Prosensa Awarded up to €5m Innovation Credit from Dutch Government for research in Myotonic Dystrophy

Leiden, The Netherlands, 25 October 2011 – Prosensa, the Dutch com­pa­ny focus­ing on RNA mod­u­lat­ing ther­a­peu­tics for rare dis­eases with unmet med­ical needs, announced today that it has received an inno­va­tion cred­it of up to €5m from AgentschapNL, a sub­sidiary of the Dutch Ministry of Economic Affairs, Agriculture and Innovation, to sup­port fur­ther devel­op­ment of a series of pre­clin­i­cal com­pounds for the treat­ment of Myotonic Dystrophy type 1 (DM1).

According to the terms of the agree­ment, Prosensa will receive €940,000 to sup­port the ini­tial stage of the project, with the poten­tial to receive addi­tion­al fund­ing of up to €5m after suc­cess­ful com­ple­tion of spe­cif­ic mile­stones and an ini­tial review in 2012.

The genet­ic muta­tion under­ly­ing DM1 is char­ac­ter­ized by expan­sion of so-called triplet repeats in the DMPK gene lead­ing to tran­scripts with tox­ic effects. Prosensa applies its RNA mod­u­la­tion plat­form to reduce the num­ber of these expand­ed repeat, tox­ic tran­scripts. One com­pound, PRO135, reversed the tran­script tox­ic effects after in vivo intra­mus­cu­lar admin­is­tra­tion in pre­clin­i­cal DM1 mod­els. Prosensa works in close col­lab­o­ra­tion with the depart­ment of Cell Biology, Nijmegen Centre for Molecular Life Sciences, Radboud University Nijmegen Medical Centre, on this project, one of the lead­ing cen­tres in DM1 research in the world.

Judith van Deutekom, Vice President of Discovery at Prosensa com­ment­ed: “We are very pleased with this grant by the Dutch gov­ern­ment, which allows us to accel­er­ate our research in this impor­tant ther­a­peu­tic area and helps us grow into a spe­cial­ist rare dis­ease com­pa­ny. The inno­va­tion cred­it from the Dutch gov­ern­ment is intend­ed to stim­u­late devel­op­ment of tech­no­log­i­cal­ly inno­v­a­tive prod­ucts. In addi­tion to Duchenne mus­cu­lar dys­tro­phy, we are also work­ing on a poten­tial ther­a­py for patients affect­ed by Myotonic Dystrophy, yet anoth­er rare dis­ease for which no treat­ment cur­rent­ly exists.”

Notes to edi­tors:

About Myotonic Dystrophy

Myotonic Dystrophy is a rare, pro­gres­sive, degen­er­a­tive dis­or­der that affects many organs in the body, but pri­mar­i­ly skele­tal mus­cle, heart and brain. It is rec­og­nized as one of the most com­mon forms of mus­cu­lar dys­tro­phy in adults and affects approx­i­mate­ly 1:8,000 indi­vid­u­als world­wide. Typical symp­toms are myoto­nia, gas­tro-intesti­nal prob­lems, res­pi­ra­to­ry insuf­fi­cien­cy and sev­er­al cog­ni­tive prob­lems.

There is cur­rent­ly no effec­tive treat­ment avail­able for DM1 patients and the con­gen­i­tal form (1:20,000 new­borns) leads to a life-long severe dis­abil­i­ty and pre­ma­ture death.

About 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. Prosensa’s cur­rent focus is on devel­op­ing treat­ments for Duchenne mus­cu­lar dys­tro­phy (DMD), Myotonic Dystrophy (DM1) and Huntington’s dis­ease. In 2009 Prosensa entered into a strate­gic alliance for part of its DMD exon skip­ping pro­gram with GlaxoSmithKline. Prosensa’s lead com­pound (GSK2402968/PRO051), being devel­oped by GSK, entered phase III clin­i­cal tri­als in January 2011.

Prosensa is a pri­vate­ly held bio­phar­ma­ceu­ti­cal com­pa­ny, backed by a con­sor­tium of Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital. For more infor­ma­tion about Prosensa, please vis­it www.prosensa.com

 Prosensa recent­ly was short­list­ed for the Biotech Company of the Year cat­e­go­ry at the 2011 Scrip Awards, as well as for the 2011 Most Innovative SME Award by EuropaBio.

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