Oxford, UK, 24 April 2012, Summit (AIM: SUMM), a UK drug discovery company, today announces
that its Phase I Clinical Trial Application (‘CTA’) for SMT C1100 has been approved by the UK
regulatory body, the Medicines and Healthcare products Regulatory Agency (‘MHRA’). SMT C1100 is
a small molecule utrophin upregulator that has the potential to be a universal treatment for the fatal
genetic disease Duchenne Muscular Dystrophy (‘DMD’).
The approval of the CTA achieves a milestone as part of the $1.5 million agreement signed between
Summit and a group of US-based DMD organisations in December 2011 and triggers a payment of
$437,500. The Phase I trial is funded by the Muscular Dystrophy Association, Parent Project
Muscular Dystrophy, Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne and the
Nash Avery Foundation.
The double-blind, placebo-controlled Phase I study will be conducted in 48 healthy male volunteers
and will evaluate a new aqueous oral formulation of SMT C1100. The study will examine both the
safety and tolerability of the new formulation, and whether it can provide consistent levels of SMT
C1100 in the blood that non-clinical efficacy studies predict would be required to have a therapeutic
benefit in DMD patients. The formulation has been developed so it will be appropriate for use by all
Results from the trial are expected by the end of 2012 and a successful outcome from the Phase I
trial is expected to lead to evaluation of SMT C1100 in DMD patients. A drug like SMT C1100 that
could treat all DMD patients has the potential to generate annual sales in excess of $1 billion.
"We're extremely pleased that SMT C1100 will now move from testing in vivo to testing in healthy
humans" said Jane Larkindale, MDA's Director of Translational Research. "We have good
evidence from laboratory studies that this drug's mechanism of action is valid for slowing the
progression of this degenerative muscle disease, and we hope that this new trial will show that it can
be effectively and safely delivered to humans."
Glyn Edwards, Chief Executive Officer of Summit added: “Our plans to commence a new Phase I
trial for SMT C1100 continue to progress well with the necessary regulatory approval now in place.
This study represents an important development milestone for this programme with a positive
outcome having the potential to add considerable value to this asset, and bring an urgently needed
treatment for this fatal disease a step closer.”
For more information, please contact:
Glyn Edwards / Richard Pye
Tel: +44 (0)1235 443 951
Singer Capital Markets
(Nominated Adviser and Joint broker)
Shaun Dobson / Claes Spång
Tel: +44 (0)203 205 7500
Claire Louise Noyce / Deepak Reddy
Tel: +44 (0)207 947 4350
(Financial public relations)
Tel: +44 (0)7879 458 364
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 the
lack of a gene required to make dystrophin, a protein, which maintains the integrity and healthy
function of muscles. One in three new cases are 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
About Utrophin Upregulation
Utrophin is a naturally occurring protein that has a similar function to dystrophin. Utrophin is produced
during foetal development but is switched off in adults. 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 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.
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
(www.nashaveryfoundation.org) 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.
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.
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