Vutrisiran

Transthyretin amyloid cardiomyopathy (ATTR-CM) has evolved from an untreatable condition to one managed with three FDA-approved disease-modifying agents: tafamidis, acoramidis, and vutrisiran.

• The FDA has approved Alnylam Pharmaceuticals' AMVUTTRA (vutrisiran) for treating cardiomyopathy in adults with wild-type or hereditary transthyretin-mediated amyloidosis, designed to reduce cardiovascular mortality and hospitalizations. • Following the approval, multiple financial institutions including Citi, Bank of America, Wells Fargo, and RBC Capital raised their price targets for Alnylam, with Citi analysts suggesting the drug is "on track to achieving mega blockbuster status." • Alnylam's stock surged 10.86% on the news, with trading volume reaching 1.8 million shares compared to the three-month daily average of 717,000, building on its impressive 19.23% year-to-date and 91.65% 52-week gains.

• Alnylam Pharmaceuticals has received FDA approval for Amvuttra (vutrisiran) to treat ATTR cardiomyopathy (ATTR-CM), significantly expanding the drug's addressable patient population beyond its original polyneuropathy indication. • The approval represents a major advancement in treatment options for ATTR-CM patients, a progressive and often fatal condition characterized by the buildup of abnormal amyloid protein in the heart muscle. • This expanded indication strengthens Alnylam's position in the ATTR treatment market, potentially increasing the company's revenue stream while providing patients with a new therapeutic option for managing cardiac manifestations of the disease.

• Alnylam's RNAi drug vutrisiran demonstrated a 28% reduction in all-cause mortality and recurrent cardiovascular events in ATTR cardiomyopathy patients, with efficacy increasing to 33% in patients not taking Pfizer's tafamidis. • The HELIOS-B trial showed vutrisiran's benefits increased over time, with a 36% reduction in the primary endpoint at 42 months, positioning the quarterly-administered injection as a potential new standard of care. • Vutrisiran, already approved as Amvuttra for ATTR polyneuropathy, could reach multibillion-dollar sales if approved for cardiomyopathy, though it faces competition from BridgeBio's acoramidis and AstraZeneca/Ionis' eplontersen.

• Alnylam showcased multiple potential blockbuster therapies in ATTR amyloidosis, cardiovascular disease, and neuroscience, including the TRITON Phase 3 program for nucresiran, which offers greater than 95% TTR knockdown with twice-annual dosing. • The company announced a pivotal Phase 3 cardiovascular outcomes trial for zilebesiran in hypertension, enrolling up to 11,000 high-risk patients across 30 countries to evaluate its impact on reducing major adverse cardiovascular events. • Alnylam presented promising data on emerging clinical programs for Huntington's disease, bleeding disorders, and type 2 diabetes, while outlining platform innovations to deliver RNAi therapeutics to all major tissue types by 2030.