Alnylam Announces Positive Phase II Data for Patisiranfor for Treatment of TTR-Mediated Amyloidosis, Initiates Phase III Trial

Alnylam Pharmaceuticals, ALNY, a leading RNAi therapeutics company, announced today the achievement of positive clinical results from its Phase II trial of patisiran (ALN-TTR02), an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The data are being presented at the IXth International Symposium on Familial Amyloidotic Polyneuropathy (ISFAP) being held in Rio de Janeiro, Brazil, November 10 – 13. Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%. Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and See full press release