Mirum Pharmaceuticals (MIRM) Morgan Stanley 24th Annual Global Healthcare Conference summary

Business performance and commercial strategy

• On track for $680M–$700M in annual revenue across three approved medicines, with strong growth in rare disease markets.

• LIVMARLI drives growth in Alagille syndrome and PFIC, with expanding adult PFIC diagnosis contributing to revenue.

• Expansion of the commercial field team aims to improve patient identification and genetic testing, especially in adult PFIC.

• Bile acid portfolio (CTEXLI, CHOLBAM) benefits from a dedicated team targeting genetics and neurology, not just hepatology.

• Launch preparations for FOP asset are underway, leveraging the existing team and infrastructure for a Q4 launch.

Pipeline developments and upcoming milestones

• Top-line data from the AZURE-1 phase III study in hepatitis delta expected later this month.

• Two additional phase III readouts and a PBC study readout are anticipated in the next two quarters.

• PDUFA date for FOP program is imminent, with launch readiness in place.

• Phase III EXPAND study for LIVMARLI targets ultra-rare cholestatic pruritus causes, with pediatric data expected next quarter.

• Fragile X phase II study is enrolling well, with data expected next year.

Regulatory and clinical strategy

• Volixibat in PSC showed strong phase II data; FDA recommended phase III, but company plans NDA submission in 1H next year based on current data.

• Ongoing engagement with FDA to address concerns and build familiarity with VISTAS data set.

• PBC program for volixibat aligns with FDA expectations, with over 300 patients enrolled and pivotal study design confirmed.

• Breakthrough designations granted for both PSC and PBC indications.