Alnylam Advancing From Platform Buzz To Late-Stage Clinical Work
RNAi pioneer is getting closer to market with NDA filing in amyloidosis expected in 2017, but ample competition is coming along, albeit far behind.
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Phase III APOLLO results finally give the beleagured firm the first late-stage success for an RNAi thereapeutic and potentially a strong lead in the rare hereditary disease.
Despite its stock price falling 48% 24 hours after the shuttering of lead candidate revusiran, Alnylam is encouraged by analyst reports indicating the failure does not read through to its overall pipeline. The biotech reaffirmed its timeline for its second Phase III candidate patisiran.
Fitusiran's mechanism of reducing antithrombin levels could apply to all hemophilia patients, as well as those with other rare bleeding disorders. Reducing antithrombin increases thrombin levels and helps to increase normal clotting.