Posted: January 23, 2009

First Dual Targeting RNAi Therapeutic Receives FDA Clearance

(Nanowerk News) Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that its investigational new drug (IND) application for ALN-VSP has been cleared by the U.S. Food and Drug Administration (FDA) to begin enrolling patients.
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 Corporation. 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.
Pre-clinical 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.
“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 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.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine.
RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
Source: Alnylam Pharmaceuticals (press release)
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