Business
Mirum Pharmaceuticals Announces Second Quarter 2020 Financial Results and Provides Clinical Program Updates
Rolling NDA submission for maralixibat in Alagille syndrome (ALGS) to be initiated in third quarter 2020; planning for potential launch in second half of
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nRolling NDA submission for maralixibat in Alagille syndrome (ALGS) to be initiated in third quarter 2020; planning for potential launch in second half of 2021.\n\n\nSubmission of European marketing authorization application for maralixibat in PFIC2 planned for fourth quarter of 2020.\n\n\nExpanded Access Program for maralixibat in ALGS planned to open for registration in September 2020.\n\n\nOrphan Drug Designation received by European Medicines Agency for maralixibat in biliary atresia.\n\n\nCash, cash equivalents and investments of $149.3 million.\n\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 business updates for the quarter ended June 30, 2020.\n\n\n“We made significant progress toward making maralixibat available as an alternative to liver transplantation in devastating pediatric cholestatic settings in the United States and Europe,” said Chris Peetz, president and chief executive officer at Mirum. “In the United States, we are initiating a rolling NDA submission for maralixibat in ALGS and are planning to start an expanded access program broadening availability to this important medicine for eligible patients. By the end of this year, we intend to submit a marketing authorization application for maralixibat in PFIC2 in Europe, dramatically shifting our timeline for a potential launch in this region.”\n\n\nSecond Quarter Highlights\n\n\n\nReleased maralixibat data demonstrating five-year transplant-free survival for pediatric patients with PFIC2. Data will be presented at the upcoming Digital International Liver Congress, August 27-29, 2020.\n\n\nCompleted pre-submission meeting with European regulators resulting in feedback enabling submission of a marketing authorization application (MAA) for maralixibat in the treatment of patients with PFIC2 in Europe in the fourth quarter of 2020.\n\n\nInvestigational new drug (IND) application cleared by FDA for the initiation of a Phase 2 study evaluating volixibat in adult patients with pruritus associated with primary sclerosing cholangitis.\n\n\nReceived orphan drug designation in Europe for maralixibat in the study of patients with biliary atresia; planning for a Phase ...