Prosensa Advances Three Exon Skipping Candidates for Duchenne Muscular Dystrophy into the Next Development Stage

Prosensa to receive up to £27M in devel­op­ment and mile­stone pay­ments from GSK
Leiden, The Netherlands, 13 September 2011 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on rare dis­eases with an unmet med­ical need, announced today that they have agreed with GlaxoSmithKline (GSK) to advance three fur­ther exon skip­ping com­pounds (PRO044, PRO045 and PRO053) into the next devel­op­ment stage under their ongo­ing col­lab­o­ra­tion rela­tion­ship in Duchenne Muscular Dystrophy (DMD).

The two com­pa­nies have agreed upon the devel­op­ment terms for the exon 44 skip­ping pro­gramme, which sees GSK pro­vid­ing addi­tion­al sup­port for PRO044, cur­rent­ly in Phase I/II. Prosensa and GSK will also work togeth­er on a pre­clin­i­cal and clin­i­cal devel­op­ment pro­gram for PRO045 and PRO053, which are expect­ed to enter clin­i­cal tri­als in the first half of 2012. The three can­di­dates PRO044, PRO045 and PRO053 are com­pounds designed to skip exons 44, 45 and 53 respec­tive­ly, in the dys­trophin gene, each tar­get­ing dif­fer­ent sub­pop­u­la­tions of DMD patients.
Under the col­lab­o­ra­tion agree­ment, Prosensa is eli­gi­ble for mile­stone pay­ments and devel­op­ment fund­ing pay­ments which could total up to £27M, depend­ing upon the course and out­come of the clin­i­cal devel­op­ment for these pro­grams. Prosensa and GSK will also embark on a nat­ur­al his­to­ry study that will pro­vide a bet­ter under­stand­ing of the dis­ease course of DMD.
Under the terms of the alliance estab­lished in 2009, GSK has an exclu­sive option to licence PRO044 and an option to licence one of either PRO045 or PRO053 for lat­er stage devel­op­ment and com­mer­cial­i­sa­tion, with Prosensa retain­ing cer­tain lim­it­ed com­mer­cial­i­sa­tion rights. For the com­pound not select­ed by GSK, Prosensa will retain full devel­op­ment and com­mer­cial­i­sa­tion rights.
“In addi­tion to the progress made with our lead com­pound tar­get­ing exon 51 (GSK2402968/PRO051) which is cur­rent­ly being inves­ti­gat­ed in a glob­al phase III study, the advance­ment of these three addi­tion­al can­di­dates under­lines the com­mit­ment of both par­ties to devel­op safe and effec­tive com­pounds for the treat­ment of DMD,” com­ment­ed Hans Schikan, Chief Executive Officer of Prosensa, adding, “The advance­ment of com­pounds tar­get­ing exons 44, 45 and 53 will allow us to use our exon skip­ping approach for the ben­e­fit of addi­tion­al patients affect­ed by this rare, pro­gres­sive dis­ease. It illus­trates our ambi­tion to help as many patients as soon as pos­si­ble.”
Giles Campion, Prosensa’s Chief Medical Officer, said: “Prosensa has devel­oped an ambi­tious plan to accel­er­ate the devel­op­ment of a port­fo­lio of ther­a­peu­tic agents for rare dis­eases with high unmet med­ical need. Our col­lab­o­ra­tion with GSK allows us to now faster inves­ti­gate addi­tion­al treat­ments for more DMD patients.”

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