Alnylam Achieves Breakthrough in Heart Study with RNAi Therapy Vutrisiran

1. Positive Study Results: Alnylam Pharmaceuticals announced positive topline results from the HELIOS-B Phase 3 study of vutrisiran, an RNAi therapeutic, for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
2. Statistical Significance: The study met its primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in both the overall and monotherapy populations.
3. Cardiovascular Benefits: Vutrisiran showed a 28% reduction in the composite endpoint in the overall population and a 33% reduction in the monotherapy population.
4. Secondary Endpoints: The study also demonstrated statistically significant improvements across all secondary endpoints, including disease progression measures such as the 6-minute walk test, Kansas City Cardiomyopathy Questionnaire, and New York Heart Association Class.
5. Potential for New Standard of Care: Alnylam CEO Yvonne Greenstreet believes vutrisiran has the potential to become the new standard of care for ATTR-CM, a debilitating and fatal disease.
6. Regulatory Submissions: Alnylam plans to submit the data to global regulatory agencies, including a New Drug Application (NDA) to the U.S. FDA with a Priority Review Voucher, later in 2024.