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

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

<0> RXi Pharmaceuticals CorporationTamara McGrillen, 508-929-3646 </0>

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.

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 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.

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  and in animal tissues to allow sd-rxRNAs to achieve efficient spontaneous cellular uptake and potent, long-lasting intracellular activity.

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 .

    

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