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RXi Pharmaceuticals Presented New Pre-Clinical Data at the Keystone RNAi Silencing Conference

WORCESTER, Mass.--(BUSINESS WIRE)--Jan 19, 2010 - RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced that new pre-clinical data using proprietary rxRNA™ compounds was presented at the Keystone Symposia's RNA Silencing: Mechanism, Biology and Application held January 14-19 in Keystone, Colorado.


“RXi has progressed substantially in advancing its technology and building a robust and differentiated RNAi therapeutic platform. Our cutting-edge innovation has led to the discovery and development of novel RNAi compounds with improved drug-like properties that may enable the development of therapeutics for a wide range of indications. By having such a broad platform, we believe that we are well positioned to focus on the most promising therapeutic areas and to select the right candidates to take forward into development,” commented Anastasia Khvorova, Ph.D., RXi's Chief Scientific Officer.


The data were presented in four posters that are available on RXi's website,




  • Novel, Chemically Modified RNAi Compounds with Improved Potency, Stability and Specificity – Describes the development of novel, chemically modified, 25-mer blunt RNAi compounds (rxRNAori™) with enhanced stability, reduced off-target effects and reduced immune stimulatory activity. This novel configuration enables rapid identification of potent RNAi compounds for potential therapeutic development due to improvements in the algorithm used to identify extremely potent candidate molecules.

  • Novel “Self-Delivering” RNAi Compounds with Enhanced Cellular Uptake and Distribution Properties – Outlines the development of “self-delivering” RNA (sd-rxRNA™) compounds that efficiently enter cells without the need for an additional delivery vehicle. sd-rxRNAs are reduced in size compared to conventional siRNAs and are chemically modified to improve cellular uptake without interfering with the ability to modulate gene expression.

  • Robust Intradermal Efficacy with Novel Chemically Modified Self-Delivering RNAi Compounds – Demonstrates efficient intracellular uptake of fluorescently-tagged sd-rxRNA into dermal cells and significant reduction of targeted mRNA using intradermal injection as a model of local delivery in vivo. The data presented establish the efficacy of locally-administered sd-rxRNAs in animal models and support the use of these novel compounds for clinical applications where direct or local administration is possible, thereby potentially enabling broad opportunities for therapeutic development using local administration routes.

  • Highly Potent RNAi Compounds Derived from a Single, Short Oligonucleotide Sequence - Illustrates the development of a 27 nucleotide RNA construct with silencing potency equal to duplex RNAs. RXi's single-oligo RNAi triggers (rxRNAsolo™) act as homo-dimers and have shown high, seed-specific potency. In addition, the poster shows that a hetero-dimer (rxRNAduo™) can simultaneously silence two different genes.


About RNA Interference (RNAi) and Self-delivering rxRNA™ (sd-rxRNA™)


Regarded as a revolutionary discovery in biology, RNA interference (RNAi) is a naturally occurring mechanism whereby short, double-stranded RNA molecules interfere with the expression of genes in living cells. This mechanism has the potential to be harnessed to “silence” or specifically block the production of disease-causing proteins before they are made. This technology can potentially be used to treat human diseases by “turning-off” genes that lead to disease in the first place. RXi Pharmaceuticals is using RNAi technology to develop RNA-derived molecules targeting disease-causing genes.


Self-delivering rxRNA™ (sd-rxRNA™) is a proprietary technology developed at RXi which has the potential to enable the efficient delivery of RNAi compounds without the requirement of an additional delivery vehicle. This technology has potential clinical applications for diseases where localized delivery is an option and also has the potential to be applied for indications requiring systemic delivery of RNAi.


About RXi Pharmaceuticals Corporation


RXi Pharmaceuticals is a discovery-stage biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi) for the treatment of human diseases. RXi has a comprehensive therapeutic platform that includes both RNAi compounds and delivery methods. RXi uses its own version of RNAi compounds -- rxRNA™ (sd-rxRNA, rxRNAori, rxRNAsolo and rxRNAduo) -- that provide an advanced alternative to conventional small interfering RNAs (siRNAs) and define the next generation of RNAi technology. rxRNA™ compounds are designed specifically for therapeutic use and contain many of the properties needed to move RNAi based drugs into the clinic. RXi Pharmaceuticals believes it is well positioned to compete successfully in the RNAi-based therapeutics market with its accomplished scientific advisors, including Dr. Craig Mello, recipient of the 2006 Nobel Prize for his co-discovery of RNAi; a management team that is experienced in developing RNAi products; and a strong early intellectual property position in RNAi chemistry and delivery.


Forward-Looking Statements


This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements about future expectations, plan and future development of RXi Pharmaceutical Corporation's products and technologies. These forward-looking statements about future expectations, plans and prospects of the development of RXi Pharmaceutical Corporation's products and technologies involve significant risks, uncertainties and assumptions, including the risk that the development of our RNAi-based therapeutics may be delayed or may not proceed as planned and we may not be able to complete development of any RNAi-based product, the risk that the FDA approval process may be delayed for any drugs that we develop, risks related to development and commercialization of products by our competitors, risks related to our ability to control the timing and terms of collaborations with third parties and the possibility that other companies or organizations may assert patent rights that prevent us from developing our products. Actual results may differ materially from those RXi Pharmaceuticals Corporation contemplated by these forward-looking statements. RXi Pharmaceuticals Corporation does not undertake to update any of these forward-looking statements to reflect a change in its views or events or circumstances that occur after the date of this release.






Contact: RXi Pharmaceuticals

Donna Falcetti, 508-929-3615



S. A. Noonan Communications

Susan Noonan, 212-966-3650



Rx Communications Group

Eric Goldman, 917-322-2563



Posted: January 2010