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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

____________________________________________________

FORM 8-K

____________________________________________________

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): October 12, 2026

____________________________________________________

Mirum Pharmaceuticals, Inc.

(Exact name of Registrant as Specified in Its Charter)

____________________________________________________


Delaware	001-38981	83-1281555
(State or Other Jurisdiction of Incorporation)	(Commission File Number)	(IRS Employer Identification No.)

989 East Hillsdale Boulevard Suite 300
Foster City, California		94404
(Address of Principal Executive Offices)		(Zip Code)

Registrant’s Telephone Number, Including Area Code: (650) 667-4085

N/A

(Former Name or Former Address, if Changed Since Last Report)

____________________________________________________

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

o Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

o Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

o Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

o Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading

Symbol(s)

Name of each exchange on which registered

Common stock, par value $0.0001 per share

MIRM

Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

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 13(a) of the Exchange Act. ☐

Item 8.01 Other Events.

Announcement of Data from the Phase 3 EXPAND Study of LIVMARLI (maralixibat) in Ultra-Rare Cholestatic Liver Diseases

On October 12, 2026, Mirum Pharmaceuticals, Inc. (the “Company”) announced topline results from the Phase 3 EXPAND study evaluating LIVMARLI (maralixibat) (“Livmarli”) for the treatment of cholestatic pruritus in patients aged 6 months or older with ultra-rare cholestatic liver diseases, including biliary atresia (“BA”). The study met its primary endpoint and achieved statistical significance across all five prespecified secondary endpoints, including the three endpoints in patients with BA and a native liver.

3期EXPAND研究纳入了52名患者，包括46名儿科患者和6名成人患者，在20周的双盲期中被随机分配接受每日两次（“BID”）285 µg/kg马拉利西巴特或安慰剂治疗。

与安慰剂相比，马拉利西巴特治疗在儿科患者的主要分析人群中显示出统计学显著的瘙痒改善。在第13至20周期间，马拉利西巴特使平均最严重每日ItchRO(Obs)严重程度评分从基线平均改善了1.81分，而安慰剂组改善了0.83分，对应的安慰剂校正治疗差异为0.98分（p=0.0004）。马拉利西巴特还使血清胆汁酸（“sBA”）相比基线减少了70.1%，而安慰剂组仅减少了3.5%，对应的治疗效果为69.0%（p<0.0001）。3期EXPAND研究的关键疗效数据如下表“疗效结果”所示。

3期EXPAND研究中的安全性发现与马拉利西巴特之前的临床经验一致，未观察到新的安全性信号。3期EXPAND研究的关键安全性数据如下表“安全性摘要”所示。

公司计划基于3期EXPAND研究结果，于2027年上半年在美国提交补充新药申请。

疗效结果 – 主要和次要终点

马拉利西巴特
285 µg/kg BID

安慰剂

治疗效果
(MRX-PBO)

p值

整体研究分析

主要终点：ItchRO(Obs)严重程度评分（SE）的变化b

-1.81 (0.18)a

-0.83 (0.18)a

-0.98

0.0004

所有儿科人群

n=22

n=24

瘙痒改善的天数百分比

86.0%

49.1%

36.9%

0.0008

所有儿科人群

n=22

n=24

总sBA从基线的相对百分比变化

-70.1%

-3.5%

-69.0%c

<0.0001

总体人群

n=25

n=27

胆道闭锁（BA）伴自体肝脏亚组

ItchRO(Obs)严重程度评分（SE）的变化b

-2.16 (0.21)a

-1.01 (0.18)a

-1.15

0.0002

n=11

n=14

瘙痒改善的天数百分比

96.4%

52.2%

44.1%

0.0045

n=11

n=14

总sBA从基线的相对百分比变化

-75.5%

-9.0%

-73.1%c

<0.0001

n=11

n=14

a 最小二乘均值（SE）。LS均值、SE和p值来自MMRM模型。

b ItchRO(Obs) = Itch报告结果（观察者），是0-4的最严重瘙痒数值评分量表。

c sBA治疗效果是使用对数转换值估算的，并转换回原始尺度；因此，它们不是通过治疗组之间的算术差计算的。

主要终点表示从基线到第13至20周平均值的变化。血清胆汁酸（sBA）终点表示从基线到第12和20周平均值的变化。瘙痒改善定义为第13至20周内最严重每日ItchRO(Obs)评分≤1或从基线下降≥1分。所有儿科人群包括

