014, which replaced the Clinical Trials Directive 2001/20/EC on January 31, 2022. The transitory provisions of the new Regulation provide that, by January 31, 2025, all ongoing clinical trials must have transitioned to the new Regulation.

The new Regulation overhauled the system of approvals for clinical trials in the EU. Specifically, it is directly applicable in all Member States (meaning that no national implementing legislation in each Member State is required), and aims at simplifying and streamlining the approval of clinical trials in the EU. The main characteristics of the new Regulation include: a streamlined application procedure via a single-entry point through the Clinical Trials Information System; a single set of documents to be prepared and submitted for the application as well as simplified reporting procedures for clinical trial sponsors; and a harmonized procedure for the assessment of applications for clinical trials, which is divided in two parts (Part I contains scientific

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and medicinal product documentation and Part II contains the national and patient-level documentation). Part I is assessed by a coordinated review by the competent authorities of all EU Member States in which an application for authorization of a clinical trial has been submitted (Member States concerned) of a draft report prepared by a Reference Member State. Part II is assessed separately by each Member State concerned. Strict deadlines have also been established for the assessment of clinical trial applications.

European Union Drug Review and Approval

In the European Economic Area, or the EEA, which is comprised of the 27 Member States of the European Union and three European Free Trade Association States (Norway, Iceland and Liechtenstein), medicinal products can only be commercialized after obtaining a Marketing Authorization, or MA. There are two types of marketing authorizations, a centralized MA and a national MA.

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The centralized MA is issued by the European Commission through the Centralized Procedure, based on the opinion of the Committee for Medicinal Products for Human Use, or CHMP, of the EMA, and is valid throughout the entire territory of the EEA. The Centralized Procedure is mandatory for certain types of products, such as biotechnology medicinal products, orphan medicinal products, advanced-therapy medicines such as gene-therapy, somatic cell-therapy or tissue-engineered medicines and medicinal products containing a new active substance indicated for the treatment of HIV, AIDS, cancer, neurodegenerative disorders, diabetes, auto-immune and other immune dysfunctions, and viral diseases. The Centralized Procedure is optional for products containing a new active substance not yet authorized in the EEA, or for products that constitute a significant therapeutic, scientific or technical innovation or which are in the interest of public health in the EU.

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National MAs, which are issued by the competent authorities of the Member States of the EEA and only cover their respective territory, are available for products not falling within the mandatory scope of the Centralized Procedure. Where a product has already been authorized for marketing in a Member State of the EEA, this National MA can be recognized in another Member States through the Mutual Recognition Procedure. If the product has not received a National MA in any Member State at the time of application, it can be approved simultaneously in various Member States through the Decentralized Procedure. Under the Decentralized Procedure an identical dossier is submitted to the competent authorities of each of the Member States in which the MA is sought, one of which is selected by the applicant as the Reference Member State, or RMS. The competent authority of the RMS prepares a draft assessment report, a draft summary of the product characteristics, or SmPC, and a draft of the labeling and package leaflet, which are sent to the other Member States (referred to as the Concerned Member States or EUCMS) for their approval. If the EUCMS raise no objections, based on a potential serious risk to public health, to the assessment, SmPC, labeling or packaging proposed by the RMS, the product is subsequently granted a national MA in all the Member States (i.e., in the RMS and the EUCMS).

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Under the above described procedures, before granting the MA, EMA or the competent authorities of the Member States of the EEA make an assessment of the risk-benefit balance of the product on the basis of scientific criteria concerning its quality, safety and efficacy. Similar to the United States patent term-restoration, SPCs serve as an extension to a patent right in Europe for up to five years. SPCs apply to specific pharmaceutical products to offset the loss of patent protection due to the lengthy testing and clinical trials these products require prior to obtaining regulatory marketing approval. Alongside patent protection, there are a number of regulatory incentives offered (including SPC extensions, regulatory data protection and orphan market exclusivity, amongst others).

Outside of the United States, ensuring coverage and adequate payment for a product also involves challenges. Pricing of prescription pharmaceuticals is subject to government control in many countries. Pricing negotiations with government authorities can extend well beyond the receipt of regulatory approval for a product and may require a clinical trial that compares the cost-effectiveness of a product to other available therapies. The conduct of such a clinical trial could be expensive and result in delays in commercialization.

