Alnylam reports positive results for RNAi therapy

Cambridge, MA-based Alnylam Pharmaceuticals ($ALNY) says it garnered positive interim data from a Phase I study of ALN-TTRsc, an RNAi therapy targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis. "These new ALN-TTRsc results are a major milestone in our ATTR program, as well as our entire pipeline of RNAi therapeutics. Specifically, we have demonstrated robust, up to 94% knockdown of circulating TTR with a very encouraging safety profile. We believe this level of consistent TTR knockdown is exceptional and unmatched, and we now aim to advance ALN-TTRsc in future clinical studies with the goal of achieving approximately 90% TTR knockdown to maximize clinical efficacy," said Dr. Akshay Vaishnaw, the chief medical officer of Alnylam. Shares of Alnylam slipped this morning. Release