Clinical Report: Alnylam’s HELIOS-B details trigger market reaction

Although Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) had disclosed top-line results from the Phase III HELIOS-B study of Amvuttra vutrisiran in June, its presentation Friday of more detailed data was still enough to provoke a market reaction, despite no major surprises. The study evaluated the gene silencing therapy to treat TTR amyloidosis with cardiomyopathy, a broader indication than that for which the treatment is already approved, the type of amyloidosis that manifests as polyneuropathy.

Alnylam had already described HELIOS-B’s readout as a “big win” scenario that positioned Amvuttra as a standard-of-care therapy for ATTR-CM. The therapy led to a benefit on all-cause mortality and recurrent cardiovascular events, meeting its primary endpoint. Friday’s presentation included subgroup data, more information about secondary endpoints, and plots showing evidence of efficacy over time...