Alnylam Pharmaceuticals (ALNY) Morgan Stanley 24th Annual Global Healthcare Conference summary

TTR franchise performance and market dynamics

• AMVUTTRA achieved over $1 billion in Q2 revenue, driven by strong HELIOS-B data and rapid adoption for TTR cardiomyopathy since its 2025 indication approval.

• Over 50% market share among prescribing physicians, with most patients accessing AMVUTTRA at $0 out-of-pocket and within 10 miles of home.

• Market fundamentals remain strong, with 80% of patients undiagnosed in a global market of 500,000; focus is on expanding first-line use.

• 2026 guidance for the TTR franchise was revised to $4.2–$4.5 billion, reflecting normalization of first-line vs. second-line demand.

• Pricing is expected to see modest, gradual net reductions as volume grows; 340B rule changes are pending, with access strategies in place.

Clinical data and competitive landscape

• HELIOS-B demonstrated superior TTR knockdown (81%) compared to eplontersen in CARDIO-TTRansform (less than 70%), supporting AMVUTTRA's efficacy.

• Early treatment yields better outcomes; both studies highlight the importance of patient selection and comprehensive endpoints.

• Physician feedback and surveys indicate no significant change in silencer use or preference for AMVUTTRA following CARDIO-TTRansform results.

• siRNA therapies are perceived as more effective than ASOs in this space.

Pipeline and future growth drivers

• Nucresiran (TRITON-CM) targets 95% TTR knockdown, with ongoing enrollment and potential study adjustments to optimize outcomes.

• Generic tafamidis is now expected in 2031, reducing near-term pricing pressure and aligning with key data readouts for TRITON-PN and TRITON-CM.

• Key growth levers include expanding first-line use, improving diagnosis rates, broadening the prescriber base, and global expansion.

• Partnerships with AI providers and organizations like the American Heart Association aim to drive diagnosis and market growth.