Alnylam Pharmaceuticals(ALNY)2023-08-03 17:40Financial Data and Key Metrics Changes - Total product revenues for Q2 2023 were $306 million, representing a 43% year-over-year growth compared to Q2 2022 [27] - Gross margin on product sales was 75%, a 9% decrease compared to Q2 2022, primarily due to fees associated with canceling manufacturing commitments for ONPATTRO [28] - Non-GAAP operating loss for the quarter was $154 million, showing a $7 million improvement compared to Q2 2022 [30] Business Line Data and Key Metrics Changes - The TTR franchise achieved $224 million in global net product revenues for ONPATTRO and AMVUTTRA, representing a 46% growth year-over-year [10] - GIVLAARI and OXLUMO together delivered $82 million in combined product sales during Q2, representing a 37% growth compared to Q2 2022 [13] Market Data and Key Metrics Changes - In the US, combined sales of ONPATTRO and AMVUTTRA increased by 72% year-over-year, marking the fourth consecutive quarter of TTR growth exceeding 70% [11] - AMVUTTRA demand in Japan is particularly encouraging, with new patient growth driven by switches from tafamidis and patients naive to therapy [10] Company Strategy and Development Direction - The company entered into a strategic partnership with Roche for the development and commercialization of zilebesiran, aiming to disrupt the global treatment paradigm for hypertension [6] - The company is focused on achieving its Alnylam P5x25 goals, positioning itself as a top-tier biotech company developing transformative medicines for rare diseases [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming catalysts, including the initial data from the KARDIA Phase 2 program and the potential launch of ONPATTRO for ATTR amyloidosis with cardiomyopathy [7] - The management highlighted the importance of maintaining high ethical standards in business conduct, especially in interactions with healthcare providers and patients [6] Other Important Information - The US Attorney's Office for the District of Massachusetts concluded its case regarding the marketing and promotion of ONPATTRO with no action taken [6] - Akshay Vaishnaw transitioned to the role of Chief Innovation Officer, focusing on the future of the R&D engine [8] Q&A Session Summary Question: Any updated thoughts on the upcoming AdCom ONPATTRO and ATTR CM? - Management is actively preparing for the advisory committee and believes the focus will be on the clinical meaningfulness of changes in the six-minute walk test and KCCQ [35] Question: Clarification on the Roche deal for zilebesiran? - The deal includes $310 million upfront and $365 million in potential development milestones, with Roche covering 60% of development costs [40] Question: Consideration by the FDA regarding the PDUFA date for ONPATTRO? - Management anticipates the PDUFA date of October 8th and is working towards that, with no changes communicated by the FDA at this time [44] Question: Timing and reasoning behind Akshay's role change? - The transition to Chief Innovation Officer allows for a more focused role in driving R&D success, with no specific reason for the timing [51] Question: Details on the expanded access program for patisiran? - The program enrolled 200 patients rapidly, highlighting the significant demand for alternative therapies for patients experiencing progression [69]