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Last update 16 Sep 2026

Maralixibat Chloride

Last update 16 Sep 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

Lopixibat, Lopixibat chloride, Lum-001 cation

+ [14]

Target

ISBT

Action

inhibitors

Mechanism

ISBT inhibitors(Ileal bile acid transporter inhibitors)

Therapeutic Areas

Cardiovascular DiseasesCongenital DisordersDigestive System Disorders+ [3]

Active Indication

progressive familial intrahepatic cholestasisAlagille SyndromeCholestatic pruritus+ [2]

Inactive Indication

Biliary AtresiaCholangitis, SclerosingCholestasis, Progressive Familial Intrahepatic 1+ [2]

Originator Organization

Pharmacia Corp.

Mirum Pharmaceuticals, Inc.

Active Organization

GC Biopharma Corp.

Mirum Pharmaceuticals, Inc.CANbridge (Suzhou) Bio-pharma Co., Ltd.

+ [4]

Inactive Organization

Halo Pharmaceutical Canada, Inc.

Shire Human Genetic Therapies, Inc.

Lumena Pharmaceuticals, Inc.

License Organization

Eversana Life Science Services LLC

Takeda Pharmaceutical Co., Ltd.Shire Pharmaceuticals LLC

+ [7]

Drug Highest PhaseApproved

First Approval Date

United States (29 Sep 2021),

Cholestatic pruritus

RegulationBreakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (Taiwan Province), Orphan Drug (Japan), Orphan Drug (South Korea)

Structure/Sequence

Molecular FormulaC40H56ClN3O4S

InChIKeyPOMVPJBWDDJCMP-RUKDTIIFSA-M

CAS Registry228113-66-4

Clinical Trials associated with Maralixibat Chloride

NCT07823127

/ Not yet recruitingPhase 2

MRX-901: An Open-label Study to Assess Safety and Tolerability of Maralixibat in Participants With Intrahepatic Cholestasis of Pregnancy (ICP) (OASIS)

This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in pregnant women with intrahepatic cholestasis of pregnancy (ICP).
This is an open-label study where all participants will receive maralixibat.

Start Date15 Jan 2027

Sponsor / Collaborator

Mirum Pharmaceuticals, Inc.

NCT07389031

/ Not yet recruitingPhase 2IIT

Maralixibat for the trEatment of inTrAhePatic cHOlestasis of pRegnancy (METAPHOR)

An open label phase 2a/b trial of maralixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability

Start Date01 Apr 2026

Sponsor / Collaborator

Imperial College London

[+1]

NCT07293897

/ RecruitingNot Applicable

A Cohort Study to Investigate the Risk of Liver Disorder of Livmarli Oral Solution in Patients With Japan Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) Registered in the Comprehensive and Informative Registry System for Childhood Liver Disease (CIRCLe)

This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
The main aim of the study is to evaluate the risk of liver disorder during the use of maralixibat in Japanese patients with ALGS or PFIC.
This database study will conduct in use of medical database called Comprehensive and Informative Registry system for Childhood Liver Disease (CIRCLe).

Start Date13 Jan 2026

Sponsor / Collaborator

Takeda Pharmaceutical Co., Ltd.

100 Clinical Results associated with Maralixibat Chloride

100 Translational Medicine associated with Maralixibat Chloride

100 Patents (Medical) associated with Maralixibat Chloride

Literatures (Medical) associated with Maralixibat Chloride

01 Jul 2026·JOURNAL OF PEDIATRICS

Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials

Article

Author: Chen, Cece ; Karnsakul, Wikrom ; Mogul, Douglas B ; Hoskins, Brett J

OBJECTIVE:

To characterize baseline xanthoma prevalence and severity in Alagille syndrome, assess their impact on quality of life, evaluate longitudinal changes in xanthoma burden with maralixibat therapy, and identify clinical and biochemical predictors of treatment response in children enrolled across 2 clinical trials.

STUDY DESIGN:

This integrated analysis from the ICONIC and ITCH trials assessed xanthoma severity using the Clinician Xanthoma Scale (CXS). Response was defined as a ≥1-point reduction from baseline in CXS. Outcomes included changes in serum lipids, serum bile acids (sBA), pruritus, and Pediatric Quality of Life Inventory scores through week 96. Baseline characteristics were compared across xanthoma severity groups using the Jonckheere-Terpstra test for continuous variables and the Cochran-Armitage trend test for categorical variables. The changes in xanthoma severity category (3 levels) from baseline to weeks 48 and 96 were tested using Bowker's test of symmetry. Change from baseline (CFB) of continuous outcomes was tested using the Wilcoxon signed-rank test. Differences in CFB of outcomes between xanthoma responders and nonresponders were evaluated using the Wilcoxon rank-sum test for continuous variables and Fisher exact test for categorical outcomes.

