Following positive feedback from patients and investigators regarding the willingness and desire of patients to go back on drisapersen and encouraging analyses of its clinical trial data, Prosensa has confirmed that it will re-dose an initial cohort of boys in the third quarter of 2014. The Company has made significant progress in the transition of the program from its former partner, GSK, and expects to communicate on a regulatory path forward for drisapersen before the end of June.
In a poster and presentation on
"We see very positive signs of the ability of drisapersen to improve the walking ability of boys with DMD. In this extension study, patients who entered the study with a less advanced stage of the disease either improved or experienced less decline in mobility with two-year continuous drisapersen therapy, and these data suggest a treatment difference from those initially on placebo," reported Dr. Goemans.
The poster (online here) included detailed data up to Week 48 (total of 96 weeks of treatment) from the second open-label extension study of drisapersen in 113 boys with DMD (DEMAND IV), who had previously completed a 48-week, double-blind, placebo-controlled treatment phase in one of two feeder studies (DEMAND II/DMD114117 and DEMAND III/DMD114044). In the six minute walk test (6MWT), boys from the DEMAND II study declined by only 5 meters over 96 weeks on continuous drisapersen (6mg/kg/week) compared with 57 meters in boys who had placebo/delayed treatment. Furthermore, boys of 7 years or younger in DEMAND IV improved from their baseline 6MWT by 8 meters with continuous drisapersen treatment compared with a 29 meter decline for patients in the placebo/delayed treatment arm. The extension study was designed to monitor the long-term safety and efficacy of drisapersen over the course of two years at more than 50 sites in 24 countries.
"These data support our hypothesis that treating
earlier in the disease and treating for a longer duration confers a
treatment benefit for boys with DMD. Early intervention is a
potentially crucial component to improving disease outcome," said
In a letter to all participating investigators
and patient groups sent earlier this week, Prosensa confirmed it is
on track with its re-dosing plans, which will take a staged
"Prosensa is dedicated to enabling long term
patient access to drisapersen and its follow-on products as novel
treatments for DMD. We are determined to accomplish this as soon as
possible, as we know time is not on patients' side" said Hans
Schikan, Prosensa's Chief Executive Officer. "We have been
following the recent communication in the DMD community and are
encouraged by the apparent regulatory flexibility and positivity
shown with respect to potential pathways for investigational
products for the treatment of DMD, especially given the very urgent
and unmet medical need of boys with this debilitating and
life-shortening disease. We are heartened by the
About drisapersen and the clinical development program
Drisapersen, (previously GSK2402968/PRO051), an
antisense oligonucleotide which induces exon skipping of exon 51,
is currently in-late stage development for DMD. Drisapersen has
orphan drug status in the EU, US,
The overall drisapersen clinical program comprises three double-blind, placebo-controlled studies (DEMAND II, DEMAND V and DEMAND III) and two long term open-label extension studies (DMD114673 and DEMAND IV).
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, myotonic dystrophy and Huntington's disease.
Forward Looking Statements
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