Mirum Pharmaceuticals, Inc.
Key statistics
from XBRL data in SEC filingsAI briefing
from the latest 10-K, 10-Q and 8-K eventsMirum Pharmaceuticals is a rare disease biopharmaceutical company with three approved products and a late-stage pipeline targeting cholestatic liver diseases and chronic hepatitis D.
What they do
Mirum commercializes LIVMARLI (maralixibat) for Alagille syndrome and PFIC, and Bile Acid Medicines (Cholbam, Ctexli) for bile acid synthesis disorders, peroxisomal disorders, and cerebrotendinous xanthomatosis. The company is advancing volixibat for PSC and PBC, brelovitug for chronic hepatitis D, and MRM-3379 for fragile X syndrome.
Revenue drivers
- LIVMARLI — Approved for cholestatic pruritus in ALGS and PFIC; Q2 2026 sales were $128.7 million, up 46% year-over-year.
- Bile Acid Medicines (Cholbam and Ctexli) — Cholbam for bile acid synthesis disorders and PBD-ZSD; Ctexli for CTX; Q2 2026 sales were $47.5 million, up 20% year-over-year.
Recent performance
Q2 2026 net product sales totaled $176.2 million, up from $127.8 million in Q2 2025. Full-year 2025 revenue was $521.3 million with a net loss of $23.4 million, improved from a net loss of $87.9 million in 2024. Operating cash flow turned positive at $55.8 million in 2025. As of June 30, 2026, cash and investments were $561.3 million, with shareholder equity of -$10.4 million.
Strategy
Management aims to expand Livmarli into additional cholestatic indications, including the EXPAND study in rare cholestatic conditions. The company acquired Bluejay Therapeutics for brelovitug and licensed zilurgisertib for FOP, adding new pipeline assets. They are preparing for potential NDA submission of volixibat for PSC in H1 2027 and expecting topline data from multiple trials in 2026 and 2027.
Risks
- Volixibat regulatory uncertainty — The FDA recommended a Phase 3 study for volixibat in PSC despite positive Phase 2b data, potentially delaying approval.
- Negative shareholder equity — At June 30, 2026, total liabilities exceeded total assets, with equity at -$10.4 million, indicating a thin balance sheet.
- Clinical trial failures — The pipeline depends on multiple Phase 2/3 trials (VANTAGE, EXPAND, AZURE) with results expected in 2026-2027, which could miss endpoints.
- Commercialization dependence — Growth relies on continued market acceptance and reimbursement for Livmarli and Bile Acid Medicines, facing competition from generics and compounded products.
Outlook
Management raised 2026 net product sales guidance to $680-700 million. Key upcoming catalysts include topline data from AZURE-1 (Q3 2026), AZURE-4 (Q4 2026), EXPAND (Q4 2026), and VANTAGE (Q1 2027). The FDA PDUFA date for zilurgisertib is September 26, 2026, and volixibat NDA submission is targeted for H1 2027.