ABINGDON, UNITED KINGDOM--(Marketwire -06/12/12)-
Summit Corporation plc('Summit' or 'the Company')
SUMMIT ACHIEVES SMT C1100 PHASE 1 DOSING MILESTONE THAT TRIGGERS MILESTONEPAYMENT
* Trial moves to Multiple Dose stage * Summit to Participate in Upcoming Industry Events
Summit (SUMM.L), a UK drug discovery company,today announced that it has successfully passed a milestone in the Phase 1trialof SMT C1100 for the treatment of the fatal genetic disease DuchenneMuscularDystrophy ('DMD'), which triggered the final payment from a $1.5 millionfundingagreement with US-based DMD organisations.
SMT C1100, an oral small molecule compound, is a potentialdisease-modifyingdrug that works by increasing, or upregulating, the amount of a naturallyoccurring protein called utrophin. The Phase 1 dose-escalation study inhealthyvolunteers was initiated in May 2012 and will now progress to the stagewhereparticipants receive multiple doses. Results from the trial are expectedby theend of this year.
The Phase 1 trial is being supported by a group of US-based DMDorganisations:the Muscular Dystrophy Association, Charley's Fund, Cure Duchenne, theFoundation to Eradicate Duchenne, Nash Avery Foundation and Parent ProjectMuscular Dystrophy.
"We are grateful for the continuing support from the DMD organisations aswemake significant progress in the Phase 1 trial of SMT C1100," said GlynEdwards,Chief Executive Officer of Summit. "The funding will enable completion ofthePhase 1 trial this year, after which we will seek an appropriate partner toadvance SMT C1100 through proof-of-concept studies to ultimately bring thispotential breakthrough therapy to patients with DMD."
The Company will be available for partnering discussions at the BIOInternational Convention June 18-21, 2012, in Boston, MA. In addition,Summitwill present at the Parent Project Muscular Dystrophy Annual ConnectConferenceJune 28-July 1, 2012, in Fort Lauderdale, FL.
SMT C1100 is designed to upregulate and maintain the production ofutrophin. Utrophin is a protein that is highly expressed in regenerating muscle, butdecreases as the muscle fibre matures and is eventually replaced bydystrophin,a protein that maintains the integrity and healthy function of muscles. Patients with DMD are unable to make dystrophin, resulting in muscle fibredegeneration. However, if utrophin is continually expressed in the maturemuscle fibre, it can replace the function of dystrophin and therebyovercome thedeficit in patients with DMD.
This approach is expected to be a universal treatment for all DMD patientsregardless of whether the disease was caused by an inherited or spontaneousgenetic mutation. Summit has demonstrated in non-clinical efficacy studiesthatSMT C1100 is capable of increasing utrophin to restore and maintain thehealthyfunction of muscles.
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For more information, please contact: Summit Glyn Edwards / Richard Pye Tel: +44 (0)1235 443 939 Singer Capital Markets (Nominated Adviser and Joint broker) Shaun Dobson / Claes Spång Tel: +44 (0)203 205 7500 Hybridan LLP (Joint broker) Claire Louise Noyce / Deepak Reddy Tel: +44 (0)207 947 4350 Peckwater PR (Financial public relations, UK) Tel: +44 (0)7879 458 364 Tarquin Edwards firstname.lastname@example.org MacDougall Biomedical Communications (U.S. media contact) Tel: +1 781-235-3060 Michelle Avery
Notes to Editors
Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder thataffects 1 in 3,500 boys with an estimated patient population of 50,000 inthedeveloped world. The disease is caused by defects in the gene required tomakedystrophin, a protein, which maintains the integrity and healthy functionofmuscles. One in three new cases are due to a spontaneous mutation wherethereis no familial history of the disease. The progressive muscle wastingbegins inearly childhood and typically leads to death in the twenties due to cardiacandrespiratory failure. Currently there is no cure for the disease.
About Utrophin Upregulation
Utrophin is a naturally occurring protein that has a similar function todystrophin. Utrophin is produced during foetal muscle development but isswitched off in mature muscle fibres. If its production could be switchedbackon, utrophin could act as a substitute for the missing dystrophin tomaintainthe healthy function of muscles. One method of turning utrophin productionbackon is through pharmacological means. Utrophin upregulation will bebeneficialto all DMD patients regardless of their specific genetic mutation and isalsoexpected to be complimentary to other therapeutic approaches indevelopment.
About SMT C1100
Discovered and developed by scientists at Summit, SMT C1100 hasdemonstrated itspotential as a disease-modifying drug in non-clinical efficacy studies.SMTC1100 disengages normal utrophin control such that utrophin RNA and proteinismade continually in muscle. It has received orphan drug designation in theUSand Europe.
About MDA Venture Philanthropy (MVP)
MVP is the Muscular Dystrophy Association's drug development program, whichoperates within MDA's translational research program. MVP is exclusivelyfocused on funding the discovery and clinical application of treatments andcures for neuromuscular diseases. For more information, visit mda.org andfollow 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 AveryFoundation(www.nashaveryfoundation.org) are independent organisations devoted todeveloping treatments for Duchenne muscular dystrophy. These groups,founded byparents of children with Duchenne, support the most promising research.
About Parent Project Muscular Dystrophy
Parent Project Muscular Dystrophy (PPMD) is a national not-for-profitorganization founded in 1994 by parents of children with Duchenne andBeckermuscular dystrophy. Our mission is to end Duchenne. We accelerateresearch,raise our voices in Washington, demand optimal care for all young men, andeducate the global community. PPMD is headquartered in Middletown, Ohiowithoffices in Fort Lee, New Jersey. For more information, visitwww.ParentProjectMD.org.
Summit is an Oxford, UK based drug discovery Company with an innovativeSeglin™ technology platform for the discovery of new medicines and aportfolio of drug programme assets. Summit's programme portfolio consistsof anumber of drug programmes targeting high-value areas of unmet medical needincluding Duchenne Muscular Dystrophy and C. difficile infection. Summitislisted on the AIM market of the London Stock Exchange and trades under theticker symbol SUMM. Further information is available at www.summitplc.com.
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Source: Summit Corporation PLC via Thomson Reuters ONE[HUG#1618978]
- Duchenne Muscular Dystrophy
- Muscular Dystrophy Association
Glyn Edwards / Richard Pye
Tel: +44 (0)1235 443 939