The Medicines Company (Nasdaq: MDCO) a leading acute/intensive hospital care company, and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that their strategic alliance has yielded a lead Development Candidate that is a subcutaneously administered RNAi therapeutic (ALN-PCSsc) targeting PCSK9 for the potential treatment of hypercholesterolemia.
New data from non-human primate studies were presented at the Oligonucleotide Therapeutics Society (OTS) meeting, and show that ALN-PCSsc leads to an up to a 90% PCSK9 knockdown and an up to 68% lowering of LDL cholesterol in the absence of statins. PCSK9 is a protein that regulates low-density lipoprotein (LDL) receptor levels on hepatocytes.
The ALN-PCSsc lead Development Candidate utilizes Alnylam’s GalNAc-siRNA conjugate delivery technology that enables subcutaneous dose administration. Pre-clinical durability data support the potential for every-two-weeks dosing, with possible every-four-weeks dosing.
Additional details of the subcutaneously administered development candidate, and the data, will be presented by John Maraganore, PhD, Chief Executive Officer of Alnylam at The Medicines Company’s Investor & Analyst Day in New York (webcast 8:30ET to noon at www.themedicinescompany.com).Dr. Maraganore said, “We have rapidly advanced the ALN-PCS collaboration to reach our first goal, which was to designate our Development Candidate by the end of this year. We anticipate submitting an investigative new drug application for ALN-PCSsc with the US Food and Drug Administration in late 2014. This is an important program within our ‘Alnylam 5x15’ product development and commercialization strategy focused on RNAi therapeutics directed toward genetically validated targets.” “These new data with subcutaneous delivery support our belief that the unique mechanism of action for ALN-PCS 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.”