Business
Mirum Pharmaceuticals Reports First Quarter 2020 Financial Results and Provides Business Updates
- On track to begin rolling submission of NDA for maralixibat in Alagille Syndrome (ALGS) in third quarter 2020 - Cash, cash equivalents and investments
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- On track to begin rolling submission of NDA for maralixibat in Alagille Syndrome (ALGS) in third quarter 2020\n\n\n- Cash, cash equivalents and investments balance of $166.1 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 reported financial results and provided business updates for the quarter ended March 31, 2020.\n\n\n“Despite the COVID-19 pandemic, we remain on track to begin the rolling submission of our new drug application to the U.S. Food and Drug Administration in the third quarter of this year with the goal of launching maralixibat for patients with Alagille syndrome in the second half of 2021,” said Chris Peetz, president and chief executive officer at Mirum. “Further, we are exploring the potential to accelerate our regulatory filings for maralixibat in PFIC2 and will be discussing the Phase 2 INDIGO study 5-year outcomes versus a natural history cohort with regulators this year.”\n\n\nFirst Quarter 2020 Highlights\n\n\n\nBolstered balance sheet with $48.0 million gross proceeds from a follow on public offering in January 2020.\n\n\nAs of March 31, 2020, Mirum had cash, cash equivalents and investments of $166.1 million; projected to fund current operations through the anticipated FDA approval of maralixibat in Alagille syndrome (ALGS) in the second half of 2021.\n\n\nCompleted Phase 1 dose-ranging and drug-drug interaction study for volixibat to inform dose regimens for planned registrational program.\n\n\n\nUpcoming Anticipated Milestones\n\n\n\nInitiate rolling submission of NDA for maralixibat in ALGS in the third quarter of 2020.\n\n\nPresentations highlighting data from maralixibat and volixibat studies planned for upcoming medical congresses including European Association for the Study of the Liver and the American Association for the Study of Liver Diseases.\n\n\nInitiate ALGS expanded access program in the United States and Canada in the fourth quarter of 2020.\n\n\nDiscuss with regulators the suitability of maralixibat INDIGO study 5-year outcomes data in PFIC2 patients compared to natural history for potential regulatory submission.\n\n\nUS launch for maralixibat in ALGS remains on track for 2021.\n\n\n\nCOVID-19 Pandemic I...