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.

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Chiesi Ventures

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Rock Springs Capital

Novo Holdings

Pappas Capital

Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. The company's lead product candidate is LIVMARLI, an investigational oral drug for the treatment of progressive familial intrahepatic cholestasis disease, as well as for the treatment of Alagille syndrome and biliary atresia disease. It also develops Volixibat drug for treatment of intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Mirum Pharmaceuticals, Inc. was incorporated in 2018 and is headquartered in Foster City, California.

COM:MIRUMPHARMA

Mirum Pharmaceuticals Inc

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39.18

31/10 17:59

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