Prosensa to Host Conference Call to Discuss Recent Drisapersen Data
Conference call and live webcast scheduled for 11:00 a.m. ET (5:00 p.m. CET)
on Tuesday, October 8, 2013
Leiden, The Netherlands, Oct. 3, 2013 (GLOBE NEWSWIRE) -- Prosensa Holding
N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on
RNA-modulating therapeutics for rare diseases with high unmet need, will hold
a conference call on Tuesday, October 8, 2013 at 11:00 a.m. ET / 5:00 p.m. CET
to discuss recently-presented data on drisapersen, an investigational
antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy
(DMD) patients with an amenable mutation, which is exclusively licensed to
Prosensa Chief Executive Officer Hans Schikan will be joined by Giles Campion,
Prosensa's Chief Medical Officer & Senior Vice-President of Research and
Development and Judith van Deutekom, Vice President of Drug Discovery to
discuss the recently presented results from studies of drisapersen. These
include results from DEMAND III (Phase III; DMD114044); DEMAND II (Phase II;
DMD114117); DEMAND V (Phase II; DMD114876) and 177 week data from the Phase
I/II extension study (DMD114673).
The data were presented at the DIA/FDA Oligonucleotide based Therapeutics
Conference in Washington DC, the 18th World Muscle Society Congress in
Asilomar, California and the 9th Annual Meeting of the Oligonucleotide
Therapeutics Society in Naples, Italy, running from Sept 25-27, October 1-5
and 6-8, respectively.
In order to participate in the conference call, please dial 1-877 280 2342 (US
domestic) and refer to conference ID 2579927. International dial-in numbers
and an audio webcast can be accessed under "Events & Presentations" through
the Investors & Media section of the Prosensa corporate website
Drisapersen, (previously GSK2402968/PRO051) an antisense oligonucleotide,
which induces exon skipping of exon 51, is currently in late stage development
GSK obtained an exclusive worldwide license to develop and commercialize
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.
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
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.
About Prosensa Holding N.V.
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.
Forward Looking Statements
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.
Prosensa Holding N.V.
Celia Economides, Director IR & Corporate Communications
Phone: +1 917 975 1983
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