Financial Data and Key Metrics Changes - Total revenues for the second quarter reached $128 million, representing a 64% increase compared to the same quarter last year [6][19] - The company raised its full-year revenue guidance for 2025 to a range of $490 million to $510 million, indicating nearly 50% top-line growth [7][13] Business Line Data and Key Metrics Changes - Net product sales for Lidmarley were approximately $128 million, driven by strong demand in both the U.S. and international markets [10][19] - In the U.S., Lidmarley generated about $57 million in net product sales, with increased patient awareness contributing to higher volumes [10][11] - Internationally, Lidmarley sales reached $31 million, supported by expanding reimbursement and strong demand [12][13] Market Data and Key Metrics Changes - The U.S. market for Lidmarley is seeing increased patient penetration, with approximately 70-75% persistence rates after one year for Alagille syndrome patients [25][59] - The company is experiencing growth in the PFIC market due to increased awareness and genetic testing, leading to more diagnoses [11][59] Company Strategy and Development Direction - The company's strategy focuses on commercial execution, scientific innovation, and financial discipline, with a commitment to delivering life-changing medicines for rare diseases [5][20] - The pipeline includes three late-stage studies expected to yield pivotal data over the next 24 months, particularly in primary sclerosing cholangitis [8][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's position and the opportunities ahead, highlighting the strong performance of its medicines and pipeline [20] - The company is optimistic about Lidmarley's potential to exceed $1 billion in revenue, driven by ongoing growth in Alagille syndrome and PFIC indications [14][62] Other Important Information - The company reported operating cash flow positivity for the quarter, with cash and investments totaling $322 million, reflecting a $29 million increase from the end of the previous year [19] Q&A Session Summary Question: What are the overall therapy persistence rates for Lidmarley? - Management indicated that persistence rates for Alagille syndrome patients are around 70-75% after one year, with lower attrition in subsequent years [25] Question: Can you provide details on the expected revenue distribution between Lidmarley and the bile acid business? - Management did not break down guidance by specific products but noted that trends observed year-to-date are expected to continue [33] Question: What are the main drivers of growth in Lidmarley sales? - Key drivers include increased awareness of PFIC and the introduction of a single tablet formulation, which has received positive feedback [39][41] Question: Are there any inventory impacts in the second quarter? - Inventory impacts are primarily relevant to Japan and Takeda, with no significant inventory issues in the U.S. or Europe [44] Question: What is the status of the Fragile X study? - The company has received clearance from the FDA and is on track to initiate the Phase II study by the end of the year [52] Question: How well penetrated is the company in the Alagille and PFIC markets? - The company estimates approximately 50% penetration in the Alagille syndrome market, with ongoing efforts to increase awareness and patient identification [59][60]

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.