Mirum Pharmaceuticals Announces Third Quarter 2025 Results and Highlights Strong Commercial Execution, Pipeline Advancement, and Strategic Positioning for 2026–2027
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company dedicated to transforming the treatment landscape for rare liver diseases, announced its financial results for the quarter ended September 30, 2025, and provided a comprehensive business update that underscores its continued commercial success and strategic pipeline progress. The company enters 2026 with notable momentum, reflecting a combination of strong top-line growth, expanded clinical activity, and a robust balance sheet poised to support upcoming clinical readouts and regulatory milestones.
Mirum’s Chief Executive Officer, Chris Peetz, emphasized the company’s continued operational strength and strategic trajectory as it advances its near- and long-term growth priorities. “Mirum is well positioned heading into 2026 with strong commercial momentum and multiple upcoming catalysts,” Peetz stated. “Our third-quarter results once again underscore the strength of our commercial medicines with continued strong performance. We also made important progress across the pipeline, including completing enrollment in the VISTAS PSC study, maintaining our momentum in VANTAGE and EXPAND, and initiating our Phase 2 study in Fragile X Syndrome.”
The combination of expanding product sales, a diversified pipeline with multiple late-stage studies, and a healthy financial position reinforces Mirum’s strategy to build a long-term rare disease franchise with significant value-creation potential.
Commercial Performance: Strong Growth and Updated Full-Year Guidance
Mirum delivered robust third-quarter commercial performance, driven by continued demand for its bile acid modulators. The company reported global net product sales of $133.0 million for Q3 2025. This performance supports its full-year revenue guidance range of $500 to $510 million, reflecting continued growth and operational execution.
LIVMARLI: Continued Expansion as Flagship Therapy
Third-quarter LIVMARLI (maralixibat) net product sales reached $92.2 million, marking a 56% increase over Q3 2024. This sustained growth highlights strong patient and prescriber uptake, expanding penetration in approved indications, and broader utilization within clinical practice. LIVMARLI is approved for the treatment of cholestatic pruritus in Alagille syndrome and continues to serve as the cornerstone of Mirum’s commercial portfolio.
The company is actively working to increase global access where regulatory frameworks permit, while simultaneously advancing development efforts aimed at broadening the reach of LIVMARLI to additional rare cholestatic disease populations. The EXPAND study, which is currently progressing, may support future indication expansions and further fuel long-term revenue growth.
Bile Acid Medicines: Steady Market Presence
Mirum reported third-quarter net product sales of $40.8 million for its bile acid product portfolio, representing 31% year-over-year growth relative to Q3 2024. These products continue to perform reliably and provide stable contribution to Mirum’s commercial base. The company remains focused on optimizing market access, patient support, and health-care provider engagement to maintain momentum within its rare liver disease franchise.
The expansion of Mirum’s cholestasis-focused commercial business reflects the rising recognition of bile acid therapies’ value among clinicians and patients managing rare hepatobiliary disorders.
Pipeline Development: Milestones Across Multiple Programs Supporting Future Growth
Mirum continued to make notable pipeline progress during the third quarter, reflecting its strategic investment in building a differentiated platform targeting rare cholestatic and genetically driven neurological conditions. The company anticipates multiple late-stage readouts across 2026 and 2027, marking a period of meaningful clinical and regulatory catalysts.
Volixibat Program Advancing in PSC and PBC
Volixibat, an oral, minimally absorbed IBAT inhibitor, remains central to Mirum’s strategy to expand treatment options for serious cholestatic liver diseases.
- VISTAS: Primary Sclerosing Cholangitis (PSC) – Enrollment Completed
In a significant milestone, Mirum completed enrollment in the VISTAS Phase 2b/3 study evaluating volixibat in primary sclerosing cholangitis (PSC). PSC is a progressive, immune-mediated bile duct disease with no approved therapies. The company expects topline data in the second quarter of 2026. Successful results could lay the foundation for volixibat to become the first approved therapy for PSC, potentially addressing major unmet need within hepatology. - VANTAGE: Primary Biliary Cholangitis (PBC) – Enrollment Ongoing
Mirum continues to progress the VANTAGE Phase 2b/3 study of volixibat in primary biliary cholangitis (PBC). Enrollment is expected to complete in 2026, with topline results anticipated in the first half of 2027. PBC is a chronic, immune-mediated liver disease where many patients fail first-line therapy or develop treatment-resistant pruritus. Volixibat may offer a new therapeutic option with differentiated clinical value.
LIVMARLI Clinical Expansion: EXPAND Study Advancing
Mirum is actively developing LIVMARLI across a broader spectrum of cholestatic indications. The EXPAND Phase 3 trial, designed to evaluate LIVMARLI in pruritus associated with rare cholestatic liver conditions beyond Alagille syndrome, remains on track to complete enrollment in 2026, with topline results expected in the first half of 2027.
If successful, EXPAND could significantly broaden the overall addressable population for LIVMARLI, reinforcing its role as a key therapy across rare liver disorders.
