PRESS RELEASE, May 5th, 2008
Prosensa announces the start of an international multi-center
phase I/II clinical study with ‘smart drug’ PRO051 in patients with Duchenne Muscular Dystrophy
On May 5th, 2008, Prosensa announces that it has started a phase I/II study to explore
the effect, safety and tolerability of systemic injections of PRO051 in Duchenne Muscular
Dystrophy (DMD) patients. This trial is performed in collaboration with UZ Leuven
(Belgium), the Queen Silvia Children’s hospital (Sweden) and the LUMC (the
Netherlands). The UZ Leuven and the Queen Silvia Children’s hospital have already
started to enroll patients. In this study, an important parameter will be the presence of
dystrophin in muscle biopsies, the protein missing in patients with Duchenne Muscular
Dystrophy (DMD). This clinical trial in patients with DMD uses an antisense
oligoribonucleotide, a ‘smart drug’ removing an unwanted segment of the faulty DMD
gene product, and represents a novel approach to combat genetic diseases like DMD.
Dr. Nathalie Goemans, the coordinating and principle investigator in Leuven says: “I am
excited that, based on the encouraging proof of concept data from the previous clinical
study, we can now proceed with this next important step in the investigations that are
required to determine whether this highly promising approach can be developed into an
effective and safe treatment for patients with this devastating disease. “
"We are proud that quickly after completion and publication of the first trial we are able to
conduct this study. We expect to provide further information on the developments later
this year. In this study, we extend our recent success proving the concept of local
dystrophin production to a study with systemic application to achieve widespread
dystrophin expression in muscles. ", says Gerard Platenburg, Prosensa's CEO.
ABOUT DMD AND EXON SKIPPING
Duchenne muscular dystrophy is a severely debilitating childhood neuromuscular
disease that affects 1 in 3500 newborn boys. The young patients suffer from progressive
loss of muscle strength due to the absence of the protein dystrophin, making them
wheelchair bound before the age of 12 and most die in early adulthood due to
respiratory and cardiac failure. Today, there is no treatment to prevent the eventual fatal
outcome. The disease is caused by mutations in the DMD gene, resulting in the absence
of the dystrophin protein, which is crucial for the integrity of muscle fiber membranes.
RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping,
are currently the most promising therapy for Duchenne Muscular Dystrophy. More
specifically, antisense oligonucleotides have the capacity to skip an exon and thereby
correct the reading frame of DMD transcripts resulting in the synthesis of a largely
functional dystrophin protein. Different mutations in the gene require different
oligonucleotide drugs. The PRO051, the first of its kind, will be suitable for 15% of all
DMD patients, because it can treat a cluster of mutations.
ABOUT PROSENSA
Prosensa is a Dutch biopharmaceutical company dedicated to the development of RNAbased
therapeutics targeting diseases with unmet medical needs, in particular
neuromuscular disorders. Prosensa’s drug development is based upon a unique and proprietary technology platform involving ‘exon skipping’, enabling correction of mutated
RNA. This ability to modulate genes selectively through RNA-based therapeutics could
provide a new way to treat a wide range of human diseases. The company has, together
with its partner LUMC, a leadership position in fundamental patents, technology, and
know-how relating to RNA-based approaches and exon skipping. Prosensa’s lead
compounds for DMD are currently in clinical phase I/II development and the company
has received orphan drug designation both in Europe and the US. Prosensa is elected
the European Venture Company of 2007. For more information on Prosensa, please visit
www.prosensa.nl.
ABOUT UZ LEUVEN
The 1 894 beds at the University Hospitals of Leuven (UZ Leuven) make this the largest
hospital in the country. UZ Leuven sees its task as caring for patients, conducting
research and providing training at academic level. The Neuromuscular Reference Center
for Children serves as a centralized clinical center for diagnosis and management of
neuromuscular patients, and provides required facilities for conducting and participating
in clinical trials and has a longstanding experience in conducting clinical trials and
translational research in Duchenne Muscular Dystrophy.
ABOUT THE QUEEN SILVIA CHILDREN’S HOSPITAL IN GÖTEBORG
The Queen Silvia Children’s Hospital in Göteborg is one of the leading neuromuscular
centers in Sweden and plays an active role in research, and offers diagnostic work-up
and yearly follow-up programs for children with all kinds of neuromuscular disorders.
ABOUT LUMC
Leiden University Medical Centre (LUMC) aims to play a leading role, nationally and
internationally, in the further improvement of health care quality. LUMC’s key tasks are
research, patient care, and academic and post-academic medical education. It performs
11,500 daytime treatments and 19,000 hospital admissions yearly. It has 800 beds and
employs 8700 people. For more information see www.lumc.nl.
For more information on Prosensa, please contact:
Gerard Platenburg, CEO, g.platenburg@prosensa.nl
For more information on the clinical study, please contact:
Ad Sitsen, CMO, a.sitsen@prosensa.nl