Data Privacy and Security

In the ordinary course of business, we collect, receive, or otherwise process personal data, including information we may collect about participants in our clinical trials. Accordingly, we are, or may become, subject to numerous data privacy and security obligations, including global, federal, state and local laws, regulations, guidance, industry standards, external and internal privacy and security policies, contractual requirements and other obligations related to privacy and data security.

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We may be subject to Swiss, European, US federal, state and foreign data protection laws and regulations (i.e., laws and regulations that address privacy and data security) which provide additional privacy restrictions. For example, in the EEA and United Kingdom, or UK, the collection and use of personal data including health information is governed by the provisions of the EU GDPR and the UK’s implementation of the UK GDPR, and collectively the GDPR, as well as national data protection laws in force in relevant EEA Member States and the UK (including the UK Data Protection Act 2018 and the UK Data (Use and Access) Act 2025). The GDPR imposes a broad range of strict requirements on companies subject to the GDPR, such as requirements relating to ensuring a legal basis or condition applies to the processing of personal data, transferring such information outside the EEA/UK, including to the United States (see below), expanded disclosures to individuals regarding the processing of their personal data, implementing safeguards to keep personal data secure, having data processing agreements with third parties who process personal data, providing information to individuals regarding data processing activities, responding to individuals’ requests to exercise their rights in respect of their personal data, if required obtaining consent of the individuals to whom the personal data relates, reporting security and privacy breaches involving personal data to the competent national data protection authority and affected individuals, appointing data protection officers, conducting data protection impact assessments, and record-keeping. The GDPR substantially increases the penalties to which we could be subject in the event of any non-compliance, including fines of up to €20,000,000 (£17.5 million for the UK GDPR) or 4% of our corporate group’s total annual global revenue from the preceding year, whichever is greater. The GDPR also confers a private right of action on data subjects and consumer associations to lodge complaints with supervisory authorities, seek judicial remedies, and obtain compensation for damages resulting from violations of the GDPR. The GDPR may impose additional responsibility and liability in relation to personal data that we process and we may be required to put in place additional mechanisms to ensure continued compliance with GDPR, which may be onerous and adversely affect our business, financial condition, results of operations and prospects. Compliance with the GDPR is a rigorous and time-intensive process that may increase our cost of doing business or require us to change our business practices, and despite those efforts, there is a risk that we may be subject to fines and penalties, litigation and reputational harm in connection with our European activities.

The UK’s data protection regime is independent from but aligned to the EU’s data protection regime. Non-compliance with the UK GDPR may result in monetary penalties of up to £17.5 million or 4% of worldwide revenue, whichever is higher.

The GDPR imposes strict rules on the transfer of personal data outside of the EEA/UK to third countries, including the United States in certain circumstances, unless a valid GDPR mechanism (e.g., the European Commission issued Standard Contractual Clauses, or SCCs, and the UK International Data Transfer Addendum/​Agreement, or UK IDTA) is put in place, or an international transfer derogation exists under the GDPR. Where relying on the SCCs or UK IDTA for data transfers, we may also be required to carry out transfer impact assessments. Further, the EU and United States have agreed to an adequacy decision for the EU-U.S. Data Privacy Framework, or the Framework, which entered into force on July 11, 2023. This Framework provides that the protection of personal data transferred between the EU and the United States is comparable to that offered in the EU. This provides a further avenue to ensuring transfers to the United States are carried out in line with GDPR. There has been an extension to the Framework to cover UK transfers to the United States. The Framework could be challenged like its predecessor frameworks. This complexity and the additional contractual burden increases our overall risk exposure, and there may be further divergence on international transfer safeguards in the future, including with regard to administrative burdens. The international transfer obligations under the EEA and UK data protection regimes will require effort and cost and may result in us needing to make strategic considerations around where EEA/UK personal data is located and which service providers we can utilize for the processing of EEA/UK personal data. Any inability to transfer personal data from the UK and EEA to the United States (and other third countries) in compliance with data protection laws may adversely affect our operations and our business and financial position. Although the UK is regarded as a third country under the EU’s GDPR, the European Commission or EC issued a decision recognizing the UK as providing adequate protection under the EU GDPR and, therefore, transfers of personal data originating in the EEA to the UK remain unrestricted. The UK government has confirmed that personal data transfers from the UK to the EEA also remain free flowing.

