Mirum Pharmaceuticals (NasdaqGM:MIRM) 2025 Conference Transcript

Mirum Pharmaceuticals Conference Call Summary Company Overview - Company: Mirum Pharmaceuticals (NasdaqGM:MIRM) - Focus: Commercial rare disease company with three approved products targeting ultra-rare genetic conditions - Recent Performance: Reported $133 million in top-line revenue for Q3 2025, achieving positive net income for the first time [3][5] Key Highlights Financial Performance - Achieved $133 million in revenue for Q3 2025, marking a significant milestone in financial performance [3] - Positive net income reported for the first time, indicating an efficient business model in rare disease settings [5] Product Pipeline and Upcoming Milestones - Velexibat: Expected top-line data from the PSC program in Q2 2026, with additional studies in PBC and label expansion for LIVMARLI [4] - LIVMARLI: Strong performance in Alagille syndrome with approximately 50% market penetration in the U.S. [8] - PFIC Launch: Exceeded expectations with strong initial uptake driven by product profile and patient identification through genetic testing [20][22] Market Dynamics - Alagille Syndrome: LIVMARLI shows high response rates in pruritus and serum bile acid reduction, leading to long-term patient retention [8][9] - PFIC Market: Early signs of growth attributed to improved patient identification and awareness of genetic testing, expanding the potential patient pool [22][25] - Competitive Landscape: Mirum holds approximately 80% market share in Alagille syndrome and about 50% in PFIC, with a focus on expanding the total market [10][27] Product Formulation and Compliance - Introduction of a tablet formulation for LIVMARLI has improved patient compliance, particularly among older patients [12][13] Pricing Dynamics - Gradual growth in pricing expected as more patients are treated, with dose adjustments contributing to revenue over time [15][19] Future Growth Potential - Long-term view for LIVMARLI projected at over $1 billion, with Alagille syndrome contributing approximately 40% and PFIC around 30% [28] Additional Insights - Genetic Testing: Increasing adoption of genetic testing is crucial for diagnosing hard-to-identify patients, particularly in adult hepatology settings [7][22] - Market Development: Efforts are underway to enhance physician awareness of pruritus in PSC patients, which is often under-discussed [47] - EXPAND Study: Aiming to address ultra-rare cholestatic pruritus, with a significant number of patients fitting the profile identified [31][33] Conclusion Mirum Pharmaceuticals is positioned for continued growth with a strong product pipeline, effective market strategies, and a focus on expanding patient access through genetic testing and improved formulations. The company is optimistic about its future prospects, particularly with LIVMARLI and velexibat, as it navigates the competitive landscape in rare diseases.