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Pharma Capital
Market: AIM
52-week High/Low: 245.00p / 30.06p
Sector: Pharma & Biotech
Market Cap: 28.33M

We are seeking to treat all boys and young men afflicted with the fatal disease Duchenne muscular dystrophy with our pioneering utrophin modulation technology.

We are also advancing a highly selective novel antibiotic for C. difficile infection.

Summit Therapeutics PLC

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Big picture - Why invest in Summit Therapeutics PLC

Summit Therapeutics PLC Snapshot

Summit Therapeutics is an international biopharmaceutical company focussed on the discovery and development of novel medicines to treat the fatal muscle wasting disease Duchenne muscular dystrophy and infections caused by the bacteria C. difficile.

Our goal is to become a fully integrated biopharmaceutical company focused on the discovery, development and commercialization of novel medicines for indications for which there are no existing or only inadequate therapies.


We are focused on developing innovative medicines to treat areas of high unmet medical need.

Our focus is on two therapy areas: the fatal muscle wasting disease Duchenne Muscular Dystrophy and infections caused by the superbug C. difficile.


Duchenne Muscular Dystrophy



Duchenne Muscular Dystrophy, or DMD, is one of the most common, fatal genetic disorders diagnosed in children around the world.  DMD is an X-linked disease, meaning that it predominantly affects males, and it results in the progressive wasting of muscles throughout the body.  The disease has an estimated incidence of 1 in 5,000 and a patient population in the developed world of approximately 50,000.  Approximately two thirds of new DMD cases are due to inherited mutations with the remaining one third resulting from spontaneous mutations where there is no family history of the disease.

DMD is caused by different mutations in the dystrophin gene that result in DMD patients being unable to produce dystrophin, a protein essential for maintaining healthy muscle function.  The absence of dystrophin results over time in the deterioration of muscles and leads to the loss of ambulation, loss of respiratory and cardiac function and ultimately death, typically by the time DMD patients reach their late twenties.  There is currently no approved therapy for the treatment of DMD applicable to all DMD patients that seeks to alter the progression of the disease.


Our utrophin modulation programme is developing oral, small molecule drugs that increase the production of a protein call utrophin.  Utrophin is a naturally occurring protein that is functionally and structurally similar to dystrophin.  Utrophin is produced during the early stages of muscle fibre development but is switched-off in maturing muscle fibres, at which point dystrophin is produced to perform the same functional role.  When a muscle fibre is damaged, utrophin is also produced during the early stages of the repair mechanism.

Our utrophin modulation approach aims to use small molecule drugs to maintain the production of utrophin to compensate for the absence of dystrophin in DMD patients and so protect healthy muscle function.  A significant advantage of utrophin modulation is that it is independent of the underlying genetic fault and therefore has the potential to treat 100% of DMD patients.  We also believe it could be complementary to other DMD treatment approaches.

The concept of utilising utrophin as a treatment for DMD was developed by our co-founder and scientific advisor Professor Kay Davies at the University of Oxford.


Summit is a leader in the field of utrophin modulation.  Our most advanced utrophin modulator, SMT C1100, is progressing through patient clinical trials. In August 2015 we announced that the Phase 1b modified diet trial of SMT C1100 achieved its primary objective with half of the patients with DMD achieving the desired plasma levels of SMT C1100 (click to read top-line results press release).  The trial also showed SMT C1100 to be safe and well tolerated at all doses tested.  With these results, we are now advancing SMT C1100 into Phase 2 trials and anticipate launching a Phase 2 open label trial before the end of 2015.

In parallel, we are also applying our know-how to develop a pipeline of second generation and future generation utrophin modulators as we seek to maintain and expand our leadership position in the field of utrophin modulation.  This research is being undertaken in collaboration with the University of Oxford.

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 a period of market exclusivity following approval and additional regulatory support.


C. difficile Infection


CDI is an infection of the colon caused by the bacteria Clostridium difficile that produces toxins that cause inflammation and severe diarrhoea. CDI can also result in serious disease complications including bowel perforation, toxic megacolon and sepsis, and it can prove fatal in the most severe cases.  CDI is a serious issue in North America and Europe with estimates of up to 700,000 cases of CDI per year in the US alone.  The US Center for Disease Control and Prevention report that CDI is responsible for 14,000 death per annum in the US and has designated C. difficile as one of three pathogens that poses an immediate public health threat and requires urgent and aggressive action.  In the US, it is estimated that CDI-related acute care costs total $4.8 billion per year.

