Mirum Pharmaceuticals Reports Third Quarter 2026 Financial Results and Provides Business Update - MIRUM PHARMACEUTICALS INC

Financial Performance:

• Third-quarter net product sales reached $186 million, a significant increase from $133 million in Q3 2020.
• Full-year net product sales guidance raised to $715–$720 million; first nine months of 2020 generated $522 million, already matching full-year 2025 revenue.
• Cash contribution margin from the commercial business was in the high 50% range of sales.
• Total operating expenses were approximately $245 million, comprising $28 million cost of sales, $70 million SG&A, and $94 million R&D (on a cash basis).
• Cash used in operations was approximately $4 million.
• R&D spending represented ~50% of revenues and is expected to remain at this level for two more quarters before declining.
• Total cash, cash equivalents, and investments stood at approximately $500 million as of September.
• The company repurchased $20 million of 2029 convertible notes for $62 million in cash in September, reducing the outstanding balance by over 80% and lowering future dilution and interest expense.
• The company anticipates becoming cash flow positive in 2027.

Guidance and Future Outlook:

• Full-year net product sales guidance increased to $715–$720 million.
• The company projects a steady revenue cadence of $10–$15 million quarter-over-quarter growth through 2027.
• Future regulatory milestones include five potential approvals or label expansions in the first half of 2027: NDAs for Velixivud (PSC/PBC), Grilobitug (HDV), Etebrios (pediatric FOP expansion), and a supplemental NDA for Livmarly (pediatric PBC).
• Commercial launch of Etebrios is underway, with adoption expected to be faster than the Livmarly trajectory.
• Peak revenue potential for the portfolio is estimated at over $4 billion.
• Specific market targets include >$1 billion in potential annual sales for HDV and significant revenue potential from PSC and PBC due to unaddressed patient populations.

Business Segments and Product Lines:

• Livmarly: Primary growth driver with Q3 sales of $133 million; steady uptake in Adult PFIC and Alagile Syndrome, with a focus on expanding diagnosis in undiagnosed adult PFIC patients (estimated 2,000 addressable in the US).
• Bile Acid Medicines: Generated $52 million in Q3 sales; includes Colesevelam and other bile acid therapies benefiting from orphan protection and steady growth.
• Etebrios: Approved in September for adults and pediatric FOP patients (ages 12+); U.S. launch commenced in October. Early engagement is strong with high receptivity from patients and physicians.
• Clinical Pipeline:
  • Grilobitug: Positive Phase 3 data for HDV (ASR1) and Phase 3 data for Biliary Atresia (EXPAND); BLA expected in H1 2027 pending Azure 04 readout this quarter.
  • Velixivud: NDA submission planned for H1 2027 for PSC and PBC; robust data supporting pruritus relief.
  • Etebrios Pediatric Expansion: SMDA planned for H1 2027 for ages 6 to <12; positive data from Cohort 2.
  • Tolvaptan (Zolingocertib): Evaluation in younger children ongoing.

Market and Competitive Landscape:

• HDV: Estimated 15,000 patients currently under management in the US with low historical testing rates; significant international opportunity exists. Competition is limited, with Grilobitug's fully human antibody profile and durable viral suppression offering differentiation.
• PSC: Approximately 20,000 addressable patients in the US for pruritus; currently no direct competition.
• PBC: Larger addressable market with ~50,000 US patients; some competition exists but strong unmet need remains.
• FOP: Highly engaged patient community; Etebrios addresses an unmet need with oral dosing and a mechanism targeting underlying disease causes.
• Competitive Moat: Bile acid franchise benefits from orphan exclusivity (though Colesevelam's orphan time is expired, the tax lead retains protection).

Risks and Challenges:

• Diagnostic Rates: Significant portion of adult PFIC and HDV patients remain undiagnosed due to low testing uptake historically; commercial success relies on increased genetic testing and provider awareness.
• Regulatory Execution: While confident, the company faces a busy regulatory timeline with multiple submissions in H1 2027, requiring careful organizational bandwidth management.
• Clinical Trial Execution: Azure 04 readout for Grilobitug is critical; outcomes will directly influence the BLA submission strategy.
• Market Acceptance: Launch speed for Etebrios and subsequent pediatric label expansions depends on payer access and physician adoption rates.
• Dilution and Debt: Although significantly reduced, the company retains convertible notes (2032 maturity), though cash position provides flexibility.

Management Commentary and Tone:

• Tone is highly confident and optimistic, emphasizing the company's transition from a clinical-stage biotech to a commercially successful entity approaching an $800M+ run rate.
• Management highlights the company's "inflection point" with four approved medicines, a late-stage pipeline, and a path to cash-flow positivity.
• CEO Chris Pietz expressed pride in the team's execution over eight years and confidence in the "greater than $4 billion" peak revenue potential.
• CFO Eric Bierkhold highlighted the disciplined capital structure management and the successful reduction of the 2029 convertible notes.

Analyst Questions and Answers:

• Takeda Purchases: Minimal revenue from Takeda in Q3; no new orders expected in Q4.
• PSC Submission Timing: Confident in NDA submission in H1 2027; interactions with FDA are ongoing for familiarity with data sets but no material changes to guidance.
• Etebrios Launch: Early adoption is strong and expected to exceed the trajectory of Livmarly; Q4 and 2027 growth cadence expected to continue at $10–$15M/quarter.
• R&D Spend: Maintained at ~50% of revenue for two more quarters, then expected to decrease rapidly as revenue grows.
• Business Development: Company remains opportunistic for in-licensing, including commercial-stage assets, despite the busy submission schedule in H1 2027.
• HDV Market: Expect increased testing interest as therapies become available; current patient identification is a key growth lever.
• Azure 04 Results: Designed to complement ASR1; expected to show consistency in virologic response and safety, which will form part of the registrational package.

Other Key Points:

• The company will present comprehensive data on Grilobitug (ASR1 full results, 48-week data, and 2-year cirrhosis data) at the AASLD conference.
• The company emphasizes the "relentless execution" across rare disease opportunities, combining approved medicines with a deep late-stage pipeline.
• Future growth is underpinned by the expansion of the Livmarly commercial footprint to capture undiagnosed adult PFIC patients and the imminent launch of Etebrios.
• The company has successfully executed a financing strategy in Q2/Q3 to secure $690M in zero-coupon convertible notes and reduce near-term debt obligations.