RXi's dermal anti-scarring candidate selected to advance into development

RXi Pharmaceuticals Corporation, a recognized leader in RNAi-based therapeutic discovery and development, today announced the selection of its first therapeutic product candidate to advance into development.

“Dermal anti-scarring is an excellent match for RXi's expertise and our novel RNAi therapeutic platform”

The development candidate, RXI-109, is a self-delivering RNAi compound (sd-rxRNA™) that will initially be evaluated for the reduction of dermal scarring in planned surgeries. The selected lead candidate and several backups silence a gene target known to modulate fibrosis and thus may be applicable in the treatment of numerous indications with a fibrotic component, including scar formation. sd-rxRNA compounds are RNAi-based therapeutics that have demonstrated efficient cellular uptake without a delivery vehicle in preclinical studies.

"Dermal anti-scarring is an excellent match for RXi's expertise and our novel RNAi therapeutic platform," said Anastasia Khvorova, Ph.D., Chief Scientific Officer at RXi. "The disease biology is well understood, the targets are well validated, and intradermal injection is an acceptable administration route. Preclinical results using intradermal injection of sd-rxRNAs demonstrate robust, dose dependent, long lasting target specific silencing. This is a very exciting time for the company, and we look forward to advancing this program and filing an IND in the second half of 2011."

Dermal anti-scarring is an attractive lead indication for RXi with a clear development precedent and the ability to employ innovative clinical trial design with early efficacy endpoints. This indication represents an area of significant unmet medical need, with no approved drugs. The generation of early clinical proof of concept data in anti-scarring may support further expansion into other indications which contain a fibrotic component. Secondary clinical applications may include pulmonary fibrosis, liver fibrosis, acute spinal cord injury, ocular scarring and restenosis, which together encompass a potential market size of more than $16 billion.

"Selecting this first development candidate is a significant milestone for RXi and demonstrates the company's commitment to achieving our corporate goals and executing on our strategy," said Noah D. Beerman, President and Chief Executive Officer of RXi. "In early June, we designated dermal anti-scarring and retinal disorders as core focus areas and throughout 2010 we have made substantial progress in transitioning RXi from a research-focused company to one that is leveraging our strategy, platform and collaborations to advance our therapeutic pipeline. In addition to accomplishing our goal of nominating RXI-109 as our first development candidate, our partnering efforts also remain a priority for the company, and we are focused on completing a corporate partnership, which we anticipate being able to execute on in the coming months."