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.

• Access initiatives ensure most patients have $0 out-of-pocket costs and 90% can access therapy within 10 miles.

• Market share exceeds 50% among prescribing physicians, with a focus on expanding first-line use.

• Guidance for the TTR franchise was revised to $4.2–$4.5 billion due to normalization of first- and second-line demand.

• 80% of the market remains undiagnosed and untreated, representing significant growth potential.

Pricing, access, and regulatory environment

• Net price is expected to decline modestly and gradually as volume increases.

• Proposed 340B rule changes are under review, with a decision expected around November; robust site-of-care networks are in place to maintain access.

• Focus remains on ensuring broad patient access regardless of regulatory changes.

Competitive landscape and clinical data insights

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

• Early treatment and patient selection are critical for optimal outcomes in TTR cardiomyopathy.

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

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