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Mirum Pharmaceuticals (MIRM) investor relations material
Mirum Pharmaceuticals H.C. Wainwright 27th Annual Global Investment Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.
Financial performance and guidance
Revenue guidance for 2025 was raised to $490–$510 million, reflecting strong first-half gains and international growth.
The business is operating cash flow positive with a strong balance sheet.
International markets, especially Japan and Europe, contributed positively to revenue surprises.
All three commercial medicines are experiencing continued growth.
Commercial portfolio and market expansion
LIVMARLI, CHOLBAM, and CHENODAL are approved for rare cholestatic and bile acid disorders.
LIVMARLI’s PFIC label expansion in 2024 drove significant revenue and patient identification, including in adults.
CHENODAL’s recent approval for CTX grants seven years of orphan exclusivity.
Ongoing efforts to expand LIVMARLI’s label via the EXPAND trial target at least 500 pediatric U.S. patients, with data expected in 2027.
Clinical pipeline and upcoming milestones
Three pivotal clinical readouts are expected by the first half of 2027.
The VISTA study for volixibat in PSC completed enrollment, with top-line data expected in Q2 2026.
Volixibat’s AVANTAGE study in PBC showed strong interim results; full enrollment to complete in 2026.
Phase 2 for MRM-3379 in Fragile X syndrome starts in Q4 2024, with a focus on both adult and pediatric populations.
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