RESULTS:

Among 63 children (ICONIC, n = 29; ITCH, n = 34), 43% had xanthomas at baseline. Higher CXS was associated with younger age (P = .03); elevated sBA (P < .001), bilirubin (P < .001), alanine aminotransferase (P = .008), and gamma-glutamyl transferase (P = .02); and worse lipid profiles (total cholesterol, P < .001; high-density lipoprotein, P < .001). Quality of life declined with increasing CXS. After 96 weeks of follow-up (ICONIC, n = 18; ITCH, n = 17), mean CXS decreased (P = .004), and CXS 0 prevalence increased from 60% to 86% (P = .03). There was a significant decline in cholesterol (mean CFB, -55 mg/dL, P < .001) and sBA (mean CFB, -85 μmol/L, P < .001). Among participants with baseline CXS ≥1 (n = 14; ICONIC, n = 8; ITCH, n = 6), 71% and 64% were xanthoma responders at weeks 48 and 96. Responders had greater cholesterol reductions at week 48 (mean CFB, -189 vs -11 mg/dL, P = .005) and more frequent pruritus improvement (94% vs 68%, P = .047).

CONCLUSIONS:

Maralixibat may reduce xanthoma burden and improve metabolic parameters, supporting therapeutic potential of the drug for xanthomas in Alagille syndrome.

CLINICAL TRIAL REGISTRATION:

This is not a clinical trial, but an integrated analysis of 2 trials; these trials are registered with ClinicalTrials.gov. • ICONIC: NCT02160782, https://clinicaltrials.gov/study/NCT02160782 • ITCH: NCT02057692, https://clinicaltrials.gov/study/NCT02057692 • IMAGINE-II (long-term extension of ITCH): NCT02117713, https://clinicaltrials.gov/study/NCT02117713.

01 Mar 2026·Liver International

Ileal Bile Acid Transporter Inhibitors in Cholestasis: Potential for More Than Just Paediatrics?

Review

Author: Boyer, James Lorenzen ; Krishna, Mahesh

ABSTRACT:

Ileal bile acid transporter inhibitors (IBATi) are a new, attractive therapeutic mechanism to alter the enterohepatic circulation through depletion of the bile acid pool by blocking bile acid reuptake in the ileum leading to improvements in pruritus and liver function in cholestatic liver diseases. These drugs may also have an impact on immunity, the gut microbiome, and motility. IBATi are approved in Japan for the treatment of idiopathic chronic constipation. There are two IBATi, maralixibat and odevixibat, that have been extensively investigated in clinical trials and are FDA approved for cholestatic pruritus in progressive familial intrahepatic cholestasis and Alagille syndrome. Clinical trials exploring IBATi in other cholestatic conditions, such as biliary atresia, primary biliary cholangitis, and primary sclerosing cholangitis, are currently ongoing. In this review, we will outline the emerging data regarding the physiology and mechanism of action for the IBATi class, an overview of clinical trials that led to the approval of maralixibat and odevixibat, ongoing clinical trials in adult cholestatic liver diseases, and the future of this drug class in systemic apical sodium bile acid transporter inhibitors.

01 Feb 2026·JPGN reports

Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome: Case reports

Article

Author: Bora, Geetanjali ; Vitola, Bernadette ; Quiros‐Tejeira, Ruben E.

Abstract:

Alagille syndrome (ALGS) is a rare, autosomal dominant disorder which presents with a broad range of clinical manifestations, including cholestatic pruritus. A unique manifestation of ALGS is the presence of xanthomas in 24%–42% of patients, which can lead to liver transplantation. Maralixibat, an ileal bile acid transporter (IBAT) inhibitor, has demonstrated improvements in both cholestatic pruritus and xanthomas in clinical trials. We report here on the use of maralixibat in two patients with ALGS and unusual manifestations of xanthomatosis, including one patient with airway xanthomas and a second patient with severe, diffuse xanthomas. In both cases, almost complete resolution of severe, debilitating xanthomas and clinically meaningful improvements in pruritus and serum bile acid levels were observed after up to 1 year of treatment with maralixibat. These cases support the utilization of maralixibat for the management of ALGS beyond cholestatic pruritus.

