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2016-02-29 13:35 CET
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Summit Therapeutics Supports Ninth Annual Rare Disease Day

Summit Therapeutics plc
("Summit" or "the Company")


Oxford, UK, 29 February 2016 - Summit Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') and Clostridium difficile infection, announces today its support for the ninth annual Rare Disease Day on 29 February 2016. The Rare Disease Day 2016 theme, Patient Voice, recognises the crucial role that patients play in voicing their needs and in initiating change that improves their lives and the lives of their families and carers.

"On this ninth annual Rare Disease Day, we at Summit take this opportunity to pay tribute to all patients and their families living with rare diseases," commented Glyn Edwards, Chief Executive Officer of Summit Therapeutics"The patient and parent voice is playing a vital role in the development of our utrophin modulator therapies that offer the potential to treat all boys and men living with the rare disease, DMD, and Summit has an unwavering commitment towards bringing hope for all those affected by this disease."

In the European Union a rare disease is defined as one that affects fewer than 5 in 10,000 of the general population while in the United States, it is defined as one that affects fewer than 200,000 people.  There are between 6,000 and 8,000 known rare diseases with around five new rare diseases described in the literature each week.  Rare diseases are often chronic and life threatening and include rare conditions such as childhood cancers and some other well-known conditions including cystic fibrosis and DMD.

Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness among the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was launched in Europe in 2008 by EURORDIS, the organisation representing rare disease patients in Europe. It is now observed in more than 80 nations, and is sponsored in the U.S. by the National Organization for Rare Disorders (NORD). For more information, please visit

About DMD and Utrophin Modulation
DMD is a progressive muscle wasting disease that affects around 50,000 boys and young men in the developed world. The disease is caused by different genetic faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscles. There is currently no cure for DMD and life expectancy is into the late twenties.

Utrophin protein is functionally and structurally similar to dystrophin. In preclinical studies, the continued expression of utrophin had a meaningful, positive effect on muscle performance. Summit believes that utrophin modulation has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin gene mutation. Summit also believes that utrophin modulation could potentially be complementary to other therapeutic approaches for DMD. The Company's lead utrophin modulator is an orally administered, small molecule called SMT C1100 that is about to enter a Phase 2 proof of concept clinical trial in patients with DMD. DMD is an orphan disease, and the US Food and Drug Administration and the European Medicines Agency have granted orphan drug status to SMT C1100. Orphan drugs receive a number of benefits including additional regulatory support and a period of market exclusivity following approval.

About Summit Therapeutics
Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at and Summit can be followed on Twitter (@summitplc).

For more information, please contact:

Glyn Edwards / Richard Pye (UK office)
Erik Ostrowski / Michelle Avery (US office)


Tel: +44 (0)1235 443 951
       +1 617 225 4455
Cairn Financial Advisers LLP
(Nominated Adviser)
Liam Murray / Tony Rawlinson



Tel: +44 (0)20 77148 7900
N+1 Singer
Aubrey Powell / Jen Boorer       



Tel: +44 (0)20 7496 3000
Peckwater PR
(Financial public relations, UK)
Tarquin Edwards


Tel: +44 (0)7879 458 364
MacDougall Biomedical Communications
(US media contact)
Chris Erdman

Tel: +1 781 235 3060

Forward-looking Statements
Any statements in this press release about Summit's future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit's product candidates, the therapeutic potential of Summit's product candidates, and the timing of initiation, completion and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit's foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit's Annual Report on Form 20-F for the fiscal year ended January 31, 2015.  Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit's views only as of the date of this release and should not be relied upon as representing Summit's views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.