Monday, Alnylam Pharmaceuticals Inc ( ALNY ) revealed topline results from its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for ATTR amyloidosis with cardiomyopathy (ATTR-CM).

ATTR-CM is a type of heart muscle disease that occurs when amyloid fibrils – clumps of misfolded proteins – are deposited into heart tissue and cause it to stiffen.

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The study met the primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular (CV) events during the double-blind period in both the overall population (HR 0.718, p-value 0.0118; n=654) and in the monotherapy population (patients not receiving tafamidis at baseline; HR 0.672, p-value 0.0162; n=395).

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 (6-MWT), Kansas City Cardiomyopathy Questionnaire (KCCQ), and New York Heart Association (NYHA) Class at Month 30 (p