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April 28, 2014
08:05 EDTALNYAlnylam, collaborators present new clinical data for patisiran
Alnylam Pharmaceuticals and collaborators announced new clinical data for patisiran, an RNAi therapeutic targeting transthyretin in development for the treatment of TTR-mediated amyloidosis. These data are being presented at the International Symposium on Amyloidosis held April 27 – May 1 in Indianapolis, Indiana. First, the company presented updated Phase 2 results in patients with Familial Amyloidotic Polyneuropathy confirming robust TTR knockdown of up to 96% with a mean TTR knockdown of approximately 80%. Further, Alnylam presented preliminary results from the open-label extension study with patisiran in patients that were enrolled in the Phase 2 study. Preliminary results from the OLE study showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the 80% target level through 168 days. Moreover, OLE results showed a favorable tolerability profile with up to eight doses administered. Finally, the company presented results of a natural history, cross-sectional analysis study of 283 FAP patients aimed at measuring the rate of neuropathy progression and its correlation with disease severity. These results provide support for Alnylam’s Phase 3 APOLLO trial where patisiran is being evaluated for its potential efficacy and safety in the FAP indication.
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November 30, 2015
07:47 EDTALNYLeerink positive on Alnylam, Juno, Karyopharm into ASH meeting
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