Three ALS Organizations Grant Funding for Research Collaboration Between RXi Pharmaceuticals and UMass Medical School

Three Lou Gehrig's Disease (ALS) Organizations Grant Funding for Research Collaboration Between RXi Pharmaceuticals and UMass Medical School
WORCESTER, Mass.--(BUSINESS WIRE)--Nov. 30, 2009-- Three prominent philanthropic organizations - The Angel Fund, The ALS Therapy Alliance and Project ALS -- dedicated to finding a treatment for amyotrophic lateral sclerosis (ALS) have financed a new collaboration between one of the world's leading ALS researchers and RXi Pharmaceuticals Corporation (Nasdaq: RXII), a biopharmaceutical company pursuing the development and commercialization of proprietary therapeutics based on RNA interference (RNAi). Robert Brown, MD, DPhil, Chair of the Department of Neurology at the University of Massachusetts Medical School in Worcester (UMMS), will study the use of RXi's self-delivering rxRNATM (sd-rxRNATM) compounds as a potential treatment for ALS.

ALS is a progressive and fatal, neurodegenerative disorder affecting the motor neurons in the central nervous system. As motor neurons die, the brain's ability to send signals to the body's muscles is compromised. This leads to loss of voluntary muscle movement, paralysis and eventually death from respiratory failure. The cause of most cases of ALS is not known. Approximately 10 percent of cases are inherited. In 1993, a team of researchers led by Dr. Brown discovered the first gene linked to familial ALS, a protein anti-oxidant known as superoxide dismutase, or SOD1.

As part of this collaboration, RXi will provide its next generation RNAi technology and materials to Dr. Brown and his team of researchers, and Brown's laboratory will investigate the level of gene silencing and extended survival in a mouse model of familial ALS in which a human mutated version of the SOD1 gene is over-expressed.

Dr. Brown commented, "We have been searching for an RNAi treatment for ALS for many years and while this approach is very promising, the limiting factor critical to an effective therapeutic has been delivery. I am impressed with RXi's RNAi platform and believe that RXi's sd-rxRNAs are the unique solution that could be the key to treating neurological disorders such as ALS."

Noah D. Beerman, President and Chief Executive Officer of RXi, stated, "RXi is extremely excited about the collaboration with Dr. Brown and his choice to use the company's sd-rxRNAs to develop potential treatments for ALS. RXi has a long standing interest in ALS and we hope that this collaboration will enable the evaluation of the clinical potential of the sd-rxRNA technology platform for the treatment of ALS and other diseases of the central nervous system."

The Angel Fund was established in 1997 by Ginny Delvecchio, an ALS patient whose mother and brother died of the same illness. Its mission is to support research and scientific investigations in the fight against ALS at the Cecil B. Day Laboratory for Neuromuscular Research at the University of Massachusetts Medical School. The ALS Therapy Alliance was founded in 2000 to coordinate research for a diverse group of scientists and clinicians working on ALS. In 2002, the ALS Therapy Alliance invited CVS to join the fight against ALS by asking its customers to donate to the cause at the checkout counters. Six years later, that partnership is still thriving and has raised more than $11 million. Project ALS was founded in 1998 by Jenifer Estess and her family and friends, when Jenifer was diagnosed with the disease at the age of 35. To date, Project ALS has raised over $42 million, directing 81 percent to research programs.

The support from these three prominent charities for this research opens a new chapter in the investigation of gene silencing as a therapeutic intervention. UMass Medical School recently broke ground on a new laboratory research facility that will include the RNA Therapeutics Institute, part of Governor Deval Patrick's Massachusetts Life Sciences Initiative; the Institute is co-directed by Craig C. Mello, PhD, a Howard Hughes Medical Institute Investigator and the Blais University Professor of Molecular Medicine at the University of Massachusetts Medical School, who was a co-recipient of the 2006 Nobel Prize in Medicine for his discovery of "RNA interference" - gene silencing by double stranded RNA. Mello is also the Chairman of RXi's scientific advisory board.

About RNA Interference (RNAi) and sd-rxRNATM

Regarded as a revolutionary discovery in biology, RNA interference (RNAi) is a naturally occurring mechanism whereby short, double-stranded RNA molecules interfere with the expression of genes in living cells. This mechanism has the potential to be harnessed to "silence" or specifically block the production of disease-causing proteins before they are made. This technology can potentially be used to treat human diseases by "turning-off" genes that lead to disease in the first place. RXi Pharmaceuticals is using RNAi technology to develop RNA-derived molecules targeting disease-causing genes.

sd-rxRNA is a proprietary technology recently developed at RXi which enables the efficient delivery of RNAi compounds without the requirement of an additional delivery vehicle. This technology has an immediate clinical application for diseases where localized delivery is an option and also has the potential to be applied for indications requiring systemic delivery of RNAi.

About RXi Pharmaceuticals Corporation

RXi Pharmaceuticals is a discovery-stage biopharmaceutical company pursuing the development and potential commercialization of proprietary therapeutics based on RNA interference (RNAi) for the treatment of human diseases. RXi has a comprehensive therapeutic platform that includes both RNAi compounds and delivery methods. RXi uses its own version of RNAi compounds -- rxRNATM -- that provide an advanced alternative to conventional small interfering RNAs (siRNAs) and define the next generation of RNAi technology. rxRNA compounds are designed specifically for therapeutic use and contain many of the properties needed to move RNAi based drugs into the clinic. RXi Pharmaceuticals believes it is well positioned to compete successfully in the RNAi-based therapeutics market with its accomplished scientific advisors, including Dr. Craig Mello, recipient of the 2006 Nobel Prize for his co-discovery of RNAi; a management team that is experienced in developing RNAi products; and a strong early intellectual property position in RNAi chemistry and delivery.

About the University of Massachusetts Medical School

The University of Massachusetts Medical School (UMMS) attracts more than $200 million in research funding annually, and its innovative programs are the centerpiece of the Massachusetts Life Sciences Initiative. Consistently ranked by U.S.News & World Report as one of the leading medical schools in the nation for primary care education, UMMS comprises a medical school, graduate school of nursing, graduate school of biomedical sciences and an active research enterprise, and is a leader in health sciences education, research and public service. UMMS is the academic partner of UMass Memorial Health Care. For more information, visit

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 Pharmaceutical Corporation's products and technologies. These forward-looking statements about future expectations, plans and prospects of the development of RXi Pharmaceutical Corporation's products and technologies involve significant risks, uncertainties and assumptions, including 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 FDA approval process may be delayed for any drugs that we develop, 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 RXi Pharmaceuticals Corporation contemplated by these forward-looking statements. RXi Pharmaceuticals Corporation 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.


Source: RXi Pharmaceuticals Corporation

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