CDI typically develops following the use of broad spectrum antibiotics that can cause widespread damage to the natural gut flora to allow overgrowth of C. difficile bacteria.  The current standard of care CDI treatments are the broad spectrum antibiotics vancomycin and metronidazole.  While effective at reducing levels of C. difficile, these antibiotics  also cause significant collateral damage to the gut flora as a result of their broad spectrum activity and leave patients vulnerable to disease recurrence, the primary clinical issue.  Each additional episode of the disease is associated with greater disease severity and higher mortality rates.  It has been reported that approximately 25% of CDI patients suffer a second episode of the infection, and the risk of further recurrence rises to 65% after a patient suffers a second episode of CDI.  Recurrent disease is associated with an increased burden on the healthcare system.


SMT19969 is our novel antibiotic for the treatment of CDI SMT19969.  In preclinical studies and clinical trials conducted to date, SMT19969 has demonstrated potent bactericidal activity for C. difficile with a minimal antibiotic effect against the bacterial groups that comprise the natural gut flora.  We believe that this profile means SMT19969 has the potential to selectively target C. difficile without causing collateral damage to the natural gut flora and thereby reduce CDI recurrence rates.

We have completed a Phase 1 clinical trial of SMT19969 in healthy volunteers.  In this trial, SMT19969 was well tolerated at all doses tested.  The results from this trial were also consistent with SMT19969’s highly selective profile, as SMT19969 had a minimal antibiotic effect on the natural gut flora other thantotal clostridia.

We have completed enrolment into a Phase 2 placebo controlled proof of concept clinical trial that is evaluating SMT19969 against vancomycin, the current standard of care.  This trial is being conducted in the US and Canada and we expect to report top-line data in the fourth quarter of 2015.

The US Food and Drugs Administration, or FDA, has designated SMT19969 as a Qualified Infectious Disease Product or QIDP.  The QIDP incentive are provided through the US GAIN Act and include eligibility for fast-track status, priority review and an extension of marketing exclusivity for an additional five years upon FDA approval.

The development of SMT19969 is being supported by the Wellcome Trust following the award to Summit of a prestigious £2.3 million Seeding Drug Discovery Award and a Translational Research Award worth up to £4.0 million..


For information on UltroDMD Alliance please click here


Dr Frank Armstrong (aged 57) was appointed a Non-Executive Director of Summit in November 2012.  Dr Armstrong has held Chief Executive roles with five biotechnology companies (public and private) one of which was Fulcrum Pharma, an AIM-listed Professional Services Company that was sold to Private Equity Investors in 2009. Most recently, Dr Armstrong led Medical Research and Innovation (MSI) at Merck Serono and previously was Head of Worldwide Product Development at Bayer and Senior Vice President of Medical Research and Communications Group at Zeneca.  Dr. Armstrong is currently the Chairman of Xceleron and Cardiorentis, and is a Non-Executive Director of Actino Pharma, Columbia Laboratores Inc. and Entelos.  Dr Armstrong is a Member of the Scientific Advisory Board of Healthcare Royalty Partners and adviser to Phase 4 Partners. Dr Armstrong is a Fellow of the Royal College of Physicians.


Mr Edwards (aged 58) was appointed to the Board of Directors as Chief Executive Officer in April 2012.  Mr Edwards has a wealth of experience garnered from a thirty-year career in the life sciences industry, during which time he has held a number of senior executive and business development roles.  Prior to joining Summit, he was interim Chief Executive Officer of the UK trade body the BioIndustry Association (BIA) and Chief Executive Officer at Antisoma plc for 13 years, and Vice President of Business Development at Therapeutic Antibodies Ltd.  Mr Edwards holds a BSc in Biochemistry from Bristol University and an MSc in Economics from the London Business School.


