Business
Mirum Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Corporate Update
New Drug Application Rolling Submission for Alagille Syndrome Planned to Begin in Third Quarter 2020 Cash, Cash Equivalents and Investments Balance of $140.0
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\nNew Drug Application Rolling Submission for Alagille Syndrome Planned to Begin in Third Quarter 2020\n\n\nCash, Cash Equivalents and Investments Balance of $140.0 million before gross proceeds of $48.0 million from a follow-on public offering in January 2020\n\n\nOn Track for PFIC Phase 3 Top-Line Data and Long-term Outcomes Natural History Comparisons by Year End\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 and business results for the quarter and year ended December 31, 2019.\n\n\n“2019 was a year of ground-breaking achievements for Mirum,” said Chris Peetz, president and chief executive officer of Mirum. “We established Mirum as a leading company in cholestatic liver diseases and have positioned ourselves to deliver our first NDA submission, planned to begin in the third quarter of this year. Maralixibat has potential to alter the course of disease in pediatric cholestasis and we are excited to be one step closer to delivering medicines to children with debilitating liver diseases. With the additional capital raised in January 2020 we are well positioned to grow our pipeline including executing on the upcoming NDA, maralixibat launch preparation, completing our ongoing studies and launching potentially registrational studies for volixibat.”\n\n\nKey Operational Highlights in Fourth Quarter 2019\n\n\n\nSuccessful completion of a pre-NDA meeting with the Food and Drug Administration (FDA) for maralixibat with alignment on submission of rolling NDA starting Q3 2020.\n\n\nBreakthrough Therapy Designation granted by the FDA for maralixibat for the treatment of pruritus associated with Alagille syndrome in patients 1 year of age and older.\n\n\nRare Pediatric Disease Designation granted by the FDA for Alagille syndrome. If maralixibat’s NDA is approved prior to October 1, 2022, the company may be eligible to receive a priority review voucher, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred.\n\n\nPresented data demonstrating the long-term durability of maralixibat in children with cholestatic liver disease. At the Liver Meeting in November 2019, data were presented s...