Summit Therapeutics Supports Ninth Annual Rare Disease Day

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 www.rarediseaseday.org .

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 www.summitplc.com and Summit can be followed on Twitter (@summitplc).

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