Summit to Extend Ongoing PhaseOut DMD Clinical Trial of Ezutromid in Patients With DMD
March 27 2017 - 7:00AM
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 it will proceed with the planned extension phase of
PhaseOut DMD, a Phase 2 clinical trial evaluating the utrophin
modulator ezutromid, subject to regulatory approval. This follows
an interim review of the safety and tolerability data from the
ongoing trial by an independent Data Monitoring Committee (‘DMC’)
and its support of Summit’s plans to extend the clinical
trial.
Summit has now applied for regulatory approval
to extend PhaseOut DMD from the UK Medicines and Healthcare
products Regulatory Agency and Ethics Committee, and has submitted
the necessary regulatory updates to the US Food and Drug
Administration. These submissions are intended to facilitate the
transition of patients participating in PhaseOut DMD onto an
open-label extension phase at the end of the initial 48-weeks of
dosing with ezutromid without a cessation in dosing. The extension
phase will be used to gather long term safety and efficacy data and
is expected to last until ezutromid either receives marketing
approval in the relevant country or its development is
discontinued.
“The proposed extension phase will allow us to
gather important long term safety and efficacy data of ezutromid in
patients with DMD that we believe will comprise part of a data
package necessary for future applications for regulatory approval
of ezutromid,” Ralf Rosskamp, MD, Chief Medical Officer of
Summit commented. “While the PhaseOut DMD trial continues
with the aim of establishing proof of concept for ezutromid, we are
pleased to see that the DMC supports the trial’s extension based on
their review of the safety and tolerability data to date, which
includes data from patients dosed over longer periods of time than
have previously been tested.”
In addition to the extension phase of the trial,
the regulatory submissions also include the addition of a safety
arm to allow for the enrolment of patients who have previously
taken part in Phase 1 clinical trials of ezutromid, but who did not
meet the inclusion criteria for PhaseOut DMD. Although regulatory
approval for the safety arm is being sought, the timing for
inclusion of these patients into the trial is to be determined.
About PhaseOut DMDPhaseOut 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. Each patient will receive two biopsies,
one at baseline and the second either at 24 or 48 weeks. Further
information is available at:
https://clinicaltrials.gov/ct2/show/NCT02858362 and
www.utrophintrials.com.
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: +44
(0)1235 443 951 |
Erik
Ostrowski / Michelle Avery (US office) |
+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) |
Tel: +1
781 235 3060 |
Chris Erdman
/ Karen Sharma |
cerdman@macbiocom.com |
|
ksharma@macbiocom.com |
|
|
Consilium Strategic Communications |
Tel: +44
(0)20 3709 5700 |
(Financial
public relations, UK) |
summit@consilium-comms.com |
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, 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, 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 contains inside information for the purposes
of Article 7 of EU Regulation 596/2014 (MAR).
-END-
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