Prosensa Completes Enrollment in Natural History Study of Duchenne Muscular Dystrophy

Leiden, The Netherlands, June 25, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on RNA-mod­u­lat­ing ther­a­peu­tics for rare dis­eases with high unmet need, today announced the suc­cess­ful com­ple­tion of enroll­ment in its prospec­tive Natural History Study designed to increase the under­stand­ing of Duchenne mus­cu­lar dys­tro­phy (DMD).
The study enrolled in total 269 boys with con­firmed DMD between the ages three and 18 from 10 coun­tries across 16 cen­ters in North & South America and Europe, 80% of whom are ambu­la­to­ry and 20% non-ambu­la­to­ry.
The pur­pose of the study is to char­ac­ter­ize the nat­ur­al his­to­ry and pro­gres­sion of DMD, which will help inform the design of future stud­ies, to cap­ture bio­mark­ers of safe­ty and dis­ease pro­gres­sion, and to pro­vide com­par­a­tive data for patients with rare exon deletions/mutations for which for­mal con­trolled tri­als are not fea­si­ble. No med­ica­tion or device is being test­ed in this study. Moreover, the study will be impor­tant in the con­fir­ma­to­ry pro­gram for Prosensa’s lead DMD com­pound dris­apersen, for which a New Drug Application is expect­ed to be sub­mit­ted lat­er this year to the FDA under an Accelerated Approval path­way.
Dr. Brenda Wong of Cincinnati Children’s Hospital, who has enrolled patients in the study said, “While we are pleased by the high lev­el of world-wide atten­tion and com­mit­ment by reg­u­la­tors giv­en to DMD, an exist­ing obsta­cle for deliv­er­ing promis­ing treat­ments to patients relies on accept­ed clin­i­cal end­points for human stud­ies. By col­lect­ing and ana­lyz­ing this Natural History Study data from hun­dreds of boys, we are bet­ter equipped to expe­dite the devel­op­ment of promis­ing ther­a­pies and offer them to DMD patients anx­ious­ly await­ing treat­ment options to slow dis­ease pro­gres­sion and improve the out­come of this dis­ease.”
“We con­tin­ue to work dili­gent­ly to pio­neer research and devel­op­ment in DMD, and com­plet­ing enroll­ment in our Natural History Study is a major mile­stone and a crit­i­cal com­po­nent of basic research to under­stand the dis­ease,” said Dr. Giles Campion, Prosensa’s Chief Medical Officer. “This study will be a cru­cial resource for our con­fir­ma­to­ry stud­ies with dris­apersen and for our fol­low-on exon skip­ping pro­grams. Outcomes of this study will con­tribute to a grow­ing body of sci­en­tif­ic knowl­edge and are fun­da­men­tal to our abil­i­ty to devel­op inno­v­a­tive treat­ment options for boys who suf­fer from this dev­as­tat­ing dis­ease.”
All boys par­tic­i­pat­ing in the study will be fol­lowed for a peri­od of three years, with site vis­its and assess­ments every 6 months. During these vis­its, inves­ti­ga­tors will eval­u­ate the boys on a num­ber of phys­i­cal tests, includ­ing the six minute walk test, climb­ing stairs, breath­ing in a tube to mea­sure lung func­tion and per­form­ing upper limb move­ments. These para­me­ters are meant to assess how the dis­ease affects their over­all qual­i­ty of life as the con­di­tion evolves over time in addi­tion to con­tribut­ing to the under­stand­ing of the nat­ur­al pro­gres­sion of the dis­ease. Furthermore, blood and urine sam­ples will be tak­en to inves­ti­gate a pan­el of bio­mark­ers that may be use­ful in fol­low­ing dis­ease pro­gres­sion.
The study, which began in September 2012 and is spon­sored by Prosensa, is expect­ed to com­plete in November 2017. This autumn, 80 boys will have com­plet­ed one year of obser­va­tion and this inter­im analy­sis will be avail­able lat­er this year. Further infor­ma­tion on the study can be found here: – Study ID: NCT01753804

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