Research Update: SMT C1100 Receives Orphan Drug Status from the US FDA for the Treatment of DMD

 
TIDMSUMM 
 
Summit Corporation plc 
('Summit' or 'the Company') 
 
 
SMT C1100 RECEIVES ORPHAN DRUG STATUS FROM THE US FDA FOR THE TREATMENT OF 
DUCHENNE MUSCULAR DYSTROPHY 
 
Oxford, UK, 24 November 2011, Summit (AIM: SUMM), a UK drug discovery company, 
is pleased to announce that the United States Food &  Drug Administration ('the 
US FDA') has granted orphan drug status to the clinical candidate SMT C1100 for 
the treatment of Duchenne Muscular Dystrophy ('DMD'), a fatal genetic 
neuromuscular disorder for which there is currently no cure.  The European 
Medicines Agency had granted SMT C1100 orphan drug status in December 2008. 
 
Commenting on the news, Barry Price, PhD, Executive Chairman of Summit said: 
"We are pleased the US FDA has designated SMT C1100 as an orphan drug for DMD as 
this recognises its potential to treat a major unmet medical need.  Our clinical 
candidate has now been granted orphan drug status both in Europe and the US, and 
this status will provide additional regulatory support and various commercial 
benefits including extended periods of market exclusivity. 
 
"Summit believes that SMT C1100 can become a first-in-class treatment for DMD 
and it is our intention to commence a clinical study that will evaluate a new 
formulation of this drug that will benefit all patients with this fatal 
disease." 
 
About Orphan Drug Designation 
An orphan drug is a pharmaceutical agent that has been developed specifically to 
treat a rare medical condition, the condition itself being referred to as an 
orphan disease. The US Orphan Drug Act and European Orphan legislation are 
designed to assist and encourage companies in the development of urgently needed 
drugs for the treatment of rare diseases.  Orphan drug designation has a number 
of benefits including a valuable seven-year period of market exclusivity in the 
US following approval.  In Europe, a ten-year period of exclusivity is provided. 
 Orphan products can also expect to receive additional regulatory support, 
reduced fees and accelerated approval.  Further information about orphan drug 
designation can be found on the FDA's website,www.fda.com/oprhan. 
 
About DMD and SMT C1100 
DMD is caused by the absence of a protein called dystrophin which results in the 
degeneration of all skeletal and other vital muscles such as the heart.  There 
is no cure for DMD with the only available treatments being steroids that 
provide symptomatic relief. 
 
SMT C1100 is a small molecule drug that works by increasing (upregulating) 
production of a similar, naturally occurring protein called utrophin to replace 
the missing dystrophin.  Summit recently published compelling preclinical data 
showing SMT C1100 can increase utrophin production to restore and maintain 
healthy muscle function.  A major advantage of utrophin upregulation is it will 
benefit all patients with DMD regardless of their specific genetic mutation and 
is expected to be complementary with other therapeutic approaches currently in 
development. 
 
                                    - END - 
 
For more information, please contact: 
 
 Summit 
 Barry Price, PhD, 
 Richard Pye, PhD                       Tel: [phone] 
 
 
 Singer Capital Markets 
 (Nominated Adviser and Joint broker) 
 Shaun Dobson / Claes Spång             Tel: [phone] 
 
 
 Hybridan LLP 
 (Joint broker) 
 Claire Louise Noyce                    Tel: [phone] 
 
 
 Peckwater PR 
 (Financial public relations)           Tel: [phone] 
 Tarquin Edwards                        [e-mail] 
 
 
 
Notes to Editors 
 
About Summit 
Summit is an Oxford, UK based drug discovery company with an innovative 
technology platform called Seglins for the discovery of new medicines, a 
portfolio of drug programme assets and a commercial strategy of signing multiple 
early-stage deals. 
 
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 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 infection. 
 
Summit's commercial strategy focuses on signing multiple early-stage drug 
programme and technology platform deals that generate upfront cash, transfer 
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 atwww.summitplc.com. 
 
 
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Source: Summit Corporation PLC via Thomson Reuters ONE 
 
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