245

News (Medical) associated with Maralixibat Chloride

15 Sep 2026

·www.biospace.com

MitoRx Therapeutics Appoints Clinical & Scientific Advisory Board to Guide the Development of MTRX31

Chaired by Mo Qatanani, PhD, Clinical & Scientific Advisory Board brings deep expertise in obesity, cardiometabolic disease and drug development Board will guide MitoRx's development strategy for lead asset MTRX31 as it advances towards the clinic for the treatment of metabolically unhealthy obesity Harwell Oxford, UK – 15 September 2026 : MitoRx Therapeutics (“MitoRx”), a biotech company targeting the restoration of healthy mitochondrial metabolism in high-risk obesity, today announces the appointment of a Clinical & Scientific Advisory Board (CSAB) to guide the Company’s clinical development strategy for its lead asset, MTRX31. The CSAB comprises several senior external scientific and clinical leaders, plus MitoRx’s Chief Scientific Officer, Xavier Jacq, PhD, and Consultant Chief Medical Officer, László Tankó, MD, PhD, DMSc, eMBA. The Board brings deep expertise spanning obesity, cardiometabolic disease and broader drug development, from discovery through to late-stage development including for assets that became blockbuster drugs, such as semaglutide. Jon Rees, PhD, CEO of MitoRx Therapeutics said: “We are delighted to welcome Mo, Rury, Arne, Thomas, Sven, Rie and Mads to our cardiometabolic Clinical and Scientific Advisory Board. Their experience leading clinical development strategies for innovative assets in obesity and cardiometabolic disease will be invaluable as we progress MTRX31 as a differentiated, mitochondrial-targeted approach to metabolically unhealthy obesity.” “I would also like to take this opportunity to pay tribute to Professor Ele Ferrannini, who was a member of our prior Scientific Advisory Board before his sad passing earlier this year. Ele was a pioneer and leader in the field of diabetes research, we all miss not only his scientific expertise, but his kindness and generosity as a colleague and friend.” Mo Qatanani, PhD, Chair of MitoRx’s Clinical & Scientific Advisory Board, added: “The emergence of effective weight-loss therapies has transformed obesity treatment, but substantial unmet need remains among patients with metabolic dysfunction and elevated cardiovascular risk. MTRX31 represents an innovative mitochondrial-targeted approach that has generated compelling preclinical results. MitoRx has assembled a world-class group of experts across obesity, metabolism, and drug development, and I look forward to working closely with the team to shape the clinical strategy for this differentiated program.” In addition to Xavier Jacq and László Tankó, the following have been appointed to the CSAB. Full biographies are available on the MitoRx website here . Mo Qatanani, PhD (Chair) Mo Qatanani brings more than 20 years of experience in drug discovery, translational research and development across the biotechnology and pharmaceutical industries. Most recently, Mo served as Chief Scientific Officer of Scholar Rock, where he led the company's research and preclinical organization and contributed to the advancement of programs across neuromuscular and metabolic diseases. Previously, he was a founding R&D leader at Dyne Therapeutics and held scientific and leadership roles at Alexion Pharmaceuticals, Synageva BioPharma and Merck. His experience spans cardiometabolic and rare diseases, translational research, and the advancement of novel therapeutics from discovery through late-stage clinical development. Professor Arne Astrup, MD, DMSc, FASN (Deputy Chair) Professor Astrup is a world-leading researcher in nutrition, obesity and cardiometabolic disease, and co-discoverer of GLP-1 as a satiety hormone. He has authored over 1,500 peer-reviewed papers, with his research having informed international public health policy, such as Denmark's ban on industrial trans-fat. Professor Astrup is a former President of the World Obesity Federation and founding Editor-in-Chief of Obesity Reviews and currently serves on the boards of several international foundations and biotechs, including Eolo Pharma and Concepteq. Professor Rury Holman, MB, ChB, FRCP, FMedSci Professor Holman is a globally recognised authority on diabetes and