10 July 2018 - - Cambridge, Massachusetts-based investigational RNAi therapeutics company Alnylam Pharmaceuticals (NASDAQ: ALNY) has published the results from the APOLLO phase 3 trial of patisiran in The New England Journal of Medicine (NEJM), the company said.
The study showed that patisiran improved measures of polyneuropathy, quality of life, activities of daily living, ambulation, nutritional status, and autonomic symptoms relative to placebo in patients with hereditary transthyretin-mediated (hATTR) amyloidosis, an inevitably progressive and generally fatal disease.
The APOLLO Phase 3 trial was a randomized, double-blind, placebo-controlled, global study designed to evaluate the efficacy and safety of patisiran in hATTR amyloidosis patients with polyneuropathy. The trial enrolled 225 hATTR amyloidosis patients in 19 countries with 39 genotypes who were randomized 2: 1, with patisiran administered at 0.3 mg/kg once every three weeks for 18 months.
Patisiran is an investigational, intravenous RNAi therapeutic targeting transthyretin in development for the treatment of hereditary ATTR amyloidosis.
It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid in peripheral tissues and potentially restore function to these tissues.
Alnylam is translating RNA interference (RNAi) into a new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, and hepatic infectious diseases.