Mirum Pharmaceuticals to Highlight LIVMARLI Data from its ALGS and PFIC Programs at the NASPGHAN Annual Meeting

- 15 abstracts accepted for presentation showcasing LIVMARLI data from ALGS and PFIC programs

- LIVMARLI PFIC primary analysis from MARCH study awarded prestigious Gerard Odell Prize for Excellence in Pediatric Hepatology Research

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that data from its LIVMARLI^® (maralixibat) oral solution studies as well as data from real- world experience will be presented at the upcoming North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) annual meeting taking place October 4-7, 2023, in San Diego, California.

Highlighted below are the titles that have been accepted for presentation during the meeting. The abstracts are available via the NASPGHAN website. Full analyses will be available following their presentation within the Publications & Presentations section on Mirum’s website.

Oral Presentation

Abstract 531: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH): A randomized placebo-controlled phase 3 study

Saturday, October 7, 2023, between 10:30am-12:00pm during the ‘Spill the Tea on Liver Disease’ session

**Gerard Odell Prize for Excellence in Pediatric Hepatology Research Recipient**

Poster Presentations

Alagille syndrome (ALGS)

Abstract 69: Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 71: Maralixibat persistency and adherence for the treatment of cholestatic pruritus in Alagille Syndrome: Real-world experience in the United States

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 72: Maralixibat impact on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 88: Impact of maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 89: Maralixibat, an ileal bile acid transporter inhibitor, delays the need for liver transplant in patients with Alagille syndrome: Real-world experience

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 626: Real-world safety experience in patients with Alagille syndrome treated with maralixibat

Saturday, October 7, 2023, between 12:00-2:00 p.m. PT during Poster Session III

Abstract 632: Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: An integrated analysis from two clinical trials

Saturday, October 7, 2023, between 12:00-2:00 p.m. PT during Poster Session III

Progressive Familial Intrahepatic Cholestasis (PFIC)

Abstract 65: Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH trial

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 85: 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

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 86: Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: Data from the MARCH trial

Thursday, October 5, 2023, between 5:00-7:00 p.m. PT during Poster Session I

Abstract 347: Analysis of long-term safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from MARCH-ON

Friday, October 6, 2023, between 12:00-2:00 p.m. during Poster Session II

Abstract 625: Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from the MARCH trial

Saturday October 7, 2023, between 12:00-2:00 p.m. during Poster Session III

Abstract 630: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: Data from the MARCH trial

Saturday, October 7, 2023, between 12:00-2:00 p.m. during Poster Session III

ALGS and PFIC

Abstract 363: Safety and tolerability of maralixibat in infants from 2 months of age with Alagille syndrome or progressive familial intrahepatic cholestasis: Results from the RISE study

Friday, October 6, 2023, between 12:00-2:00 p.m. during Poster Session II

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 cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. 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. 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 three investigational treatments for debilitating liver diseases. The LIVMARLI development program includes the Phase 2b EMBARK study for biliary atresia. Mirum’s second investigational IBAT inhibitor is volixibat, which 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, is being evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX.

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

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 therapeutic potential and/or commercial viability of our product candidates and technologies in PFIC and ALGS. 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230926619683/en/