The UK data protection regime is independent from but currently still aligned with the EEA’s data protection regime. However, going forward, there is increasing risk for divergence in application, interpretation and enforcement of the data protection laws as between the UK and EEA, creating additional regulatory uncertainty.

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For example, the UK Data (Use and Access) Act 2025, now in force, further differentiates the UK and EU data protection regimes. In December 2025, the European Commission adopted a decision determining that the UK continues to provide a level of data protection that is “essentially equivalent” to the EU standards and extended the validity of the UK adequacy decision for six years, through to December 2031. While this renewal reduces immediate adequacy concerns, uncertainty remains regarding how UK data protection laws will evolve in the medium to longer term. The lack of clarity on future UK laws and regulations and their interaction with EU laws and regulations may affect our efforts to maintain a harmonized approach to processing European personal data and expose us to two parallel regimes where the UK GDPR and EU GDPR both apply with differing interpretation and enforcement approaches. This could increase our legal risk, uncertainty, complexity and compliance cost associated with the handling of European personal data, and may require us to adapt our privacy and data security compliance programs to account for legal and regulatory divergence between the UK and EEA. Further, EU Member States have adopted implementing national laws to implement the EU GDPR which may partially deviate from the EU GDPR and the competent authorities in the EU Member States may interpret EU GDPR obligations slightly differently from country to country, so that we do not expect to operate in a uniform legal landscape in the EEA.

In Switzerland, we may be subject to data protection requirements including the Swiss Federal Act on Data Protection which imposes comprehensive rules on the processing of personal data including health related information.

In the United States, numerous federal and state laws and regulations, including federal health information privacy laws, state data breach notification laws, state health information privacy laws and federal and state consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act), that govern the collection, use, disclosure and protection of health-related and other personal information could apply to our operations or the operations of our collaborators. For example, the California Consumer Privacy Act, or the CCPA, is a comprehensive law that creates individual privacy rights and protections for California consumers, places increased privacy and security obligations on entities handling personal data of consumers or households, and provides for civil penalties for violations and a private right of action for data breaches. The CCPA requires covered companies to provide certain disclosures to consumers about its data collection, use and sharing practices, and to provide affected California residents with ways to opt-out of certain sales or transfers of personal information.

Further, as of January 1, 2023, the California Privacy Rights Act, or the CPRA, amended the CCPA and created additional obligations with respect to processing and storing personal information and sensitive personal information. While the CCPA contains an exception for activities that are subject to HIPAA, we cannot yet determine the impact the CCPA and other such future laws, regulations and standards may have on our business.

Numerous U.S. states have passed similar consumer privacy laws. Like the CCPA, these laws grant consumers rights in relation to their personal information and impose new obligations on regulated businesses, including, in some instances, broader data security requirements. Such legislation adds additional complexity, variation in requirements, restrictions and potential legal risk, requiring additional investment of resources in compliance programs, impacting our strategies and the availability of previously useful data which may result in increased compliance costs and/or changes in business practices and policies. The existence of comprehensive privacy laws in different states in the country make our compliance obligations more complex and costly and may increase the likelihood that we may be subject to enforcement actions or otherwise incur liability for noncompliance. State laws are changing rapidly and there have been discussions in the U.S. Congress of comprehensive federal data privacy laws to which we could become subject to, if enacted.

Furthermore, other states have proposed or enacted legislation that is focused on more narrow aspects of privacy. In the state of Washington, for example, the My Health My Data Act requires regulated entities to obtain consent to collect health information, grants consumers certain rights, including to request deletion of their information, and provides for robust enforcement mechanisms, including enforcement by the Washington state attorney-general and a private right of action for consumer claims. Additionally, a small number of states, including Illinois and Texas, have enacted laws that specifically target the collection and use of biometric information.

