Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, and collaborators announced today new clinical data for patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of TTR-mediated amyloidosis (ATTR). These data are being presented at the International Symposium on Amyloidosis (ISA) held April 27 – May 1, 2014 in Indianapolis, Indiana. First, the company presented updated Phase 2 results in patients with Familial Amyloidotic Polyneuropathy (FAP) 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 (OLE) 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.
“We continue to make strong progress in our patisiran development program, which is focused on making a difference in the lives of ATTR patients with polyneuropathy. Several highlights from our efforts are being presented at ISA, including updated Phase 2 study results, initial data from our Phase 2 OLE study, and results from a natural history study of neuropathy progression in FAP. Notably, our initial results from the Phase 2 OLE confirm a sustained TTR knockdown at the 80% target level through up to 168 days. Further, we are very encouraged by the tolerability data, especially the significant reduction in infusion reactions due to the use of our proprietary micro-dosing regimen. With this early update on the OLE study, we still expect to present clinical endpoint data results later this year consistent with our external guidance,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “Another notable highlight is the presentation of FAP natural history study results. This cross-sectional analysis evaluated the neuropathy progression rate in a multinational population of patients with FAP, and demonstrates a rapid progression in NIS and a high correlation of this measurement with disease severity. Moreover, these results give us confidence that our APOLLO Phase 3 trial of patisiran in FAP patients is robustly powered to show the potential impact of TTR lowering on the mNIS+7 endpoint used in that study.”