Dr Price (aged 70) has a wealth of industry and board-level expertise in the pharmaceutical and life sciences industry. He spent 28 years with the Glaxo Group of companies and held several executive positions including Managing Director of Glaxochem Ltd. Dr Price was a Non-Executive Director of Shire plc and during his 13 years with the company, he was involved in Shire developing into one of the UK's largest and most successful life science companies. Dr Price was appointed Non-Executive Chairman of Summit in 2006 and has previously held directorships at Chiroscience plc, Celltech Group plc, Pharmagene plc, Antisoma plc and BioWisdom Ltd.


Professor Davies (aged 64) is a distinguished academic who has been professor at the University of Oxford for over 20 years and was elected to the Waynflete Chair of Chemistry in 2006, one of the most prestigious academic posts in UK science. His areas of expertise include medicinal and asymmetric chemistry and he has received numerous awards for his contribution to chemistry. A serial entrepreneur, Professor Davies is a co-founder of Summit and also founded a number of other spin-out companies. These included Oxford Asymmetry and Oxford Diversity which would later combine for the IPO of Oxford Asymmetry International. This subsequently merged with Evotec for £316 million. Professor Davies has held a number of board positions and he currently holds directorships with OxStem Limited, Isis Innovation Limited and Sci-Ink Limited.


Mr Leopoldo Zambeletti (aged 45), a highly respected figure within the life sciences sector, joined the board of directors in June 2014.  During a 19 year career as an investment banker, he led the European Healthcare Investment team at JP Morgan for eight years before taking up the same position at Credit Suisse for a further five years.  Since 2013 Mr Zambeletti has been an independent strategic advisor to life science companies on Merger and Acquisitions, out-licensing deals and financing strategy.  He is a Non-Executive Director of  Nogra Pharma and an advisor to the US medtech company Qardio.  He serves as a trustee to Barts and the London Charity, which helps to fund the hospitals of the Barts NHS Trust including St Bartholomew, the Royal London and the London Chest Hospitals, and he is a founder of the cultural initiative 5x15 Italy.  Mr Zambeletti started his career at KPMG as an accountant.


Ms Valerie Andrews was appointed to the board of directors in September 2014 and has extensive skills in business and legal matters related to the healthcare industry.  Ms Andrews has held executive and non-executive roles within publically listed biotechnology companies, major hospitals and in legal practice and has garnered expertise across a number of areas including strategic transactions, corporate governance, risk management and compliance.  Ms Andrews served as the General Counsel for Vertex Pharmaceuticals Inc until May 2014. Prior to joining Vertex in 2002, she was an Executive Director of Licensing for Massachusetts General and The Brigham and Women’s Hospitals in Boston, and a partner in the Boston law firm Hill & Barlow. She served as a law clerk to the Chief Judge Levin H Campbell of the US Court of Appeals for the First Circuit from 1988 to 1989. Ms Andrews has also been a Non-Executive Director of the drug development company Columbia Laboratories since 2005. Ms Andrews is a member of the Massachusetts Bar, and holds a JD in law (Boston College) and a BA in chemistry and psychology (Duke University).


Mr David Wurzer joined the board of directors in February 2015 and has extensive experience in business and financial matters relating to the pharmaceutical and biotechnology sector having held a number of senior executive and board level positions.  Mr Wurzer was Executive Vice President, Treasurer and Chief Financial Officer of the NASDAQ listed biotechnology company CuraGen Corporation from 1997 to 2008.   He also held a number of positions at Value Health Inc., from 1991 to 1997 including Senior Vice President, Treasurer and Chief Financial Officer.  He is currently the Executive Vice President and Chief Investment Officer at Connecticut Innovations, a state funded venture capital fund.  Mr. Wurzer is currently a Non-Executive Director on the boards of Response Genetics and Special Diversified Opportunities Inc, and from 2010 to 2012 he was a Non-Executive Director on the board of DUSA Pharmaceuticals.  Mr. Wurzer is a Certified Public Accountant and began his career with Coopers & Lybrand, which is now part of PricewaterhouseCoopers. He received a BBA from the University of Notre Dame.


Summit Therapeutics plc
85b Park Drive
Milton Park
OX14 4RY

Tel: +44 (0)1235 443 939
Fax: +44 (0)1235 443 999


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