Phase 1 study of ALN-VSP for liver cancer

Alnylam Pharmaceuticals announced that it has initiated a Phase 1 human clinical trial of ALN-VSP for the treatment of patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement. This trial is a multi-center, open label, dose escalation study designed to enroll approximately 55 patients with advanced solid tumors with liver involvement, who have failed to respond to or have progressed after standard treatment. The study’s primary objective is to evaluate the safety, tolerability, and pharmacokinetics of intravenous ALN-VSP, including demonstration of the maximum tolerated dose.

ALN-VSP is comprised of two small interfering RNAs (or siRNAs) formulated in a lipid nanoparticle. ALN-VSP is designed to target two genes critical in the growth and development of cancer cells: kinesin spindle protein (KSP), which is required for tumor proliferation; and vascular endothelial growth factor (VEGF), which is required for tumor growth.

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