Tekmira's LNP Delivery Technology Enables Proof of RNAi in Man & Clinical Activity
Tekmira Pharmaceuticals Corporation recently provided comments on presented data from a Phase I human clinical trial with ALN-VSP conducted by Alnylam Pharmaceuticals, Inc. ALN-VSP, an RNAi therapeutic for the treatment of liver cancers that utilizes Tekmira’s LNP technology, the only RNAi delivery technology supporting multiple clinical candidates being advanced by Tekmira and its partners in several different disease indications.
The most recent ALN-VSP data was presented at the American Society of Clinical Oncology (ASCO) meeting in a poster titled Phase I Dose-Escalation Study of ALN-VSP02,Âa Novel RNAi Therapeutic for Solid Tumors With Liver Involvement. Alnylam disclosed that ALN-VSP was generally well tolerated, demonstrated evidence for anti-tumor activity, and was found to mediate RNAi activity in both hepatic and extra-hepatic tumors.
“Earlier this year, we were excited to see Alnylam’s interim clinical data with ALN-VSP, which demonstrated RNAi activity in human tumor biopsies and confirmed that our LNP technology enables bona fide RNAi activity in man. Now, another important advancement has been made in the RNAi field as Alnylam reports that its ALN-VSP data demonstrates anti-tumor activity,” said Dr. Mark J. Murray, Tekmira’s President and CEO.
“To date, this is the second completed human clinical trial that has reported Tekmira’s LNP technology is safe and well tolerated. We anticipate further clinical data being presented from a number of LNP-enabled products over the remainder of 2011. Building upon this momentum, Tekmira continues to innovate and protect our LNP technology with a focus on improvements to LNP potency and tolerability, as well as combining new RNAi payloads with LNP delivery,” he added.
Tekmira’s LNP technology is enabling the systemic RNAi product pipeline of Alnylam Pharmaceuticals, including the products ALN-VSP, ALN-TTR, and ALN-PCS. As Alnylam disclosed, ALN-VSP was administered to 41 patients at doses ranging from 0.1 to 1.5 mg/kg; a total of 182 doses have been administered, including to one patient who has received 24 doses at 0.7 mg/kg over the course of more than 1 full year, and continues to receive treatment in the study. The Phase I trial was designed as a multi-center, open label, dose escalation study in patients with advanced solid tumors with liver involvement who have failed to respond to or have progressed after standard treatment. The primary objective was to evaluate the safety, tolerability, and pharmacokinetics of intravenous ALN-VSP.
RNAi therapeutics have the potential to treat a broad number of human diseases by “silencing” disease-causing genes. The discoverers of RNAi, a gene-silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi therapeutics, such as siRNAs, require delivery technology to be effective systemically. LNP technology is one of the most widely used siRNA delivery approaches for systemic administration. Tekmira’s LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles, which are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. Tekmira’s LNP formulations are manufactured by a proprietary method that is robust, scalable, and highly reproducible, and LNP-based products have been reviewed by multiple FDA divisions for use in clinical trials. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.
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