Mirum Pharmaceuticals (NasdaqGM:MIRM) FY Conference Transcript

Summary of Mirum Pharmaceuticals FY Conference Call Company Overview - Company: Mirum Pharmaceuticals (NasdaqGM:MIRM) - Focus: Rare disease company delivering life-changing medicines to patients with rare diseases - Key Products: Livmarli, Volixibat, Chenodal, Cholbam, Brilovatug Financial Performance - 2025 Product Revenue: Estimated at $520 million - 2026 Revenue Guidance: Expected between $630 million and $650 million - Potential Revenue from Pipeline: Over $4 billion across current commercial and pipeline programs [3][25] Key Products and Pipeline Livmarli - Indications: Approved for Alagille syndrome and PFIC, with a third indication expected to read out later this year - 2025 Revenue: $359 million, with strong growth driven by pediatric patient starts and label expansion into PFIC [8][30] - Future Potential: Estimated billion-plus potential opportunity [6][30] - EXPAND Study: Aiming for label expansion into other cholestatic pruritic indications, with top-line data expected in Q4 [10][12] Volixibat - Indications: Studied for PSC and PBC, targeting a significant unmet need in cholestatic pruritus [12][13] - Market Size: Approximately 30,000 patients with PSC, with two-thirds experiencing pruritus [13] - Upcoming Data: Top-line data from the VISTAS study expected next quarter, with NDA filing anticipated in the second half of the year [16][25] Brilovatug (Acquisition of BlueJ Therapeutics) - Indication: Targeting hepatitis delta, a rare co-infection with HBV - Market Size: Approximately 15,000 diagnosed and insured patients in the U.S., with potential for higher prevalence in other regions [22][48] - Phase 2 Results: Achieved 100% virologic response, with a 65%-82% response rate on FDA approval endpoints [23][47] - Phase 3 Program: AZURE studies currently enrolling, with top-line data expected in the second half of the year [24][25] Market Dynamics - Livmarli: Strong growth driven by increased awareness and diagnosis in adult PFIC settings, with a focus on genetic testing [9][28] - Volixibat: Positioned as the only potential therapy for PSC, with a significant market opportunity due to the lack of approved therapies [41][42] - PBC Market: More competitive with recent approvals of PPARs, but still presents opportunities for IBAT inhibitors [43] Commercial Strategy - Geographic Focus: Direct commercialization in North America and Western Europe, with partnerships in smaller countries [5] - Field Expansion: Plans to increase the U.S. field force by 30-40 people to target community physicians and GI specialists [44] Risks and Considerations - Regulatory Risks: Ongoing monitoring of clinical trial outcomes and regulatory approvals for pipeline products [2][25] - Market Competition: Need to navigate competitive landscapes, especially in the PBC market [42][43] Conclusion - Mirum Pharmaceuticals is positioned for significant growth with a strong pipeline and commercial strategy, focusing on rare diseases and addressing unmet medical needs in cholestatic conditions and hepatitis delta. The company anticipates pivotal data readouts in the coming year that could further enhance its market position and revenue potential [25][26].