Mirum Pharmaceuticals to Present at JMP Life Sciences Conference

Mirum Pharmaceuticals, Inc., a biopharmaceutical company based in Foster City, California, has announced its participation in the upcoming Citizens JMP Life Sciences Conference. This event will be held in New York, NY on Tuesday, May 14th at 1:30 PM EDT. More details and webcast links can be accessed through the Investors and Media section of Mirum’s official website.

Mirum Pharmaceuticals, Inc. is committed to developing and bringing to market innovative treatments for rare diseases that impact both children and adults. The company currently has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor that treats two rare liver diseases in both pediatric and adult patients. It is approved for managing cholestatic pruritus in individuals with Alagille syndrome in the United States for patients aged three months and older, in Europe for those two months and older, and in various other regions globally. Additionally, it is approved in the United States for treating cholestatic pruritus in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) aged five years and older. An application is currently under review in Europe to extend this treatment to PFIC patients two months of age and older.

CHOLBAM, another product from Mirum, is approved by the FDA for treating bile acid synthesis disorders caused by single enzyme deficiencies and as an adjunct treatment for peroxisomal disorders in patients who exhibit symptoms or liver disease. CHENODAL has been recognized by the FDA for its medical necessity in treating patients with cerebrotendinous xanthomatosis (CTX).

In addition to its approved products, Mirum is also advancing a late-stage pipeline focused on debilitating liver diseases. The investigational IBAT inhibitor Volixibat is undergoing evaluation in two potentially registrational Phase 2b studies: the VISTAS study for primary sclerosing cholangitis and the VANTAGE study for primary biliary cholangitis. Furthermore, CHENODAL has been assessed in a Phase 3 clinical study called RESTORE, aimed at treating CTX, with positive topline results reported in 2023.

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