CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 4, 2012-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the achievement of positive preliminary results from its ongoing clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia. ALN-PCS demonstrated statistically significant RNAi silencing of PCSK9 of up to 66% and reductions of up to over 50% in levels of low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol, a clinically validated endpoint. Alnylam believes that the preliminary results of this study, presented today at the Brigham and Women’s Hospital in Boston, also document for the first time major advances in delivery of RNAi therapeutics with second generation lipid nanoparticles (LNPs) in human studies. This Phase I study continues with planned dose escalation, given the favorable safety profile and positive efficacy achieved to date. Additional results of the study are expected to be reported in the first half of 2012.
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