Prosensa Completes Enrollment in Natural History Study of Duchenne Muscular
Leiden, The Netherlands, June 25, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding
N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on
RNA-modulating therapeutics for rare diseases with high unmet need, today
announced the successful completion of enrollment in its prospective Natural
History Study designed to increase the understanding of Duchenne muscular
The study enrolled in total 269 boys with confirmed DMD between the ages three
and 18 from 10 countries across 16 centers in North & South America and
Europe, 80% of whom are ambulatory and 20% non-ambulatory.
The purpose of the study is to characterize the natural history and
progression of DMD, which will help inform the design of future studies, to
capture biomarkers of safety and disease progression, and to provide
comparative data for patients with rare exon deletions/mutations for which
formal controlled trials are not feasible. No medication or device is being
tested in this study. Moreover, the study will be important in the
confirmatory program for Prosensa's lead DMD compound drisapersen, for which a
New Drug Application is expected to be submitted later this year to the FDA
under an Accelerated Approval pathway.
Dr. Brenda Wong of Cincinnati Children's Hospital, who has enrolled patients
in the study said, "While we are pleased by the high level of world-wide
attention and commitment by regulators given to DMD, an existing obstacle for
delivering promising treatments to patients relies on accepted clinical
endpoints for human studies. By collecting and analyzing this Natural History
Study data from hundreds of boys, we are better equipped to expedite the
development of promising therapies and offer them to DMD patients anxiously
awaiting treatment options to slow disease progression and improve the outcome
of this disease."
"We continue to work diligently to pioneer research and development in DMD,
and completing enrollment in our Natural History Study is a major milestone
and a critical component of basic research to understand the disease," said
Dr. Giles Campion, Prosensa's Chief Medical Officer. "This study will be a
crucial resource for our confirmatory studies with drisapersen and for our
follow-on exon skipping programs. Outcomes of this study will contribute to a
growing body of scientific knowledge and are fundamental to our ability to
develop innovative treatment options for boys who suffer from this devastating
All boys participating in the study will be followed for a period of three
years, with site visits and assessments every 6 months. During these visits,
investigators will evaluate the boys on a number of physical tests, including
the six minute walk test, climbing stairs, breathing in a tube to measure lung
function and performing upper limb movements. These parameters are meant to
assess how the disease affects their overall quality of life as the condition
evolves over time in addition to contributing to the understanding of the
natural progression of the disease. Furthermore, blood and urine samples will
be taken to investigate a panel of biomarkers that may be useful in following
The study, which began in September 2012 and is sponsored by Prosensa, is
expected to complete in November 2017. This autumn, 80 boys will have
completed one year of observation and this interim analysis will be available
later this year. Further information on the study can be found here:
www.ClinicalTrials.gov- Study ID: NCT01753804
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, 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 the genetic code 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.
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 (DMD), Myotonic dystrophy and Huntington's
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
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 the Company's 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 the Company's exon-skipping drug pipeline
and financial position. 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 the Company's Annual Report on Form 20-F. In addition,
any forward-looking statements represent its views only as of today and should
not be relied upon as representing its views as of any subsequent date. While
the Company may elect to update these forward-looking statements at some point
in the future, the Company specifically disclaims any obligation to do so,
even if its views change.
CONTACT: Celia Economides
Senior Director, IR & Corporate Communications
Prosensa Holding B.V. Logo
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