Summit Corporation plc
("Summit" or "the Company")
POSTIVE RESULTS IN DMD PROGRAMME:
* SMT C1100 increases utrophin in DMD patient muscle cells to therapeutically
Oxford, UK, 14 March 2011, Summit (AIM: SUMM), a UK drug discovery company,
today announces positive non-clinical efficacy results for SMT C1100, an orally
available drug that has the potential to be a disease modifying treatment for
the fatal disorder Duchenne muscular dystrophy ('DMD').
DMD is caused by the loss of a protein called dystrophin which results in the
degeneration of all skeletal muscles and causes damage to the heart. Currently
there is no cure for this disease. Scientists have previously shown that by
increasing (upregulating) production in the body of a similar, naturally
occurring protein called utrophin, it can compensate for the missing dystrophin
and can restore and maintain healthy muscle function.
Summit is pleased to report that new results from studies evaluating the effect
of SMT C1100 on dystrophin deficient muscle cells taken from DMD patients have
been positive. Dosing of these human myoblast cells with only low
concentrations of SMT C1100 resulted in increased utrophin protein levels, which
if translated into DMD patients, are anticipated to be of significant
The studies were conducted at Oxford University by Professor Dame Kay Davies
FRS, a world-leading expert who pioneered utrophin upregulation as a therapeutic
approach for DMD. Commenting on the results, Professor Davies said "The use of
utrophin protein to substitute for the missing dystrophin is an approach that
has the potential to benefit all DMD patients, regardless of their genetic
mutation. It is also expected to be complementary to the other therapeutic
approaches in development. These significant results provide further
encouragement about the potential of SMT C1100 as a utrophin upregulator drug."
A leading DMD clinician, Francesco Muntoni, Professor of Paediatric Neurology
and Head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child
Health in London added: "These results in DMD muscle cells demonstrate that SMT
C1100 can increase utrophin to levels, which if replicated in DMD patients,
would undoubtedly make it a disease modifying therapy."
The results will be presented at the Muscular Dystrophy Association's National
Scientific Conference being held in Las Vegas, US on March 13-16 2011.
Enhancing a compelling data package
These new data supplement a compelling data package generated from a range of
earlier studies, including those conducted in the 'gold standard' in vivo model.
These showed that SMT C1100:
* Increases the amount of utrophin and reduces muscle degeneration, fibrosis
and chronic inflammation.
* Significantly improves resistance to muscle fatigue in a forced exercise
model. This is a surrogate for the six minute distance walk test which is
the accepted primary efficacy end-point in human clinical trials.
In a healthy volunteer clinical trial, SMT C1100 was shown to be safe and well
tolerated with no adverse events reported. In addition, blood plasma levels of
SMT C1100 did exceed levels that are anticipated to produce a therapeutic effect
in some individuals, although there was variability in the results with lower
exposure levels reported for others. Summit believe alternative formulations of
SMT C1100 can produce consistently higher results and is seeking to evaluate
these in future clinical studies.
Commenting on the results, Dr Barry Price, Executive Chairman of Summit said:
"These results are important as they show treatment of DMD patient muscle cells
with SMT C1100 increases utrophin protein to levels that we anticipate will have
significant therapeutic benefit if replicated in patients. These data reinforce
Summit's belief about the potential of SMT C1100 as a first-in-class disease
modifying treatment for all DMD patients."
- END -
For more information, please contact:
Barry Price, PhD,
Richard Pye, PhD Tel: +44 (0)1235 443 939
Singer Capital Markets (Nominated Adviser)
Shaun Dobson / Claes Spång Tel: +44 (0)20 3205 7500
Tarquin Edwards Tel: +44 (0)7879 458 364
Notes to Editors
About Duchenne Muscular Dystrophy and Utrophin Upregulation
Duchenne muscular dystrophy is a fatal neuromuscular disorder that affects 1 in
3,500 boys with an estimated patient population of over 40,000 in the developed
DMD is caused by a genetic defect meaning DMD patients lack an important protein
called dystrophin, which is crucial to maintaining muscle integrity and
function. The absence of dystrophin results in extensive muscle wasting in all
voluntary muscles as well as the heart and breathing muscles and causes severe
restriction in the mobility of DMD patients by their early teens and is
ultimately fatal, generally in their twenties. Currently there is no cure for
DMD; corticosteroid treatment is the only frontline therapy and acts to only
delay the progression of the disease.
Summit has identified SMT C1100, a proprietary, orally available small molecule
with a novel mechanism of action for DMD. SMT C1100 acts to modify the
progression of DMD by replacing dystrophin with an endogenous, functionally
similar protein called utrophin. Summit believes the primary advantage of SMT
C1100 is that it offers the potential to treat the entire DMD patient
Due to the low patient population and high unmet medical need, DMD is designated
as an orphan indication by the regulatory agencies. Orphan products can expect
to receive additional regulatory support and accelerated approval in addition to
seven and ten years of market exclusivity in the US and EU respectively upon
designation by the FDA and the EMEA.
Summit is an Oxford, UK based drug discovery company with an innovative
technology platform called Seglins(TM) for the discovery of new medicines, a
portfolio of drug programme assets and a commercial strategy of signing multiple
Seglin(TM) technology is using new chemistry to access biological drug targets
that cannot be exploited by conventional drug discovery approaches. Summit's
internal research is currently focussed in the high-value therapy areas and the
Company will further exploit the technology's wider potential through strategic
alliances. Summit's programme portfolio consists of a number of drug programmes
targeting high-value areas of unmet medical need including Duchenne muscular
dystrophy and C. difficile infections.
Summit's commercial strategy focuses on signing multiple early-stage drug
programme and technology platform deals that generate upfront cash, remove
development costs from the Company, and retain valuable upside potential.
Summit is listed on the AIM market of the London Stock Exchange and trades under
the ticker symbol SUMM. Further information is available at www.summitplc.com.
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Source: Summit Corporation PLC via Thomson Reuters ONE