Alnylam Pharmaceuticals (ALNY) investor relations material

Company overview and strategic direction

• Transitioned from a loss-making to a profitable company, leveraging Nobel Prize-winning RNAi technology and launching multiple products over the past six to seven years.

• Set new 2030 goals to drive growth beyond the TTR franchise, with significant pipeline investment planned over the next five years.

• Focused on expanding beyond TTR, aiming for top-line growth and broader therapeutic impact.

AMVUTTRA performance and guidance

• Provided 2026 sales guidance for TTR products at $4.4–$4.7 billion, with nearly all revenue from AMVUTTRA.

• Q1 revenue for TTR reached $910 million, a 150% year-over-year increase, despite lower sequential growth due to international pricing adjustments and U.S. seasonality.

• International performance exceeded expectations outside Germany, with strong CM launch in Japan and stable PN business.

• U.S. growth impacted by insurance reauthorizations and distribution timing, but foundational access and logistics improvements support future growth.

• Confident in achieving guidance, expecting step-up in growth for the remainder of the year, driven by increased physician adoption and expanded prescriber base.

Market dynamics and competition

• Ex-U.S. price adjustments occur as new indications launch, typically benchmarking to existing products like tafamidis.

• Market is becoming more competitive, with new entrants and ongoing launches, but access and reimbursement dynamics remain favorable.

• Combination therapy with silencers and stabilizers is expected to grow, especially after tafamidis goes generic around 2031.

• Upcoming competitor data (Ionis/AstraZeneca) may be viewed as a class benefit, with limited immediate commercial impact due to payer restrictions on combination use.