Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The company is reporting that ALN-TTRsc achieved robust and statistically significant (p<0.01) knockdown of serum TTR protein levels of greater than 80% in healthy volunteer subjects, in line with results for ALN-TTRsc previously reported in non-human primates. In addition, to date ALN-TTRsc was found to be generally safe and well tolerated. Dose escalation in this trial continues and results will be presented at the Annual Scientific Meeting of the Heart Failure Society of America (HFSA), being held September 22 – 25, 2013 in Orlando, Fla. These human study results are the first to be reported for Alnylam’s proprietary GalNAc-siRNA conjugate delivery platform, enabling subcutaneous dosing of RNAi therapeutics with a wide therapeutic index.
“These clinical results with ALN-TTRsc establish human translation for RNAi therapeutics that utilize our GalNAc-siRNA conjugate delivery platform. This platform enables subcutaneous dose administration with a wide therapeutic index and has now become our primary approach for development of RNAi therapeutics. As a result, we believe these data are very meaningful not only for the continued advancement of ALN-TTRsc, but also for the continued execution on our entire ‘Alnylam 5x15’ product strategy,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Specifically, we are very excited to report top-line results from the study showing statistically significant knockdown of serum TTR to levels greater than 80% in treated subjects, results which are in line with our non-human primate experience. We look forward to continued advancement of our ALN-TTRsc program, including presentation of data from the Phase I trial at the HFSA meeting in September, start of a Phase II study in familial amyloidotic cardiomyopathy patients by the end of this year, and – assuming positive results – start of a pivotal Phase III trial for ALN-TTRsc in 2014.”