Mirum Pharmaceuticals to Present at the Goldman Sachs 45th Annual Global Healthcare Conference

Mirum Pharmaceuticals to Present at the Goldman Sachs 45th Annual Global Healthcare Conference

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases, today announced that it will present at the Goldman Sachs 45th Annual Global Healthcare Conference in Miami on Wednesday, June 12 th at 10:00 AM EDT.

Visit the Investors and Media section of Mirum's corporate website for webcast links and additional information.

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. 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) .

Investor Contact:
Andrew McKibben
ir@mirumpharma.com

Media Contact:
Erin Murphy
media@mirumpharma.com

News Provided by Business Wire via QuoteMedia

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

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

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Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older. The CHMP also concluded that LIVMARLI in PFIC brings significant clinical benefit based on improved efficacy and a major contribution to patient care compared to the existing approved treatment for PFIC. The CHMP positive opinion is the scientific recommendation to the European Commission for marketing authorization in Europe.

In addition, the Committee for Orphan Medicinal Products (COMP), has also recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC. The European Commission's endorsement of the CHMP and COMP opinions is expected in the third quarter of 2024.

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