cardiometabolic diseases. As an Emeritus Professor of Diabetic Medicine at the University of Oxford and former Director of the University of Oxford Diabetes Trials Unit, he has led groundbreaking clinical research for improving the management of diabetes and cardiometabolic diseases. His accolades include the ADA Harold Rifkin Award for Distinguished International Service, the Diabetes UK Robert Turner Award for Research Impact and the 2024 ADA Banting Medal for Scientific Achievement. Thomas Jaecklin, MD Thomas brings 25 years of experience as an executive drug development leader, currently serving as fractional CMO and board member in several biotech companies. Prior to this he served as CMO for NodThera, advancing a NLRP3 inhibitor pipeline in cardiometabolic indications, SVP Clinical Development at Mirum Pharmaceuticals, leading the successful registration of Livmarli® in pediatric liver disease, and Vice President, Global Project Head at Galapagos, leading a JAK inhibitor project in both commercial and Phase 3 indications. Sven Moosmang, MD, PhD Sven is an experienced physician-scientist and global clinical development leader with over 15 years of experience across cardiovascular–renal–metabolic (CVRM) disorders and obesity. He is VP of Nonclinical Sciences at Philip Morris International (PMI), where he leads a team of over 180 scientists and physicians. Prior to joining PMI, Sven served as Therapeutic Area Head, Translational Medicine & Clinical Pharmacology at Boehringer Ingelheim, leading clinical project teams across obesity, diabetes, renal and cardiovascular portfolios. Rie Schultz Hansen, PhD Rie is Chief Scientific Officer at Augustine Therapeutics, where she leads early-stage drug discovery and development initiatives. She has more than 20 years of experience in drug discovery and translational research. Rie also spent almost 12 years at Zealand Pharma in roles including Interim CSO and Head of Discovery and Innovation, leading programs across cardiometabolic and inflammatory diseases. Mads Axelsen, MD, CPharmMed, MBA With 35+ years of pharmaceutical and biotech experience with a particular focus on cardiometabolic disease, Mads recently retired from his position as the Chief Medical Officer at Gubra at the end of 2025. He supported Gubra's IPO on Nasdaq Copenhagen and played a key role in securing a landmark $2.2B licensing deal with AbbVie for the GUBamy obesity program. Mads has also held Chief Medical Officer and medical leadership roles at companies including Novo Nordisk, Vaccibody (now Nykode) and NatImmune. At Novo Nordisk, he was the medical director for major assets including semaglutide, insulin detemir and liraglutide, and managed development plans, program execution and regulatory interactions with FDA, EMA and PMDA. - Ends - About MitoRx Therapeutics MitoRx is a biotech company targeting the restoration of healthy mitochondrial metabolism in high-risk obesity. High-risk obesity is present in about 25-40% of the population with obesity and is characterized by visceral adiposity, insulin resistance, lipotoxicity and mitochondrial dysfunction, which is often described as metabolically unhealthy obesity. These patients are at disproportionate risk of metabolic-associated fatty liver disease, metabolic-associated steatohepatitis, type 2 diabetes and atherosclerotic cardiovascular disease. Our first-in-class small molecule asset, MTRX31, is designed to reduce ectopic fat by restoring healthy mitochondrial metabolism rather than suppressing appetite. This differentiated approach has the potential to deliver durable, high-quality weight loss characterized by preferential fat loss and preservation of lean mass, while improving broader measures of metabolic health. In a preclinical diet-induced obesity model, MTRX31 demonstrated significant reductions in body weight and fat mass, preservation of lean mass and improvements in metabolic parameters, with greater and more durable weight loss than tirzepatide at the doses evaluated. Find out more about our work on LinkedIn or visit our website at mitorxtherapeutics.com . For more information about MitoRx Therapeutics, please contact: MitoRx Therapeutics info@mitorxtherapeutics.com ICR Healthcare Tracy Cheung, Ashley Tapp, Phillip Marriage mitorx@icrhealthcare.com

