Alnylam Pharmaceuticals(ALNY) - 2021 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - In Q3 2021, Alnylam Pharmaceuticals generated $120.3 million in net revenue from ONPATTRO, reflecting a 6$167 million, representing a 68% growth versus Q3 2020 [37] - Cash, cash equivalents, and marketable securities stood at $2.3 billion as of September 30, 2021, up from$1.9 billion at the end of 2020 [38] Business Line Data and Key Metrics Changes - ONPATTRO saw a 12% growth in demand in the U.S., with over 1,875 patients on commercial treatments [13] - GIVLAARI achieved $31.8 million in net revenue, a 4$14.9 million in net revenue, a 9% decrease from Q2 due to the transition of patients from monthly loading doses to quarterly maintenance doses [36] Market Data and Key Metrics Changes - Market access for ONPATTRO has been achieved in over 30 countries, with significant growth in Europe, Canada, and Japan [15] - GIVLAARI's U.S. sales were flat versus Q2 2021, but the product is seeing strong progress in establishing Value-Based Agreements with commercial payers [17] - OXLUMO is experiencing steady geographic expansion, with recent launches in Germany and France [19] Company Strategy and Development Direction - The company aims to bring its fifth RNAi therapeutic to market and expand its TTR franchise significantly [11] - Alnylam is focused on sustainable innovation and plans to file a CTA for ALN-APP, targeting Alzheimer's disease, by the end of 2021 [32] - The P5x25 strategy remains a guiding roadmap for delivering transformative medicines and achieving excellent financial performance [73] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the positive results from the HELIOS-A study of Vutrisiran, which strengthens the outlook for future studies [45] - The leadership transition is seen as timely, with the company in a strong position to continue its growth trajectory [71] - The management team remains committed to executing the existing strategy without loss of momentum [73] Other Important Information - The company plans to present full results from the HELIOS-A study at a medical conference in early 2022 [39] - Alnylam is on track to file a CTA for ALN-XDH for gout treatment by late 2021 [32] Q&A Session Summary Question: Contextualization of HELIOS-A data relative to APOLLO-A - Management highlighted that the current data strengthens confidence in Vutrisiran, with encouraging exploratory data on cardiac endpoints [45] Question: Definition of the cardio subpopulation in HELIOS-A - The majority of patients in HELIOS-A would have New York Heart Association class one or two level disease [50] Question: Enrollment progress in the KARDIA-1 trial - Enrollment in KARDIA-1 is progressing well, with a clear line of sight on the last patient in before further guidance is provided [55] Question: Statistical plan for APOLLO-B regarding tafamidis drop-in - The study design accounts for potential drop-ins, with conservative assumptions made to ensure robustness [66] Question: CNS delivery capabilities for ALN-APP - The company has shown promising CNS delivery in preclinical studies, with plans to file an IND for ALN-APP by the end of the year [69]