CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and collaborators announced today new results from the company’s ongoing Phase 2 open-label extension (OLE) study of patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic polyneuropathy (FAP). In collaboration with investigators at The Scripps Research Institute and Misfolding Diagnostics, Inc., patisiran administration was shown to reduce pathogenic, misfolded TTR monomers and oligomers in FAP patients in the Phase 2 OLE study. Specifically, patisiran administration resulted in a rapid and sustained reduction of approximately 90% in serum non-native conformations of TTR (NNTTR). Since NNTTR is pathogenic in ATTR amyloidosis, these results provide direct mechanistic evidence supporting the therapeutic hypothesis that TTR knockdown has the potential to result in clinical benefit. In addition, Alnylam presented complete 12-month data from all 27 patients initially enrolled in the patisiran OLE study, showing a mean 3.1-point decrease in the modified Neuropathy Impairment Score (mNIS+7) at 12 months as well as sustained mean maximum reductions in total serum TTR of 91% for over 18 months. Moreover, patisiran administration was found to be generally well tolerated out to 21 months of treatment.
Help employers find you! Check out all the jobs and post your resume.
