Alnylam wins FDA clearance to initiate Phase I liver cancer study

The proposed Phase I study is a multi-center, open label, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ALN-VSP in patients with advanced solid tumors with liver involvement. Additional study design details will be provided upon initiation of the clinical study.

ALN-VSP is designed to target two genes critical in the growth and development of cancer: kinesin spindle protein, or KSP, required for tumor proliferation; and vascular endothelial growth factor, or VEGF, required for tumor growth.

Preclinical data in mouse tumor model studies have demonstrated robust efficacy of ALN-VSP, including suppression of targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival, the company said.

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, formulated in a lipid nanoparticle developed by Tekmira Pharmaceuticals.

Akshay Vaishnaw, senior vice president of clinical research at Alnylam, said: We expect to initiate patient dosing in the first half of 2009, which positions us solidly on track to meet our goal of having three programs in clinical trials in 2009.