DGAP-News: Prosensa Announces 1st Quarter 2014 Financial Results and Recent Corporate Developments

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Regulatory Discussions Ongoing; Drisapersen Re-Dosing to Start in Q3

LEIDEN, The Netherlands, 2014-05-20 13:00 CEST (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 reported financial results for the first quarter ending March 31, 2014 and provided an update on the next steps for its exon-skipping platform for the treatment of Duchenne Muscular Dystrophy (DMD).

Hans Schikan, CEO of Prosensa said, 'We have made excellent progress during this period, and are very pleased that we are now in a position to commence re-dosing with drisapersen of boys that have previously participated in drisapersen trials beginning in the third quarter of 2014. In addition, our 250-patient Natural History study, designed to better understand the disease progression of DMD, has almost completed enrollment.'

'We are encouraged by our ongoing interactions with the regulatory authorities, in both the United States and Europe, and remain on track to communicating a potential regulatory path forward for drisapersen by the end of June,' he added. 'Prosensa is dedicated to enabling long term patient access to drisapersen and its follow-on candidates as novel treatments for DMD. We are determined to accomplish this as soon as possible.'

Recent Corporate Highlights

-- Updates on the Drisapersen Development Program

-- On January 13, we announced that we regained the rights to drisapersen from GSK and retained the rights to all other programs for the treatment of DMD. Prosensa now has full, unencumbered rights to continue the development of drisapersen in addition to each of our other DMD programs. Since then, we have made substantial progress in the transfer of the drisapersen program from GSK.

-- On March 17, encouraging 48-week data from Prosensa's U.S. Phase II placebo-controlled study of drisapersen for the treatment of boys with DMD (DEMAND V, DMD114876) were presented by the principal investigator, Craig M. McDonald, M.D., Professor and Chair of Physical Medicine & Rehabilitation and Professor of Pediatrics at the University of California, Davis School of Medicine, at the Muscular Dystrophy Association 2014 Clinical Conference in Chicago, Illinois (March 16-19). The results of this 51 patient-study indicated that, compared to placebo, boys in the higher-dose drisapersen group (6 mg/kg once weekly) experienced stabilization and even improvements in their muscle function and physical activity as measured by the six-minute walk test (6MWT) for the 24-week treatment phase and maintained this improvement during the 24-week no-treatment follow-up period. Additionally, when evaluating the percent-predicted six-minute walk distance (6MWD), a clinically meaningful treatment difference of 5.2% was observed at week 24 and 4.8% at week 48.

-- On April 30, Dr. Nathalie Goemans, Head of the Neuromuscular Reference Center for Children at the University Hospitals Leuven (UHL) in Belgium and a key investigator in the drisapersen clinical program presented 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, DMD114349), 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), during the 66th American Academy of Neurology (AAN) Annual Meeting in Philadelphia, PA. The data are supportive of the hypothesis that treating earlier in the disease and treating for a longer duration confers a treatment benefit for boys with DMD.

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DGAP-News: Prosensa Announces 1st Quarter 2014 Financial Results and Recent Corporate Developments