Alnylam Pharmaceuticals (ALNY) Status update summary

Strategic growth, launch momentum, 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 and $2.1B in total global TTR revenues since launch.

• Over 12,000 patients treated globally, with ~1,600 unique U.S. prescribers and 35% new-to-brand market share in the first year.

• 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.

• 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 and other trial data showing up to 42% reduction in all-cause mortality, significant improvements in cardiac structure, and 22% amyloid regression.

• Patients report 40–65% fewer GI symptoms, and nearly halved extra-cardiac adverse effects, supporting a differentiated clinical profile.

• Once-quarterly dosing drives >90% persistence and adherence, with a 2x advantage in persistence over daily oral stabilizers.

• AMVUTTRA rated highly by physicians for reducing mortality (84%), CV hospitalizations (86%), and improving quality of life (78%).

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

Market opportunity and category expansion

• U.S. ATTR-CM prevalence estimated at 200,000, with 80% untreated; global prevalence at 500,000.

• Annual new-to-treatment patients increased to 15,000+, and 15,000 stabilizer progressors represent significant expansion opportunities.

• Diagnosis and treatment rates are accelerating, driven by increased awareness, improved options, and investments in accessible diagnostics.

• Category growth is robust, with 77% growth since competitor launches and 56% growth before competition.

• U.S. potential for 75,000 treated patients in the TTR category by 2030.