Alnylam releases data on its Phase II study of TTR-mediated amyloidosis patients

Alnylam Chief Medical Officer Akshay Vaishnaw

At the American Neurological Association's 2014 Annual Meeting in Baltimore, Alnylam ($ALNY) released 6-month data on its Phase II open-label extension (OLE) study of patisiran (ALN-TTR02), a clinical-stage RNAi candidate for the treatment of TTR-mediated amyloidosis in patients with familial amyloidotic polyneuropathy. The 19 patients performed better than baseline on their change in the modified Neuropathy Impairment Score. "We believe the approximate one point decrease in neuropathy impairment score is an encouraging result in light of multiple historical data sets that would have predicted an increase of 7 to 10 points for untreated patients with similar baseline characteristics. These data will be increasingly meaningful as we monitor neuropathy progression in patisiran-treated patients over time, and we look forward to sharing those results at least once annually hereafter," said Chief Medical Officer Dr. Akshay Vaishnaw in a statement. The company also announced data from its Phase I candidate for investigational RNAi therapeutic targeting transthyretin for TTR-mediated amyloidosis at the Annual Meeting of the Oligonucleotide Therapeutics Society in San Diego. It achieved knockdown of TTR serum of up to 96.2%. Release | Release