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RXi Pharmaceuticals Presented Data from Animal Trials Demonstrating the Utility of Self-Delivering RNAi at IBC's Oligonucleotide Therapeutics Conference

WORCESTER, Mass.--(BUSINESS WIRE)--Aug 5, 2009 - RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi), today announced positive data demonstrating the utility of RXi's self-delivering rxRNA™ (sd-rxRNA™) compounds, which are compounds that are delivered to cells without a delivery vehicle. The pharmacological data establish that in vivo administration of sd-rxRNA™ compounds result in preferential distribution to the liver in a mouse model, compared to the distribution of many other RNAi compounds that are rapidly filtered from the body via the kidneys.

The data presented indicate that RXi's sd-rxRNAs enter many cell types spontaneously, and that sd-rxRNAs also show dramatically improved distribution to tissues, specifically the liver. These sd-rxRNA™ compounds offer drug-like properties more similar to small molecules, rather than those offered by other RNAi therapeutics, as they act as stand-alone compounds that do not require an additional vehicle to deliver them inside the body.

Tod Woolf, Ph.D., President and CEO of RXi Pharmaceuticals commented, “These data are very significant to the RNAi therapeutics field because the liver is an essential organ, and there are many indications that could potentially be treated with successful delivery to the liver. One of the important advantages of our sd-rxRNA™ approach is that it does not require a separate delivery component potentially allowing for lower manufacturing costs and more patient friendly routes of administration.”

The development of sd-rxRNA™ compounds represents the combination of technology in-licensed from Advirna LLC and internal development work performed at RXi. Self-delivering rxRNAs have the following advantages over traditional RNAi therapeutics currently in development:


  • Spontaneous cellular uptake: delivery of RNAi therapeutics without the added expense of liposomes, complexes, nanoparticles or other delivery vehicles
  • Potentially patient-friendly administration routes: subcutaneous administration to treat patients without the need of medical professional supervision
  • Bio-distribution to targeted tissues: distribution of rxRNAs to certain tissues while reducing renal clearance
  • Reduced side effect profiles: reduced side effects that are often associated with formulations that contain an added delivery vehicle

A subset of slides presented at IBC's Oligonucleotide Therapeutics Conference on August 3-4 in Boston, MA can be found on the company's website

About RNA Interference (RNAi)

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.

About RXi Pharmaceuticals Corporation

RXi Pharmaceuticals is a discovery-stage biopharmaceutical company pursuing the development and potential 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™ -- 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
SAN Group
Susan Noonan, 212-966-3650
Rx Communications Group
Eric Goldman, 917-322-2563


Posted: August 2009