Business Context and Reporting Period
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) is a biopharmaceutical company focused on rare diseases. This Form 10-K covers the fiscal year ended December 31, 2025. The company commercializes three approved medicines: LIVMARLI (maralixibat) for Alagille syndrome and PFIC; CHOLBAM (cholic acid) for bile acid synthesis disorders; and CTEXLI (chenodiol) for cerebrotendinous xanthomatosis (CTX). In January 2026, Mirum completed the acquisition of Bluejay Therapeutics, Inc., adding the late-stage product candidate brelovitug for chronic hepatitis D virus (HDV) infection.
Key Financial Metrics (Year Ended Dec 31, 2025)
| Metric | 2025 | 2024 |
|---|---|---|
| Total Revenue | $521.3 million | $336.9 million |
| Net Loss | $(23.4) million | $(87.9) million |
| Operating Loss | $(22.1) million | $(87.6) million |
| Net Cash from Operating Activities | $55.8 million | $10.3 million |
| Cash, Cash Equivalents & Investments | $391.4 million | $292.8 million |
| Convertible Notes (Principal) | $316.2 million | $316.3 million |
| Accumulated Deficit | $(667.5) million | $(644.2) million |
Revenue Breakdown: Livmarli contributed $360.0 million (up 69% YoY), while Bile Acid Medicines (Cholbam and Ctexli) contributed $161.3 million (up 31% YoY).
Material Changes vs. Prior Period
- Revenue Growth: Total revenue increased by $184.4 million (55%) driven by expanded commercialization of Livmarli in the U.S. and international markets, and the launch of Ctexli for CTX in February 2025.
- Profitability Improvement: Net loss narrowed significantly from $87.9 million in 2024 to $23.4 million in 2025, primarily due to revenue growth outpacing expense increases.
- Expense Increases:
- R&D Expenses: Increased $45.5 million to $186.2 million, driven by volixibat clinical trials (PSC/PBC) and MRM-3379 development.
- SG&A Expenses: Increased $54.8 million to $257.0 million, reflecting headcount growth for commercialization and increased stock-based compensation.
- Acquisition Activity: Completed the Bluejay Acquisition in January 2026 (subsequent event) for approximately $224.2 million in cash and stock, plus potential milestones.
Guidance, Outlook, and Risks
Outlook and Milestones:
- Volixibat: Topline data for PSC (VISTAS trial) expected Q2 2026; PBC (VANTAGE trial) enrollment expected to complete H2 2026.
- Livmarli: EXPAND study topline data expected Q4 2026.
- Brelovitug: Topline data from AZURE-1 and AZURE-4 trials expected H2 2026.
- MRM-3379: Topline data for Fragile X Syndrome expected in 2027.
Liquidity: Management believes existing cash and investments ($391.4 million as of Dec 31, 2025) are sufficient to fund operations for at least 12 months. The company recently raised approximately $268.5 million via a private placement in January 2026 to support the Bluejay acquisition.
Key Risks:
- Patent Litigation: Mirum initiated litigation in December 2025 against four generic manufacturers (Sandoz, Hetero, Biophore, Zydus) regarding Livmarli patents. A 30-month stay on generic approval is currently in effect through March 2029.
- Convertible Notes: $316.2 million in 4.00% Convertible Senior Notes due 2029 are convertible at the option of holders during Q1 2026 due to stock price performance.
- Regulatory & Clinical: Risks associated with clinical trial outcomes, regulatory approvals for product candidates, and potential pricing/reimbursement pressures.
Investor Verification Checklist
- Patent Litigation Status: Verify the progress of the LIVMARLI Patent Litigations filed in December 2025 and the potential impact of the 30-month stay expiration in March 2029.
- Convertible Note Conversion: Monitor Q1 2026 for potential conversion of the $316.2 million convertible notes, which could result in significant equity dilution or cash outflow.
- Bluejay Integration: Assess the financial impact and integration progress of the Bluejay Therapeutics acquisition completed in January 2026.
- Clinical Trial Data: Track the release of topline data for volixibat (Q2 2026) and brelovitug (H2 2026) as critical value drivers.
- Revenue Sustainability: Evaluate the consistency of Livmarli revenue growth given the company's reliance on large periodic orders from distributors and partners like Takeda.