RXi Pharmaceuticals Granted US Notice of Allowance Covering Self-Delivering (sd-rxRNA) Technology Platform

WESTBOROUGH, Mass., May 22, 2014 /PRNewswire/ -- RXi Pharmaceuticals Corporation (NASDAQ: 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 received the Notice of Allowance from the United States Patent and Trademark Office on its novel, self-delivering RNAi platform (sd‑rxRNA®). The patent, titled "Reduced Size Self-Delivering RNAi Compounds", broadly covers both the composition and methods of use of RXi's self-delivering platform technology.  The patent, once issued, will be scheduled to expire in 2029.

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This core patent, within RXi's intellectual property portfolio, is the foundation for RXI's self-delivering platform. The patent broadly protects RXi's sd‑rxRNA technology, which facilitates the delivery of RNAi compounds to tissues and cells of interest in the absence of a delivery vehicle. sd‑rxRNA oligonucleotides 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. This allows sd‑rxRNAs to achieve efficient cellular uptake and potent, long-lasting intracellular activity. 

"We are pleased to have our novel technology recognized by the United States Patent and Trademark Office," said Dr. Geert Cauwenbergh, President and CEO of RXi Pharmaceuticals. He added that, "This key patent is what differentiates RXi's technology platform from the competition. It provides us with a powerful advantage to deliver our RNAi compounds without delivery vehicles, which are commonly used with the other siRNA compounds in clinical development. The use of our proprietary sd‑rxRNA technology platform may lead to improved tolerability and thereby potentially broadening of the therapeutic window for these compounds."

About RXi's Clinical Trials

RXi Pharmaceuticals' first clinical program involves RXI‑109, an sd‑rxRNA compound, developed for the reduction of dermal scarring. RXI‑109 is designed to reduce the expression of connective tissue growth factor (CTGF), a critical regulator of biological pathways involved in fibrosis, including scar formation in the skin. The first clinical trials with RXI‑109 showed excellent safety and tolerability with ascending single and multiple doses, as well as dose dependent effects on the CTGF protein and on the mRNA that controls production of this protein. 

Late last year the Company started its first Phase 2a study (RXI-109-1301) in patients who had pre-existing hypertrophic scars present on their lower abdomen. In this study, patients undergo scar revision surgery, after which they are treated with RXI-109 on one end of the scar and placebo on the opposite end of the scar. Earlier this year, the Company announced the initiation of a second Phase 2a study (RXI-109-1401) for RXI-109 treatment to prevent recurrence of keloids in patients undergoing keloidectomy (removal of keloid). In this study, two keloids of similar size and location are selected for keloidectomy, and after removal, one site is treated with RXI-109 and one with placebo for a direct in-patient comparison.

About RXi Pharmaceuticals Corporation
RXi Pharmaceuticals Corporation (NASDAQ: RXII) is a biotechnology company focused on discovering, developing and commercializing innovative therapies based on its proprietary, self-delivering RNAi platform. Therapeutics that use RNA interference, or "RNAi," have great  promise because of their ability to down-regulate the expression of specific genes that may be over-expressed in disease conditions. Building on the pioneering work of scientific founder and Nobel Laureate Dr. Craig Mello, a member of the Company's Scientific Advisory Board, RXi's first RNAi product candidate, RXI‑109, entered into human clinical trials in June 2012 and is currently in Phase 2. RXI-109 targets connective tissue growth factor (CTGF) to reduce dermal scarring (fibrosis).  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, planned and future development of RXi Pharmaceuticals Corporation's products and technologies. Forward-looking statements about expectations and development plans of RXi's products involve significant risks and uncertainties:  the risk that we may not be able to successfully develop our candidates, or that development of RNAi-based therapeutics may be delayed or not proceed as planned, or that we may not develop any RNAi-based products; risks that the development process for our product candidates may be delayed, risks related to the development and commercialization of products by our competitors, the risk 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 preventing us from developing our products. Actual results may differ from those contemplated by these forward-looking statements. RXi does not undertake to update forward-looking statements to reflect a change in its views, events or circumstances that occur after the date of this release.

Contacts
RXi Pharmaceuticals Corporation
Tamara McGrillen, 508-929-3646
tmcgrillen@rxipharma.com

SOURCE RXi Pharmaceuticals Corporation



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