DGAP-News: GSK and Prosensa Announce Primary Endpoint Not Met in Phase III Study of Drisapersen in Patients With Duchenne Muscular Dystrophy
(firmenpresse) - Prosensa Holding N.V.
20.09.2013 13:00
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LONDON and LEIDEN, Netherlands, 2013-09-20 13:00 CEST (GLOBE NEWSWIRE) --
GlaxoSmithKline (GSK) and Prosensa today announced that GSK's Phase III
clinical study of drisapersen, an investigational antisense oligonucleotide,
for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an
amenable mutation, did not meet the primary endpoint of a statistically
significant improvement in the 6 Minute Walking Distance (6MWD) test compared
to placebo.
A total of 186 boys were randomised to this double-blind, placebo-controlled
study (DMD114044) and received drisapersen at a dose of 6mg/kg/week (N=125) or
placebo (N=61) via subcutaneous injection over 48 weeks. The difference in 6MWD
(mean (CI) 10.33m (-14.65, 35.31), p=0.415) between drisapersen and placebo
groups did not reach statistical significance. There was no treatment
difference in key secondary assessments of motor function: 10-meter walk/run
test, 4-stair climb and North Star Ambulatory Assessment. The most commonly
reported adverse events included injection site reactions (78% for drisapersen
vs 16% for placebo) and renal adverse events (including subclinical
proteinuria; 46% for drisapersen vs 25% for placebo). No patients had
thrombocytopenia.
Full evaluation of the benefit-to-risk profile of drisapersen treatment across
all studies is anticipated to be completed by year end. This may include
analyses of pooled results from various drisapersen studies.
'We appreciate that these results will be disappointing for boys with DMD and
their families. We would like to sincerely thank all those who participated in
the study for their commitment,' commented Carlo Russo, Senior Vice President,
Head of GSK Rare Diseases Research&Development. 'We are committed to
evaluating the outcome of this study in the context of the overall development
programme with experts in the field, and we expect such evaluation to help
inform our next steps for drisapersen. It is our hope that progress will be
made in an effort to help boys with DMD.'
'While we are disappointed that this study did not meet its primary endpoint,
we remain committed to the overall programme and will continue to work closely
with GSK,' said Hans Schikan, Chief Executive Officer of Prosensa. 'With no
long term disease modifying therapies available for DMD patients, research and
development of possible treatment options is of critical importance for boys
and their families affected by this debilitating disease.'
Results have been submitted for presentation at forthcoming scientific meetings
and will also be submitted for publication in a scientific peer-reviewed
journal.
Drisapersen is not approved or licensed for use anywhere in the world.
About drisapersen
Drisapersen, (previously GSK2402968/PRO051) an antisense oligonucleotide, which
induces exon skipping of exon 51, is currently in late stage development for
DMD.
GSK obtained an exclusive worldwide license to develop and commercialise
drisapersen from Prosensa in 2009. Drisapersen has been designated orphan drug
status in the EU, US and Japan. In June 2013, drisapersen was granted
Breakthrough Therapy designation by the US Food and Drug Administration.
For more information regarding the ongoing clinical studies involving
drisapersen visit www.clinicaltrials.gov.
About drisapersen clinical development program
The overall clinical programme comprises three early phase studies (PRO051-01,
PRO051-02 and DMD114673) and three double-blind, placebo-controlled studies
(DMD114117, DMD114876 and DMD114044). Phase II results from DMD114117 were
presented at Cold Spring Harbor in April 2013 and are posted on
www.gsk-clinicalstudyregister.com. The Phase II results of DMD114876 will be
presented at DIA/FDA oligonucleotide meeting, 25-27 Sept 2013, Washington, USA
and other scientific congress meetings this year.
The clinical programme also includes an open-label extension study DMD114349
for boys completing DMD114117 and DMD114044.
About DMD
Duchenne Muscular Dystrophy (DMD) is a severely debilitating childhood
neuromuscular disease that affects up to 1 in 3,500 live male births. This rare
disease is caused by mutations in the dystrophin gene, resulting in the absence
or defect of the dystrophin protein.
Patients suffer from progressive loss of muscle function due to the absence or
defect of the dystrophin protein, often making them wheelchair bound before the
age of 12. Respiratory and cardiac muscle can also be affected by the disease.
Few patients survive the age of 30.
About exon skipping
The dystrophin gene is the largest gene in the body, consisting of 79 exons.
Exons are small sequences of genetic code which lead to the manufacture of
sections of protein. In DMD, when certain exons are mutated/deleted, the RNA
cannot read past the fault. This prevents the rest of the exons being read,
resulting in a non-functional dystrophin protein and the severe symptoms of
DMD.
