RXi Pharmaceuticals Awarded NCI SBIR Funding to Advance Self-Delivering RNAi Technology

WESTBOROUGH, Mass.--(BUSINESS WIRE)--RXi Pharmaceuticals Corporation (OTC: RXII), a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted technologies, today announced that it was awarded a Small Business Innovation Research (SBIR) grant by the National Cancer Institute (NCI) of the National Institutes of Health (NIH). The grant provides funding for a project enabling the discovery and preclinical development of sd-rxRNAs® as potential therapy for retinoblastoma, a pediatric ocular malignancy. The project will be completed in collaboration with Dr. David Cobrinik and colleagues at the Memorial Sloan-Kettering Cancer Center.

“The grant process is highly competitive, and this award serves to recognize and support the therapeutic potential of RXI’s proprietary RNAi platform.”

Dr. Cobrinik commented, "Prior studies performed by RXi and their collaborators at the University of Massachusetts, demonstrated that self-delivering sd-rxRNAs can penetrate the retina and regulate retinal gene expression. As retinoblastomas form within the retina, we hypothesized that sd-rxRNAs might also regulate gene expression in intraocular retinoblastoma cells. This SBIR award will enable us to test this hypothesis, possibly extending the application of sd-rxRNAs from ophthalmic diseases to the cancer setting."

RXi has been awarded approximately $300,000 to fund the collaborative project over six months. Under this grant (R43CA165899), entitled “Development of sd-rxRNAs® as Therapy for Retinoblastoma and Other Malignancies”, sd-rxRNA compounds will be developed to silence one or more targets critical to retinoblastoma cell growth and survival. The sd-rxRNA compounds will be evaluated for efficacy in in vivo models of retinoblastoma by the Cobrinik lab.

“We are honored to have the opportunity to collaborate with the team at Memorial Sloan-Kettering, and are very pleased to have received financial support from the NCI to further the development of our novel RNAi compounds as potential therapeutics for ocular cancer,” said Pamela Pavco, Chief Development Officer at RXi Pharmaceuticals. “The grant process is highly competitive, and this award serves to recognize and support the therapeutic potential of RXI’s proprietary RNAi platform.”

The Small Business Innovation Research (SBIR) program was created by the U.S. Congress to strengthen the role of small, innovative companies in federally supported research and development. It is one of the largest sources of early-stage technology financing in the U.S. At the National Cancer Institute (NCI), these programs seek small business participation in the development and commercialization of technologies for the treatment and diagnosis of cancer.

About sd-rxRNA

RXi’s sd-rxRNA compounds are designed for therapeutic use and have drug-like properties, such as high potency, target specificity, serum stability, reduced immune response activation, and efficient cellular uptake. They are hybrid oligonucleotide compounds that combine the beneficial properties of both conventional RNAi and antisense technologies. The sd-rxRNAs have a single-stranded phosphorothioate region, a short duplex region, and contain a variety of nuclease-stabilizing and lipophilic chemical modifications. The combination of these features has been shown in vitro and in animal tissues to allow sd-rxRNAs to achieve efficient spontaneous cellular uptake and potent, long-lasting intracellular activity.

About RXi Pharmaceuticals Corporation

RXi Pharmaceuticals Corporation (OTC: RXII) is a biotechnology company focused on discovering, developing and commercializing innovative therapies based on its proprietary, next-generation RNAi platform. Therapeutics that use RNA interference, or “RNAi,” have great promise because of their ability to “silence,” or down-regulate, the expression of a specific gene that may be over-expressed in a disease condition. Building on the pioneering work of scientific founder and Nobel Laureate Dr. Craig Mello, a member of the RXi Scientific Advisory Board, RXi’s first RNAi product candidate, RXI-109, which targets CTGF (connective tissue growth factor), entered into a Phase 1 clinical trial in June 2012 to evaluate its safety, tolerability and potential efficacy for scar prevention. For more information, please visit www.rxipharma.com.

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 Pharmaceuticals Corporation’s products and technologies. These forward-looking statements about future expectations, plans and prospects of the development of RXi’s products and technologies involve significant risks, uncertainties and assumptions, including the risk that RXi may not be able to successfully develop its candidates, 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 development process for our product candidates may be delayed, 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 contemplated by these forward-looking statements. RXi 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.