Alnylam’s Liver Cancer Drug Cleared for Phase I Study

Alnylam Pharmaceuticals’ IND application for ALN-VSP has been cleared by the FDA to begin enrolling patients in a Phase I trial. ALN-VSP, an RNAi therapeutic for the treatment of liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement, contains two small interfering RNAs (siRNAs, the molecules that mediate RNAi), formulated in a lipid nanoparticle developed by Tekmira Pharmaceuticals.

Preclinical data in mouse tumor model studies have demonstrated efficacy of ALN-VSP, including suppression of targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival. The drug is designed to target two genes critical in the growth and development of cancer: kinesin spindle protein (KSP), required for tumor proliferation; and vascular endothelial growth factor (VEGF), required for tumor growth.

“ALN-VSP represents Alnylam’s first IND for a systemically delivered RNAi therapeutic, which is a testament to the very strong progress we have made in achieving delivery of siRNAs,” said Akshay Vaishnaw, M.D., Ph.D., senior vice president, clinical research at Alnylam. “We are very excited about reaching yet another important milestone in this program and in our overall efforts. As planned, we expect to initiate patient dosing in the first half of this year, which positions us solidly on track to meet our goal of having three programs in clinical trials in 2009.”

The Phase I study is a multi-center, open label, dose escalation trial that will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ALN-VSP in patients with advanced solid tumors related to the liver.