Mirum Pharmaceuticals, Inc. Q3 FY2025 earnings call

Commercial Performance

• Third quarter revenue was $133 million, a nearly 50% YOY increase. Driven by strong performance of LIVMARLI in US and international markets, and bile acid portfolio.

R&D Progress

• VISTAS Phase IIb study in PSC has completed enrollment, expecting top line data in Q2 2026. VANTAGE study of volixibat in PBC progressing well. EXPAND study of LIVMARLI in ultra-rare cholestatic conditions enrolling well. Phase II study of MRM-3379 in Fragile X syndrome initiated.

Financial Milestones

• Cash balance grew significantly, and first positive net income achieved, highlighting operating leverage in commercial model.

In the third quarter, total net product revenue was $133 million. LIVMARLI contributed $92 million in net product sales, with $64 million in the US (Alagille syndrome and PFIC) and $28 million internationally. Bile Acid Medicines (CHOLBAM and CTEXLI) generated $41 million in net product sales. There was also approximately $5 million in sales to partner Takeda in Japan. Revenue contribution: LIVMARLI made up ~69.2% of the total, Bile Acid Medicines ~30.8%, and Takeda ~3.7%.

• Expect to land in upper end of 2025 guidance range with $500 million to $510 million in revenues.
• Peak revenue potential for LIVMARLI, volixibat, and MRM-3379 each exceed $1 billion.

• Forward-looking statements involve risks and uncertainties that may cause actual results to differ.
• Regulatory uncertainties in clinical trials and approvals.
• Competition in the rare disease space.
• Potential IP challenges related to product patents.

Q: What are the key drivers of LIVMARLI's performance ahead?

A: Continued growth in US PFIC launch, international market expansion, and switch to solid tablet formulation.

Q: What trends in LIVMARLI solid tablet adoption?

A: Substantial proportion of eligible patients switching from liquid formulation.

Q: Thoughts on volixibat pricing?

A: Monitoring market dynamics, considering analogs of other approved products in PBC but still analyzing.

Q: Expectations on Paragraph IV filers for LIVMARLI?

A: Confident in IP position, particularly method patents for LIVMARLI dosing in indications.

Q: Endpoints for Fragile X study and PSC safety?

A: Focus on pruritus endpoint for PSC, preclinical data in Fragile X supports efficacy, and PSC study has no safety issues raised by data monitoring committee.