Mirum(US:MIRM)2025-05-21 14:00Summary of Mirum Pharmaceuticals Inc (MIRM) Conference Call Company Overview - Company: Mirum Pharmaceuticals Inc (MIRM) - Date of Conference: May 21, 2025 - Speakers: Eric Viarholt (CFO), Andrew McKibbin (SVP of Finance and IR) Key Points Commercial Performance - Record Revenue: In Q1, LIVMARLI generated approximately $73 million in revenue, with about two-thirds from the U.S. and one-third from international markets [3][4] - Market Penetration: The company is nearing 50% penetration in the U.S. for Alagille syndrome, with an addressable population of 1,000 to 1,200 patients [5][6] - Growth Expectations: Significant growth opportunities are anticipated for both Alagille syndrome and PFIC (Progressive Familial Intrahepatic Cholestasis) throughout the patent life, which extends to February 2040 [5][6] International Expansion - Geographic Reach: LIVMARLI is now available in over 25 countries, with plans for continued expansion, particularly in major Western European countries [6][7] - Partnerships: Takeda has recently received approval to launch LIVMARLI in Japan, with expectations for a successful market entry [7] Clinical Studies and New Formulations - EXPAND Study: A basket study is underway for additional indications, including cholestatic pruritus, with an estimated 500 addressable patients [9][10] - Tablet Formulation: A new tablet formulation has been approved, which is expected to enhance market uptake by providing a more convenient option for older patients transitioning from liquid formulations [11][12] Bile Acid Portfolio - Revenue Growth: The bile acid portfolio, including Cetexly and Colban, generated $37 million in Q1, marking a 50% increase compared to the previous quarter [18][19] - Patient Diagnosis: Currently, only about 10% of potential patients for CTX (Cholestanol Storage Disease) are diagnosed, indicating a significant opportunity for patient finding efforts [19] Pruritus in PSC and PBC - Market Opportunity: There are approximately 30,000 patients in the U.S. with PSC (Primary Sclerosing Cholangitis), with pruritus being a major issue for these patients [21][22] - Treatment Landscape: Current treatments for pruritus are largely ineffective, creating a substantial opportunity for IBAT inhibitors like LIVMARLI [23][24] - Clinical Data: Over half of the patients in previous studies using LIVMARLI for PSC reported resolution of itching, indicating strong efficacy [25] Regulatory Pathway - Approval Strategy: The company is optimistic about the regulatory pathway for pruritus in PSC, as it is considered an outcome measure that can lead to full approval [33][34] Intellectual Property - Patent Protection: LIVMARLI has IP protection until 2040, with the new tablet formulation extending IP to 2043 [48][49] Competitive Landscape - Dosing Optimization: The company emphasizes the importance of dosing in achieving effective treatment outcomes, particularly in comparison to competitors [40][41] Additional Insights - Durability of Effects: Long-term data indicates consistent and durable responses in patients treated with LIVMARLI, with improvements in both pruritus and fatigue [36][37] - Payer Landscape: The payer environment is described as receptive, particularly for treatments that provide tangible benefits to patients [42][43] This summary encapsulates the key insights and developments discussed during the conference call, highlighting Mirum Pharmaceuticals' strategic direction, market opportunities, and clinical advancements.