PARSIPPANY, NJ -- (Marketwired) -- The Medicines Company and Alnylam Pharmaceuticals, Inc. announced today the presentation of new pre-clinical data from ALN-PCSsc, targeting PCSK9 for the treatment of hypercholesterolemia. These data are being presented at the Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2014 Scientific Sessions being held May 1-3 in Toronto, Ontario.

In an oral presentation titled "A Subcutaneous, Potent and Durable RNAi Platform Targeting Metabolic Diseases, Genes PCSK9, ApoCIII and ANGPLT3" new pre-clinical data utilizing Alnylam's GalNAc-siRNA conjugate platform demonstrated robust, durable, and clamped knockdown of PCSK9 of up to 96% and reduction of LDL-C of up to 77% with single dose of ALN-PCSsc in the absence of statins in Non-Human Primates.

Alnylam also presented new single-dose durability data for ALN-PCSsc showing robust knockdown of PCSK9 and reductions in LDL-C that support once-monthly and possibly once-quarterly subcutaneous dosing regimens. Specifically, a single dose of ALN-PCSsc maintained greater than 50% LDL-C reductions for over 3 months in the absence of statin co-administration.

Alnylam plans to file an investigational new drug (IND) application, or IND equivalent, for ALN-PCSsc by late 2014 or early 2015.

ALN-PCSsc is being developed as a subcutaneously delivered RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia. The ALN-PCS program is partnered with The Medicines Company, where, under the terms of the agreement, Alnylam will complete certain pre-clinical studies and a Phase 1 clinical study of ALN-PCSsc and The Medicines Company is responsible for leading and funding development from Phase 2 forward and commercializing the ALN-PCS program, if successful.

"These new data support our belief that the unique mechanism of action for ALN-PCSsc holds great promise for a differentiated and potentially best-in-class strategy for PCSK9 antagonism," said Clive Meanwell, MD, PhD, Chairman and Chief Executive Officer of The Medicines Company. "Subcutaneous dosing holds the potential for administration of a therapeutic in patients less frequently and less invasively than intravenous dosing. We are very excited about the potential of ALN-PCSsc as a key asset in our acute cardiovascular care portfolio."

John Maraganore PhD, Chief Executive Officer of Alnylam said, "These new pre-clinical results demonstrate significant knockdown of PCSK9 and reductions in LDL-C with very durable effects, lasting over three months. We believe that this pharmacological profile could support a once-monthly and possibly a once-quarterly subcutaneous dosing regimen, which would be highly competitive with anti-PCSK9 monoclonal antibodies. Working with our colleagues at The Medicines Company, we look forward to advancing ALN-PCSsc toward clinical trials by the end of this year or early next."

Source: The Medicines Company