Alnylam Pharmaceuticals (ALNY) ESC Congress Presentation summary

TTR leadership and strategic vision

• Positioned for durable leadership in TTR with AMVUTTRA and nucresiran, leveraging strong clinical data and broad access in the U.S.

• AMVUTTRA is established as a foundational, first-line therapy for ATTR-CM, with >50% share of new starts among experienced prescribers.

• Over 1,700 new prescribers since launch and >90% first-line access with minimal out-of-pocket costs for most patients.

• Ambition to achieve global TTR market leadership and launch next-gen silencer nucresiran in CM by 2030.

• Targeting 25%+ total revenue CAGR and ~30% non-GAAP operating margin through 2030.

Clinical innovation and pipeline progress

• HELIOS-B data support AMVUTTRA’s efficacy, showing significant reductions in all-cause mortality and cardiovascular events, especially when initiated early.

• TRITON-CM trial designed for success with deep TTR knockdown, early patient population, and event-driven endpoints.

• Nucresiran aims for highest-ever TTR knockdown, with modeling suggesting deeper knockdown correlates with better outcomes.

• RNAi therapeutics have demonstrated additive benefit on top of stabilizer therapy in multiple studies.

• Multiple late-stage readouts expected in 2H26, including programs in Huntington’s disease and obesity.

Market performance and growth drivers

• Quarterly sales post-AMVUTTRA CM launch show strong revenue growth, with AMVUTTRA leading among products.

• ATTR-CM category expected to grow, with ~15,000 new patients annually and ~75,000 treated by 2030.

• Driving breadth of prescribers and broad payer access are key to unlocking next growth wave.

• Continued investment in R&D (~30% of revenues) to deliver new transformative medicines beyond TTR.

• RNAi platform offers differentiated clinical and commercial advantages over ASO competitors.