Mirum Pharmaceuticals (MIRM) 44th Annual J.P. Morgan Healthcare Conference summary

Commercial performance and financial outlook

• Achieved $520 million in product revenue for 2025, with 2026 guidance of $630–$650 million.

• Portfolio potential exceeds $4 billion, driven by three approved medicines and pipeline assets.

• Livmarli reached $359 million in 2025 net revenue, showing strong growth from Alagille syndrome and PFIC indications.

• Bile acid replacement products, Cholbam and Chenodal, are growing, with Chenodal's CTX approval boosting diagnoses.

• Well-financed balance sheet, expecting to be cash flow positive by 2027.

Pipeline and clinical development updates

• Four pivotal clinical readouts expected in the next 18 months, including Livmarli EXPAND, Volixibat VISTAS (PSC), Volixibat VANTAGE (PBC), and Brilovatug AZURE (HDV).

• Livmarli's EXPAND study targets ultra-rare cholestatic pruritus, with top-line data in Q4 and label expansion opportunity for 2025.

• Volixibat's VISTAS study (PSC) and VANTAGE study (PBC) are fully enrolled, with interim analyses showing strong pruritus reduction.

• MRM-3379 for Fragile X is in phase 2, with first patients randomized and data expected in 2027.

• Brilovatug for hepatitis delta is in phase 3 (AZURE), with interim and top-line data expected in 2024.

Market opportunities and commercialization strategy

• Livmarli is a billion-plus brand, with roughly equal opportunity across Alagille, PFIC, and EXPAND indications.

• Volixibat targets 20,000 PSC patients and 31,000 PBC patients with pruritus in the U.S., with high unmet need in PSC.

• Brilovatug addresses 15,000 diagnosed hepatitis delta patients in the U.S., with potential for higher prevalence in Europe and Asia.

• Commercial team covers North America and Western Europe, with expansion planned for Volixibat and Brilovatug launches.

• Field force expansion of 30–40 in the U.S. and similar scale-up in Europe planned for new launches.