08 May 2007
('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 biotechnology
company, announces that it has selected a candidate to enter into preclinical
development in the Company's Duchenne Muscular Dystrophy ('DMD') drug discovery
VASTox has a unique therapeutic approach that targets the underlying cause of
this disease. Owing to a genetic defect, DMD patients lack an important protein
called dystrophin, which results in severe muscle wasting and is ultimately
fatal for patients. VASTox is developing a small molecule treatment that
replaces the missing dystrophin by increasing levels of a functionally similar
protein called utrophin.
A significant benefit of VASTox's approach of 'up-regulating' utrophin is that
it should be effective in treating all DMD patients and, importantly, the
Company expects any drug it develops to be complementary with current palliative
treatments and the variety of other scientific approaches currently aimed at
discovering effective DMD therapies.
VASTox's preclinical candidate, called VOX C1100, was selected from a series of
promising compounds after it significantly reduced levels of muscle degeneration
during in vivo studies. Other key secondary symptoms of DMD relating to the
chronic inflammation and fibrosis of muscles also showed significant improvement
in the same investigations. Higher levels of utrophin were identified in
treated muscles with these data providing validation of VASTox's unique approach
for developing a treatment for DMD.
The novel candidate, VOX C1100, has advantageous chemical and pharmaceutical
properties suitable for a small molecule oral drug. Furthermore, it has
successfully completed preliminary safety and toxicity testing after being
screened through VASTox's zebrafish chemical genomics platform. VASTox expects
to submit an Investigational New Drug ('IND') filing for VOX C1100 by mid 2008
with the compound entering first-in-man Phase I clinical trials during the
second half of 2008.
Orphan drug designation has been granted for utrophin up-regulation as a
mechanism of treating DMD by the European Medicines Agency (EMEA) and this
status will provide VASTox with considerable support during drug development by
accelerating this process and reducing the associated costs. The Company has
also built up a strong patent estate to protect the increasing value of this
drug discovery programme with several patents, either granted or filed, covering
all of the World's major markets.
Orphan disease indications are rare diseases with relatively small patient
populations but are commercially attractive with current marketed orphan drugs
generating average annual sales in excess of $500 million. VASTox is actively
pursuing commercial partnering collaborations for the Company's increasingly
valuable DMD preclinical programme.
Richard Storer, DPhil, VASTox's Chief Scientific Officer, commented: 'The
selection of this candidate for development is the culmination of over two years
of dedicated efforts by our research teams. It is particularly gratifying that
a compound initially identified as a result of in vitro utrophin up-regulation
produces the benefits observed in vivo and thereby supports our fundamental
Steven Lee, PhD, CEO of VASTox added 'The selection of VOX C1100 represents the
achievement of a major milestone for our DMD programme and illustrates the
excellent progress this programme has made over the past two years. With the
progression of DMD into preclinical development, VASTox now has five programmes
across a range of therapeutic areas that are either in clinical or preclinical
development and indicates the strength, depth and ever increasing value of our
- ends -
For more information please contact:
Steven Lee, PhD, Chief Executive Officer Tel: +44 (0)1235 443951
Richard 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 is
currently no effective treatment. Patients rarely survive beyond the age of 25
VASTox has demonstrated in vivo up-regulation (increased production) of the
protein utrophin by a number of small molecules from their proprietary chemical
library. This is a significant development as utrophin has been demonstrated to
replace the function of dystrophin, which is missing in DMD patients.
Up-regulation of utrophin is widely viewed by the scientific community as a
highly promising approach for the development of an effective treatment for DMD.
About VASTox plc
VASTox is a leading UK biotechnology company that discovers and develops
proprietary new drugs. The Company's internal drug development programmes are
underpinned by its advanced chemistry and drug screening (chemical genomics)
technology platforms, which it also provides on a collaborative or
fee-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 diseases
with 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 in
both medicinal and carbohydrate chemistry with high-volume, high-content
screening using its proprietary zebrafish and fruitfly technologies (chemical
genomics). These whole organism screens have the potential to dramatically
decrease the time and cost of drug discovery and development by delivering data
that are highly predictive of the efficacy and toxicity of potential drug
compounds in humans.
The company listed on the AIM market of the London Stock Exchange in October
2004 - symbol: VOX
Further information about the company is available at www.vastox.com
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