Mirum Pharmaceuticals to Present LIVMARLI® (maralixibat) Analyses and Host a Scientific Symposium at NASPGHAN 2022 Annual Meeting

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present three posters and host a scientific symposium during the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) Annual Meeting taking place October 12-16, 2022, in Orlando, Florida.

Analyses Presented During the Congress

Poster #290: Maralixibat Improves Growth in Patients with Alagille Syndrome: A 4-Year Analysis (p. 210)

Friday, October 14, 2022 from 12:00-2:30pm ET, Poster Session II

Presented by: Binita M. Kamath, MBBChir, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada

Poster #291: Maralixibat-Treated Patients with Alagille Syndrome Demonstrate Improved Event-Free Survival in a Natural History Comparison with Patients from the GALA Database: Application of Real-World Evidence Analytics (p. 291)

Friday, October 14, 2022 from 12:00-2:30pm ET, Poster Session II

Presented by: Binita M. Kamath, MBBChir, The Hospital for Sick Children (SickKids), Toronto, Ontario, Canada

Poster #552: Caregiver Burden Associated with Caring for a Child with Alagille Syndrome: A Multi-National Quantitative Analysis (p.403)

Saturday, October 15, 2022 from 12:00-2:30pm ET

Presented by: Robin Howard, Mirum Pharmaceuticals, Inc.

The abstracts are now available via the NASPGHAN website and full presentations will be available upon presentation at the congress, which starts on October 12.

Scientific Symposium

Mirum will host a scientific symposium, “Beyond Itch: Exploring the Clinical Benefits of Treating Cholestasis in Children,” featuring Drs. William F. Balistreri (chair), Binita M. Kamath, Regino P. Gonzalez-Peralta, and Saeed Mohammad.

The event will take place on Thursday, October 13, 2022 from 7:15-8:45pm ET in Sebastian L 1/2.

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) one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and 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 bone fractures and gastrointestinal bleeding.

Prescribing Information

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare 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 one year of age and older. Please view the warnings and precautions available in the Prescribing information or, for more information, visit LIVMARLI.com.

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study 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 has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

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

Learn more about Mirum by visiting www.mirumpharma.com. Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

Forward-Looking Statements

This press release includes forward-looking statements or efficacy 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, and the therapeutic potential thereof, the continuation of our clinical trials, and the success of our collaborations with partners and any potential future collaborations. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading “Risk Factors” included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements, except as required by law.

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