At the federal level, the FTC has used its authority over “unfair or deceptive acts or practices” to impose stringent requirements on the collection and disclosure of sensitive categories of personal information, including health information. Moreover, the FTC’s expanded interpretation of a “breach” under its Health Breach Notification Rule could impose new disclosure obligations that would apply in the event of a qualifying breach.

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Regulators and legislators in the United States are also increasingly scrutinizing and restricting certain personal data transfers and transactions involving foreign countries. For example, the Department of Justice’s January 8, 2025, rule on “Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons, prohibits data brokerage transactions involving certain sensitive personal data categories, including health data, genetic data and biospecimens, to countries of concern, including China. The regulations also restrict certain investment agreements, employment agreements and vendor agreements involving such data and countries of concern, absent specified cybersecurity controls. Actual or alleged violations of these regulations may be punishable by criminal and/or civil sanctions, and may result in exclusion from participation in federal and state programs.

The uncertainty surrounding the implementation of recent and emerging state privacy and other similar laws, regulations and standards that may be adopted in other jurisdictions exemplifies the vulnerability of our business to the evolving regulatory environment related to personal data and protected health information. Compliance with U.S. and international data protection laws and regulations could require us to take on more onerous obligations in our contracts, restrict our ability to collect, use and disclose data, or in some cases, impact our ability to operate in certain jurisdictions. Failure to comply with these laws and regulations could result in government enforcement actions (which could include civil, criminal and administrative penalties), private litigation, and/or adverse publicity and could negatively affect our operating results and business. Moreover, clinical trial subjects, employees and other individuals about whom we or our potential collaborators obtain personal information, as well as the providers who share this information with us, may limit our ability to collect, use and disclose the information. Claims that we have violated individuals’ privacy rights, failed to comply with data protection laws, or breached our contractual obligations, even if we are not found liable, could be expensive and time-consuming to defend and could result in adverse publicity that could harm our business.

Many jurisdictions outside of Europe where we do business directly or through master resellers today and may seek to expand our business in the future, are also considering and/or have enacted comprehensive data protection legislation. We also continue to see jurisdictions imposing data localization laws. These and similar regulations may interfere with our intended business activities, inhibit our ability to expand into those markets, require modifications to our products or services or prohibit us from continuing to offer services in those markets without significant additional costs.

Compliance With Other Federal and State Laws or Requirements; Changing Legal Requirements

If any products that we may develop are made available to authorized users of the Federal Supply Schedule of the General Services Administration, additional laws and requirements apply. Products must meet applicable child-resistant packaging requirements under the U.S. Poison Prevention Packaging Act. Manufacturing, labeling, packaging, distribution, sales, promotion and other activities also are potentially subject to federal and state consumer protection and unfair competition laws, among other requirements to we may be subject.

The distribution of pharmaceutical products is subject to additional requirements and regulations, including extensive record-keeping, licensing, storage and security requirements intended to prevent the unauthorized sale of pharmaceutical products.

The failure to comply with any of these laws or regulatory requirements subjects firms to possible legal or regulatory action. Depending on the circumstances, failure to meet applicable regulatory requirements can result in criminal prosecution, fines or other penalties, injunctions, exclusion from federal healthcare programs, requests for recall, seizure of products, total or partial suspension of production, denial or withdrawal of product approvals, relabeling or repackaging, or refusal to allow a firm to enter into supply contracts, including government contracts. Any claim or action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses and divert our management’s attention from the operation of our business. Prohibitions or restrictions on marketing, sales or withdrawal of future products marketed by us could materially affect our business in an adverse way.

Changes in regulations, statutes or the interpretation of existing regulations could impact our business in the future by requiring, for example: (i) changes to our manufacturing arrangements; (ii) additions or modifications to product labeling or packaging; (iii) the recall or discontinuation of our products; or (iv) additional record-keeping requirements. If any such changes were to be imposed, they could adversely affect the operation of our business.

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Other U.S. Environmental, Health and Safety Laws and Regulations

We may be subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. From time to time and in the future, our operations may involve the use of hazardous and flammable materials, including chemicals and biological materials, and may also produce hazardous waste products. Even if we contract with third parties for the disposal of these materials and waste products, we cannot completely eliminate the risk of contamination or injury resulting from these materials. In the event of contamination or injury resulting from the use or disposal of our hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties for failure to comply with such laws and regulations.

We maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees, but this insurance may not provide adequate coverage against potential liabilities. However, we do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us.

In addition, we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. Current or future environmental laws and regulations may impair our research, development or production efforts. In addition, failure to comply with these laws and regulations may result in substantial fines, penalties or other sanctions.

Government Regulation of Drugs Outside of the United States

To market any product outside of the United States, we would need to comply with numerous and varying regulatory requirements of other countries regarding safety and efficacy and governing, among other things, clinical trials, marketing authorization or identification of an alternate regulatory pathway, manufacturing, commercial sales and distribution of our products.

Whether or not we obtain FDA approval of a product, we must obtain the requisite approvals from regulatory authorities in foreign countries prior to the commencement of clinical trials or marketing of the product in those countries. If we fail to comply with applicable foreign regulatory requirements, we may be subject to, among other things, fines, suspension or withdrawal of regulatory approvals, product recalls, seizure of products, operating restrictions and criminal prosecution.

Non-Clinical Studies and Clinical Trials

Similarly to the United States, the various phases of non-clinical and clinical research in the EU are subject to significant regulatory controls.

Non-clinical studies are performed to demonstrate the safety and non-toxicity of new chemical (or biological) substances. Non-clinical studies intended to be used to support regulatory filings for medicinal products, both in vitro and in vivo, must be planned, performed, monitored, recorded, reported and archived in accordance with the principles of GLP, which define a set of rules and criteria for a quality system for the organizational process and the conditions for non-clinical studies. These GLP standards reflect the Organization for Economic Co-operation and Development requirements.

Clinical trials of medicinal products in the EU must be conducted in accordance with EU and national regulations and the International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use, or ICH, guidelines on GCP, as well as the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.

In April 2014, the EU adopted the Clinical Trials Regulation (EU) No 536/2014, or the Clinical Trials Regulation, which replaced the Clinical Trials Directive 2001/20/EC on January 31, 2022. The Clinical Trials Regulation, which is directly applicable in all EU Member States (meaning no national implementing legislation in each EU Member State is required), aims to simplify and streamline the approval of clinical trials in the EU, for example by providing for a streamlined application procedure via a single entry point and simplifying reporting procedures for clinical trial sponsors.

Marketing Authorizations

In the EU, medicinal products can only be placed on the market after obtaining an MA. This process depends, among other things, on the nature of the medicinal product, but the two routes are either the centralized authorization procedure or one of the national authorization procedures.

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Centralized procedure — Under the centralized procedure, following the opinion of the EMA’ CHMP, the European Commission issues a single MA valid across the EU as well as the additional states of the EEA (Iceland, Norway and Liechtenstein). The centralized procedure is compulsory for human medicines derived from biotechnology processes, such as genetic engineering, or advanced therapy medicinal products (i.e. gene therapy, somatic cell therapy and tissue engineered products), products that contain a new active substance indicated for the treatment of certain diseases, (i.e. HIV/AIDS, cancer, neurodegenerative disorders, diabetes, autoimmune diseases and other immune dysfunctions and viral diseases), and officially designated orphan medicinal products. For medicines that do not fall within these categories, an applicant has the option of submitting an application for a centralized MA to the EMA, as long as the medicine concerned contains a new active substance not yet authorized in the EU, is a significant therapeutic, scientific or technical innovation, or if its authorization would be in the interest of public health in the EU. Under the centralized procedure, the maximum timeframe for the evaluation of an MAA by the EMA is 210 days, excluding clock stops, when additional written or oral information is to be provided by the applicant in response to questions asked by the CHMP. Clock stops may extend the timeframe of evaluation of an MAA considerably beyond 210 days. Where the CHMP gives a positive opinion, the EMA provides the opinion together with supporting documentation to the European Commission, who makes the final decision to grant an MA, which is issued within 67 days of receipt of the EMA’s recommendation. In exceptional cases, the CHMP might perform an accelerated review of an MAA in no more than 150 days (not including clock stops). Innovative products that target an unmet medical need and are expected to be of major public health interest may be eligible for certain expedited development and review programs, such as the EMA’s PRIME scheme, which provides incentives similar to the Breakthrough Therapy Designation in the U.S. PRIME is a voluntary scheme aimed at enhancing the EMA’s support for the development of medicines that target unmet medical needs. It is based on increased interaction and early dialogue with companies developing promising medicines, to optimize their product development plans and speed up their evaluation to help them reach patients earlier. Product developers that benefit from support under the PRIME scheme can expect to be eligible for accelerated assessment, however this is not guaranteed. The benefits of the PRIME scheme include the appointment of a CHMP rapporteur before submission of an MAA, early dialogue and scientific advice at key development milestones, and the potential to qualify products for accelerated review earlier in the application process. National authorization procedures — There are also two other possible routes to authorize products for therapeutic indications in several EU Member States, which are available for products that fall outside the mandatory scope of the centralized procedure.

