RXi Pharmaceuticals Corporation (Nasdaq: RXII), a recognized leader in RNA interference (RNAi) based therapeutic discovery and development, today announced that new preclinical data using proprietary self-delivering RNAi (sd-rxRNA™) compounds, including RXI-109, will be presented at the Keystone Symposia’s Mechanism and Biology of Silencing conference being held March 20-25, 2011 in Monterey, California. “RXi has made significant scientific progress and the data to be presented at this conference will highlight important advancements in our technology, platform and therapeutic programs. We have developed a novel class of self-delivering RNAi compounds with improved drug-like properties that may enable the development of therapeutics for a wide range of indications, including our lead development candidate, RXI-109, for the reduction of dermal scarring,“ said Anastasia Khvorova, Ph.D., RXi’s Chief Scientific Officer. The data will be presented in three posters and one speaker presentation, and will be available on RXi’s website following the conference, www.rxipharma.com/scientific_publications_presentations.html.
- Speaker Presentation – Karen Bulock, Ph.D., Associate Director of Discovery at RXi Pharmaceuticals, will present preclinical in vivo data showing robust, dose dependent, long-lasting, target-specific silencing data with an sd-rxRNA compound targeting CTGF (connective tissue growth factor). Additional data demonstrating that CTGF silencing results in reduction of myofibroblasts and collagen deposition, downstream effects related to abrogation of scar formation, will also be presented.
- Poster: Development of an sd-rxRNA™ Based Therapy for Treatment of Fibrosis: Preclinical Development Abstract - Outlines the results of several sd-rxRNA compounds targeting CTGF for the reduction of dermal scarring. Preclinical results using intradermal injection of CTGF targeting sd-rxRNAs show strong silencing for more than a week, as well as downstream effects related to abrogation of scar formation. RXi has initiated a preclinical development program for RXI-109, an sd-rxRNA compound designed to reduce the expression of CTGF, with a goal of submitting an IND in the second half of 2011.
- Poster: Self-Delivering RNAi Compounds (sd-rxRNA™) Demonstrate Robust Efficacy in vitro and in vivo in the Eye – Describes the preclinical studies used to establish the general utility of sd-rxRNA as a potential treatment for ocular disease. Data from these studies show that sd-rxRNA compounds not only successfully distribute to retinal cells without a delivery vehicle after an intravitreal injection, but also maintain statistically significant silencing of the mRNA target for up to 21 days. In addition, a pilot safety evaluation in mouse reveals no overt morphological, functional or microscopic signs of ocular toxicity.
- Poster: A Novel Class of Self-Delivering RNAi Compounds (sd-rxRNA™) with Robust in vitro and in vivo Efficacy: Mechanism of Action – Describes the development of a novel class of “self-delivering” RNAi (sd-rxRNA) compounds which demonstrate robust cellular uptake and silencing of target genes without the requirement for a delivery vehicle. sd-rxRNAs combine features of conventional RNAi and antisense technology and are heavily chemically modified to improve stability and lipophilicity. Preclinical data demonstrating the mechanism of uptake as well as efficacy of sd-rxRNA compounds using local and systemic administration in vivo will be presented.