随机分配的年龄在18岁或以下的受试者，至少接受了一剂研究药物，并且至少有一个ItchRO(Obs)评分。

安全性总结 – 20周双盲期

安全性人群包括所有随机分配的受试者，他们至少接受了一剂马拉利西巴特或安慰剂；受试者根据所接受的治疗进行分析。


有，n (%)	马拉利西巴特 285 µg/kg BID (n=25)	安慰剂 (n=27)
任何治疗中出现的不良事件（“TEAE”）	24 (96.0)	26 (96.3)
3级或更高级别的TEAE	6 (24.0)	6 (22.2)
与研究药物相关	1 (4.0)	0
严重的TEAE	5 (20.0)	5 (18.5)
导致死亡的TEAE	0	0
导致提前中止研究的TEAE	1 (4.0)	0
关注的TEAE
腹泻	16 (64.0)	3 (11.1)
腹痛	5 (20.0)	4 (14.8)
ALT升高	2 (8.0)	1 (3.7)

MRX治疗患者的严重不良事件（“SAEs”）：肺炎、发热、结肠感染、急性肾损伤、病毒性支气管炎；均无相关性。安慰剂治疗患者的SAEs：肺炎、全血细胞减少、败血症、前臂骨折、胆管炎。

患者人群信息

公司认为，在美国和欧洲，针对Alagille综合征（“ALGS”）患者胆汁淤积性瘙痒的Livmarli的潜在患者人群约为4,000 - 5,500名儿科ALGS患者，其中约2,000名儿科ALGS患者可被覆盖，根据公司的当前预期和信念，这代表了超过5亿美元的市场机会。ALGS估计影响每30,000个出生中的1个。

公司认为，在美国和欧洲，针对进行性家族性肝内胆汁淤积症（“PFIC”）患者胆汁淤积性瘙痒的Livmarli的可覆盖患者人群约为1,000名儿科PFIC患者和超过4,000名成人PFIC患者。

公司认为，在美国和欧洲，针对胆汁淤积症（“BA”）的Livmarli的可覆盖患者人群约为2,000名BA患者。

公司估计，美国约有300名患者被诊断患有进行性骨化性肌炎（“FOP”），全球潜在患者人群约为900名FOP患者。

前瞻性声明

本报告中包含的一些陈述属于前瞻性声明，涉及许多风险和不确定性。此类前瞻性声明包括但不限于以下内容：关于maralixibat在真实世界环境中对胆汁淤积性瘙痒患者（涵盖III期EXPAND研究的环境）的临床相关性的陈述，关于III期EXPAND研究后maralixibat持续开发的预期，任何额外的maralixibat监管批准的商业影响，基于III期EXPAND研究数据的任何潜在监管提交时间表，公司已批准药物和产品候选人的目标疾病预计总可触及市场的估计，以及关于公司已批准药物和产品候选人的目标疾病患病率的假设。不应将前瞻性声明的包含视为公司对任何此类结果将实现的陈述。由于与药品候选药物研发相关的风险和不确定性，以及公司业务中固有的风险和不确定性（包括公司向美国证券交易委员会提交的其他文件中描述的风险和不确定性），实际结果可能与本报告中提出的有所不同。请谨慎不要过度依赖这些前瞻性声明，这些声明仅反映本文件日期的情况，公司不承担更新或修改任何前瞻性声明以反映本文件日期后事件或情况的义务。所有前瞻性声明均受本警示声明的全部限制。

本警示声明是根据1995年《私人证券诉讼改革法案》第21E条的安全港条款作出的。

签名

根据1934年《证券交易法》的要求，注册人已 duly 促使本报告由 duly 授权的代表签署。


	Mirum Pharmaceuticals, Inc.

日期：2026年10月13日	签署人：	/s/ Christopher Peetz
		Christopher Peetz
		首席执行官

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