Prosensa Receives £7.5M Milestone Payment as Part of Its Program with GlaxoSmithKline in Duchenne Muscular Dystrophy

LEIDEN, The Netherlands--(BUSINESS WIRE)-- Prosensa, the Dutch biopharmaceutical company focusing on RNA modulating therapeutics, has announced that it received a £7.5m milestone payment from GlaxoSmithKline (GSK) as a result of achieving a data milestone in its Phase IIa open label extension trial of GSK2402968 (PRO051), being developed to treat Duchenne Muscular Dystrophy (DMD) under its agreement with GSK. The milestone payment was based upon achievement of a successful safety review, with no serious safety signals observed.

GSK2402968 (PRO051) is an investigational antisense oligonucleotide which induces exon skipping of exon 51. The six-month data from the open label Phase IIa extension study in 12 patients with DMD, receiving a weekly 6mg/kg systemic delivery by subcutaneous injection, will be presented at the 15th International Congress of the World Muscle Society in Japan, October 12-16. The data will be presented by Dr. Nathalie Goemans, MD, from the Department of Pediatric Neurology, University of Leuven, Belgium.

Under their collaboration, Prosensa and GSK have several further late-stage clinical trials underway.

“It is with great enthusiasm that we take this compound, and other products to treat DMD, forward with GSK,” said Dr. Giles Campion, Chief Medical Officer of Prosensa. “Our joint dedication to rare diseases is integral to the success of the programme.”

“I am pleased with achieving this important milestone. It is an additional validation and further confirmation of our technology and platform as well as of our excellent collaboration with GSK,” said Hans Schikan, Chief Executive Officer of Prosensa.

Notes to editors:

About Prosensa

Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics correcting gene expression in diseases with large unmet medical needs, in particular neuromuscular disorders. Prosensa’s focus is on developing a treatment for DMD. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline in a licensing deal that could yield up to GBP 428 million plus double-digit royalty payments.

Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital.

For more information about Prosensa, please visit

About DMD and exon skipping

Duchenne Muscular Dystrophy is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 newborn boys. The young patients suffer from progressive loss of muscle strength due to the absence of the protein dystrophin, often making them wheelchair bound before the age of 12. Most patients 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 amongst the most promising therapies in development for DMD. More specifically, antisense oligonucleotides have the capacity to skip an exon and thereby correct the reading frame of DMD transcripts aiming at the synthesis of a novel dystrophin protein. Different mutations in the gene require different oligonucleotide drugs.


Hans Schikan, Chief Executive Officer
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Luc Dochez, Chief Business Officer
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College Hill Life Sciences
Sue Charles/Adam Michael/Anastasios Koutsos
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T: +44 20 7457 2020

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