Alnylam Pharmaceuticals(US:ALNY)2023-05-04 16:22Financial Data and Key Metrics Changes - Total product revenues for Q1 2023 were $276 million, representing a 48% increase compared to Q1 2022, primarily driven by growth in TTR product revenues from the AMVUTTRA launch [36][37] - Net revenue from collaborations was approximately $36 million, a 41% increase year-over-year, attributed to increased activities with Regeneron [36] - Gross margin on product sales was 85%, a 2% decrease from Q1 2022, mainly due to increased royalties on AMVUTTRA sales [37] - Non-GAAP operating loss for Q1 2023 was $110 million, showing a $7 million improvement compared to Q1 2022 [38] Business Line Data and Key Metrics Changes - The TTR franchise achieved $204 million in global net product revenues, a 49% growth compared to Q1 2022, with U.S. market growth at 75% [10][12] - Ultra-rare products GIVLAARI and OXLUMO generated $72 million in combined product sales, reflecting a 45% increase year-over-year [13] - AMVUTTRA's launch contributed significantly to the TTR franchise, with over 3,160 patients on treatment worldwide, marking a 6% quarterly growth [10][11] Market Data and Key Metrics Changes - In international markets, total TTR product sales increased 9% compared to the previous quarter, driven by strong demand in Japan [12] - The U.S. market saw a 5% increase in combined sales of ONPATTRO and AMVUTTRA, with a 7% increase in demand [11] Company Strategy and Development Direction - The company aims to expand its TTR franchise and address more common disease areas beyond rare diseases [8] - Alnylam is focused on sustainable innovation through new platform enhancements and genetically validated targets to drive pipeline expansion [9] - The company is committed to advancing its clinical-stage product candidates for ATTR amyloidosis and exploring new therapeutic areas such as hypertension with zilebesiran [30][34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing growth trajectory of AMVUTTRA and the overall TTR franchise, highlighting strong patient uptake and prescriber expansion [11][65] - The company anticipates continued strong performance from its pipeline, particularly with upcoming data presentations and clinical milestones [40] Other Important Information - The FDA has accepted the supplemental New Drug Application for patisiran for cardiomyopathy, with a PDUFA date set for October 8 [31] - The company has received regulatory approval to begin Part B of the ALN-APP Phase 1 study in Canada, while a partial clinical hold is in place in the U.S. [27][28] Q&A Session Summary Question: Can you talk about the upcoming 18-month data from APOLLO-B? - Management confirmed that the data will include ongoing follow-up and key secondary endpoints, with high retention rates in the study [44][46] Question: What is the market share impact of AMVUTTRA on ONPATTRO? - Management indicated a healthy switch rate from ONPATTRO to AMVUTTRA while also adding new patients, suggesting a positive growth trajectory for the TTR franchise [49][51] Question: Can you discuss the timelines for the CAA program? - Management highlighted the potential for ALN-APP in both Alzheimer's and CAA, with ongoing studies to establish safety and efficacy [53][55] Question: What are the expectations for zilebesiran in hypertension? - Management expressed excitement about zilebesiran's potential, with ongoing Phase 2 studies expected to provide pivotal data for future registrational programs [57][62]