Mirum Pharmaceuticals, Inc. Q1 FY2026 earnings call

1. Progress in rare liver disease: Uptake of Live Marley remains strong, especially in PFIC. Recent clinical readouts in PSC and hepatitis delta show potential expansions. 2. Rare genetic disease: Addition of Zolurgocertib licensed from Insight, which has an NDA accepted with priority review and PDUFA date of September 26th. 3. Commercial execution: Strong first quarter with total net product sales of $160 million, and plans to expand teams for rare liver disease into adult settings.

Total net product sales for the first quarter were approximately $160 million. Live Marley had U.S. net product sales of $84 million and international sales of $30 million, with bile acid medicines contributing $46 million. Rare liver disease and rare genetic disease are key focus areas. Revenue guidance for full-year 2026 is raised to $660 to $680 million.

1. Raised full-year 2026 net product sales guidance to $660 to $680 million. 2. Zolurgocertib NDA with PDUFA date of September 26th, expecting launch by year-end. 3. R&D expense expected to step up in 2026 for Berlovitug ahead of BLA submission next year, fully funded.

1. Forward-looking statements involve risks and uncertainties that may cause actual results to differ materially. 2. Risks related to regulatory approval, clinical trial outcomes, and market adoption of new products.

Q: About FOP patient count and treatable patients at launch.

A: Approximately 300 identified patients in U.S., NDA for Zolurgocertib is for age 12 and over.

Q: Differentiation of Zolurgocertib vs other programs.

A: Oral once-daily ALK2 inhibitor with strong tolerability and activity profile.

Q: BD strategy and pricing for FOP.

A: Continue to find good programs, pricing to be decided closer to approval.

Q: HDV efficacy measures.

A: Focus on composite of virologic response and ALT normalization.

Q: FOP market penetration and OUS filing.

A: European filing upcoming, peak sales estimate is global.