Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data on the pharmacology of GalNAc-siRNA conjugates at the 12 th US-Japan Symposium on Drug Delivery Systems held December 16 – 20, 2013 in Lahaina, Maui, Hawaii. In a presentation titled “ Advances in Systemic Delivery of RNAi Therapeutics,” Alnylam scientists presented new data on tissue drug levels and sustained target knockdown achieved with long-term chronic dosing of GalNAc-siRNA conjugates. GalNAc-siRNA conjugates are a proprietary Alnylam delivery platform, and are designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor. This targeted delivery platform enables subcutaneous dose administration with a wide therapeutic index, and has demonstrated potent and durable gene silencing, as well as a favorable tolerability profile, in clinical and pre-clinical studies from multiple programs in the company’s “Alnylam 5x15” product pipeline.
“Our GalNAc-siRNA conjugate delivery platform is being employed in the majority of the pipeline programs within our “Alnylam 5x15” product development strategy. At Alnylam, we are at the forefront of optimizing the delivery of RNAi therapeutics using this proprietary, now clinically validated delivery approach,” said Muthiah (Mano) Manoharan, Ph.D., Senior Vice President of Drug Discovery at Alnylam. “These new data show that weekly subcutaneous dosing of GalNAc conjugates results in mean steady state drug levels that compare very favorably with other oligonucleotide platforms that require 100 to 1000 times greater tissue drug levels to achieve clinically relevant gene silencing. Furthermore, these data show that chronic dosing results in sustained target gene knockdown with a high level of consistency in the absence of any evidence for tachyphylaxis or sensitization. We view these findings as very encouraging, as they will likely have significant implications for RNAi-mediated silencing with chronic dosing in the clinical setting.”