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Alnylam Files to Begin Phase I Trial of Hypercholesterolemia Drug


NEW YORK (GenomeWeb) — Alnylam Pharmaceuticals this week announced that it has filed an application with UK regulators to begin a Phase I trial of ALN-PCSsc, a subcutaneously administered siRNA treatment for hypercholesterolemia.

The placebo-controlled, single-blind study is expected to enroll up to 74 individuals with elevated baseline low-density lipoprotein levels. Single ascending and multiple doses of ALN-PCSsc will be evaluated.

The study is expected to begin in late 2014 or early 2015, with initial data available by mid-2015.

ALN-PCSsc is designed to silence proprotein convertase subtilisn/kexin type 9, or PCSK9, which has been shown to regulate low-density lipoprotein receptor levels and play a role in plasma low-density lipoprotein clearance.

The drug uses Alnylam's proprietary GalNAc conjugation technology to enable subcutaneous delivery. The company has also tested a different version of the drug that uses lipid-based delivery nanoparticles, but has been focusing its efforts on GalNAc recently.

Under the terms of a 2013 deal, The Medicines Company holds the rights to both drug candidates.