14 Sep 2026

·www.biospace.com

Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - September 14, 2026

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that on September 10, 2026, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 28,512 shares of common stock and 37,375 restricted stock units (“RSUs”) to 14 new employees under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the awards as an inducement material to the new employees’ employment in accordance with Nasdaq Listing Rule 5635(c)(4). Each stock option has an exercise price of $100.61 per share, which is equal to the closing price of Mirum’s common stock on September 10, 2026, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date, and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The RSUs will vest over three years, with 33% of the underlying shares vesting on each anniversary of the applicable initial vesting date, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum’s 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

08 Sep 2026

·www.biospace.com

Mirum Pharmaceuticals Appoints Dr. Rob Myers as Chief Medical Officer

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced the appointment of Rob Myers, M.D., as Chief Medical Officer, effective September 21, 2026. Dr. Myers will join the company’s leadership team and lead Mirum's global medical and clinical development functions. Dr. Myers is an accomplished physician-scientist and drug development leader with more than two decades of experience developing novel medicines. A widely recognized hepatologist, he has held senior development leadership roles in industry and academia, contributed to the advancement of numerous clinical programs, and built a distinguished scientific record that includes more than 200 peer-reviewed publications. His development experience spans primary sclerosing cholangitis (PSC) and broader hepatology, including contributions to the understanding of PSC natural history, diagnosis, and management, and extends beyond liver disease through recent work in metabolism and endocrine-related disorders. Dr. Myers joins Mirum from OrsoBio, where he served as Chief Medical Officer and Head of Development. Prior to OrsoBio, he held senior leadership positions at Gilead Sciences, overseeing clinical development programs across liver fibrosis, cholestatic liver diseases and gastrointestinal inflammation. Dr. Myers earned his M.D. and completed his Internal Medicine training at Western University, followed by a Gastroenterology fellowship at the University of Calgary and a Hepatology fellowship at Université de Paris VI. He additionally earned a master's degree in Epidemiology from the University of Calgary. “Rob is a proven development leader whose experience is exceptionally well aligned with both Mirum’s portfolio today and where we are headed,” said Chris Peetz, Chief Executive Officer of Mirum. “His deep expertise in liver disease, combined with broad clinical development leadership, comes at an important moment in Mirum’s evolution with expected growth in our commercial portfolio and an increasingly diverse late-stage pipeline. Rob will play a critical role in helping us bring breakthrough medicines forward for people living with rare diseases.” ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); "Mirum has built a distinctive rare disease company by combining deep drug development and delivery expertise with a clear focus on conditions where patients have historically had too few options," said Dr. Myers. "I am excited to join at such an important stage of the company’s growth and to work with the team to advance a pipeline of medicines with the potential to make a meaningful difference for patients and families." The company also announced that Joanne Quan, M.D. will be departing Mirum. Mirum thanks Dr. Quan for her leadership and many contributions to advance Mirum’s medicines for rare disease patients. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, expected growth in our commercial portfolio, the anticipated benefits of our pipeline of medicines for patients and families, the anticipated hiring of our incoming Chief Medical Officer and the impact that Dr. Myers is expected to have on our medicines and our business. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “anticipate,” “expected,” “will,” “could,” “would,” “guidance,” “target,” “intend,” “plan,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, risks and uncertainties associated with pharmaceutical development and commercialization in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended December 31, 2025 and subsequent filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. ezstandalone.cmd.push(function () { ezstandalone.showAds(133); }); Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