RNA-based therapeutics, specifically antisense oligonucleotides inducing exon
skipping, are currently in development for DMD. This technology uses synthetic
antisense oligonucleotides to skip an exon next to a deletion and thereby
correct the reading frame, enabling the production of a novel dystrophin
protein. Up to 13% of boys with DMD have dystrophin gene mutation/deletions
amenable to an exon 51 skip.
GlaxoSmithKline - one of the world's leading research-based pharmaceutical and
healthcare companies - is committed to improving the quality of human life by
enabling people to do more, feel better and live longer. For further
information please visit www.gsk.com
Prosensa (Nasdaq:RNA) is a Dutch biotechnology company engaged in the discovery
and development of RNA-modulating therapeutics for the treatment of genetic
disorders. Its primary focus is on rare neuromuscular and neurodegenerative
disorders with a large unmet medical need, including Duchenne muscular
dystrophy, myotonic dystrophy and Huntington's disease.
Prosensa's current portfolio includes six compounds for the treatment of DMD,
all of which have received orphan drug status in the United States and the
European Union. The compounds use an innovative technique called exon-skipping
to provide a personalized medicine approach to treat different populations of
DMD patients.
www.prosensa.com
GlaxoSmithKline Enquiries:
UK Media enquiries: David Mawdsley +44 (0) 20 8047 (London)
5502
Simon Steel +44 (0) 20 8047 (London)
5502
David Daley +44 (0) 20 8047 (London)
5502
Catherine +44 (0) 20 8047 (London)
Hartley 5502
US Media enquiries: Stephen Rea +1 215 751 4394 (Philadelphia)
Kevin Colgan +1 919 483 2933 (NorthCarolina)
Melinda Stubbee +1 919 483 2510 (North
Carolina)
Mary Anne Rhyne +1 919 483 0492 (North
Carolina)
Sarah Alspach +1 202 715 1048 (Washington,
DC)
Jennifer +1 215 751 5664 (Philadelphia)
Armstrong
Analyst/Investor enquiries: Ziba Shamsi + 44 (0) 20 8047 (London)
3289
Lucy Budd +44 (0) 20 8047 (London)
2248
Tom Curry + 1 215 751 5419 (Philadelphia)
Gary Davies + 44 (0) 20 8047 (London)
5503
James Dodwell + 44 (0) 20 8047 (London)
2406
Jeff McLaughlin + 1 215 751 7002 (Philadelphia)
Prosensa Enquiries:
Analyst/Investor/Media Celia Economides + 1 917 975 1983 (New York)
enquiries:
Luc Dochez + 31 6 2436 8381 (Leiden)
Prosensa Solo Conference Call Information:
Prosensa will host a conference call today at 8:00 am Eastern Time to discuss
the results of the DMD114044 study. To participate by telephone, please dial
877-280-2296 (US) or 0800 020 2576 (Netherlands) or 0800 279 5004 (UK). The
conference ID number is 7133371.
Additional dial-in numbers and an archived replay of this call will be
accessible via the Investors&Media section of Prosensa's website at
www.prosensa.com. The replay will remain available for seven (7) days following
the live call.
GSK cautionary statement regarding forward-looking statements
GSK cautions investors that any forward-looking statements or projections made
by GSK, including those made in this announcement, are subject to risks and
uncertainties that may cause actual results to differ materially from those
projected. Factors that may affect GSK' s operations are described under Item
3.D 'Risk factors' in the company's Annual Report on Form 20-F for 2012.
Forward Looking Statements Prosensa
This press release contains certain forward-looking statements. All statements,
other than statements of historical facts, contained in this press release,
including statements regarding our strategy, future operations, future
financial position, future revenues, projected costs, prospects, plans and
objectives of management, are forward-looking statements. The words
'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'plan,'
'predict,' 'project,' 'target,' 'potential,' 'will,' 'would,' 'could,'
'should,' 'continue,' and similar expressions are intended to identify
forward-looking statements, although not all forward-looking statements contain
these identifying words. Forward-looking statements in this press release
include statements around our exon-skipping drug candidates and our
collaboration with GlaxoSmithKline (GSK). Actual results may differ materially
from those projected or implied in such forward-looking statements. Such
forward-looking information involves risks and uncertainties that could
significantly affect expected results. These risks and uncertainties are
discussed in the Company's SEC filings, including, but not limited to, the
Company's Form 6-K containing this press release and certain sections of the
Company's Registration Statement on Form F-1. In addition, any forward-looking
statements represent our views only as of today and should not be relied upon
as representing our views as of any subsequent date. While we may elect to
update these forward-looking statements at some point in the future, we
specifically disclaim any obligation to do so, even if our views change.
Registered in England&Wales:
No. 3888792
Registered Office:
980 Great West Road
Brentford, Middlesex
TW8 9GS
News Source: NASDAQ OMX
20.09.2013 Dissemination of a Corporate News, transmitted by DGAP -
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Language: English
Company: Prosensa Holding N.V.
Netherlands
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