OXFORD, UNITED KINGDOM--(Marketwire - May 25, 2012) - Summit (AIM: SUMM), a UK drug
discovery company,
today announced that it has dosed the first cohort of patients in a Phase 1
study of SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD),
a
fatal, rare genetic disease characterized by rapidly worsening muscle
weakness.
SMT C1100, an oral small molecule compound, is a potential
disease-modifying
drug that works to increase, or upregulate, the amount of a naturally
occurring
protein called utrophin.
"There is currently no known cure for DMD, and the only treatments
available
mask the symptoms of the disease," said Glyn Edwards, Chief Executive
Officer of
Summit. "SMT C1100 has the potential to modify the underlying disease, and
the
initiation of this Phase 1 trial represents a great step forward in
bringing our
breakthrough science to patients suffering from DMD. We expect to
report top-line data from the full trial before the end of this year."
SMT C1100 has been extensively evaluated in non-clinical efficacy and
safety
studies and has demonstrated its ability to restore and maintain the
function of
muscles. This Phase 1 dose-escalating clinical trial in healthy volunteers
will
evaluate if the Company's aqueous formulation of SMT C1100 can provide the
consistent levels of drug in blood that non-clinical efficacy studies
predicted
would be required to confer therapeutic benefit in DMD patients, while also
assessing its safety and tolerability. A successful outcome from this trial
is
expected to lead to a Phase 2 trial of SMT C1100 in DMD patients.
The Phase 1 trial is being supported by $1.5 million from a group of
US-based
DMD organisations: the Muscular Dystrophy Association, Charley's Fund, Cure
Duchenne, the Foundation to Eradicate Duchenne, Nash Avery Foundation and
Parent
Project Muscular Dystrophy.
SMT C1100 is designed to upregulate and maintain the production of
utrophin.
Utrophin is a protein that is highly expressed in regenerating muscle, but
decreases as the muscle fibre matures and is eventually replaced by
dystrophin,
a protein that maintains the integrity and healthy function of muscles.
Patients
with DMD are unable to make dystrophin, resulting in muscle fibre
degeneration.
However, if utrophin is continually expressed in the mature muscle fibre,
it can
replace the function of dystrophin and thereby overcome the deficit in
patients
with DMD. This approach is expected to be a universal treatment for all DMD
patients regardless of whether the disease was caused by an inherited or
spontaneous genetic mutation. Summit has demonstrated in non-clinical
efficacy
studies that SMT C1100 is capable of increasing utrophin to restore and
maintain
the healthy function of muscles.
About Summit
Summit is an Oxford, UK based drug discovery Company with an innovative
Seglin™ technology platform for the discovery of new medicines and a
portfolio of drug programme assets. 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 infection. 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.
Forward Looking Statements
This document contains "forward-looking statements" within the meaning of
the
U.S. Private Securities Litigation Reform Act of 1995. Forward-looking
statements can be identified by words such as "anticipates", "intends",
"plans",
"seeks", "believes", "estimates", "expects" and similar references to
future
periods, or by the inclusion of forecasts or projections. Forward-looking
statements are based on the Company's current expectations and assumptions
regarding our business, the economy and other future conditions. Because
forward-looking statements relate to the future, by their nature, they are
subject to inherent uncertainties, risks and changes in circumstances that
are
difficult to predict. The Company's actual results may differ materially
from
those contemplated by the forward-looking statements. The Company cautions
you
therefore that you should not rely on any of these forward-looking
statements as
statements of historical fact or as guarantees or assurances of future
performance. Important factors that could cause actual results to differ
materially from those in the forward-looking statements and regional,
national,
global political, economic, business, competitive, market and regulatory
conditions.
Notes to Editors
About DMD
Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder that
affects 1 in 3,500 boys with an estimated patient population of 50,000 in
the
developed world. The disease is caused by defects in the gene required to
make
dystrophin, a protein, which maintains the integrity and healthy function
of
muscles. One in three new cases is due to a spontaneous mutation where
there is
no familial history of the disease. The progressive muscle wasting begins
in
early childhood and typically leads to death in the twenties due to cardiac
and
respiratory failure. Currently there is no cure for the disease.
About Utrophin Upregulation
Utrophin is a naturally occurring protein that has a similar function to
dystrophin. Utrophin is produced during foetal muscle development but is
switched off in mature muscle fibres. If its production could be switched
back
on, utrophin could act as a substitute for the missing dystrophin to
maintain
the healthy function of muscles. One method of turning utrophin production
back
on is through pharmacological means. Utrophin upregulation will be
beneficial to
all DMD patients regardless of their specific genetic mutation and is also
expected to be complimentary to other therapeutic approaches in
development.
About SMT C1100
Discovered and developed by scientists at Summit, SMT C1100 has
demonstrated its
potential as a disease-modifying drug in non-clinical efficacy studies. SMT
C1100 disengages normal utrophin control such that utrophin RNA and protein
is
made continually in muscle. It has received orphan drug designation in the
US
and Europe.
About MDA Venture Philanthropy (MVP)
MVP is the Muscular Dystrophy Association's drug development program, which
operates within MDA's translational research program. MVP is exclusively
focused
on funding the discovery and clinical application of treatments and cures
for
neuromuscular diseases. For more information, visit mda.org and follow MDA
on
Facebook (facebook.com/MDANational) and Twitter (@MDAnews).
About Charley's Fund, Cure Duchenne, Foundation to Eradicate Duchenne,
and Nash Avery Foundation
Charley's Fund (www.charleysfund.org), Cure Duchenne
(www.cureduchenne.org),
Foundation to Eradicate Duchenne (www.duchennemd.org), and Nash Avery
Foundation
are independent organisations devoted to
developing treatments for Duchenne muscular dystrophy. These groups,
founded by
parents of children with Duchenne, support the most promising research.
About Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit
organization founded in 1994 by parents of children with Duchenne and
Becker
muscular dystrophy. Our mission is to end Duchenne. We accelerate research,
raise our voices in Washington, demand optimal care for all young men, and
educate the global community. PPMD is headquartered in Middletown, Ohio
with
offices in Fort Lee, New Jersey. For more information, visit
www.ParentProjectMD.org.
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Source: Summit Corporation PLC via Thomson Reuters ONE
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