Alnylam Pharmaceuticals (ALNY) Wells Fargo 21st Annual Healthcare Conference summary

Commercial performance and market dynamics

• AMVUTTRA's cardiomyopathy launch has surpassed expectations, with over $1 billion in quarterly TTR franchise revenue and strong fundamentals for sustainable growth, especially in first-line treatment.

• Access to AMVUTTRA is robust, with most patients paying $0 out-of-pocket and about 1,700 physicians initiating use since the cardiomyopathy indication launch.

• The market is shifting toward first-line use, with normalized second-line inflow and balanced growth between community centers and centers of excellence.

• Broad access is supported by a network enabling 90% of patients to receive treatment within 10 miles of home.

• Category growth is driven by both share gains and increased diagnosis, with ongoing initiatives to expand awareness and patient identification.

Competitive landscape and pricing outlook

• Loss of exclusivity for tafamidis is expected in 2031, reducing midterm pricing pressure and allowing more time to compete for first-line preference.

• The failure of CARDIO-TTRansform removes a competitor and further reduces pricing risk, supporting optimism for continued share and volume growth.

• Combination therapy is present but remains a minority; most of the market is monotherapy, with combo use mainly in second-line settings.

• The HELIOS-B dataset supports AMVUTTRA's efficacy both as monotherapy and in combination, with future competition expected from mivelsiran.

Clinical data and differentiation

• HELIOS-B data shows a 40% reduction in all-cause mortality over four years, robust cardiovascular outcomes, and improved functional capacity with quarterly dosing.

• MRI and safety analyses indicate positive effects on cardiac structure, function, and extra-cardiac manifestations, including reduced GI issues.

• Real-world evidence confirms that patients on stabilizers often progress, highlighting the need for alternative mechanisms like AMVUTTRA.

• Early intervention with AMVUTTRA leads to better outcomes, and adherence is higher with quarterly dosing compared to daily oral treatments.