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Long-Term Data from Mirum’s LIVMARLI Studies in ALGS and PFIC Highlighted at EASL Congress

- Data showcases sustained clinical benefit following seven years of LIVMARLI treatment in patients with Alagille syndrome

- Long-term extension data from LIVMARLI PFIC studies highlight improvement in key liver markers and growth

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from new analyses showing long-term treatment with LIVMARLI were presented during the European Association for the Study of the Liver (EASL) Annual Congress, June 5-8, 2024, in Milan, Italy. Presentations from studies evaluating LIVMARLI® (maralixibat) oral solution in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) included:

THU-094: Clinical Benefits with Maralixibat for Patients with Alagille Syndrome are Durable Through 7 Years of Treatment: Data from the MERGE Study

Presented by Doug Mogul, MD, PhD – Mirum Pharmaceuticals, Inc., Foster City, CA, USA

An analysis of ALGS studies evaluating LIVMARLI for up to seven years of treatment were presented to demonstrate the durability of response to LIVMARLI across multiple clinical and laboratory outcomes. Data demonstrated that nearly all patients who remained on LIVMARLI for seven years experienced benefit including clinically meaningful reductions in pruritus and serum bile acids, along with improvements in height.

View the full presentation.

THU-129: Maralixibat Improves Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials

Presented by Professor Richard J. Thompson, MD, PhD – King's College, London, UK

This analysis reported on the long-term impact of LIVMARLI on improvements in growth across a variety of PFIC types in 60 patients from the All-PFIC cohort of the MARCH/MARCH-ON clinical trials. Patients treated with LIVMARLI from the All-PFIC cohort had a statistically significant improvement in height and weight z-scores which persisted through 82 weeks of treatment.

View the full presentation.

THU-158: Improvements in Serum Bile Acid Levels are Associated with Improvements in Key Markers of Liver Health After Maralixibat Treatment in Children with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials

Presented by Lorenzo D’Antiga, MD - Papa Giovanni XXIII Hospital, Bergamo, Italy

This analysis aimed to assess correlations between serum bile acid (sBA) improvements and key liver parameters, including bilirubin. These data showed that reductions in sBA after treatment with LIVMARLI are correlated with reductions in bilirubin, a key marker of liver disease. These results indicate the potential for LIVMARLI to have disease modifying effects and improvements in liver health for patients with PFIC.

View the full presentation.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and for progressive familial intrahepatic cholestasis (PFIC) five years of age and older.

LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.

LIVMARLI has received positive opinion by CHMP in Europe for the treatment of PFIC in patients three months of age and older. A decision by the European Commission is expected by the third quarter of 2024.

LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. 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 Alagille syndrome 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 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, CHOLBAM® (cholic acid) capsules, and CHENODAL® (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 five years of age and older. LIVMARLI has received positive opinion by CHMP in Europe for the treatment of PFIC in patients three months of age and older. A decision by the European Commission is expected by the third quarter of 2024. 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 or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, including data presentation title and synopsis, which may include discussion of the Company’s clinical and research data regarding patient growth data while on LIVMARLI, the reliability of long-term data, and the therapeutic potential and/or commercial viability of LIVMARLI in various liver disease indications and in patient populations that are investigational only. 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 “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The accuracy of such statements is subject to a number of risks, uncertainties and assumptions including, but are not limited to, the following factors: the uncertainties inherent in research and development; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Mirum’s business and prospects, adverse developments in our focused markets, or adverse developments in the U.S. or global regulatory environment or economies generally; the continued impact of COVID-19 on our business, operations and financial results; and competitive developments. Other factors that might cause such a difference include those discussed in the Company’s filings with the SEC. 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.

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