ALS research collaboration receives philanthropic funding

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 rxRNA™ (sd-rxRNA™) 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.”