Study Shows Intradigm's RNAi Nanoplex Technology To Be Effective Systemic siRNA Delivery Platform for the Treatment of Various Cancer Models

Tuesday, April 15, 2008 General News J E 4
PALO ALTO, Calif., April 14 Intradigm Corporation, aleading developer of targeted, systemic RNA interference (RNAi) therapeutics,today announced study results that demonstrated the company's RNAi Nanoplex(NPX) delivery technology platform is capable of effectively delivering smallinterfering RNAs (siRNAs) targeting both vascular endothelial growth factor(VEGF) and epidermal growth factor (EGF) pathways, two clinically validatedpathways in the treatment of various cancers. Study data demonstrated thatmice treated with Intradigm's proprietary peptide-based, biodegradable polymer(PolyTran(TM)) carrying each of VEGF-, VEGFR2- orEGFR-targeted siRNAs experienced significant tumor growth inhibition that wassimilar to that of mice receiving the study's positive control (VEGF antibodyBevacizumab or EGFR tyrosine kinase inhibitor Erlotinib). In addition, mice inthe active siRNA treatment groups demonstrated no apparent toxicity orsignificant weight loss.

These study findings, which also confirmed the gene-silencing mechanism ofaction of Intradigm's technology, were presented by Intradigm scientists onSunday, April 13 at the 2008 American Association for Cancer Research (AACR)Annual Meeting in San Diego.

"We are very pleased with these study results as they confirm thegene-silencing mechanism of our RNAi NPX delivery technology, as well as itsability to systemically deliver siRNAs to tumors in a safe and effectivemanner," said Mohammad Azab, M.D., president and chief executive officer ofIntradigm. "Furthermore, by effectively targeting VEGF, VEGFR2, and EGFR,three established oncology targets, with our siRNAs, we have demonstrated theversatility of the RNAi NPX delivery technology which could be translated intomultiple product opportunities for Intradigm's pipeline of RNAi therapeutics."

The findings presented today support data from several other in vivomodels, including xenograft models of different cancers, which havedemonstrated the efficacy of the RNAi NPX delivery technology.


To address the issue of effective and targeted delivery of RNAitherapeutics, Intradigm has developed and in-licensed an extensiveintellectual property portfolio around its proprietary RNAi Nanoplex (NPX)delivery technology. The RNAi NPX delivery technology is a modular,multi-component delivery vector that carries active siRNA molecules in itscore with the flexibility to attach a Polyethylene Glycol (PEG) layer and/or atargeting ligand to the polymer-siRNA NPX to improve circulation half life andachieve selective distribution to the target tissue. At the core of the RNAiNPX delivery technology is Intradigm's proprietary peptide-based biodegradablepolymer known as PolyTran(TM). PolyTran enables safe systemic delivery of thesiRNAs. Additionally, this platform is unique in its ability to offer tissuespecific targeting of siRNA through the attachment of specific ligandsdirected to target cell receptors. An additional key strength of the RNAi NPXdelivery technology is its ability to incorporate multiple siRNAs in a singledrug product. These multi-targeted therapeutics can simultaneously silenceseveral mRNAs that represent parallel or synergistic elements of the samedisease pathway resulting in a more comprehensive and/or novel therapeuticpotential.

Recent technology licensing agreements with institutions such as theUniversity of Massachusetts Medical School and the Massachusetts Institute ofTechnology have enhanced Intradigm's existing intellectual property portfolioand provided the company with the rights and know-how to leverage a broadfamily of polymers and polymer structures, as well as next generation siRNAstructures, in connection with its RNAi NPX delivery technology.


Intradigm is a private biotechnology company dedicated to the developmentof targeted, systemic RNA inter


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