Prosensa announces com­mence­ment of re-dos­ing of dris­apersen in North America in patients with Duchenne mus­cu­lar dys­tro­phy

Leiden, The Netherlands, Sept. 17, 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 a com­pre­hen­sive pro­gram of re-dos­ing has com­menced, with the first patients now re-dosed in the United States. All dos­ing in the dris­apersen clin­i­cal pro­gram had been placed on hold by GSK on September 20, 2013, upon announce­ment of the DEMAND III study results.
The re-dos­ing pro­gram in North America will include up to 72 patients across 14 sites who had par­tic­i­pat­ed in the dris­apersen DEMAND V (Phase II) & DEMAND III (Phase III) stud­ies. Prosensa regained the rights to dris­apersen from its pre­vi­ous part­ner GSK in January 2014, and soon there­after com­mit­ted to re-dose patients as quick­ly as pos­si­ble, accept­ing that the trans­fer of data and clin­i­cal mate­ri­als, and the re-engage­ment with clin­i­cal sites would be a com­plex process.
“We are very pleased that we have been able to keep our promise to com­mence re-dos­ing patients with dris­apersen in the third quar­ter of this year, which is tak­ing a staged approach,” said Dr. Giles Campion, Prosensa’s Chief Medical Officer. “While we know the wait has been incred­i­bly dif­fi­cult for the boys (and their fam­i­lies), we have been work­ing dili­gent­ly on this achieve­ment since regain­ing the rights to dris­apersen and are grate­ful to all that have helped us in attain­ing this goal. While today is a major mile­stone for the Company, it is an even greater one for these first patients and their fam­i­lies, who have been anx­ious­ly await­ing dris­apersen treat­ment since dos­ing was stopped almost one year ago” he added.
Drisapersen is admin­is­tered sub­cu­ta­neous­ly, with once week­ly dos­ing. Prosensa is aware of the bur­den that clin­i­cal tri­als can place on the fam­i­lies, and in this pro­to­col efforts have been made to alle­vi­ate bur­den and facil­i­tate par­tic­i­pa­tion. Prosensa aims to enable home-dos­ing where fea­si­ble, and cost-free trans­port with straight­for­ward logis­tics for clin­i­cal cen­ters, through col­lab­o­ra­tion with Greenphire and the Medical Research Network (MRN).
Greenphire is the industry’s lead­ing provider of clin­i­cal pay­ment tech­nol­o­gy and has made its pro­pri­etary ClinCard System avail­able for patients in the US and Canada. The ClinCard sys­tem with Travel Module will help Prosensa man­age the com­plex trav­el arrange­ments required for the patients involved in this study, which will main­tain a tru­ly patient-cen­tric envi­ron­ment. Patients, along with their care­givers and fam­i­ly mem­bers, can make use of Greenphire’s expert trav­el ser­vices to arrange trav­el for expect­ed study vis­its with min­i­mal out-of-pock­et costs. Similarly, Greenphire’s logis­ti­cal sup­port allows clin­i­cian inves­ti­ga­tors to focus their time and atten­tion where it’s need­ed: patient care and study exe­cu­tion.
MRN is the world’s lead­ing provider of home health­care for patients in clin­i­cal tri­als and will be mak­ing home-dos­ing options avail­able to patients in the study.
“Given the week­ly dos­ing reg­i­men for dris­apersen and the trav­el time and costs asso­ci­at­ed with these, we are pleased to col­lab­o­rate with both Greenphire and MRN to ease the bur­den of this process to patients and their fam­i­lies, said Hans Schikan, Prosensa’s Chief Executive Officer. “While mak­ing treat­ment options avail­able for DMD patients glob­al­ly is our num­ber one pri­or­i­ty, we also want to do this in a man­ner that is as seam­less as pos­si­ble for patients and their fam­i­lies.”
Prosensa is also in in prepa­ra­tions for sim­i­lar pro­grams of re-dos­ing and sup­port for all pre­vi­ous­ly treat­ed dris­apersen patients.
In June, Prosensa announced that the United States Food and Drug Administration (FDA) out­lined a reg­u­la­to­ry path for­ward for dris­apersen, under an accel­er­at­ed approval path­way, based upon exist­ing data, includ­ing the DEMAND II study, which was recent­ly pub­lished in the Lancet Neurology. Prosensa remains on track to pur­su­ing reg­u­la­to­ry fil­ings for dris­apersen, ini­tial­ly in the US and Europe, with an FDA sub­mis­sion planned before the end of the year and an EMA sub­mis­sion short­ly there­after.

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