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

Mirum Pharmaceuticals, Inc., headquartered in Foster City, California, is a biopharmaceutical company focused on developing and commercializing innovative therapies for rare and orphan diseases. The firm recently announced its participation at the Goldman Sachs 45th Annual Global Healthcare Conference in Miami, scheduled for June 12th at 10:00 AM EDT. Additional details and webcast links are available on the company's website.

Mirum Pharmaceuticals is devoted to transforming the treatment landscape for rare diseases affecting both pediatric and adult populations. The company boasts a portfolio of three FDA-approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.

LIVMARLI is an ileal bile acid transporter (IBAT) inhibitor, approved for treating two rare liver conditions that affect children and adults. In the United States, it is sanctioned for the treatment of cholestatic pruritus in patients with Alagille syndrome who are three months of age or older. In Europe, it is approved for patients aged two months and older, and it is available in various other global regions. Additionally, in the U.S., LIVMARLI is approved for managing cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) in patients aged five years and older.

CHOLBAM is another FDA-approved treatment, aimed at managing bile acid synthesis disorders caused by single enzyme deficiencies and serving as an adjunctive treatment for peroxisomal disorders in patients displaying signs or symptoms of liver disease.

CHENODAL, the third approved medication, is recognized by the FDA as a necessary treatment for patients with cerebrotendinous xanthomatosis (CTX), a rare genetic disorder.

Mirum's pipeline also features promising late-stage investigational treatments targeting debilitating liver diseases. Volixibat, another IBAT inhibitor, is under 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 subject to a Phase 3 clinical trial named RESTORE, aimed at treating patients with CTX, with positive topline results announced in 2023.

In summary, Mirum Pharmaceuticals is committed to offering novel therapies for rare diseases, with a strong focus on liver conditions. The company's efforts encompass both the commercialization of approved treatments and the continued development of investigational therapies to address unmet medical needs within the rare disease community. For further information, visit Mirum’s website and follow their social media channels.