The announcement of the early halt of Alnylam Pharmaceuticals Inc.'s Phase III ENDEAVOR clinical trial of interfering RNA (RNAi) drug revusiran in 206 patients with hereditary AATR amyloidosis with cardiomyopathy should have been a sector-moving event of Hillary Clinton tweet-like proportions. (Also see "Alnylam Ends Revusiran In Phase III, But Was Failure Due To Safety Or Efficacy?" - Scrip, 6 October, 2016.) Despite revusiran's failure and that of many other nucleic acid drugs, there was only a latent correction last week that resulted in the NASDAQ Biotech Index falling 1.3% against the 0.3% drop in the broad S&P 500 index. Alnylam's stock price was cut in half over the week.
Stockwatch: Alnylam Helps Nucleic Acid Drugs Degrade
Clinical trial failures and holds for drugs derived from nucleic acids from Alnylam and Ionis join those of Dicerna and Genta as the hit rate for these drugs continues to degrade. The real question is whether the failures have been due to a lack of efficacy or safety.
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