Summit Therapeutics Presents Preclinical DMD Data at the 21st International Congress of the World Muscle Society
06 Octobre 2016 - 1:00PM
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 21st
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™) 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™ 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)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 (Broker)Aubrey Powell / Jen Boorer
|
Tel: +44 (0)20 7496 3000 |
MacDougall Biomedical Communications(US media
contact)Chris Erdman / Karen Sharma |
Tel: +1
781 235 3060cerdman@macbiocom.com /ksharma@macbiocom.com |
Consilium
Strategic Communications (Financial public relations, UK)
Mary-Jane Elliott / Sue Stuart / Jessica Hodgson / Lindsey
Neville |
Tel: +44 (0)20 3709
5700summit@consilium-comms.com |
|
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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
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product candidates, the therapeutic potential of Summit’s product
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containing the words "anticipate," "believe," "continue," "could,"
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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
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