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Dec 31, 2020
Mirum Pharmaceuticals Q4 2020 Earnings Report
Mirum Pharmaceuticals reported results for the fourth quarter of 2020 and provided business updates.
Key Takeaways
Mirum Pharmaceuticals reported a net loss of $37.2 million for the fourth quarter of 2020, with total operating expenses of $37.0 million. The company is preparing for the potential launch of maralixibat for Alagille syndrome in the U.S. in the second half of 2021, pending FDA approval, and has initiated Phase 2b programs for volixibat in various cholestatic conditions.
Completed rolling NDA submission to FDA for maralixibat for the treatment of pruritus in patients with Alagille syndrome (ALGS) one year of age and older in January 2021.
Received validation (acceptance) of Mirum’s marketing authorization application (MAA) to the European Medicines Agency (EMA) for maralixibat for the treatment of patients with Progressive Familial Intrahepatic Cholestasis Type 2 (PFIC2).
Initiated Phase 2b VISTAS study evaluating volixibat in patients with primary sclerosing cholangitis, and the Phase 2b OHANA study evaluating volixibat in patients with intrahepatic cholestasis of pregnancy.
As of December 31, 2020, Mirum had cash, cash equivalents, and investments of $231.8 million.
Mirum Pharmaceuticals
Mirum Pharmaceuticals
Forward Guidance
Mirum anticipates several milestones including the potential launch of maralixibat for ALGS in the US and for PFIC2 in Europe, as well as the completion of enrollment for the Phase 3 MARCH PFIC study.
Positive Outlook
- Anticipated launch of maralixibat for the treatment of cholestatic pruritus in patients with ALGS one year of age and older in the second half of 2021, if approved by the FDA.
- Launch of maralixibat for the treatment of PFIC2 in Europe planned for early 2022, if approved by the EMA.
- Enrollment completion for Phase 3 MARCH PFIC study expected in the second quarter 2021.
- Phase 2b study in primary biliary cholangitis planned for the second half of 2021.
- Financial runway expected to support maralixibat launch in U.S. and Europe and pipeline expansion over the next three years