Prosensa and CureDuchenne Strengthen Long-term Collaboration with an Additional €5M to Advance DMD Pipeline

Leiden, The Netherlands, Aug. 11, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced that an affil­i­ate of CureDuchenne, a US nation­al non­prof­it orga­ni­za­tion ded­i­cat­ed to find­ing a cure for Duchenne Muscular Dystrophy (DMD), will pro­vide Prosensa with up to €5 mil­lion by means of con­vert­ible promis­so­ry notes to sup­port the com­pa­ny and accel­er­ate the devel­op­ment and patient access of much need­ed DMD ther­a­pies. The clos­ing of €4.5 mil­lion of the notes is con­tin­gent upon spec­i­fied mile­stones in the advance­ment of dris­apersen and the Company’s oth­er exon skip­ping can­di­dates in Prosensa’s DMD port­fo­lio.
“The abil­i­ty for indus­try and patient orga­ni­za­tions to work col­lab­o­ra­tive­ly is cru­cial to devel­op­ing much need­ed treat­ment options for rare dis­eases such as DMD,” said Hans Schikan, CEO of Prosensa. “CureDuchenne has been a ded­i­cat­ed sup­port­er of Prosensa since the company’s incep­tion, and we are very appre­cia­tive of the addi­tion­al fund­ing for our exten­sive DMD pro­gram.”
The fund­ing arrange­ment will assist the Company in a num­ber of efforts that are core to Prosensa’s mis­sion of devel­op­ing inno­v­a­tive, RNA-based ther­a­peu­tics to address unmet med­ical needs for patients with rare genet­ic dis­or­ders includ­ing:
• Progressing the sec­ond exon skip­ping can­di­date for the treat­ment of DMD, PRO044, by ini­ti­at­ing a Phase II clin­i­cal exten­sion study in Europe by the end of 2014 and a place­bo-con­trolled tri­al in the US in the first half of 2015, which may serve as one of two con­fir­ma­to­ry stud­ies to sup­port a poten­tial accel­er­at­ed approval for dris­apersen.
• Supporting re-dos­ing efforts for dris­apersen clin­i­cal tri­al par­tic­i­pants in North America and Europe and facil­i­tate the drug’s New Drug Application (NDA) fil­ing in the US in 2014.
• Supporting the devel­op­ment of oth­er Prosensa’s oth­er exon skip­ping com­pounds, PRO045 and PRO053.
Prosensa and CureDuchenne have been col­lab­o­rat­ing since 2004 when CureDuchenne pro­vid­ed valu­able finan­cial sup­port to help advance the company’s exten­sive pipeline of DMD prod­ucts.
“We are very encour­aged by the pio­neer­ing progress that Prosensa has made in Duchenne and are com­mit­ted to the long-stand­ing col­lab­o­ra­tion we have with Prosensa to sup­port the devel­op­ment of treat­ments for boys diag­nosed with this rare and dev­as­tat­ing genet­ic dis­ease,” said Debra Miller, CEO & Founder of CureDuchenne. “This part­ner­ship under­scores the impor­tant role that patient groups play in accel­er­at­ing the research and devel­op­ment for Duchenne and oth­er dis­eases,” she added.
In June Prosensa received pos­i­tive feed­back from the United States Food and Drug Administration (FDA) enabling it to pur­sue an NDA fil­ing for its lead DMD ther­a­py, dris­apersen, under an accel­er­at­ed approval path­way based on exist­ing data. The FDA’s guid­ance also request­ed that the com­pa­ny com­mence a con­fir­ma­to­ry ran­dom­ized, place­bo con­trolled study in a fol­low-on exon skip­ping drug with a sim­i­lar mech­a­nism of action pri­or to a poten­tial approval for dris­apersen. The place­bo con­trolled tri­al with PRO044 antic­i­pat­ed to start in the first half of 2015 may serve this pur­pose.

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