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LEIDEN, The Netherlands, Aug. 28, 2013 (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 quarter ended June 30, 2013.
"We had a transformative second quarter, culminating with our successful IPO at the end of June, raising $89.7M. On July 3, 2013, the date we received our IPO proceeds, our cash and cash equivalents amounted to €94.3 million ($122.2 million), providing a substantial base to support the development of our portfolio especially our rapidly advancing Duchenne muscular dystrophy (DMD) product candidates. We were also very pleased with the results of the phase II DMD114117 double-blind, placebo-controlled study with drisapersen, which we exclusively licensed to GlaxoSmithKline (GSK). The study, which met its primary endpoint, was presented by GSK at the Cold Spring Harbor Conference in April 2013," said Hans Schikan, CEO of Prosensa.
Mr. Schikan continued, "We look forward to an incredibly busy and exciting remainder of the year, particularly as phase III data will become available for drisapersen. To date, more than 300 patients have participated in clinical studies with drisapersen at more than 50 trial sites in 25 countries. We believe that drisapersen, which was recently granted 'Breakthrough Therapy Designation' by the U.S. Food and Drug Administration, may potentially offer DMD patients with an amenable mutation an important, much-needed treatment option and we are dedicated to advancing the rest of our portfolio to help other sub-populations of boys with this devastating neuromuscular disease."
Prosensa's current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union. The compounds utilize an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients.