Business
Mirum Pharmaceuticals Provides Third Quarter 2020 Financial Results and Business Update, and Announces Virtual Investor Day
- Initiated rolling NDA submission and launched Expanded Access Program for maralixibat in Alagille syndrome. - Presented five-year transplant-free survival
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Initiated rolling NDA submission and launched Expanded Access Program for maralixibat in Alagille syndrome. \n\n- Presented five-year transplant-free survival data for patients with PFIC2 at Digital International Liver Congress (EASL).\n\n- European Marketing Authorization Application submission for maralixibat in PFIC2, planned by year-end 2020.\n\n- Cash, cash equivalents and investments of $133.7 million.\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced financial results and a corporate update for the quarter ended September 30, 2020.\n\n“This quarter marked several milestones toward providing better treatment options for Alagille syndrome and PFIC, with the initiation of our rolling NDA submission, the launch of an expanded access program, and presentation of five-year transplant free survival data in PFIC2,” said Chris Peetz, president and chief executive officer of Mirum. “Looking forward to next year, we are planning for the U.S. launch of maralixibat in Alagille syndrome and the expansion of our programs, with upcoming study starts in biliary atresia, primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy, all settings with high disease burden and no currently approved therapies.”\n\nKey Operational Highlights\n\n\nPresented five-year transplant-free survival data for patients with PFIC2 at Digital International Liver Congress (EASL).\n\n\nInitiated rolling submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).\n\n\nLaunched maralixibat Expanded Access Program (EAP) for the treatment of cholestatic pruritus associated with ALGS in United States, Canada, Australia, and 10 countries in Europe.\n\n\nAnnounced partnership with EVERSANA™ to support the planned launch and commercialization of maralixibat in ALGS in the United States, if approved.\n\n\nReceived Orphan Drug Designation from the U.S. FDA for maralixibat in biliary atresia; Phase 2 study initiation planned for the first quarter of 2021.\n\n\nReceived rare pediatric disease designation for maralixibat for the treatment of PFIC.\n\n\nTh...