VASTox [now Summit plc], a leading UK biotechnology company, has presented exciting progress in its spinal muscular atrophy (SMA) drug discovery programme. VASTox’s Head of Biology, Dr Jon Tinsley, presented the data at the Society for Neuroscience annual meeting, Neuroscience 2006, held in Atlanta, GA, USA, from the 14-18 October 2006.

The Company has discovered a number of promising ‘hits’ from a proprietary collection of drug-like molecules, which have been shown to improve the symptoms of SMA in an in vivo fruitfly (Drosophila melanogaster) screen designed to model the disease. The speed with which these hit molecules were identified by screening directly in a genetically-modified fruitfly is an important validation of VASTox’s innovative approach towards drug discovery. This progress will allow the Company to rapidly advance the SMA programme into the lead optimisation phase of pre-clinical development early in 2007, only 18 months after the programme was initiated.

SMA is a severe genetic neurological disease that causes a progressive loss of motor neurons in the spinal cord leading to severe muscle atrophy. SMA patients either do not acquire or eventually lose the ability to move and death occurs primarily as a result of fatal respiratory insufficiency.

SMA is the leading genetic cause of mortality in infants and toddlers in the World. It affects 1 in 6,000 newborns, an incidence comparable to that of other ‘common’ rare diseases, including Cystic Fibrosis, Duchenne Muscular Dystrophy and Sickle Cell Anaemia. There are an estimated 50,000 SMA sufferers in the developed World.

Steven Lee, PhD, CEO of VASTox said: ”These results from our spinal muscular atrophy programme illustrate the ‘in vivo advantage’ of VASTox’s approach to drug discovery. By using fruitflies and zebrafish at the earliest stages of drug discovery, we are dramatically reducing the time and resources needed and we believe, significantly increasing the chances of producing a drug which is safe in man. SMA is a deadly genetic disease affecting children and our approach towards developing a therapy is both innovative and unparalleled.”
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