• Seven abstracts accepted including four oral presentations
• Phase 3 MARCH PFIC data selected as one of highest scoring abstracts and will be presented during the plenary session on May 18
• RISE study evaluating LIVMARLI in infants with ALGS or PFIC included among highest scoring posters

FOSTER CITY, Calif. / May 17, 2023 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it will present data at the upcoming 55^th Annual European Society for Pediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting, taking place May 17-20, 2023, in Vienna, Austria. The company will present data from its LIVMARLI^® (maralixibat) oral solution clinical studies in Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC).

Data from Mirum’s studies of LIVMARLI^® (maralixibat) oral solution include oral and poster presentations:

Oral presentations

Abstract 477 – H-O027: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH-PFIC): A randomized placebo-controlled Phase 3 study
Thursday, May 18, 2023 - 11:45am-1:15pm CET, Plenary Session, Highest Scoring Abstracts, Hall A
Presented at 1:02pm CET by Professor Richard J. Thompson, King’s College, London

Abstract 468- H-O029: Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH-PFIC trial
Friday, May 19, 2023 – 12:00-1:00pm - Parallel Session: Hepatology-Abstract Session 02, Hall G
Presented at 12:00pm CET by Professor Richard J. Thompson, King’s College, London

Abstract 472 – H-O010: Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: an integrated analysis from two clinical trials
Friday, May 19, 2023 – 12:00-1:00pm - Parallel Session: Hepatology-Abstract Session 02, Hall G
Presented at 12:32pm CET Dr. Brett Hoskins, The Johns Hopkins Hospital School of Medicine, Baltimore, Maryland

Abstract 463 – H-O008: Real-world safety experience in patients with Alagille syndrome treated with maralixibat
Friday, May 19, 2023 – 12:00-1:00pm, Parallel Session: Hepatology-Abstracts 02, Hall G
Presented at 12:40pm CET by Dr. Regino P. Gonzalez-Peralta, AdventHealth for Children and AdventHealth Transplant Institute, Orlando, Florida

Poster Presentations

Abstract 473: Safety and tolerability of maralixibat in infants from two months of age with Alagille syndrome or progressive familial intrahepatic cholestasis: Results from the RISE study
Friday, May 19, 2023 – 2:40-3:30pm CET – Poster Walk Hepatology 02: General Hepatology I, Exhibition Area
Presented at 2:56pm CET by Professor Emanuel Gonzales, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP, Université Paris-Saclay, Le Kremlin-Bicêtre, France

Abstract 469: Maralixibat, an ileal bile acid transporter inhibitor, delays the need for liver transplant in patients with Alagille syndrome: Real-world experience
Friday, May 19, 2023 – 2:40-3:30pm CET –Hepatology 02: General Hepatology I, Exhibition Hall
Presented by Dr. Douglas B. Mogul, Mirum Pharmaceuticals, Inc. Foster City, California USA

Abstract 470: Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome
Friday, May 19, 2023 – 2:40-3:30pm CET –Hepatology 02: General Hepatology I, Exhibition Hall
Presented by Dr. Bernadette Vitola, Children’s National Hospital, Washington, DC USA

All abstracts will be available on the ESPGHAN website via the ESPGHAN website on May 17, 2023.

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) 3 months of age and older. LIVMARLI is also approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older. It is the only approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information for U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older).

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.

IMPORTANT SAFETY INFORMATION

LIVMARLI can cause side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite.

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were gastrointestinal bleeding and bone fractures.

US Prescribing Information
EU SmPC

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved medication is LIVMARLI^® (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and in Europe for the same indication in patients two months of age and older.

Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe (in PFIC for patients two months of age and older).

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.

Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.

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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, including the discovery, development, and commercialization of our product candidates and technologies, the therapeutic potential of Company products, the continuation of our clinical trials, and any potential future collaborations.. 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. 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 the COVID-19 pandemic, and the other risks described in Mirum’s 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.