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Alnylam Pharmaceuticals (NasdaqGS:ALNY) 2025 Conference Transcript

Summary of Alnylam Pharmaceuticals Conference Call Company Overview - Company: Alnylam Pharmaceuticals (NasdaqGS:ALNY) - Event: UBS Healthcare Conference - Date: November 10, 2025 Key Highlights from the Conference Call Financial Performance - Third Quarter Earnings: Alnylam reported $300 million in cardiomyopathy (CM) revenue for Q3, doubling the $150 million from Q2, leading to an upgraded full-year guidance by $275 million [6][11][24] - Five-Year Goals: The company has achieved its five-year goals set for 2025, including: - 500,000 patients on RNAi therapeutics - Six commercial products launched - 20 clinical programs - 40% compound annual growth rate (CAGR) in top-line growth - Non-GAAP profitability [10][11] Product Launch and Market Dynamics - Cardiomyopathy Launch: The launch has seen strong early momentum, with successful access to 170 health systems and first-line access to payers, primarily in a Medicare population (80%) [15][18][29] - Pricing Strategy: Alnylam anticipates a mid-single-digit year-over-year price decline for the TTR business in 2025, while volume growth is expected to offset the price decline [24][26] Pipeline and Future Goals - 2030 Goals: The company plans to announce new goals at the JPMorgan conference, focusing on TTR leadership, pipeline progression, and financial profile evolution [12][14] - Next-Generation Products: Zilebesiran is expected to achieve over 95% TTR reduction with a twice-annual dosing regimen, with ongoing studies in both cardiomyopathy and hereditary polyneuropathy [32][33] Competitive Landscape - Market Positioning: Alnylam is focused on first-line positioning against competitors, with expectations of expanding the market pie rather than just taking market share [30][45] - Combination Therapy: The company is preparing for a potential shift towards combination therapies as Tafamidis goes generic, leveraging data from ongoing studies [45][46] Early-Stage Pipeline - Mivelsiran: Targeting amyloid precursor protein for Alzheimer's and cerebral amyloid angiopathy, with promising early results [48][49] - Huntington's Disease: The company is optimistic about its differentiated approach targeting both full-length and variant proteins [50][52] - ALN-6400: Aiming to be a universal hemostatic agent, with initial focus on hereditary hemorrhagic telangiectasia [54] Additional Insights - Value-Based Agreements: Alnylam has implemented value-based agreements with payers to ensure patient compliance and reduce costs associated with non-compliance [29] - Patient Enrollment: The company is confident in its ability to enroll patients in ongoing studies without significant impact from competitive dynamics [36] This summary encapsulates the key points discussed during the conference call, highlighting Alnylam Pharmaceuticals' financial performance, product launches, pipeline developments, and strategic positioning in the market.