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 Paediatric 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.

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.