Mirum Pharmaceuticals (NasdaqGM:MIRM) FY Conference Transcript

Summary of Mirum Pharmaceuticals FY Conference Call Company Overview - Company: Mirum Pharmaceuticals (NasdaqGM:MIRM) - Industry: Biopharmaceuticals focused on rare diseases - Products: Three commercialized small molecule drugs: LIVMARLI, CHOLBAM, and CHENODAL [1][2] Financial Performance - Revenue Guidance: Management raised revenue guidance for 2025 to a range of $490 million to $510 million, reflecting robust revenue gains in the first half of 2025 compared to 2024 [1][2] - Cash Flow: The company is operating cash flow positive and has a strong balance sheet [2] Product Details LIVMARLI - Indications: Approved for Alagille syndrome and PFIC (Progressive Familial Intrahepatic Cholestasis) [3] - Market Growth: The PFIC indication has shown unexpected growth, particularly in adult populations, leading to increased patient identification [4][5] - Clinical Profile: LIVMARLI has a differentiated clinical profile, showing benefits in pruritus, growth, and bilirubin improvements [4] CHENODAL - Indication: Approved for CTX (Cerebrotendinous Xanthomatosis) with seven years of orphan exclusivity [3] CHOLBAM - Indication: Approved for bile acid synthesis disorders [3] International Expansion - Partnerships: Strong performance from international business, particularly in Japan with partner Takeda, who received approvals for Alagille and PFIC [6] Clinical Pipeline EXPAND Trial - Objective: Expanding the label of LIVMARLI to include additional settings of cholestatic pruritus, targeting at least 500 pediatric patients in the U.S. [7][8] VISTA Study - Indication: Evaluating volixibat for PSC (Primary Sclerosing Cholangitis) with an expected top-line readout in Q2 of the following year [9][10] - Market Size: Approximately 30,000 patients in the U.S., with a significant unmet need for effective treatments [9][10] AVANTAGE Study - Indication: Evaluating volixibat for PBC (Primary Biliary Cholangitis) with positive interim data showing significant improvements in pruritus and fatigue [17][18] MRM-3379 - Indication: Targeting Fragile X syndrome, a genetic mutation leading to autism spectrum disorder, with a market opportunity of around 25,000 patients in the U.S. [22][23] - Phase 2 Study: Expected to start in Q4 of the current year, focusing on different age groups [25] Competitive Landscape - Volixibat Positioning: Positioned as a first-line treatment option for pruritus in PBC, differentiating from existing therapies that primarily target second-line settings [20][21] Future Outlook - Clinical and Commercial Catalysts: Continued growth of all three medicines, initiation of Phase 2 in Fragile X, and upcoming data readouts for PSC and PBC expected in 2026 [27] - Portfolio Expansion: The company is actively looking for opportunities in the rare disease space, focusing on adding value and ensuring alignment with current capabilities [28][29]