Mirum Pharmaceuticals, Inc. Q1 FY2025 earnings call

Regulatory Approvals

• CTEXLI was FDA approved for the treatment of CTX in February.
• LIVMARLI was approved in Japan for PFIC and Alagille syndrome through partner Takeda.
• A convenient single tablet form of LIVMARLI was approved by the FDA.

Pipeline Progress

• VISTAS study of Volixibat in PSC is close to completing enrollment, expecting enrollment in Q3 2025 and top-line data in Q2 2026.
• VANTAGE study in PBC to present updated 28-week interim data at EASL, showing durable pruritus improvement.
• Phase 2 study for MRM-3379 in Fragile X syndrome to start this year.
• LIVMARLI EXPAND trial and Volixibat VANTAGE study to complete enrollment in 2026.

Commercial Execution

• Strong growth in commercial medicines, leading to updating full-year revenue guidance to $435 million to $450 million.

Total revenues for the first quarter of 2025 reached $111.6 million, representing a 61% growth over the first quarter of the previous year. For LIVMARLI, total global net product sales grew to $73.2 million in Q1 2025, an increase of over 70% compared to Q1 2024. U.S. LIVMARLI sales were $49.5 million, and international sales were $23.7 million. The bile acid products had $38.4 million of net product sales in Q1 2025, representing 47% growth over the same quarter last year. CTEXLI, now approved for CTX, contributed to the revenue growth with its sales performance.

Revenue Guidance

• Full-year revenue guidance updated to $435 million to $450 million, driven by robust growth from all three commercial medicines.

Cash Flow

• Expected to be cash flow positive for the full year, with the cash contribution margin from the commercial business improving from approximately 47% in Q1 2024 to approximately 53% in Q1 2025.

• Forward-looking statements involve risks and uncertainties from Form 10-Q and subsequent SEC filings.
• Inventory dynamics in international markets with new partners holding stock impacting sales numbers.
• Milestones tied to development and regulatory progress may vary, affecting future financial performance.

Q: For the additional Volixibat PBC data, speak to pruritus benefit deepening and discontinuations due to diarrhea.

A: Chris Peetz mentions the data matured with deepened pruritus response and a positive note on discontinuations due to diarrhea being a testament to the program's benefit.

Q: On LIVMARLI tablet formulation, frame IP and commercial adoption.

A: Chris Peetz talks about novel IP from the tablet formulation with a patent expected to grant soon, and Peter Radovich expects the tablet to be an attractive option for older children, adolescents, and adults.

Q: How do you think your interim Phase 2 PBC data compares to linerixibat and placebo response?

A: Chris Peetz states Volixibat's program stands out with strong change from baseline and placebo-adjusted difference, attributing it to the dose of Volixibat being maximally efficacious.

Q: Update on LIVMARLI access versus competitors and step-through policies in Alagille and PFIC.

A: Peter Radovich states LIVMARLI access in the U.S. is strong, with preferential step-through policies in Alagille indication being beneficial, and differentiating factors being clinical value proposition and support.

Q: Expectations for VISTAS study pruritus reduction and Volixibat's positioning in PSC.

A: Chris Peetz refers to VANTAGE interim result as a robust data set, optimistic about Volixibat's activity level in PSC, and Peter Radovich mentions PSC is an unsatisfied market with Volixibat having a strong position without meaningful competition.

Q: Insight on CTEXLI patient identification since approval and go-forward expectations.

A: Peter Radovich mentions a nice uptick in patient identification since FDA approval, with efforts in disease awareness across specialties like neurology, ophthalmology, and GI.

Q: PFIC launch in U.S. and ex-U.S. comparison to expectations, and profitability sustainability.

A: Chris Peetz states PFIC launch has been better than expectations, and Eric Bjerkholt mentions cash flow positive for the quarter and expected for the full year, but not expecting GAAP profitability soon.

Q: Impact of LIVMARLI tablet formulation on demand and competitive positioning, and proportion of Alagille patients opting for tablet.

A: Chris Peetz mentions the tablet is attractive for older patients, with a substantial minority likely to opt for it, potentially driving switch demand.