Mirum(US:MIRM)2024-05-11 18:25Financial Data and Key Metrics - Total revenue for Q1 2024 was $69.2 million, a significant increase from $31.6 million in Q1 2023 [43] - Net product sales for Q1 2024 were $68.9 million, representing a 137% increase from Q1 2023 [31] - LIVMARLI global net product sales grew to $42.8 million in Q1 2024, up from $41.4 million in Q4 2023 and $29.1 million in Q1 2023 [34] - U.S. LIVMARLI sales were $30.8 million, while international sales were $12.1 million [34] - Net loss for Q1 2024 was $25.3 million or $0.53 per share [45] - Cash, cash equivalents, and investments increased to $302.8 million as of March 31, 2024, up from $286.3 million at the end of 2023 [45] Business Line Data and Key Metrics - LIVMARLI continues to show strong performance with demand growth across all geographies [27][34] - CHOLBAM and CHENODAL net product sales were $26.1 million in Q1 2024 [29] - The company is advancing volixibat in PSC and PBC, with interim analyses scheduled for June 2024 [32][37] Market Data and Key Metrics - The U.S. business was impacted by a healthcare cybersecurity incident, resulting in a temporary $3 million disruption to Q1 sales [28] - The company is preparing for the European market launch of LIVMARLI for PFIC, pending EMA approval [17][105] Company Strategy and Industry Competition - The company is focused on expanding the indications of its commercial medicines, including LIVMARLI for PFIC and CHENODAL for CTX [24][25] - Volixibat is being developed for PSC and PBC, with no approved therapies currently available for these conditions [32] - The company remains disciplined in its business development approach, focusing on rare disease settings [81] Management Commentary on Operating Environment and Future Outlook - Management is confident in achieving the full-year revenue guidance of $310 million to $320 million, driven by demand growth and new approvals [27][31] - The company expects reimbursement and payer discussions for LIVMARLI in PFIC to materialize over the next few quarters [35] - The interim analysis for volixibat in PSC and PBC is expected to provide meaningful treatment effect data and dose selection [39][40] Other Important Information - The company is preparing for the NDA submission of CHENODAL for CTX treatment [25] - The interim analysis for volixibat in PSC and PBC will focus on pruritus improvement, safety, and serum bile acids [40] Q&A Session Summary Question: Impact of the Change Healthcare cybersecurity incident on revenue - The $3 million impact was a one-time effect, with no lingering effects expected in future quarters [58][59] - The impact was balanced across products, proportional to their size [60] Question: Expectations for volixibat studies - The studies are designed to be broadly applicable, allowing patients on or off ursodiol and with varying levels of alphos [52] - The interim analysis will focus on pruritus improvement, safety, and serum bile acids, with no statistical significance expected [40][83] Question: European market preparation for LIVMARLI in PFIC - The company is ready to launch LIVMARLI in Europe upon EMA approval, with prescribers identical to those for Alagille syndrome [105] Question: Revenue contribution from PFIC in Q1 2024 - There was no significant PFIC contribution in Q1 2024, with revenue expected to grow in the back half of the year [103] Question: Drivers for bile acid product sales - The Change Healthcare cyberattack impacted the entire portfolio, with quarter-to-quarter volatility due to the ultra-rare nature of the diseases [106]