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Alnylam Pharmaceuticals (ALNY) FY Conference Transcript

Summary of Alnylam Pharmaceuticals (ALNY) FY Conference Call - June 09, 2025 Company Overview - Company: Alnylam Pharmaceuticals (ALNY) - Industry: Biotechnology - Key Product: Ambutra for TTR cardiomyopathy Core Points and Arguments 1. European Approval: Alnylam secured European approval for Ambutra for cardiomyopathy, enhancing its global momentum in the TTR franchise [3][4] 2. Five-Year Strategy: The company is on track to achieve its 2021 five-year goals, including sustainable non-GAAP profitability by the end of 2025 [5][6] 3. Revenue Growth: Alnylam's revenues are primarily driven by the TTR franchise, with a robust pipeline for future growth [6][7] 4. Pipeline Programs: - Zarbisiran for hypertension and a program for Huntington's disease are highlighted as significant growth levers [10][11] - ARN 6400 targeting plasminogen is noted for its potential as a vessel hemolytic agent [12] 5. Market Dynamics: The company is experiencing rapid progress in treating patients with TTR cardiomyopathy, with over 50% of priority health systems having Ambutra on formulary [16][17] 6. Financial Guidance: Alnylam provided guidance for TTR revenue between $1.6 billion and $1.725 billion, representing approximately 36% year-over-year growth at the midpoint [22] 7. Polyneuropathy Growth: The polyneuropathy segment is growing at a rate of 28-34% year-over-year, with expectations for continued growth alongside cardiomyopathy [23][24] 8. Patient Access: The company reports broad access across payer types, with the majority of patients paying $0 out of pocket [33][35] 9. Patient Services: Alnylam has a comprehensive patient services organization to support access and adherence, with over 95% adherence rates in polyneuropathy [31][38] 10. Pricing Strategy: The company anticipates modest adjustments to Ambutra pricing over time, but specifics are not yet available [42] 11. Global Launch Plans: Alnylam plans to launch Ambutra in Germany and Japan in the second half of 2025, with additional launches expected throughout 2026 [45] 12. Clinical Data: The HELIOS B trial data shows a 36% reduction in all-cause mortality and a 33% reduction in cardiovascular mortality, reinforcing the drug's value proposition [47][48] 13. Pipeline Expansion: Alnylam is committed to advancing its pipeline, with multiple programs in various stages of development, including obesity and diabetes treatments [62][64] Additional Important Content - Regulatory Strategy: The orphan drug designation for Alnylam's products has positioned the company favorably in negotiations related to pricing and access [14] - Commercial Strategy: The company emphasizes a "buy and build" approach for its products, focusing on establishing a strong commercial footprint [20] - Innovation Engine: Alnylam aims to deliver 2-4 new INDs per year, expanding its siRNA technology across various tissues [59][60] - Capital Allocation: The company plans to focus on progressing its existing pipeline rather than pursuing significant business development opportunities at this time [64]