Alnylam Reports Positive Topline 18-Month Results From HELIOS-A Phase 3 Study of Vutrisiran in Patients With hATTR Amyloidosis With Polyneuropathy

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months, including statistically significant improvements in neuropathy as measured by the modified Neuropathy Impairment Score (mNIS+7), quality of life (QOL), gait speed, nutritional status and overall disability, relative to external placebo data from the APOLLO Phase 3 study of patisiran.

The final secondary endpoint, reduction in serum TTR levels with vutrisiran, demonstrated non-inferiority relative to the within-study patisiran arm, as expected. In addition, patients treated with vutrisiran showed improvements in exploratory endpoints, including the biomarker NT-proBNP and certain echocardiographic parameters, relative to placebo, and an improvement in technetium uptake relative to baseline in a majority of patients in a planned cohort, providing potential evidence for reduced cardiac amyloid burden.