Alnylam reports results from its HELIOS-B Phase 3 study of vutrisiran

Alnylam Pharmaceuticals announced positive topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy, ATTR-CM. The study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events during the double-blind period in both the overall population and in the monotherapy population. The study also demonstrated statistically significant improvements across all secondary endpoints in both the overall and monotherapy populations. This includes key measures of disease progression: 6-minute walk test, Kansas City Cardiomyopathy Questionnaire and New York Heart Association Class at Month 30. Importantly, treatment with vutrisiran also reduced all-cause mortality in the overall population and in the monotherapy population up to Month 42. This was a pre-specified, intent-to-treat analysis that included up to six months of data from the open-label extension.