Alnylam Pharmaceuticals(US:ALNY)2026-02-12 14:32Financial Data and Key Metrics Changes - Total global net product revenues for 2025 were nearly $3 billion, representing an 81% growth compared to 2024, driven by a more than doubling of revenue in the TTR franchise [25][26] - The company achieved GAAP profitability for the full year 2025 and expects to sustain profitability going forward [7][29] - Non-GAAP operating income for 2025 was $850 million, a $755 million increase compared to the previous year [28] Business Line Data and Key Metrics Changes - The TTR franchise generated $858 million in net revenues in Q4 2025, up 151% year-over-year [12] - The rare disease portfolio generated $136 million in net revenue in Q4, up 26% year-over-year [11] - GIVLAARI and OXLUMO together became a $500 million franchise in 2025, reflecting continued growth more than five years post-launch [11] Market Data and Key Metrics Changes - U.S. net revenues for the TTR franchise grew 20% compared to Q3 2025 and 222% compared to Q4 2024 [12] - International revenues grew 13% quarter-over-quarter and 47% year-over-year, indicating strong global momentum [14] - The company anticipates launching AMVUTTRA for ATTR cardiomyopathy in additional international markets throughout 2026 [14] Company Strategy and Development Direction - The company has set new five-year goals, Alnylam 2030, focusing on achieving global TTR leadership, sustainable innovation, and disciplined growth [8][9] - Plans include launching nucresiran in 2028 for polyneuropathy and 2030 for cardiomyopathy, with an aim for over 25% revenue CAGR through 2030 [10] - The company expects to invest approximately 30% of revenues in non-GAAP R&D to accelerate organic innovation and selectively access external innovation [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the size and growth of the ATTR-CM category, noting that the majority of patients remain untreated [15] - The early momentum of AMVUTTRA is encouraging, with strong customer demand and broad access expected to drive future growth [17] - Management highlighted the importance of improving diagnosis and treatment rates to expand the category further [18] Other Important Information - The company ended 2025 with cash, cash equivalents, and marketable securities of $2.9 billion, an increase from $2.7 billion at the end of 2024 [29] - Collaboration revenue for 2025 was $553 million, an 8% growth compared to 2024, including a significant milestone payment [26] Q&A Session Summary Question: Insights on new patient additions and first-line mix for patisiran versus tafamidis - Management expressed satisfaction with the AMVUTTRA launch and noted strong fundamentals supporting continued growth [41][43] Question: Addressing choppiness in scripts and pricing dynamics - Management indicated confidence in access and pricing, with a mid-single-digit net price decline expected in 2026 [49][61] Question: Seasonality effects in Q4 and potential tailwinds for Q1 2026 - Management acknowledged typical industry seasonality but emphasized that underlying momentum remains strong [53][54] Question: Gross to net pattern over 2026 - Management confirmed a gradual mid-single-digit net price decrease expected throughout the year [59][61] Question: External innovation strategy and potential partnerships - Management highlighted a focus on complementary technologies that enhance the existing portfolio and pipeline [66][98] Question: Impact of nucresiran on operating margins - Management anticipates significant positive margin impacts post-2030 if nucresiran demonstrates a best-in-class profile [70][71] Question: Updates on the Huntington's program and trial initiation - Management is optimistic about the Huntington's program and plans to accelerate it responsibly based on initial data [78][79] Question: Prioritization of ACVR1C asset in obesity pipeline - Management explained the prioritization based on preclinical data indicating ACVR1C's potential for greater efficacy compared to other targets [86][87]