MRM-3379: Phase 2 Initiation in Fragile X Syndrome
Mirum initiated a Phase 2 clinical trial evaluating MRM-3379 in Fragile X Syndrome (FXS), a genetic neurodevelopmental condition characterized by intellectual disability, behavioral symptoms, and cognitive impairment. The study launch marks an important step in expanding Mirum’s therapeutic footprint beyond liver disease. The company expects that insights from this early-stage program will inform broader strategies in genetically based neurological conditions.
Overall, Mirum’s pipeline is strategically balanced, with near-term late-stage milestones alongside earlier programs designed to address significant unmet medical needs. These initiatives are expected to drive value creation and support continued growth through the end of the decade.
Financial Results: Solid Quarter with Strengthened Balance Sheet
Mirum reinforced its financial position in the third quarter, reflecting strong commercial execution and disciplined investment in R&D programs.
Revenue Growth
Total revenue for the quarter ended September 30, 2025, was $133.0 million, compared with $90.4 million in Q3 2024. The year-over-year growth was driven primarily by continued expansion in LIVMARLI utilization and steady contribution from the bile acid medicines franchise.
Operating Expenses
Mirum reported total operating expenses of $130.4 million for Q3 2025, compared to $103.1 million in Q3 2024. The increase reflects growth-related investments across commercial operations, clinical development, and corporate infrastructure.
Approximately $24.0 million of Q3 2025 operating expenses were attributable to non-cash stock-based compensation, intangible amortization, and other non-cash items, compared with $18.0 million in Q3 2024. The higher non-cash expense contribution aligns with portfolio expansion and increased R&D investment.
Cash Position
As of September 30, 2025, Mirum held $378.0 million in unrestricted cash, cash equivalents, and investments, compared with $292.8 million as of December 31, 2024. The strengthened balance sheet provides substantial financial flexibility to support commercial execution and advance late-stage clinical programs across 2026–2027.
Mirum’s disciplined operating framework positions the company to efficiently allocate capital toward high-priority programs while maintaining the necessary foundation for long-term operational success.
Corporate Update: Continued Strategic Execution
Throughout the quarter, Mirum continued to enhance commercial infrastructure, advance late-stage clinical programs, and broaden its potential treatment landscape. In addition to clinical progress, the company remained focused on improving operational efficiency and strengthening organizational capabilities to support future product growth.
Mirum’s executive team highlighted ongoing efforts to increase access to existing therapies and support clinical trial participation. Work is also underway to deepen market access partnerships and health-care provider engagement to optimize uptake, especially as new indications potentially come online in future years.
The company intends to maintain prudent spending levels, balancing investment into clinical development with its commitment to financial discipline. With multiple upcoming catalysts and a solid resource base, Mirum remains well placed to continue executing on its strategic priorities.
Upcoming Milestones: A Catalyst-Rich Near-Term Outlook
Mirum expects several major clinical milestones between mid-2026 and 2027:
| Program | Indication | Milestone | Expected Timing |
|---|---|---|---|
| Volixibat (VISTAS) | PSC | Topline Data | Q2 2026 |
| Volixibat (VANTAGE) | PBC | Enrollment Completion | 2026 |
| Volixibat (VANTAGE) | PBC | Topline Data | 1H 2027 |
| LIVMARLI (EXPAND) | Rare cholestatic pruritus | Enrollment Completion | 2026 |
| LIVMARLI (EXPAND) | Rare cholestatic pruritus | Topline Data | 1H 2027 |
| MRM-3379 | Fragile X Syndrome | Phase 2 trial ongoing | Initiated Q3 2025 |
These milestones represent a significant pipeline opportunity, particularly within rare hepatology, where approved treatment options are limited and medical need remains substantial.
Successful clinical readouts from VISTAS and VANTAGE, in particular, could potentially position volixibat as a cornerstone therapy for multiple cholestatic liver disorders. Meanwhile, LIVMARLI’s expansion through EXPAND may significantly increase the company’s revenue base.
Business Update Conference Call
To discuss third-quarter results and recent business developments, Mirum will host a conference call on:
📅 November 4, 2025
🕒 1:30 p.m. PT / 4:30 p.m. ET
Conference call dial-in details:
- U.S. Toll-Free: +1 833-470-1428
- International: +1 646-844-6383
- Access Code: 299722
A webcast of the call will be accessible via the Events & Presentations section of Mirum’s website. A recording will be available for replay for 30 days.
Positioned for Sustainable Growth and Innovation
Mirum Pharmaceuticals continues to demonstrate significant and sustainable growth as it advances a focused strategy centered on serving patients with rare liver and neurological diseases. The company’s performance in Q3 2025 highlights:
- Strong commercial expansion, particularly of LIVMARLI
- Durable revenue growth supported by its bile acid medicine portfolio
- Momentum across multiple late-stage clinical programs
- A strengthened balance sheet enabling continued investment
- Strategic planning that aligns near- and long-term value drivers
With multiple pivotal readouts anticipated in 2026–2027, Mirum stands at an important inflection point. Its combination of commercial assets, diversified pipeline, and meaningful near-term catalysts positions the company to continue delivering therapeutic innovation, commercial value, and potential shareholder returns.
Mirum remains committed to advancing transformative therapies for underserved patient populations worldwide and entering 2026 with strong confidence in its commercial durability and scientific momentum.
About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets
LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com.
LIVMARLI has received orphan designation for the treatment of ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus.
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