Mirum Pharmaceuticals, Inc. posts full-year 2025 financial results and outlines key business updates and strategic priorities for 2026.
Mirum Pharmaceuticals, Inc. a leading biopharmaceutical company focused on rare diseases, reported strong fourth quarter and full-year 2025 financial results, while outlining an ambitious 2026 outlook driven by commercial momentum and multiple late-stage clinical catalysts. The company emphasized that 2025 marked a year of disciplined execution, revenue expansion, regulatory progress, and strategic pipeline strengthening, positioning the organization for what management described as a “pivotal” 2026.
Transformational Year Sets the Stage for 2026
Chris Peetz, Chief Executive Officer of Mirum, highlighted 2025 as a year of meaningful operational and strategic progress. According to Peetz, the company’s expanding commercial footprint, combined with pipeline advancements and the addition of a new hepatitis delta virus (HDV) program, has significantly strengthened Mirum’s long-term growth trajectory.
A key strategic development was the acquisition of Bluejay Therapeutics, which added worldwide rights to brelovitug for chronic HDV. This transaction expands Mirum’s presence beyond cholestatic liver diseases into viral liver disease, diversifying its clinical portfolio and creating additional late-stage value inflection points.
Looking ahead, Mirum expects four potentially registrational readouts over the next 18 months. The first of these catalysts will be topline results from the Phase 3 VISTAS study evaluating volixibat in primary sclerosing cholangitis (PSC), expected in the second quarter of 2026. Management believes this catalyst-rich period, alongside continued revenue growth and financial discipline, positions the company to deliver meaningful value to both patients and shareholders.
2026 Financial and Clinical Expectations
For 2026, Mirum projects global net product sales in the range of approximately $630 million to $650 million, reflecting continued growth across its commercial portfolio.
On the clinical front, multiple key milestones are anticipated:
- Volixibat VISTAS study in PSC: Topline data expected in Q2 2026.
- LIVMARLI EXPAND Phase 3 study: Enrollment completion anticipated in the first half of 2026, with topline results expected in Q4 2026. The study evaluates pruritus in additional rare cholestatic conditions.
- Volixibat VANTAGE study in primary biliary cholangitis (PBC): Enrollment expected to complete in the second half of 2026.
- Brelovitug AZURE program in chronic HDV: Interim data from AZURE-1 are expected in Q2 2026, with topline data from AZURE-1 and AZURE-4 projected in the second half of 2026.
These upcoming milestones represent significant regulatory and commercial opportunities, particularly as PSC, PBC, and HDV remain areas of high unmet medical need.
Commercial Performance: Accelerating Rare Disease Impact
Mirum delivered substantial commercial growth in 2025, reporting total net product sales of $521.3 million, representing a significant increase from $336.4 million in 2024.
The company’s flagship product, LIVMARLI® (maralixibat), continued to drive performance. Full-year 2025 LIVMARLI net product sales totaled $360.0 million, including $244.7 million in U.S. net product sales. This reflects 69% year-over-year growth compared to 2024, underscoring strong demand and successful global expansion efforts.
Bile Acid Medicines also contributed meaningfully, generating $161.3 million in 2025 net product sales, representing 31% year-over-year growth.
Mirum expanded its global reach to 33 countries with commercial access, further strengthening its leadership position in rare cholestatic liver diseases. The company’s growing international footprint demonstrates its ability to execute across multiple regulatory jurisdictions while scaling commercial infrastructure effectively.
Regulatory and Pipeline Momentum
Mirum achieved several important regulatory milestones during 2025, reinforcing its leadership in rare liver diseases.
LIVMARLI tablets received approval from the U.S. Food and Drug Administration (FDA) and marketing authorization from the European Commission for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In addition, Health Canada authorized LIVMARLI oral solution and tablets for PFIC, and approved the tablet formulation for ALGS.
In Japan, LIVMARLI oral solution was approved for cholestatic pruritus in patients with ALGS and PFIC, marking further geographic expansion in a key international market.
Beyond LIVMARLI, the FDA approved CTEXLI® for cerebrotendinous xanthomatosis (CTX), broadening Mirum’s rare metabolic disease portfolio.
Pipeline advancement also continued. Enrollment was completed in the volixibat VISTAS Phase 3 study in PSC, positioning the program for its upcoming 2026 readout. The company initiated the MRM-3379 BLOOM Phase 2 study in Fragile X syndrome (FXS), and MRM-3379 received FDA Fast Track designation, highlighting the potential of the program in a serious neurodevelopmental disorder with limited treatment options.
The acquisition of Bluejay Therapeutics added brelovitug, a promising therapy for chronic HDV, further diversifying Mirum’s pipeline into viral hepatitis. Chronic HDV represents one of the most severe forms of viral hepatitis, and effective treatments remain limited, making this an important strategic addition.
Financial Strength and Capital Discipline
Mirum maintained a strong financial position throughout 2025. Total operating expenses for the year ended December 31, 2025 were $543.4 million, compared to $424.5 million in 2024. The increase reflects investment in commercial expansion, research and development programs, and organizational scaling to support growth.
Operating expenses in 2025 included $95.0 million in non-cash stock-based compensation, intangible amortization, and other non-cash items, compared to $79.4 million in 2024.
Despite increased investments, Mirum strengthened its balance sheet. Unrestricted cash, cash equivalents, and investments totaled $391.4 million as of December 31, 2025, up from $292.8 million at the end of 2024.
In January 2026, concurrent with the closing of the Bluejay acquisition, Mirum completed two private placements generating aggregate gross proceeds of $268.5 million. These financings further enhance liquidity and provide capital to support ongoing pipeline development, commercial expansion, and potential regulatory submissions.
