Summit Therapeutics plc Summit Presents Preclinical Dmd Data At 21st Congress Of World Muscle Society

 
TIDMSUMM 
 
   Summit Therapeutics plc 
 
   ("Summit" or "the Company") 
 
   SUMMIT THERAPEUTICS PRESENTS PRECLINICAL DMD DATA AT THE 21(ST) 
INTERNATIONAL CONGRESS OF THE WORLD MUSCLE SOCIETY 
 
 
   -- Summit reports on development of new, automated biomarker tools to 
      evaluate disease status of muscle biopsies 
 
 
   Oxford, UK, 6 October 2016 - Summit Therapeutics plc (AIM: SUMM, NASDAQ: 
SMMT), the drug discovery and development company advancing therapies 
for Duchenne muscular dystrophy ('DMD') and Clostridium difficile 
infection, announces the presentation of preclinical data at the 21(st) 
International Congress of the World Muscle Society, which is taking 
place in Granada, Spain from 4-8 October 2016.  The data include further 
findings from Summit's programme to develop tools to measure biomarkers 
of muscle health for use in patient clinical trials evaluating utrophin 
modulator therapies. 
 
   "Our approach to DMD aims to maintain production of utrophin, a protein 
typically found in young and repairing muscle fibres, so that it can 
replace the missing dystrophin protein in mature muscle fibres," 
commented Dr Ralf Rosskamp, Chief Medical Officer of Summit. "The 
development of new, fully automated biomarker tools capable of 
evaluating the disease status of muscle biopsies will play an important 
role in clinical trials, including our ongoing PhaseOut DMD trial. In 
this trial, we seek to establish the potential of utrophin modulation, 
including our most advanced candidate ezutromid, as an effective 
treatment for all patients with this disease." 
 
   The findings reported at WMS highlight progress in the development of 
fully automated quantification techniques for muscle biopsies capable of 
measuring structural proteins such as utrophin and biomarkers of muscle 
regeneration. The techniques described are immunohistochemical based 
assays and digital tissue image analysis tools that can robustly measure 
utrophin, beta-dystroglycan (a member of the dystrophin protein complex) 
and developmental myosin (a biomarker of muscle fibre maturity) in 
individual muscle fibres across a whole biopsy section. 
 
   Patients with DMD have higher levels of immature, regenerating muscle 
fibres because the absence of dystrophin leads to a continual cycle of 
muscle fibre damage and repair. By seeking to replace missing dystrophin 
with utrophin, utrophin modulators such as ezutromid aim to maintain the 
integrity of muscle fibres and allow them to mature. The automated 
approaches described at WMS make it feasible to look at associations 
between fibre maturity and integrity and utrophin expression in 
individual fibres. This provides a basis to distinguish new and 
beneficial expression of utrophin caused by a utrophin modulating drug, 
from the expression of utrophin seen generally during fibre 
regeneration. This is an important step in understanding and 
characterising the activity of these drugs. 
 
   This biomarker research is being conducted in partnership with Flagship 
Biosciences using their computational Tissue Analysis (cTA(TM)) approach 
to accelerate precise muscle fibre biomarker quantification. This work 
builds on a recently published manual quantification approach that was 
developed by Summit and researchers at the Institute of Child Health, 
London with financial support from the charity Joining Jack. Further 
validation work to optimise the Flagship Biosciences' cTA(TM) approach 
is ongoing; it is expected the tools will be used in Summit's Phase 2 
proof of concept trial of ezutromid, PhaseOut DMD. 
 
   Additional presentations at WMS 2016 reported preclinical data 
highlighting the potential of utrophin modulation as a disease-modifying 
treatment for all patients with DMD, regardless of the underlying fault 
in the dystrophin gene. This research was conducted at the University of 
Oxford under the UtroDMD alliance. 
 
   Copies of the presentations given at WMS 2016 are available from the 
'Programmes' section of Summit's website, www.summitplc.com. 
 
   About PhaseOut DMD 
 
   PhaseOut DMD aims to provide proof of concept for ezutromid and utrophin 
modulation by measuring muscle fat infiltration, as well as by measuring 
utrophin protein and muscle fibre regeneration in muscle biopsies. The 
primary endpoint of the open-label trial is the change from baseline in 
magnetic resonance imaging parameters related to fat infiltration and 
inflammation of the leg muscles. Exploratory endpoints include the 
six-minute walk distance, the North Star Ambulatory Assessment and 
patient reported outcomes. PhaseOut DMD is a 48-week open-label trial 
expected to enrol up to 40 boys ranging in age from their fifth to their 
tenth birthdays at sites in the UK and the US. 
 
   Further information is available at: www.utrophintrials.com and 
https://clinicaltrials.gov/ct2/show/NCT02858362. 
 
   About Utrophin Modulation in DMD 
 
   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 has 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, ezutromid, is an orally 
administered, small molecule. DMD is an orphan disease, and the US Food 
and Drug Administration ('FDA') and the European Medicines Agency have 
granted orphan drug status to ezutromid. Orphan drugs receive a number 
of benefits including additional regulatory support and a period of 
market exclusivity following approval. In addition, ezutromid has been 
granted Fast Track designation and Rare Pediatric Disease designation by 
the FDA. 
 
   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 programs 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). 
 
   For more information, please contact: 
 
 
 
 
Summit 
 Glyn Edwards / Richard Pye (UK office)         Tel: [phone] 
 Erik Ostrowski / Michelle Avery (US office)    [phone] 
Cairn Financial Advisers LLP 
 (Nominated Adviser)                            Tel: [phone] 
 Liam Murray / Tony Rawlinson 
N+1 Singer 
 (Broker)                                       Tel: [phone] 
 Aubrey Powell / Jen Boorer 
MacDougall Biomedical Communications 
 (US media contact)                            Tel: [phone] 
 Chris Erdman / Karen Sharma                   [e-mail] / 
                                               [e-mail] 
Consilium Strategic Communications            Tel: [phone] 
 (Financial public relations, UK)             [e-mail] 
 Mary-Jane Elliott / Sue Stuart / 
 Jessica Hodgson / Lindsey Neville 
 
   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 
utility of tools developed to evaluate 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 ongoing 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, 2016. 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. 
 
   -- 
 
   This announcement is distributed by Nasdaq Corporate Solutions on behalf 
of Nasdaq Corporate Solutions clients. 
 
   The issuer of this announcement warrants that they are solely 

responsible for the content, accuracy and originality of the information 
contained therein. 
 
   Source: Summit Therapeutics plc via Globenewswire 
 
 
  http://www.summitplc.com/ 
 

(END) Dow Jones Newswires

October 06, 2016 07:00 ET (11:00 GMT)

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