Mirum Pharmaceuticals (MIRM) H.C. Wainwright 27th Annual Global Investment Conference summary

Financial performance and guidance

• Revenue guidance for 2025 raised to $490 million-$510 million, reflecting strong first-half growth and international performance.

• Business is operating cash flow positive with a strong balance sheet.

• International markets, especially Japan and Europe, contributed positively to revenue surprises.

Commercial portfolio and market expansion

• LIVMARLI, CTEXLI, and CHOLBAM are approved for rare cholestatic and metabolic diseases.

• LIVMARLI's PFIC label expansion in 2024 drove significant growth, revealing a larger-than-expected patient pool, including adults.

• Genetic testing initiatives are identifying new PFIC patients, expanding the addressable market.

• The EXPAND trial aims to further broaden LIVMARLI's use in pediatric and adult cholestatic pruritus, with data expected in the first half of 2027.

Clinical pipeline and upcoming catalysts

• Three pivotal readouts are expected between now and the first half of 2027.

• The VISTAS study for volixibat in PSC completed enrollment, with top-line data expected in Q2 2026; the market includes 30,000+ U.S. patients.

• Volixibat's VANTAGE study in PBC showed strong interim results for pruritus and fatigue; full enrollment to complete in 2026.

• Phase II trial for 3379 in Fragile X syndrome starts in Q4 2025, targeting both adult and pediatric populations.