Executive ChangeDrug Approval

100 Deals associated with Maralixibat Chloride

External Link

KEGG	Wiki	ATC	Drug Bank
D10951 D10952	-	-	DB16226

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
progressive familial intrahepatic cholestasis	European Union	Mirum Pharmaceuticals, Inc.	08 Jul 2024
progressive familial intrahepatic cholestasis	Iceland	Mirum Pharmaceuticals, Inc.	08 Jul 2024
progressive familial intrahepatic cholestasis	Liechtenstein	Mirum Pharmaceuticals, Inc.	08 Jul 2024
progressive familial intrahepatic cholestasis	Norway	Mirum Pharmaceuticals, Inc.	08 Jul 2024
Alagille Syndrome	European Union	Mirum Pharmaceuticals, Inc.	09 Dec 2022
Alagille Syndrome	Iceland	Mirum Pharmaceuticals, Inc.	09 Dec 2022
Alagille Syndrome	Liechtenstein	Mirum Pharmaceuticals, Inc.	09 Dec 2022
Alagille Syndrome	Norway	Mirum Pharmaceuticals, Inc.	09 Dec 2022
Cholestatic pruritus	United States	Mirum Pharmaceuticals, Inc.	29 Sep 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Cholestatic liver disease	Phase 3	United States	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Brazil	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Canada	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	France	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Germany	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Italy	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Lebanon	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Poland	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	Spain	Mirum Pharmaceuticals, Inc.	14 Oct 2024
Cholestatic liver disease	Phase 3	United Kingdom	Mirum Pharmaceuticals, Inc.	14 Oct 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02117713 \| NCT02057692 \| NCT02160782 (IMAGINE-II, Pubmed \| J Pediatr)	Phase 2	Alagille Syndrome	63	Maralixibat	eqhtvsevcd(izslbmqvld) = opfkkpducr rzmnqoiexu (qyhjhfhxne ) View more	Positive	01 Jul 2026
NCT04729751 (RISE, CTgov)	Phase 2	Alagille Syndrome \| progressive familial intrahepatic cholestasis \| Cholestatic liver disease	27	Maralixibat (Maralixibat: ALGS)	vwxhzpaahp = pybqrqiqzj yegeewocih (jetgvzkiku, bdncqshigm - ysjapysmpy) View more	-	02 Jun 2026
				PFIC+Maralixibat (Maralixibat: PFIC)	vwxhzpaahp = rglwmvbbad yegeewocih (jetgvzkiku, lclknwwngk - xpufedsvjp) View more
NCT04168385 (MRX-800 \| MERGE, CTgov)	Phase 2	Cholestatic liver disease	52	Maralixibat (Maralixibat: ALGS)	vptzhtrbmp = xrkggxqvzk mpxecnweed (fzadbvjwwr, qkhkqhdfkt - nclvaelknt) View more	-	08 Dec 2025
				PFIC+Maralixibat (Maralixibat: PFIC)	vptzhtrbmp = yjrytxeacw mpxecnweed (fzadbvjwwr, gcpgppofko - tmrvsxghkq) View more
NCT02160782 (FDA_CDER) Manual	Phase 2	Alagille Syndrome \| Pruritus	29	Maralixibat	yrgffekaux(vimzlkdcdb) = eaoazvpvek jdtlswkwpp (cgoyhzgpsy, -0.3 to 0.7)	Positive	10 Apr 2025
				Placebo	yrgffekaux(vimzlkdcdb) = ntjjxtvbyi jdtlswkwpp (cgoyhzgpsy, 1.2 - 2.1)
NCT03905330 (FDA_CDER) Manual	Phase 3	progressive familial intrahepatic cholestasis	64	Maralixibat	vgrkadiinb(lrcgdkaxjw) = mawqevustw jyzipdptfv (cwgnwofbrb, -2.2 to -1.4)	Positive	10 Apr 2025
				Placebo	vgrkadiinb(lrcgdkaxjw) = ijpmqsjjoo jyzipdptfv (cwgnwofbrb, -1.0 to -0.2)
NCT04524390 (EMBARK, CTgov)	Phase 2	Biliary Atresia	75	Maralixibat (Double Blind - Maralixibat)	kqbtkfsccp(mpehmvjelu) = brjxsvumok tnekyjggtj (bduxyhnjvf, 0.853) View more	-	17 Feb 2025
				Placebo (Double Blind - Placebo)	kqbtkfsccp(mpehmvjelu) = obfpsjfyhj tnekyjggtj (bduxyhnjvf, 0.947) View more
NCT05543174 (CTgov)	Phase 3	Alagille Syndrome	7	TAK-625	pvxqjmibdz(fptxcrnizc) = aslxdicugl wwcdjtmzvf (esaisyiyhf, fpkxehtkhi - wqyfcojcyo) View more	-	15 Oct 2024
NCT05543187 (CTgov)	Phase 3	progressive familial intrahepatic cholestasis	5	TAK-625 (Primary Cohort: TAK-625)	aeuvbvxfor(mtuqkykvge) = jbvvwqyjsq ixxdglfroc (nlkaqojxig, wrxxvwzuka - ipqvtznagw) View more	-	24 Sep 2024
				TAK-625 (Supplemental Cohort: TAK-625)	aeuvbvxfor(mtuqkykvge) = rutabsgnzi ixxdglfroc (nlkaqojxig, tdsvhgarwb - qkhtdukmii) View more
NCT04168385 (MERGE, NEWS)	Phase 2	Alagille Syndrome	76	马昔巴特	llgcvqdlav(swacyblpko) = vvoiytkolf mkvhocucep (pxfzngpioi ) View more	Positive	07 Jun 2024
MARCH-ON (Biospace) Manual	Not Applicable	progressive familial intrahepatic cholestasis	-	LIVMARLI	mbowtrimln(jabnejorft) = in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study. xnoqtugzte (nztgyotqxt )	Positive	18 May 2024

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