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 that a series of presentations will be given by Summit
and its collaborators at the 22nd International Congress of the
World Muscle Society, which is taking place in Saint-Malo, France,
from 3-7 October 2017.
The presentations include validation data of
muscle biopsy biomarkers designed to assess utrophin modulator
activity in clinical trials that have been developed in
collaboration with Flagship Biosciences (‘Flagship’), a leader in
quantitative tissue-based biomarkers. In addition, baseline
characteristics of patients enrolled into Summit’s ongoing Phase 2
clinical trial, called PhaseOut DMD, will be presented.
“Developing new biomarkers capable of more
reliably analysing muscle biopsies in clinical trials has been a
major priority for Summit, and I am delighted with the progress
made in our collaboration with Flagship. These automated techniques
are capable of analysing thousands of muscle fibres in whole muscle
biopsy sections, and we believe that their use could help us
establish proof of mechanism for our utrophin modulator, ezutromid,
in our ongoing PhaseOut DMD clinical trial,” commented Dr
David Roblin, Chief Operating and Medical Officer of
Summit. “Summit is looking forward to an important period
in our utrophin modulation programme: we expect to report the first
24-week biopsy, MRI and functional data from PhaseOut DMD in Q1
2018, with top-line data from the complete 48-week clinical trial
expected in Q3 2018.”
Dr David Young, CEO of Flagship
Biosciences, added: “We proudly support Summit’s critical
work towards a therapy potentially applicable to all patients with
DMD with our advanced tissue analysis solutions. The progress we
have made in developing these tools could advance the understanding
of how patients are responding to treatment with ezutromid in this
challenging rare disease.”
Posters P392, P393 and P469: Validation
Data and Handling of Muscle Biopsy Biomarkers Continuous
utrophin expression by utrophin modulator therapies represents a
potential disease modifying treatment for all patients with DMD,
irrespective of their underlying genetic mutation. A series of
presentations to be given at the WMS Congress details the
development and validation of three biomarkers that allow for fibre
identification, measurement of utrophin protein and measurement of
muscle fibre regeneration.
The presentations detail an automated
immunohistochemical analysis process that accurately and precisely
quantifies utrophin protein and developmental myosin, a biomarker
of muscle fibre immaturity, in whole section muscle biopsies each
comprising several thousand individual fibres. High levels of
consistency were observed between images from consecutive biopsy
sections, and biopsy sections separated by 110μm or 150μm. High
levels of consistency were also observed when the analysis of
samples was performed within the same batch or performed over three
days.
Use of these biomarkers requires taking biopsy
samples of muscle from patients via a surgical procedure. Knowing
how burdensome and precious these biopsies are for patients, Summit
has developed a robust standardised practise to handle, process and
ship muscle biopsy samples. This protocol builds on the processes
developed by Sarepta Therapeutics Inc.
These muscle biopsy biomarker techniques and the
biopsy handling protocol are being used in PhaseOut DMD.
Poster P403: PhaseOut DMD - Baseline
Characteristics and MRI MeasuresPhaseOut DMD is seeking to
establish mechanistic proof of concept for the small molecule
utrophin modulator ezutromid. The clinical trial design, endpoints
and inclusion/exclusion criteria are presented. A total of 40 boys
have been enrolled aged between their 5th and 10th birthdays and
are being dosed with ezutromid twice daily for a total of 48 weeks.
There is an optional extension phase at the end of this 48-week
dosing period. The baseline characteristics of the enrolled
patients, including age and body weight, are detailed, along with
the baseline pharmacodynamics measures related to the magnetic
resonance spectroscopy endpoint assessing muscle health by fat
fraction percentage.
A further series of posters is being presented
by the research group of Professor Kay Davies as part of Summit’s
strategic alliance with the University of Oxford. This includes
reporting known serum biomarkers and emerging micro RNA biomarkers
that are elevated in the dystrophin deficient mdx mouse disease
model but found at close to normal levels in this disease model
where utrophin is expressed continually (Posters P235 and P239).
The continued development of future generation utrophin modulators
is also presented (Poster P308).
