UMMS, RXi receive MLSC grant to develop RNAi therapeutics for ALS

RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biotechnology company focused on discovering, developing and commercializing innovative therapies addressing major unmet medical needs using RNA-targeted and immunotherapy technologies, today announced that the company's proposed research collaboration with the University of Massachusetts Medical School (UMMS) has been selected for Cooperative Research Matching Grant funding by the Massachusetts Life Sciences Center.

The Center awarded the grant to contribute funding to an ongoing collaboration between UMMS and RXi Pharmaceuticals to develop a new treatment for ALS (amyotrophic lateral sclerosis) using RXI's proprietary self-delivering RNAi therapeutic platform (sd-rxRNA™), a novel class of RNAi compounds that do not require a delivery vehicle to enter cells and has improved pharmacology compared to traditional RNAs. The work focuses on delivery to the spinal cord and brain to silence the SOD1 gene in ALS, and completion of the project is expected to generate supportive data for clinical development of anti-SOD1 sd-rxRNA therapy. ALS is currently incurable and almost 100% fatal within 5 years.

"This highly competitive grant underwent rigorous review by the Life Sciences Center's Scientific Advisory Board and recognizes the potential of RXi's technology to advance novel treatments for ALS, which is a devastating disease and unmet medical need," said Mark Ahn, Ph.D., President and CEO of RXi Pharmaceuticals. "The partnership with UMass Medical School comprises a unique combination of resources, talents and technologies that offers a promising path towards clinical development."

Bob Brown, MD, will be the Principal Investigator at UMMS leading this project. Dr. Brown is Professor and Chair of Neurology University of Massachusetts Medical School, and world leading expert in ALS.

The MLSC's Cooperative Research Grant Program funds collaborations among scientists, academic institutions and industry that promise significant commercial potential in the near term and are scientifically meritorious. The RXi Pharmaceuticals/UMMS collaboration was one of two projects funded. The MLSC grant amount is $250,000 per year for two years, and subject to a formalized agreement between the parties, will be matched dollar for dollar by RXi, totaling to up to $500,000 in funding for this project.

"This is the Life Sciences Center's second grant to support collaborative research between RXi and UMass Medical School, and we are pleased to be supporting this important research into new treatments for ALS," said Dr. Susan Windham-Bannister, President & CEO of the Center. "By supporting translational research, the Center seeks to accelerate the commercialization of science and technology, with the goal of strengthening the Massachusetts life sciences ecosystem through economic development and job creation," The Cooperative Research Matching Grant Program builds on the Center's strategy of using public investments to leverage private sector resources as we pursue our dual mission of job creation and support for good science that will improve the human condition."

"After rigorous review by me and other members of the Life Sciences Center's Scientific Advisory Board, we recommended two grants as holding great potential for both scientific advancement and commercialization," said Dr. Harvey Lodish, Chair of the Center's Scientific Advisory Board, Member of the Whitehead Institute for Biomedical Research, and Professor of Biology and Professor of Bioengineering at MIT. "Important advances in medicine can only be realized and treatments can only be developed and brought to market if the relevant research is funded. These are sensible investments for the Commonwealth that will both create jobs and advance scientific knowledge."