Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) is one of the best performing large cap stocks to buy. On September 29, Alnylam Pharmaceuticals announced new data analyses from the HELIOS-B Phase 3 study of its RNAi therapeutic, called AMVUTTRA (vutrisiran), presented at the Heart Failure Society of America/HFSA Annual Scientific Meeting 2025.
The findings showed a reduction in gastrointestinal/GI events, which is a multisystem manifestation of transthyretin-mediated amyloidosis cardiomyopathy/ATTR-CM. Compared to placebo, treatment with vutrisiran led to a 42% lower rate of GI events in the overall population, a 37% lower rate in the vutrisiran monotherapy group, and a 49% lower rate in the group treated with tafamidis at baseline.
Importantly, individual symptoms like diarrhea, nausea, and vomiting saw reductions of greater than 50% across all three groups, with the lower rate of GI events observed as early as three months into treatment. Vutrisiran is an RNAi therapeutic that achieves rapid knockdown of transthyretin/TTR, approved for treating the cardiomyopathy of wild-type or hereditary ATTR-CM and the polyneuropathy of hereditary ATTR-PN in adults.
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) is a biotech company that discovers, develops, and commercializes therapeutics based on ribonucleic acid interference.
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Disclosure: None. This article is originally published at Insider Monkey.