Mirum Pharmaceuticals (MIRM) TD Cowen 46th Annual Health Care Conference summary

Strategic overview and financial outlook

• Building a rare disease leader with three approved medicines and 2024 revenue guidance of $630M–$650M.

• Four pivotal trial readouts expected over the next 18 months, supporting a robust late-stage pipeline.

• 2025 product sales exceeded guidance, with strong 2026 outlook and consensus-beating performance.

Commercial performance and market penetration

• LIVMARLI has achieved about 50% penetration in Alagille syndrome in the U.S., with 80%+ market share among newly diagnosed patients.

• PFIC market penetration is growing, with at least 50% of new treatment-naive patients starting therapy and higher drug consumption per patient.

• Ex-U.S. markets show growth in Alagille and initial PFIC uptake, with distributor markets showing more revenue variability.

Pipeline and clinical development updates

• Key 2024 milestones include unblinding of the PSC trial with volixibat and interim analysis of brelovitug in Q2, plus AZURE-1, AZURE-4, and EXPAND trial readouts in H2.

• EXPAND basket study for LIVMARLI targets ultra-rare cholestatic indications, with about half of enrolled patients having post-Kasai biliary atresia.

• LIVMARLI's peak sales projected at $1B+, split roughly equally among Alagille, PFIC, and EXPAND populations.