Mirum Pharmaceuticals (MIRM) Leerink Global Healthcare Conference 2026 summary

Key business updates and financial guidance

• Maintains $630-$650 million commercial guidance, with durable trends supporting R&D programs.

• Four pivotal data readouts expected over the next 18 months, including VISTAS PSC, brelovitug, and EXPAND.

• EXPAND population could represent a third of peak sales for LIVMARLI, with at least 500 pediatric patients in the U.S. and similar numbers in Europe.

• International expansion ongoing, with sales in 30+ countries and further opportunities in Middle East and Latin America.

• Adult PFIC market is in early stages, with increased genetic testing but more education needed for broader adoption.

Clinical development and pipeline milestones

• VISTAS PSC top-line data expected in Q2, potentially leading to NDA filing by year-end.

• BreloVitug phase 2b interim readout from AZURE-1 in Q2, with phase 3 top-line data in the second half of the year and BLA filing in early 2027.

• EXPAND top-line readout planned for Q4 2026; volixibat PBC data expected in first half of next year.

• PSC and PBC launches will leverage overlapping commercial infrastructure and prescriber base.

• Launch of new products expected to drive substantial top-line inflection in 2027-2028.

Market development and commercialization strategy

• EXPAND study designed as a basket trial for ultra-rare cholestatic diseases, driven by physician demand.

• Market development includes education, patient testimonials, and advocacy collaborations to raise awareness of pruritus burden.

• Reflex testing for delta hepatitis is key to market growth, with European experience showing significant increases in diagnosis rates.

• U.S. market could see similar prevalence rates for delta with broader testing, supporting brelovitug's commercial potential.

• Commercial rollout focuses on U.S. and Europe, with distributor networks and licensed partners extending reach globally.