Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy

Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, has select­ed clin­i­cal can­di­dates for two more com­pounds for the treat­ment of Duchenne mus­cu­lar dys­tro­phy (DMD) and has been grant­ed orphan drug des­ig­na­tion for two addi­tion­al DMD com­pounds in its pipeline.
Prosensa iden­ti­fied suit­able oligonu­cleotide can­di­dates for PRO052 and PRO055, designed for the skip­ping of exons 52 and 55 of the dys­trophin gene. PRO052 and PRO055 are cur­rent­ly in pre-clin­i­cal devel­op­ment and will be moved to clin­i­cal tri­als as soon as pos­si­ble.
Prosensa has also received orphan drug des­ig­na­tion by the European Commission for PRO045 and PRO053. These com­pounds are designed to skip exons 45 and 53 of the dys­trophin gene and are expect­ed to enter clin­i­cal devel­op­ment with­in the next 6 months. In par­al­lel, Prosensa and GSK have ini­ti­at­ed a large glob­al nat­ur­al his­to­ry study in order to gen­er­ate impor­tant data on the progress of DMD that will help to facil­i­tate the devel­op­ment path­ways for these com­pounds.
Together with dris­apersen (PRO051/GSK2402968) and PRO044, already in clin­i­cal devel­op­ment, the Prosensa port­fo­lio of six DMD com­pounds has the poten­tial to treat more than 40% of all DMD patients.
Hans Schikan, CEO of Prosensa, com­ment­ed: “Achieving orphan drug sta­tus for PRO045 and PRO053 is an impor­tant mile­stone that we believe will allow us to accel­er­ate devel­op­ment of these com­pounds and ini­ti­ate clin­i­cal tri­als with­in the next half year. While GSK has an option to license one com­pound, PRO045 or PRO053, the oth­er com­pound will be devel­oped and even­tu­al­ly com­mer­cial­ized by Prosensa in our ambi­tion to become a ful­ly inte­grat­ed bio­phar­ma­ceu­ti­cal play­er in rare dis­eases. Together with PRO052 and PRO055, where the same prin­ci­ple applies, Prosensa will be in a posi­tion to tar­get rare muta­tions in spe­cif­ic DMD sub­pop­u­la­tions and may pro­vide treat­ment to more patients with this debil­i­tat­ing dis­ease.”
Prosensa has the most advanced port­fo­lio of drug can­di­dates for the treat­ment of DMD, and has also pre­clin­i­cal com­pounds for Myotonic Dystrophy and Huntington’s Disease. Prosensa’s DMD com­pounds are based on its pro­pri­etary exon-skip­ping tech­nol­o­gy that uses anti­sense oligonu­cleotides to restore expres­sion of a func­tion­al dys­trophin pro­tein and to pro­vide poten­tial treat­ment for patients affect­ed by this pro­gres­sive­ly debil­i­tat­ing neu­ro­mus­cu­lar dis­ease.

Leave a Reply

Your email address will not be published. Required fields are marked *