Prosensa Provides Update on Drisapersen

Re-dos­ing plans are under­way and 96-week data from an open-label exten­sion study of dris­apersen for the treat­ment of DMD pre­sent­ed at the American Academy of Neurology (AAN)

Leiden, The Netherlands, May 1, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA) the Dutch bio­phar­ma­ceu­ti­cal com­pa­ny focus­ing on rare dis­eases with a high unmet med­ical need, yes­ter­day pro­vid­ed the data analy­sis of its sec­ond open-label exten­sion study (DEMAND IV/DMD114349) of dris­apersen in boys with Duchenne Muscular Dystrophy (DMD) dur­ing the 66th American Academy of Neurology (AAN) Annual Meeting in Philadelphia, PA. The Company also pro­vid­ed an update on the dris­apersen clin­i­cal pro­gram ear­li­er this week in a let­ter sent to patient group rep­re­sen­ta­tives and clin­i­cal inves­ti­ga­tors and will present a cor­po­rate update dur­ing an investor brief­ing on May 1.
Following pos­i­tive feed­back from patients and inves­ti­ga­tors regard­ing the will­ing­ness and desire of patients to go back on dris­apersen and encour­ag­ing analy­ses of its clin­i­cal tri­al data, Prosensa has con­firmed that it will re-dose an ini­tial cohort of boys in the third quar­ter of 2014. The Company has made sig­nif­i­cant progress in the tran­si­tion of the pro­gram from its for­mer part­ner, GSK, and expects to com­mu­ni­cate on a reg­u­la­to­ry path for­ward for dris­apersen before the end of June.
In a poster and pre­sen­ta­tion on Wednesday, April 30 at the AAN, Dr. Nathalie Goemans, Head of the Neuromuscular Reference Center for Children at the University Hospitals Leuven (UHL) in Belgium and one of the key inves­ti­ga­tors in var­i­ous dris­apersen stud­ies con­firmed the ear­li­er pre­lim­i­nary analy­sis of the results of DEMAND IV/DMD114349, which include data from 96 weeks of con­tin­u­ous dris­apersen treat­ment.
“We see very pos­i­tive signs of the abil­i­ty of dris­apersen to improve the walk­ing abil­i­ty of boys with DMD. In this exten­sion study, patients who entered the study with a less advanced stage of the dis­ease either improved or expe­ri­enced less decline in mobil­i­ty with two-year con­tin­u­ous dris­apersen ther­a­py, and these data sug­gest a treat­ment dif­fer­ence from those ini­tial­ly on place­bo,” report­ed Dr. Goemans.
The poster (online here) includ­ed detailed data up to Week 48 (total of 96 weeks of treat­ment) from the sec­ond open-label exten­sion study of dris­apersen in 113 boys with DMD (DEMAND IV), who had pre­vi­ous­ly com­plet­ed a 48-week, dou­ble-blind, place­bo-con­trolled treat­ment phase in one of two feed­er stud­ies (DEMAND II/DMD114117 and DEMAND III/DMD114044). In the six minute walk test (6MWT), boys from the DEMAND II study declined by only 5 meters over 96 weeks on con­tin­u­ous dris­apersen (6mg/kg/week) com­pared with 57 meters in boys who had placebo/delayed treat­ment. Furthermore, boys of 7 years or younger in DEMAND IV improved from their base­line 6MWT by 8 meters with con­tin­u­ous dris­apersen treat­ment com­pared with a 29 meter decline for patients in the placebo/delayed treat­ment arm. The exten­sion study was designed to mon­i­tor the long-term safe­ty and effi­ca­cy of dris­apersen over the course of two years at more than 50 sites in 24 coun­tries.
“These data sup­port our hypoth­e­sis that treat­ing ear­li­er in the dis­ease and treat­ing for a longer dura­tion con­fers a treat­ment ben­e­fit for boys with DMD. Early inter­ven­tion is a poten­tial­ly cru­cial com­po­nent to improv­ing dis­ease out­come,” said Dr. Giles Campion, Chief Medical Officer of Prosensa.
In a let­ter to all par­tic­i­pat­ing inves­ti­ga­tors and patient groups sent ear­li­er this week, Prosensa con­firmed it is on track with its re-dos­ing plans, which will take a staged approach. Dr. Nathalie Goemans (UHL, Belgium) and Dr. Craig McDonald (UC Davis, Sacramento, USA), who will be par­tic­i­pat­ing at an investor brief­ing on May 1, will be amongst inves­ti­ga­tors work­ing with Prosensa to under­take re-dos­ing pro­to­cols with dris­apersen. The first wave of re-dos­ing in the third quar­ter will focus on sites in North America and Europe. Patients in North America will be re-dosed under exist­ing exten­sion pro­to­cols and patients in Europe will either par­tic­i­pate in a new treat­ment pro­to­col or via an appro­pri­ate expand­ed access pro­gram. “We real­ize that this has been an incred­i­bly dif­fi­cult peri­od for the boys and their fam­i­lies as they eager­ly await any updates. We are work­ing with all key stake­hold­ers as dili­gent­ly and quick­ly as pos­si­ble to pro­vide access to dris­apersen and to deter­mine a path for­ward” con­firmed Dr. Campion.
“Prosensa is ded­i­cat­ed to enabling long term patient access to dris­apersen and its fol­low-on prod­ucts as nov­el treat­ments for DMD. We are deter­mined to accom­plish this as soon as pos­si­ble, as we know time is not on patients’ side” said Hans Schikan, Prosensa’s Chief Executive Officer. “We have been fol­low­ing the recent com­mu­ni­ca­tion in the DMD com­mu­ni­ty and are encour­aged by the appar­ent reg­u­la­to­ry flex­i­bil­i­ty and pos­i­tiv­i­ty shown with respect to poten­tial path­ways for inves­ti­ga­tion­al prod­ucts for the treat­ment of DMD, espe­cial­ly giv­en the very urgent and unmet med­ical need of boys with this debil­i­tat­ing and life-short­en­ing dis­ease. We are heart­ened by the FDA’s com­mit­ment to DMD and its will­ing­ness to work close­ly with all key stake­hold­ers, and to con­sid­er amenable approach­es to expe­dite the avail­abil­i­ty of treat­ment options for DMD.”

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