Alnylam: ALN-PCS Phase I Study Results Published In The Lancet

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY), an RNAi therapeutics company, Thursday announced 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 is 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."

It 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 led to a rapid, dose-dependent reduction in plasma PCSK9 of up to 84 percent relative to baseline and placebo, with a corresponding reduction in serum levels of low-density lipoprotein cholesterol of up to 57 percent 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. According to the company, this new paper documents the first human proof of concept for an RNAi therapeutic impacting a clinically validated endpoint.