Alnylam Presents New 18-Month Results From Exploratory Cardiac Endpoints in HELIOS-A Phase 3 Study of Investigational Vutrisiran

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced new positive results from an 18-month analysis of exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The 18-month findings show that in a pre-defined cardiac subpopulation, treatment with vutrisiran was associated with improvements in exploratory cardiac endpoints relative to external placebo, including levels of NT-proBNP, a measure of cardiac stress, and a trend towards improvement in echocardiographic parameters.

Consistent beneficial effects on cardiac measures were observed in the modified intent-to-treat (mITT) group. Additionally, in a planned cohort of patients from the mITT population, vutrisiran treatment reduced cardiac uptake of technetium on scintigraphy imaging relative to baseline in a majority of assessable patients, including those with Perugini grade >=2 at baseline, suggesting that patients with the highest degrees of cardiac amyloid burden may recognize benefit from a TTR silencer.

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