Mirum(US:MIRM)2024-08-10 01:10Financial Data and Key Metrics - Net product revenue for Q2 2024 was $77.8 million, a 139% increase compared to $32.5 million in Q2 2023 [4][13] - Total operating expenses for Q2 2024 were $102 million, including R&D expenses of $32.7 million, SG&A expenses of $49.2 million, and cost of sales of $20.2 million [13] - Net loss for Q2 2024 was $24.6 million, or $0.52 per share [14] - Cash, cash equivalents, and investments totaled $295.4 million as of June 30, 2024, a decrease of $7.4 million from the prior quarter [14] Business Line Performance - LIVMARLI global net product sales grew to $47.2 million in Q2 2024, a 45% increase compared to the same quarter last year [8] - U.S. sales of LIVMARLI were $35.5 million, while international sales were $11.7 million [8] - CHOLBAM and CHENODAL combined net product sales were $30.5 million in Q2 2024 [9] Market Performance - LIVMARLI demand growth was strong internationally, with favorable price negotiations in Germany [8] - The European Commission endorsed LIVMARLI for PFIC patients aged 3 months and older, highlighting its significant benefit [9] Strategic Direction and Industry Competition - The company is focused on growing its commercial business, expanding indications for approved medicines, advancing volixibat in adult cholestasis, and seeking pipeline growth opportunities [14] - The company submitted an NDA for chenodiol in CTX, aiming to address an underdiagnosed population and secure orphan exclusivity [4] - The VISTAS PSC and VANTAGE PBC studies for volixibat showed positive interim results, positioning the company to bring the first medicine to the PSC patient community [7] Management Commentary on Operating Environment and Future Outlook - The company is on track to achieve its full-year revenue guidance of $310 million to $320 million [7][9] - Management expressed confidence in the continued growth of LIVMARLI, supported by new patient additions and label expansions [8] - The company is optimistic about the potential of volixibat to set a new standard in addressing cholestatic pruritus [11] Other Important Information - The company launched the EXPAND study to evaluate LIVMARLI in patients with cholestatic pruritus across various rare conditions, targeting enrollment of approximately 45 patients by 2026 [12] - The VISTAS PSC study is expected to complete enrollment in the second half of 2025, while the VANTAGE PBC study aims to complete enrollment in 2026 [10][11] Q&A Session Summary Question: How does the company plan to broaden LIVMARLI's reach within the PFIC segment? - The company has seen strong initial reception for LIVMARLI in both Alagille syndrome and PFIC indications, with equal footing in the U.S. and leadership in Europe [16] Question: What are the enrollment strategies for VISTAS and VANTAGE trials? - Enrollment is progressing well, with investigators excited about the potential impact of volixibat for PBC and PSC patients [18] Question: What is the duration and time horizon for the EXPAND study? - The EXPAND study is expected to complete enrollment in 2026, with results anticipated to support wider use of LIVMARLI in cholestatic pruritus [20] Question: How does the company view seasonality in its commercial business? - The company does not identify strong seasonality effects due to the ultra-rare nature of its products, though quarter-to-quarter variability can occur [21] Question: What is the timeline for OUS pricing reference dynamics? - The pricing reference impact is expected to play out in Q2 and Q3, with normalization anticipated by Q4 [22] Question: How was the sample size for the EXPAND study determined? - The sample size was based on powering for the primary endpoint, with confidence derived from prior experience with LIVMARLI [24] Question: What data supports enrolling biliary atresia patients in the EXPAND trial? - The EXPAND study targets a different patient population than EMBARK, focusing on chronic liver disease patients with cholestatic pruritus [26] Question: Where will the interim PBC data for volixibat be presented? - The company is preparing an abstract for submission to an upcoming congress, with additional details to be included [29] Question: What is the company's business development strategy? - The company is focused on rare disease opportunities, particularly in pediatric rare diseases, with a high bar for new opportunities [31] Question: Will the company pursue a label expansion for PFIC patients below 12 months? - The company is evaluating the potential for another sNDA based on infant data but feels the current label captures most targeted patients [33] Question: How does the FDA view the EXPAND trial for label expansion? - The EXPAND study was suggested by the FDA and is designed to address cholestatic pruritus across various conditions [36] Question: How will pruritus be measured in the EXPAND trial? - Pruritus will be measured using an observer-rated ItchRO for pediatric patients and self-reported endpoints for adults [37][39] Question: What is the formulation used in the EXPAND trial? - The EXPAND trial will use the same liquid formulation of LIVMARLI that is commercially available [40] Question: Are biliary atresia patients from EMBARK eligible for EXPAND? - The EXPAND study targets a different patient population, with no overlap expected from EMBARK [42] Question: What is driving demand growth for CHOLBAM and CHENODAL? - Demand growth is steady, with Q1 2024 impacted by a cyberattack, and no major plans for CHOLBAM label expansion [45] Question: How is the PFIC launch progressing in the U.S.? - The PFIC launch is progressing well, with modest revenue contribution expected in 2024 and stronger growth anticipated in 2025 [48] Question: What is the commercial opportunity for CHENODAL in CTX? - The CTX opportunity includes increasing diagnosis rates and promoting CHENODAL to a broader patient population [53] Question: Has the FDA or EMA reviewed interim data for VANTAGE and VISTAS? - Regulatory agencies have not reviewed interim data, with discussions planned after final results are available [55]