23 Sep, 2013 Positive Clinical Results from Phase I Trial of ALN-TTRsc
We have reported positive clinical results from our Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). Data were presented at the Heart Failure Society of America 17th Annual Scientific Meeting being held September 22 – 25. These results demonstrated that ALN-TTRsc achieved robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR was found to be rapid, dose-dependent, and durable. ALN-TTRsc was 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. We believe these results establish a new benchmark for consistent TTR knockdown of approximately 90% for RNA therapeutics for the treatment of ATTR. 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 – and position us well for continued execution on this program.