Summit Highlights Scientific Rigour of its PhaseOut DMD Clinical Trial at MDA Clinical Conference
14 March 2018 - 10:00PM
Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM) highlighted the
rigour being utilised in the collection and analysis of muscle
biopsies and magnetic resonance spectroscopy (‘MRS’) related data
in its PhaseOut DMD clinical trial, at the 2018 MDA Clinical
Conference in Arlington, VA.
PhaseOut DMD is a Phase 2 open-label trial of
the Company’s utrophin modulator, ezutromid, in patients with
Duchenne muscular dystrophy (‘DMD’). Through this study, Summit is
compiling a deep dataset for each patient in the trial, which
includes the analysis of several thousand muscle fibres per biopsy
sample. As part of this effort, steps are being taken to remove
human bias through the automated reading of biopsy and MRS
measurements at central sites.
“Since DMD is a muscle-wasting disorder, it is
critical for us to have high quality data from objective muscle
health measurements as we assess ezutromid’s activity. As the
collection of muscle biopsy and MRS measurements are a major
intervention for patients in clinical trials, it means it is
crucial this is done in a robust and reliable way,” said Dr
David Roblin, Chief Medical Officer and President of R&D of
Summit. “Our data collection and analysis methods used in
PhaseOut DMD helped yield a high-quality dataset from all evaluable
boys in the trial after 24-weeks of treatment. We are grateful to
all the boys in the trial and excited about the interim findings
that showed compelling signs of ezutromid activity.”
The recently announced positive interim data
from PhaseOut DMD showed that after 24 weeks of treatment, there
was a significant and meaningful reduction in muscle damage, as
well as a significant reduction in muscle inflammation, in patients
treated with ezutromid. These data provide early evidence that
ezutromid is modulating the production of utrophin protein and
having its intended effect of stabilising muscle membranes, which
has in turn led to early improvements in muscle health. Utrophin is
a protein that can substitute for dystrophin, the protein that is
missing in those with DMD the absence of which results in disease
progression and premature death. Because Summit’s utrophin
modulation approach is independent of the patient’s underlying
dystrophin gene mutation, it could be applicable to all patients
with DMD.
Details of the Methodologies from the
Poster PresentationOne of the methods being used to
measure muscle damage in PhaseOut DMD is biopsy analysis. Summit
has made a significant effort to ensure that these biopsies are
handled, processed and analysed with great care. For the
biopsy procedure, two pieces of muscle are taken during each
biopsy. These pieces are frozen and shipped separately to a central
reading facility. Multiple sections from each biopsy are assayed
for muscle damage, yielding a total of six biopsy sections for each
patient for both the baseline and post-treatment samples. After
quality control checks, the muscle sections are then read via an
automated system to remove human bias. For the 24-week assessment,
several thousand muscle fibres were analysed per sample, producing
a robust dataset for each patient with high concordance across
individual samples.
Muscle inflammation is measured by T2-
relaxation time using MRS. MRS of the soleus (calf) and vastus
lateralis (thigh) muscles is performed at central imaging centres
following a training and certification process. The MRS
quantification is then performed by an automated blinded
process.
A copy of the poster is available in the
Publications section of Summit’s website, www.summitplc.com.
About PhaseOut DMDPhaseOut DMD
is an open-label, multi-centre trial that has enrolled 40 patients
in the US and UK, aged from their fifth to their tenth birthdays.
PhaseOut DMD is 48 weeks in length after which patients have the
option of enrolling into an extension phase and continuing to be
dosed with ezutromid. The primary endpoint is the change from
baseline in magnetic resonance spectroscopy parameters related to
the leg muscles. Biopsy measures evaluating utrophin and muscle
damage are included as secondary endpoints. Exploratory endpoints
include the six-minute walk distance, the North Star Ambulatory
Assessment and patient reported outcomes. Top-line 48-week results
are expected to be reported 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 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 patient’s underlying
dystrophin gene mutation. Summit also believes that utrophin
modulation could 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 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 programmes 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).
Contacts
Summit |
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Glyn
Edwards / Richard Pye (UK office) |
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Erik
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Cairn Financial Advisers LLP (Nominated
Adviser) |
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+44
(0)20 7213 0880 |
Liam
Murray / Tony Rawlinson |
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N+1 Singer (Joint Broker) |
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(0)20 7496 3000 |
Aubrey Powell / Jen Boorer |
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Panmure Gordon (Joint Broker) |
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+44
(0)20 7886 2500 |
Freddy Crossley, Corporate Finance |
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Tom
Salvesen, Corporate Broking |
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MacDougall Biomedical Communications (US) |
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+1
781 235 3060 |
Karen
Sharma |
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ksharma@macbiocom.com |
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Consilium Strategic Communications (UK) |
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+44
(0)20 3709 5700 |
Mary-Jane Elliott / Jessica Hodgson / |
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summit@consilium-comms.com |
Philippa Gardner |
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for the purposes of Article 7 of EU Regulation 596/2014
(MAR).-END-
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