Maralixibat Chloride

New data reinforce Mirum’s commitment to advancing therapies and improving the lives of patients across diverse rare liver diseases. FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that multiple presentations featuring data from its LIVMARLI (maralixibat) and volixibat clinical programs will be shared during the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting®, taking place November 7-11, 2025, in Washington, D.C. The presentations highlight Mirum’s continued progress in expanding the scientific and clinical foundation for new approaches to treating adult and pediatric rare liver diseases, including new findings from the VANTAGE trial and real-world studies. Oral Presentation A key highlight at this year’s meeting will be an oral presentation featuring additional analyses from the VANTAGE trial evaluating volixibat in adults with primary biliary cholangitis (PBC). Abstract #0215: Volixibat Leads to Improvements in Fatigue and Sleep for Adults with Primary Biliary Cholangitis: Data from VANTAGE Trial Monday, November 10, 2025, 3:00-3:15 pm during the ‘Novel Therapies and Monitoring in Cholestatic Liver Disease’ session, Room 145, Level 1, Convention Center Presented by Kris Kowdley, Liver Institute Northwest, Seattle, Washington, USA Poster Presentations In addition to new volixibat data, several abstracts highlight real-world experience with maralixibat and insights from genetic testing in cholestasis. Abstract #4411: Maralixibat, an Ileal Bile Acid Transporter Inhibitor, Reduces Cholestatic Pruritus in PSC: Real-World Experience Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Adrielly Martins, University of Miami, Florida, USA Abstract #4412: Clinical Utility of Comprehensive Genetic Testing in Adult Cholestasis: Insights from 856 Panel Tests Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Tiziano Pramparo, Mirum Pharmaceuticals, Inc., Foster City, California, USA Abstract #4415: Exploring the Role of Ileal Bile Acid Transporter Inhibitors, Like Volixibat, in the Regulation of IL-31 and Serum Bile Acids in Patients with Primary Biliary Cholangitis: Preliminary Results from the VANTAGE Trial Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Alan Bonder, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA Abstract #4511: Physician Reported Assessment of Pruritus Amongst Patients with PSC: Results from a Real-World Survey in France, Germany, Italy and the United States Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Marlyn Mayo, UT Southwestern Medical Center, Dallas, Texas, USA Abstract #4532: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis: Baseline and 6-month Follow-up Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Natasha Dilwali, Columbia University Irving Medical Center, New York, New York, USA Fireside Discussion Mirum will also host an educational fireside discussion addressing current approaches to Progressive Familial Intrahepatic Cholestasis (PFIC) diagnosis and management. PFIC Diagnosis and Treatment Fireside Discussion (Mirum-Sponsored) During The Liver Meeting, Mirum will host a session featuring Dr. Robert Gish, Loma Linda University, focused on the recognition and diagnosis of PFIC, including the role of genetic testing and disease management considerations for adults. The discussion will also include a review of clinical data for LIVMARLI in PFIC. Sunday, November 9, 2025, 1:30-2:00 pm Exhibit Hall TLM Theater Mirum’s presence at The Liver Meeting underscores the company’s commitment to advancing therapies that improve patient outcomes across rare liver diseases. Full abstracts are available on the AASLD website. Following the conference, detailed analyses will be posted in the Publications and Presentations section of Mirum’s website. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI® (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is conducting the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum has also initiated a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (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, the Company’s planned participation at a scientific congress, Mirum’s leadership in liver disease and some anticipated scientific presentations of data from Mirum’s LIVMARLI and volixibat studies at the American Association for the Study of Liver Diseases. 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 “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” 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, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. 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. Source: Mirum Pharmaceuticals, Inc. Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

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