Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today positive interim results from its Phase I clinical trial of ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The data are being presented today at the Heart Failure Society of America 17 th Annual Scientific Meeting being held September 22 – 25, 2013 in Orlando, Fla. Results show that ALN-TTRsc administration led to robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR, the disease causing protein in ATTR, was found to be rapid, dose dependent, and durable. To date, ALN-TTRsc has been found to be generally safe and well tolerated in this study. These human data are the first to be presented for Alnylam’s proprietary GalNAc-siRNA conjugate delivery platform, enabling subcutaneous dosing of RNAi therapeutics with a wide therapeutic index, and demonstrate human translation for this platform. Moreover, these results establish a new benchmark for consistent TTR knockdown of approximately 90% for RNA therapeutics in development for the treatment of ATTR.
“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 Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “These new data support our belief that ALN-TTRsc has the potential to be an important therapeutic for the treatment of familial amyloidotic cardiomyopathy (FAC) – a disease for which there are no approved therapies. With these results in hand, we are well positioned for continued execution on this program, which includes the initiation of a pilot Phase II study in FAC patients by the end of this year, and – assuming positive results – start of a pivotal Phase III trial with ALN-TTRsc by the end of 2014.”