Alnylam Pharmaceuticals (ALNY) Status update summary

Strategic growth and market leadership

• Achieved rapid adoption of AMVUTTRA in ATTR cardiomyopathy, doubling TTR franchise revenues in 2025 and guiding for 83% growth in 2026, with a target of $4.4–$4.7 billion in 2026 revenues.

• Built a strong foundation with broad physician preference, 90%+ first-line access, and a robust provider network, enabling 90% of U.S. patients to receive treatment within 10 miles of home.

• Secured approximately 35% new-to-brand market share within the first year of launch, with over 12,000 patients treated globally and more than 1,600 unique U.S. prescribers.

• 75% of prescribers are new, indicating successful expansion beyond the initial base.

• Targeting leadership in TTR by 2030, aiming for the largest share of 75,000 treated U.S. patients and 25% company revenue CAGR through 2030.

Clinical differentiation and patient outcomes

• AMVUTTRA demonstrates strong clinical value, with HELIOS-B trial data showing up to 40% reduction in all-cause mortality and significant improvements in cardiac structure and function.

• Patients report 40–65% fewer GI symptoms, and 22% show amyloid regression, supporting a differentiated clinical profile.

• Once-quarterly dosing drives >90% persistence and adherence, with real-world data supporting superior adherence compared to stabilizers.

• Ongoing studies (e.g., DEMONSTRATE) aim to further validate effectiveness and safety in broader populations.

Competitive landscape and payer dynamics

• Maintained 70%+ share in hATTR-PN despite new silencer competition, with AMVUTTRA’s access, adherence, and clinical profile driving preference.

• Anticipated genericization of tafamidis seen as a tailwind, expected to unlock dual-mechanism therapy and not disrupt AMVUTTRA’s access or growth.

• Over 90% of patients have first-line access, most pay $0 out of pocket, and Medicare coverage is expected to remain stable post-tafamidis LOE.

• Competition is expected to expand the category, with AMVUTTRA positioned for sustained leadership.