Mirum(US:MIRM)2022-05-07 20:18Financial Data and Key Metrics Changes - Total revenue for Q1 2022 was $12.9 million, including $10.9 million from net product revenue of LIVMARLI and $2 million in licensing revenue from a partner in China [22] - Operating expenses for the quarter totaled $45.6 million, with R&D expenses at $24.1 million and SG&A expenses at $19.1 million [23] - The company had cash, cash equivalents, and investments of $239.9 million at the end of Q1 2022 [23] Business Line Data and Key Metrics Changes - LIVMARLI generated net product revenue of $10.9 million in Q1 2022, reflecting strong demand dynamics and refill cadence [7][12] - Approximately 75% of expenses were reimbursed in Q1, with expectations for this rate to increase to 90% or higher in Q2 and beyond [12] Market Data and Key Metrics Changes - The payer mix for LIVMARLI currently stands at approximately 50% Medicaid and 50% commercial [12] - The company is preparing for international launches of LIVMARLI, with anticipated approval in Europe later in 2022 and early access programs being initiated [15] Company Strategy and Development Direction - Mirum Pharmaceuticals aims to capitalize on a greater-than-$500 million U.S. market opportunity in Alagille syndrome while progressing with five other late-stage clinical programs [9] - The company is focused on innovative research for rare diseases to expedite the development of new medicines [8] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth throughout the year, driven primarily by the U.S. market [28][40] - The company is optimistic about the potential for LIVMARLI to address unmet medical needs in Alagille syndrome and other cholestatic conditions [24] Other Important Information - The MARCH-PFIC Phase III clinical trial is fully enrolled, with top-line data expected in Q4 2022 [16] - The INDIGO Phase II data has been published, showing significant improvements in patient outcomes [19] Q&A Session Summary Question: What drove the strong revenue of $10.9 million? - Management noted strong demand dynamics and refill cadence, with expectations for continued growth above the previously stated floor of $50 million for the year [27][30] Question: What is the current status of drug reimbursement? - Management expects to achieve a reimbursement rate of 90% in Q2 and beyond [31] Question: How is the LIVMARLI launch progressing in the U.S.? - The conversion of expanded access patients to commercial product was rapid, and the company is seeing strong adoption among targeted key accounts [33] Question: What are the timelines for the European launch? - The first launch will be in Germany following EMA approval, with additional Western European countries targeted for 2023 [35][36] Question: How does the company plan to maintain brand awareness for LIVMARLI? - The focus will be on rolling out access for as many patients as possible and publishing data from the clinical program [45] Question: What is the expected R&D spend trend? - R&D spending is anticipated to remain flat throughout the year, with some studies winding down while others ramp up [42] Question: Is the company seeking collaborators for additional PFIC genetic subtypes? - The company is actively looking for opportunities to grow and develop underappreciated programs in pediatric rare diseases [73]