Alnylam Pharmaceuticals (ALNY) and Collaborators Publish Clinical Trial Results With ALN-PCS, an RNAi Therapeutic Targeting PCSK9 for the Treatment of Hypercholesterolemia, in The Lancet
10/3/2013 10:16:43 AM
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the publication in The Lancet of complete study results from a Phase I trial with ALN-PCS, a systemically administered RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia. The paper, titled “Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial,” (Fitzgerald, et al., The Lancet, doi:10.1016/S0140-6736(13)61914-5) reports the results of a study evaluating single intravenous dose administration of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins. Specifically, ALN-PCS administration resulted in a rapid, dose-dependent reduction in plasma PCSK9 of up to 84% relative to baseline and placebo, with a corresponding reduction in serum levels of low-density lipoprotein cholesterol (LDL-C) – a clinically validated endpoint – of up to 57% relative to baseline and placebo. The knockdown of PCSK9 and lowering of LDL-C were also found to be durable, with effects lasting for weeks after a single dose. This new paper documents the first human proof of concept for an RNAi therapeutic impacting a clinically validated endpoint.
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