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Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from two RNAi therapeutic programs for cardiovascular disease, including: ALN-PCSsc, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; and ALN-ANG, an RNAi therapeutic targeting ANGPTL3 for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia. These data were presented at the American Heart Association (AHA) Scientific Sessions held November 16 – 20, 2013 in Dallas, Texas. In a presentation titled “
A Subcutaneous Platform for RNAi Therapeutics Targeting Metabolic Diseases: PCSK9 and ANGPTL3,”
Alnylam scientists and collaborators presented updated non-human primate (NHP) data showing that subcutaneous administration of ALN-PCSsc led to an up to 95% knockdown of plasma PCSK9 and an up to 67% reduction of low density lipoprotein cholesterol (LDL-C) – in the absence of statin co-administration. In addition, new results were presented for ALN-ANG, showing a greater than 95% reduction of triglycerides and greater than 85% reduction of LDL-C in a rodent model of mixed hyperlipidemia. ALN-PCSsc and ALN-ANG utilize the company’s proprietary GalNAc-siRNA conjugate delivery platform, which is designed to achieve targeted delivery of RNAi therapeutics to hepatocytes through uptake by the asialoglycoprotein receptor, enabling subcutaneous dose administration with a wide therapeutic index. ALN-PCSsc and ALN-ANG are programs in the company’s “Alnylam 5x15” product development and commercialization strategy, where the company aims to advance at least five RNAi therapeutic programs toward genetically validated disease targets into clinical development, including programs in advanced stages, by the end of 2015.
“Our recent progress with GalNAc-siRNA conjugates enables advancement of subcutaneously administered RNAi therapeutics for the treatment of cardiovascular disease, and our new data presented at the AHA meeting feature our efforts with ALN-PCSsc and ALN-ANG. With ALN-PCSsc, our new study results in non-human primates show an up to 95% knockdown of plasma PCSK9 and an up to 67% reduction of LDL-C – in the absence of statin co-administration, with a very durable knockdown of more than 50 days after the last dose. We believe these results support a highly competitive target product profile, and we look forward to advancing ALN-PCSsc toward clinical trials in 2014 with our partner The Medicines Company,” said Rachel Meyers, Ph.D., Vice President of Research and RNAi Lead Development at Alnylam. “In addition, our scientists presented new data from our discovery program with ALN-ANG. Specifically, a lead GalNAc-siRNA conjugate targeting ANGPTL3 showed a greater than 95% reduction of triglycerides and a greater than 85% reduction of LDL-C in a mouse model of mixed hyperlipidemia. These pre-clinical results phenocopy the human genetics for loss of function in ANGPTL3, and are supportive of our efforts to advance this new RNAi therapeutic program into later stages of pre-clinical development.”