Summit Therapeutics plc Summit Joins Ctap In Effort To Enhance Development Of Utrophin Modulators And Other Treatments For Dmd

 
TIDMSUMM 
 
 
   Summit Therapeutics plc 
 
   ('Summit', or 'the Company') 
 
   SUMMIT JOINS cTAP IN COLLABORATIVE EFFORT TO ENHANCE THE DEVELOPMENT OF 
UTROPHIN MODULATORS AND OTHER TREATMENTS FOR DUCHENNE MUSCULAR DYSTROPHY 
 
   Oxford, UK, 25 September 2017 - Summit Therapeutics plc (NASDAQ: SMMT, 
AIM: SUMM), the drug discovery and development company advancing 
therapies for Duchenne muscular dystrophy ('DMD') and Clostridium 
difficile infection, today announces it has joined the Collaborative 
Trajectory Analysis Project ('cTAP') to support cTAP's mission of 
accelerating the development of drugs to treat DMD through a coalition 
of Duchenne clinical experts, patient advocates and biopharmaceutical 
companies. 
 
   "In the Duchenne field, there is a strong community amongst the families, 
patient organisations, caregivers and industry that comes together to 
improve the lives of patients living with this disease, where time is of 
the essence," said Dr David Roblin, President of R&D of Summit. "cTAP is 
leveraging the natural history and clinical data collected within this 
community to create more predictive models of disease progression with 
the aim of enabling companies to interpret data and improve the design 
of clinical trials in DMD. We believe this could potentially benefit the 
development of our utrophin modulators for the treatment of all patients 
with DMD. In addition, we have the opportunity to contribute our own 
data to aid others as we all seek to improve the lives of DMD patients 
and their families." 
 
   Debra Miller, founder and CEO of CureDuchenne and initial funder of cTAP 
commented: "cTAP was started with the single mission of helping 
biopharmaceutical companies to bring treatments to our children living 
with DMD as quickly as we possibly can. This mission is only made 
possible through a collaboration of clinicians, patient advocates and 
the biopharma industry, and we welcome Summit in this effort." 
 
   Professor Eugenio Mercuri, Neurology and Pediatrics, Università 
Cattolica del Sacro Cuore, Rome, Italy, added: "cTAP's initiative has 
brought together a wealth of data from clinicians and biopharmaceutical 
companies. This resource could assist Summit in developing its 
potentially universal treatments for patients with DMD, and in turn, 
Summit's data could contribute to the wider cTAP collaboration." 
 
   Summit's lead utrophin modulator candidate for the treatment of DMD, 
ezutromid, is currently in a Phase 2 clinical trial called PhaseOut DMD. 
The trial aims to establish proof of concept of ezutromid through a 
range of muscle structure, muscle health and functional endpoints. The 
trial uses quantitative magnetic resonance to measure fat fraction in 
leg muscles as its primary endpoint. This technique is emerging as a 
tool to measure muscle health since the fat fraction increases as the 
disease progresses over time. In addition, the Company plans to measure 
utrophin and a biomarker of muscle regeneration (developmental myosin) 
from muscle biopsies provided at baseline and again after either 24 or 
48 weeks of treatment. These measurements could provide early evidence 
of ezutromid's activity. Finally, PhaseOut DMD assesses functional 
measures, such as the six minute walk distance and North Star Ambulatory 
Assessment, as exploratory endpoints. Summit expects to report 24-week 
data in the first quarter of 2018, and data from the full 48-week trial 
are expected in the third quarter of 2018. 
 
   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 had 
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 cTAP 
 
   Driven by a shared mission to overcome the challenges of developing 
drugs for diseases characterized by heterogeneous progression, cTAP 
brings advanced data science to a dynamic alliance of all stakeholders 
in the ecosystem - a first in Duchenne. The Collaborative Trajectory 
Analysis Project, or cTAP, is enabling clinical experts to solve the 
most critical problems in drug development for Duchenne muscular 
dystrophy. The first community-wide coalition in Duchenne, cTAP has 
forged an alliance between clinical experts, drug companies developing 
therapies, patient advocacy organizations and collaborating registries 
and clinical centers across Europe and the US. cTAP brings advanced data 
science to the fight against Duchenne through a partnership with 
outcomes research experts at Analysis Group Inc. 
(http://www.analysisgroup.com/). cTAP is curating and growing what is 
already the largest natural history database of patient data in 
Duchenne. This rich resource enables cTAP to develop solutions with the 
urgency necessary to enhance clinical trial design and analysis, 
near-term. http://ctap-duchenne.org 
 
   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] 
cTAP 
 Susan J. Ward, PhD                             617-448-2617 
                                                [e-mail] 
Cairn Financial Advisers LLP 
 (Nominated Adviser)                            Tel: [phone] 
 Liam Murray / Tony Rawlinson 
N+1 Singer 
 (Broker)                                       Tel: [phone] 
 Aubrey Powell / Lauren Kettle 
MacDougall Biomedical Communications 
 (US media contact)                            Tel: [phone] 
 Karen Sharma                                  [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 
therapeutic potential of Summit's product candidates, and the timing of 
initiation, completion and availability of data from clinical trials, 
the potential benefits of Summit's collaborative work with cTAP 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, 2017. 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. 
 
   -END- 
 
   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

September 25, 2017 07:00 ET (11:00 GMT)

Copyright (c) 2017 Dow Jones & Company, Inc.