Prosensa and GlaxoSmithKline ini­ti­ate devel­op­ment of four addi­tion­al prod­ucts under exist­ing alliance in Duchenne Muscular Dystrophy – Broadened pro­gram marks key inflex­ion in Prosensa’s progress to a ful­ly inte­grat­ed spe­cial­ty-phar­ma

Leiden, June 23, 2010 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA mod­u­lat­ing ther­a­peu­tics, announces the ini­ti­a­tion of two fur­ther pro­grammes under its exist­ing alliance with GSK cov­er­ing nov­el RNA-based treat­ments for Duchenne Muscular Dystrophy (DMD). The ini­ti­a­tion of these addi­tion­al pro­grams under the terms of the exist­ing alliance agree­ment is a val­i­da­tion of both the poten­tial of Prosensa’s “exon skip­ping” plat­form and the ongo­ing rela­tion­ship.

Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA mod­u­lat­ing ther­a­peu­tics, announces the ini­ti­a­tion of two fur­ther pro­grammes under its exist­ing alliance with GSK cov­er­ing nov­el RNA-based treat­ments for Duchenne Muscular Dystrophy (DMD). The ini­ti­a­tion of these addi­tion­al pro­grams under the terms of the exist­ing alliance agree­ment is a val­i­da­tion of both the poten­tial of Prosensa’s “exon skip­ping” plat­form and the ongo­ing rela­tion­ship.

The two new pro­grams are includ­ed under the exist­ing alliance agree­ment between both par­ties and will address the devel­op­ment of four com­pounds which tar­get dif­fer­ent sub­pop­u­la­tions of patients suf­fer­ing from DMD. To access these new pro­grams, GSK has made two ini­ti­a­tion pay­ments to Prosensa and Prosensa becomes eli­gi­ble for fur­ther pre-option mile­stone pay­ments based on research progress. Within these new pro­grams, Prosensa and GSK will focus on the skip­ping of four exons (i.e. exon 45, 52, 53, and 55), in addi­tion to their exist­ing pro­grams which tar­get skip­ping of exon 51 and 44 (PRO051/GSK2402968 and PRO044). The ini­ti­a­tion of these pro­grams con­firms the joint com­mit­ment of both com­pa­nies to find treat­ments for DMD.

Under the terms of the col­lab­o­ra­tion, GSK has an option to select two of these addi­tion­al four com­pounds for lat­er-stage devel­op­ment and com­mer­cial­iza­tion. Prosensa will retain cer­tain lim­it­ed European com­mer­cial­iza­tion rights along­side GSK for the two com­pounds select­ed by GSK. For the two com­pounds not select­ed by GSK, Prosensa will retain full com­mer­cial­iza­tion rights.

“We are very pleased with this news. Prosensa and GSK’s com­mit­ment to progress fur­ther devel­op­ments of addi­tion­al prod­ucts that can pro­vide for a solu­tion in DMD is encour­ag­ing and wel­comed by all of us” said Elizabeth Vroom, Chair of the United Parent Project Muscular Dystrophy, which unites dif­fer­ent par­ent project orga­ni­za­tions set up by par­ents of chil­dren with DMD in many coun­tries all over the world. “The expan­sion of our DMD port­fo­lio with four addi­tion­al com­pounds is a great step for­ward in our efforts to devel­op safe and effec­tive treat­ments for this severe­ly debil­i­tat­ing dis­ease and to help as many patients as pos­si­ble,” said Hans Schikan, Chief Executive Officer of Prosensa, adding, “Thanks to the suc­cess­ful progress in our exist­ing pro­gram with GSK, we are now able to extend our pro­duc­tive col­lab­o­ra­tion with these four fur­ther com­pounds. With Prosensa retain­ing full com­mer­cial­iza­tion rights to two of these exon skip­ping com­pounds and any oth­er prod­ucts which are not part of the alliance with GSK, this set-up
enables us to devel­op into a ful­ly inte­grat­ed spe­cial­ty-phar­ma com­pa­ny.”

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