Research Update

8 May 2007 07:01

VASTox plc08 May 2007 VASTox plc ("VASTox" or "the Company") VASTOX SELECTS PRECLINICAL CANDIDATE IN DUCHENNE MUSCULAR DYSTROPHY PROGRAMME Oxford, UK, 8 May 2007 - VASTox plc (AIM: VOX), a leading UK biotechnologycompany, announces that it has selected a candidate to enter into preclinicaldevelopment in the Company's Duchenne Muscular Dystrophy ("DMD") drug discoveryprogramme. VASTox has a unique therapeutic approach that targets the underlying cause ofthis disease. Owing to a genetic defect, DMD patients lack an important proteincalled dystrophin, which results in severe muscle wasting and is ultimatelyfatal for patients. VASTox is developing a small molecule treatment thatreplaces the missing dystrophin by increasing levels of a functionally similarprotein called utrophin. A significant benefit of VASTox's approach of 'up-regulating' utrophin is thatit should be effective in treating all DMD patients and, importantly, theCompany expects any drug it develops to be complementary with current palliativetreatments and the variety of other scientific approaches currently aimed atdiscovering effective DMD therapies. VASTox's preclinical candidate, called VOX C1100, was selected from a series ofpromising compounds after it significantly reduced levels of muscle degenerationduring in vivo studies. Other key secondary symptoms of DMD relating to thechronic inflammation and fibrosis of muscles also showed significant improvementin the same investigations. Higher levels of utrophin were identified intreated muscles with these data providing validation of VASTox's unique approachfor developing a treatment for DMD. The novel candidate, VOX C1100, has advantageous chemical and pharmaceuticalproperties suitable for a small molecule oral drug. Furthermore, it hassuccessfully completed preliminary safety and toxicity testing after beingscreened through VASTox's zebrafish chemical genomics platform. VASTox expectsto submit an Investigational New Drug ("IND") filing for VOX C1100 by mid 2008with the compound entering first-in-man Phase I clinical trials during thesecond half of 2008. Orphan drug designation has been granted for utrophin up-regulation as amechanism of treating DMD by the European Medicines Agency (EMEA) and thisstatus will provide VASTox with considerable support during drug development byaccelerating this process and reducing the associated costs. The Company hasalso built up a strong patent estate to protect the increasing value of thisdrug discovery programme with several patents, either granted or filed, coveringall of the World's major markets. Orphan disease indications are rare diseases with relatively small patientpopulations but are commercially attractive with current marketed orphan drugsgenerating average annual sales in excess of $500 million. VASTox is activelypursuing commercial partnering collaborations for the Company's increasinglyvaluable DMD preclinical programme. Richard Storer, DPhil, VASTox's Chief Scientific Officer, commented: "Theselection of this candidate for development is the culmination of over two yearsof dedicated efforts by our research teams. It is particularly gratifying thata compound initially identified as a result of in vitro utrophin up-regulationproduces the benefits observed in vivo and thereby supports our fundamentalscientific approach." Steven Lee, PhD, CEO of VASTox added "The selection of VOX C1100 represents theachievement of a major milestone for our DMD programme and illustrates theexcellent progress this programme has made over the past two years. With theprogression of DMD into preclinical development, VASTox now has five programmesacross a range of therapeutic areas that are either in clinical or preclinicaldevelopment and indicates the strength, depth and ever increasing value of ourdiscovery pipeline." - ends - For more information please contact: VASTox Steven Lee, PhD, Chief Executive Officer Tel: +44 (0)1235 443951Richard Storer, DPhil, Chief Scientific Officer Citigate Dewe Rogerson Mark Swallow / David Dible / Valerie Auffray Tel: +44 (0)207 638 9571 About VASTox's DMD programme DMD is a devastating disease that affects young males for which there iscurrently no effective treatment. Patients rarely survive beyond the age of 25years. VASTox has demonstrated in vivo up-regulation (increased production) of theprotein utrophin by a number of small molecules from their proprietary chemicallibrary. This is a significant development as utrophin has been demonstrated toreplace the function of dystrophin, which is missing in DMD patients.Up-regulation of utrophin is widely viewed by the scientific community as ahighly promising approach for the development of an effective treatment for DMD. About VASTox plc VASTox is a leading UK biotechnology company that discovers and developsproprietary new drugs. The Company's internal drug development programmes areunderpinned by its advanced chemistry and drug screening (chemical genomics)technology platforms, which it also provides on a collaborative orfee-for-service basis to the pharmaceutical industry. VASTox has a broad range of drug discovery programmes in the clinical,pre-clinical and discovery stages of development, which target serious diseaseswith a high unmet medical need. These therapeutic areas include neuro-disorders(neurodegenerative and neuromuscular), anti-infectives, ophthalmic diseases,oncology and regenerative medicines. VASTox's in-house drug development capabilities combine world-class expertise inboth medicinal and carbohydrate chemistry with high-volume, high-contentscreening using its proprietary zebrafish and fruitfly technologies (chemicalgenomics). These whole organism screens have the potential to dramaticallydecrease the time and cost of drug discovery and development by delivering datathat are highly predictive of the efficacy and toxicity of potential drugcompounds in humans. The company listed on the AIM market of the London Stock Exchange in October2004 - symbol: VOX Further information about the company is available at www.vastox.com This document contains "forward-looking statements" within the meaning of theU.S. Private Securities Litigation Reform Act of 1995. Forward-lookingstatements can be identified by words such as "anticipates", "intends", "plans","seeks", "believes", "estimates", "expects" and similar references to futureperiods, or by the inclusion of forecasts or projections. Forward-looking statements are based on the Company's current expectations andassumptions regarding our business, the economy and other future conditions.Because forward-looking statements relate to the future, by their nature, theyare subject to inherent uncertainties, risks and changes in circumstances thatare difficult to predict. The Company's actual results may differ materiallyfrom those contemplated by the forward-looking statements. The Company cautionsyou therefore that you should not rely on any of these forward-lookingstatements as statements of historical fact or as guarantees or assurances offuture performance. Important factors that could cause actual results to differmaterially from those in the forward-looking statements and regional, national,global political, economic, business, competitive, market and regulatoryconditions. This information is provided by RNS The company news service from the London Stock Exchange