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Decentralized procedure — Under the decentralized procedure, an applicant may apply for simultaneous authorization in more than one EU Member State for medicinal products that have not yet been authorized in any EU Member State.

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Mutual recognition procedure — Under the mutual recognition procedure, a medicine is first authorized in one EU Member State, in accordance with the national procedures of that country. Following this, the applicant may seek additional MAs from other EU Member States in a procedure whereby the countries concerned agree to recognize the validity of the original, national MA.

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MAs have an initial duration of five years. After these five years, the authorization may be renewed for an unlimited period on the basis of a reevaluation of the risk-benefit balance by the EMA or the relevant national competent authority, as applicable.

Data and Market Exclusivity

In the EU, upon receiving an MA, innovative medicinal products, sometimes referred to as new active substances (i.e., reference products) qualify for eight years of data exclusivity and an additional two years of market exclusivity. The data exclusivity period prevents generic or biosimilar applicants from relying on the non-clinical and clinical trial data contained in the dossier of the reference product when applying for a generic or biosimilar MA in the EU during a period of eight years from the date on which the reference product was first authorized in the EU. During the additional two-year period of market exclusivity, a generic/biosimilar MAA can be submitted, and the innovator’s data may be referenced, but no generic or biosimilar product can be marketed in the EU until the expiration of the market exclusivity period. The overall ten-year period can be extended to a maximum of eleven years if, during the first eight years of those ten years, the MA holder obtains an MA for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies. However, even if a product benefits from the prescribed period of data exclusivity, another company may market another version of the product if such company obtained a marketing authorization based on an application with a complete and independent data package of pharmaceutical tests, nonclinical tests and clinical trials.

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Orphan Medicinal Products

The criteria for designating an “orphan medicinal product” in the EU are similar in principle to those in the United States. In the EU, a medicinal product may be designated as orphan if (i) it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition; (ii) either (a) such condition affects no more than five in 10,000 persons in the EU when the application is made, or (b) it is unlikely that the product, without the benefits derived from orphan status, would generate sufficient return in the EU to justify the investment in its development; and (iii) there exists no satisfactory method of diagnosis, prevention or treatment of such condition authorized for marketing in the EU, or if such a method exists, the product will be of a significant benefit to those affected by that condition. The application for orphan designation must be submitted before the MAA. Orphan medicinal products are eligible for financial incentives such as reduction of fees or fee waivers and are, upon grant of an MA, entitled to ten years of market exclusivity for the approved therapeutic indication. During this ten-year orphan market exclusivity period, no MAA shall be accepted in the EU for the same indication in respect of a similar medicinal product to the authorized orphan product. A “similar medicinal product” is defined as a medicinal product containing a similar active substance or substances as contained in an authorized orphan medicinal product, and which is intended for the same therapeutic indication. An orphan medicinal product can also obtain an additional two years of market exclusivity in the EU for submission of data from pediatric studies in compliance with an EMA-approved pediatric investigation plan. No extension to any SPC can be granted on the basis of pediatric studies for orphan indications. Orphan designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.

