Prosensa receives £7.5m mile­stone pay­ment as part of its pro­gram with GlaxoSmithKline in Duchenne Muscular Dystrophy

Leiden, October 13, 2010 – 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.

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.

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