A total of seven posters are being presented by
Summit and its collaborators at WMS 2017 and these are now
available for download from Summit’s website. Details of the poster
presentations are as follows:
Date & Time: 17:00-18:30
CET, Thursday, 5 October 2017Session:
4Abstract Number: P.392Title:
Analytical validation (based on CLIA & CLSI standards) of
utrophin-laminin α2 and MHCd-laminin α2 duplex immunohistochemical
assays using Computational Tissue Analysis (cTA™) for evaluation of
Duchenne muscular dystrophy therapeuticsAuthors:
C. Faelan; J. Tinsley; A. Milici; S. Moore; J. Patterson-Kane
Date & Time: 17:00-18:30
CET, Thursday, 5 October 2017Poster Session:
4Abstract Number: P.393Title:
Computational alignment of duplex immunohistochemically-stained
muscle sections in support of therapies for Duchenne muscular
dystrophyAuthors: L. Cerkovnik; J. Patterson-Kane;
K. Ryall; A. Milici; J. Tinsley; S. Moore; C. Faelan
Date & Time: 17:00-18:30
CET, Thursday, 5 October 2017Session:
4Abstract Number: P.403Title:
PhaseOut DMD: A Phase 2, proof of concept, clinical study of
utrophin modulation with ezutromidAuthors: F.
Muntoni; K. Maresh; K. Davies; S. Harriman; G. Layton; R. Rosskamp;
A. Russell; B. Tejura; J. Tinsley
Date & Time: 17:00-18:30
CET, Thursday, 5 October 2017Session:
4Abstract Number: P.469Title:
Collection of high quality muscle biopsies for use in DMD clinical
trial analysis; process development and
implementationAuthors: J. Tinsley; D. Frank; J.
Dworzak; C. Faelan; J. Patterson-Kane; H. Wolff; F. Muntoni;
PhaseOut DMD Study Group
Date & Time: 15:30-17:00
CET, Thursday, 5 October 2017Session:
3Abstract Number: P.235Title:
Circulating miRs biomarkers for therapeutic monitoring in utrophin
based DMD therapyAuthors: N. Ramadan; S. Guiraud;
B. Edwards; S. Squire; S. Hemming; K. Davies
Date & Time: 15:30-17:00
CET, Thursday, 5 October 2017Session:
3Abstract Number: P.239Title:
Identification of serum protein biomarkers for utrophin based DMD
therapyAuthors: S. Guiraud; B. Edwards; S. Squire;
A. Babbs; N. Shah; A. Berg; H. Chen; K. Davies
Date & Time: 15:30-17:00
CET, Thursday, 5 October 2017Session:
3Abstract Number: P.308Title:
Discovery of small molecule utrophin modulators for the therapy of
Duchenne muscular dystrophyAuthors: G. Wynne; A.
Vuorinen; E. Emer; D. Conole; M. Chatzopoulou; S. Davies; A.
Russell; S. Guiraud; S. Squire; A. Berg; B. Edwards; S. Hemming; T.
Kennedy; L. Moir; K. Davies; S. Harriman; J. Tinsley; F. Wilson
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 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 drug. 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 PhaseOut DMD
Clinical TrialPhaseOut DMD is a 48-week open-label Phase 2
clinical trial that has enrolled 40 patients at sites in the UK and
the US. The trial aims to establish proof of concept of ezutromid
and is evaluating a range of muscle structure, muscle health and
functional endpoints. As part of the trial, each patient has two
muscle biopsies taken, a baseline biopsy on enrolment and a second
either at 24 or 48 weeks. In the first quarter of 2018, Summit
expects to report 24-week biopsy analysis from approximately 20
patients, as well as 24-week MRI and functional data from all 40
patients enrolled in the trial. Top-line data from the complete
48-week trial are expected in the third quarter of 2018.
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 7213 0880 |
|
|
N+1 Singer (Broker)Aubrey Powell / Lauren
Kettle |
Tel:
+44 (0)20 7496 3000 |
|
|
MacDougall Biomedical Communications(US media
contact)Karen Sharma |
Tel:
+1 781 235 3060ksharma@macbiocom.com |
|
|
Consilium
Strategic Communications (Financial public relations,
UK)Mary-Jane Elliott / Sue Stuart / Jessica Hodgson / Philippa
Gardner |
Tel: +44 (0)20 3709
5700 summit@consilium-comms.com |
Forward-looking StatementsAny
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, Summit’s license and collaboration
agreement with Sarepta and the expected receipt of any milestone
payments under the agreement, the therapeutic potential of Summit’s
product candidates, the potential benefit and utility of using
biomarkers to analyse muscle biopsies, 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 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-
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