The ten-year market exclusivity may be reduced to six years if, at the end of the fifth year, it is established that the product no longer meets the criteria for orphan designation, including if the product is sufficiently profitable not to justify maintenance of market exclusivity. Additionally, an MA may be granted to a similar medicinal product for the same indication as an authorized orphan product at any time if (i) it is established that a similar medicinal product is safer, more effective or otherwise clinically superior than the authorized orphan product; (ii) the MA holder for the authorized orphan product consents to the second medicinal product authorization; or (iii) the MA holder for the authorized orphan product cannot supply enough orphan medicinal product.

Pediatric Development

In the EU, MAAs for new medicinal products must include the results of trials conducted in the pediatric population, in compliance with a pediatric investigation plan, or PIP, agreed with the EMA’s Pediatric Committee, or PDCO, unless the EMA has granted a product-specific waiver, a class waiver, or a deferral for one or more of the measures included in the PIP. This requirement also applies when a company wants to add a new indication, pharmaceutical form or route of administration for a medicine that is already authorized. The PIP sets out the timing and measures proposed to generate data to support a pediatric indication of the product for which an MA is being sought. The PDCO can grant a deferral of the obligation to implement some or all of the measures of the PIP until there are sufficient data to demonstrate the efficacy and safety of the product in adults. Further, the obligation to provide pediatric clinical trial data can be waived by the PDCO when these data are not needed or appropriate because the product is likely to be ineffective or unsafe in children, the disease or condition for which the product is intended occurs only in adult populations, or when the product does not represent a significant therapeutic benefit over existing treatments for pediatric patients. Products that are granted an MA with the results of the pediatric clinical trials conducted in accordance with the PIP are eligible for a six month extension of the protection under an SPC (provided an application for such extension is made at the same time as filing the SPC application for the product, or at any point up to 2 years before the SPC expires) even where the trial results are negative. This pediatric reward is subject to specific conditions and is not automatically available when data in compliance with the PIP are developed and submitted.

Post-Approval Requirements

Similar to the United States, both MA holders and manufacturers of medicinal products are subject to comprehensive regulatory oversight by the EMA, the European Commission and/or the competent regulatory authorities of the EU Member States. The holder of an MA must establish and maintain a pharmacovigilance system and appoint an individual qualified person for pharmacovigilance who is responsible for oversight of that system. Key obligations include expedited reporting of suspected serious adverse reactions and submission of periodic safety update reports, or PSURs. All new MAAs must include a risk management plan, or RMP,

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describing the risk management system that the company will put in place, including the measures to be taken to prevent or minimize the risks associated with the product. The regulatory authorities may also impose specific obligations as a condition of the MA. Such risk-minimization measures or post-authorization obligations may include additional safety monitoring, more frequent submission of PSURs, or the conduct of additional clinical trials or post-authorization safety studies.

The advertising and promotion of medicinal products is also subject to laws concerning promotion of medicinal products, interactions with physicians, misleading and comparative advertising and unfair commercial practices. All advertising and promotional activities for the product must be consistent with the approved summary of product characteristics, and therefore all off-label promotion is prohibited in the EU. Direct-to-consumer advertising of prescription medicines is also prohibited in the EU. Although general requirements for advertising and promotion of medicinal products are established under EU Directives, the details are governed by regulations in each EU Member State and can differ from one country to another.

Failure to comply with EU and Member State laws that apply to the conduct of clinical trials, manufacturing approval, authorization of medicinal products and marketing of such products, both before and after grant of the MA, or with other applicable regulatory requirements, may result in administrative, civil or criminal penalties. These penalties could include delays or refusal to authorize the conduct of clinical trials, or to grant an MA, product withdrawals and recalls, product seizures, suspension, withdrawal or variation of the MA, total or partial suspension of production, distribution, manufacturing or clinical trials, operating restrictions, injunctions, suspension of licenses, fines and criminal penalties.

The aforementioned EU rules are generally applicable in the EEA. For other countries outside of the EU, such as countries in Latin America or Asia, the requirements governing the conduct of clinical studies, product licensing, pricing and reimbursement vary from country to country. In all cases, again, the clinical studies are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.

Should we utilize third-party distributors, compliance with such foreign governmental regulations would generally be the responsibility of such distributors, who may be independent contractors over whom we have limited control.

