Summary 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.