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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.
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H.C. Wainwright 27th Annual Global Investment Conference summary10 Sep, 2025

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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Frequently asked questions

Mirum Pharmaceuticals Inc. is a biopharmaceutical company focused on the development and commercialization of therapies for rare liver diseases. The company targets conditions that primarily affect children and adults with limited treatment options. Mirum’s therapeutic approach addresses disorders related to bile acid metabolism, aiming to improve liver function and quality of life for patients. The company's portfolio includes both approved treatments and investigational therapies designed to manage chronic liver diseases. The company is headquartered in Foster City, California, and its shares are listed on the NASDAQ.

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