Mirum Pharmaceuticals to Present New LIVMARLI Data at the 2023 EASL Congress

• Five abstracts accepted for presentation, including data demonstrating bilirubin normalization with LIVMARLI in PFIC
• Sleep improvement data from MARCH-PFIC study included among ‘Best of EASL’ presentations
• Maintenance of response in patients with PFIC selected as late-breaker presentation

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that it will present data at the upcoming International Liver Congress of the European Association for the Study of the Liver taking place June 21-24, 2023 in Vienna, Austria.

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

Abstract OS-072: Maralixibat leads to significant reductions in bilirubin for patients with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC

Oral presentation during Abstract Session 2: Immune-mediated and cholestatic diseases

Friday, June 23, 2023 from 5:45-6:00pm CET

Author: Dr. Lorenzo D’Antiga, Papa Giovanni XXIII Hospital, Bergamo, Italy

LBP-35: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study

*Late-breaker poster presentation*

June 21, 2023, throughout the day

Author: Professor Richard J. Thompson, King’s College, London, United Kingdom

WED-257: Impact of maralixibat on cholestatic pruritus in adults aged 16 years and older with Alagille syndrome

Poster presentation during Poster Session: Immune-mediated and cholestatic diseases

Wednesday, June 21, 2023, throughout the day

Author: Dr. Binita Kamath, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada

WED-252: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC

**Selected to be included as part of ‘Best of EASL’ presentation**

Poster presentation during Poster Session: Immune-mediated and cholestatic disease

Wednesday, June 21, 2023, throughout the day

Author: Professor Richard J. Thompson, King’s College, London, United Kingdom

WED-282: Analysis of safety in maralixibat-treated participants with Progressive Familial Intrahepatic Cholestasis: Data from MARCH-PFIC

*Poster tour*

Wednesday, June 21, 2023, 1:13-1:20pm CET as part of overall poster tour from 12:45-1:45pm CET

Author: Dr. Alexander Miethke, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA

The abstracts are available on the EASL website and during the congress, the presentations will be available within the Publications and Presentations section on Mirum’s website.

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, therapeutic potential, and commercialization of our product candidates and technologies, as well as, anticipated 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.

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