Reform of the Regulatory Framework in the EU

The European Commission introduced legislative proposals in April 2023 intended to replace the current regulatory framework in the EU for all medicines (including those for rare diseases and for children). In April 2024, the European Parliament adopted its position on the legislative proposals, and in June 2025, the Council of the EU adopted its position. A common position on the text has been agreed upon on December 11, 2025, in the context of subsequent inter-institutional trilogue negotiations. The proposed revisions are expected to be formally adopted in 2026, but are not expected to become applicable before 2028.

Brexit and the Regulatory Framework in the United Kingdom

Following the end of the Brexit transition period on January 1, 2021 and the implementation of the Windsor Framework on January 1, 2025, the United Kingdom, or UK, is not generally subject to EU laws in respect of medicines. The EU laws that have been transposed into UK law through secondary legislation remain applicable in the UK, however, new legislation such as the EU Clinical Trials Regulation is not applicable in the UK. As a result of the Northern Ireland protocol, different rules applied in Northern Ireland than in England, Wales, and Scotland (together, Great Britain) for a period following Brexit where Northern Ireland continued to follow the EU regulatory regime. However, on January 1, 2025 a new arrangement called the “Windsor Framework” came into effect and reintegrated Northern Ireland under the regulatory authority of the MHRA with respect to medicinal products. The Windsor Framework removes EU licensing processes and EU labeling and serialization requirements in relation to Northern Ireland and introduces a UK-wide licensing process for medicines. In particular, the MHRA is now responsible for approving medicinal products placed on the UK market (i.e., Great Britain and Northern Ireland), and the EMA no longer has a role in UK marketing authorizations. A single UK-wide MA will be granted by the MHRA for medicinal products to be sold in the UK, enabling products to be sold in a single pack and under a single authorization throughout the UK. In addition, the new arrangements require, for packs placed on the UK market on or after January 1, 2025, a “UK Only” label, indicating they are not for sale in the EU. However, although a separate authorization is now

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required to market medicinal products in the UK, since January 1, 2024, the MHRA may rely on the International Recognition Procedure, or IRP, when reviewing certain types of MAAs. Pursuant to the IRP, the MHRA will take into account the expertise and decision-making of trusted regulatory partners (e.g., the medicines regulatory authorities in Australia, Canada, Switzerland, Singapore, Japan, the United States, and the EMA in the EU) when considering an application for a UK marketing authorization. There is no pre-MA orphan designation in the UK. Instead, the MHRA reviews applications for orphan designation in parallel to the corresponding MAA. The criteria are essentially the same as in the EU, but have been tailored for the UK market, i.e., the prevalence of the condition in the UK (rather than the EU) must not be more than five in 10,000. Should an orphan designation be granted, the period of market exclusivity will be set from the date of first approval of the product in the UK.

Employees and Human Capital Resources

As of September 30, 2026, we had 21 full-time employees and six part-time consultants, and nine of our employees have M.D. or Ph.D. degrees. Within our workforce, 12 employees are engaged in research and development and eight are engaged in business development, finance, accounting, legal and general management and administration. Our human capital resources objectives include identifying, recruiting, retaining, incentivizing and integrating our existing and new employees, advisors and consultants. None of our employees are represented by a labor union or covered by a collective bargaining agreement. We consider our relationship with our employees to be good.

Facilities

We maintain an office at 265 Franklin Street, Suite 1702, Boston, Massachusetts, which serves as our principal executive office. We also maintain additional office space at our location in the United Kingdom at Suite 8, The Mansion, Chesterford Research Park, Little Chesterford, CB10 1XL, Cambridge, United Kingdom.

Legal Proceedings

From time to time, we may become involved in litigation or other legal proceedings. We are not currently a party to any litigation or legal proceedings that, in the opinion of our management, are probable to have a material adverse effect on our business. Regardless of outcome, litigation can have an adverse impact on our business, financial condition, results of operations and prospects because of defense and settlement costs, diversion of management resources and other factors.

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MANAGEMENT

Executive Officers and Directors

The following table sets forth the name, position, as well as ages as of the date of this prospectus, of each of our executive officers, significant employees and directors as of the date of this prospectus: