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Alnylam Pharmaceuticals Inc present new clinical data for Patisiran

Monday, 28 Apr 2014 08:00am EDT 

Alnylam Pharmaceuticals Inc:New clinical data for patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) in development for treatment of TTR-mediated amyloidosis (ATTR).Frst company presented updated Phase 2 results in patients with Familial Amyloidotic Polyneuropathy (FAP) confirming robust TTR knockdown of up to 96 pct with mean TTR knockdown of about 80 pct.Furthe Alnylam presented preliminary results from open-label extension (OLE) study with patisiran in patients that were enrolled in the Phase 2 study.Preliminary results from OLE study showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the 80 pct target level through 168 days.Moreover, OLE results showed favorable tolerability profile with up to eight doses administered.Finally 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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