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Dec 31, 2019
Mirum Pharmaceuticals Q4 2019 Earnings Report
Mirum Pharmaceuticals reported financial results for Q4 2019, highlighted by a net loss of $18.0 million and total operating expenses of $18.7 million.
Key Takeaways
Mirum Pharmaceuticals reported a net loss of $18.0 million for the quarter ended December 31, 2019, with total operating expenses amounting to $18.7 million. The company's cash, cash equivalents, and investments stood at $140.0 million before gross proceeds from a follow-on public offering in January 2020.
Mirum completed a pre-NDA meeting with the FDA for maralixibat, aligning on a rolling NDA submission starting in Q3 2020.
The FDA granted Breakthrough Therapy Designation for maralixibat for treating pruritus associated with Alagille syndrome in patients 1 year and older.
The FDA granted Rare Pediatric Disease Designation for Alagille syndrome, potentially entitling Mirum to a priority review voucher upon maralixibat's NDA approval before October 1, 2022.
Data presented demonstrated the long-term durability of maralixibat in children with cholestatic liver disease, showing significant effects on pruritus, xanthomas, and growth for up to four years in Alagille syndrome.