Mirum Pharmaceuticals (MIRM) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Business strategy and portfolio

• Focus on acquiring overlooked rare disease assets and developing high-impact medicines for underserved conditions.

• Commercial business is approaching $680–$700 million in annual revenue across three approved medicines.

• Multiple near-term catalysts include a PDUFA date for zilurgisertib, pivotal data for PSC, and several phase III readouts.

• Anticipates a financial inflection point as new product launches contribute to commercial growth.

LIVMARLI growth and expansion

• LIVMARLI is approved for cholestatic pruritus in Alagille syndrome and PFIC, with steady growth in patient adds and new geographies.

• Recent acceleration in PFIC due to increased identification of adult patients through improved genetic testing.

• Estimated 2,000 adult PFIC patients as an addressable market, with commercial expansion planned for broader GI clinics.

• Medical education and support for genetic testing are key to increasing diagnosis and adoption in adult hepatology.

• EXPAND phase III trial results expected in Q4, with potential to unlock further pediatric and adult patient populations.

Pipeline and clinical development

• Zilurgisertib for FOP has a PDUFA date this month, representing the next potential launch.

• Brelovitug for hepatitis delta virus has phase III readouts this quarter and next, with additional long-term data expected in the fall.

• Volixibat in PBC and PSC has upcoming data readouts, with ongoing regulatory dialogue to support NDA submission in the first half of next year.