Prosensa and CureDuchenne Strengthen Long-term Collaboration with an Additional €5M to Advance DMD Pipeline

Leiden, The Netherlands, Aug. 11, 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 an affiliate of CureDuchenne, a US national nonprofit organization dedicated to finding a cure for Duchenne Muscular Dystrophy (DMD), will provide Prosensa with up to €5 million by means of convertible promissory notes to support the company and accelerate the development and patient access of much needed DMD therapies. The closing of €4.5 million of the notes is contingent upon specified milestones in the advancement of drisapersen and the Company’s other exon skipping candidates in Prosensa’s DMD portfolio.
“The ability for industry and patient organizations to work collaboratively is crucial to developing much needed treatment options for rare diseases such as DMD,” said Hans Schikan, CEO of Prosensa. “CureDuchenne has been a dedicated supporter of Prosensa since the company’s inception, and we are very appreciative of the additional funding for our extensive DMD program.”
The funding arrangement will assist the Company in a number of efforts that are core to Prosensa’s mission of developing innovative, RNA-based therapeutics to address unmet medical needs for patients with rare genetic disorders including:
• Progressing the second exon skipping candidate for the treatment of DMD, PRO044, by initiating a Phase II clinical extension study in Europe by the end of 2014 and a placebo-controlled trial in the US in the first half of 2015, which may serve as one of two confirmatory studies to support a potential accelerated approval for drisapersen.
• Supporting re-dosing efforts for drisapersen clinical trial participants in North America and Europe and facilitate the drug’s New Drug Application (NDA) filing in the US in 2014.
• Supporting the development of other Prosensa’s other exon skipping compounds, PRO045 and PRO053.
Prosensa and CureDuchenne have been collaborating since 2004 when CureDuchenne provided valuable financial support to help advance the company’s extensive pipeline of DMD products.
“We are very encouraged by the pioneering progress that Prosensa has made in Duchenne and are committed to the long-standing collaboration we have with Prosensa to support the development of treatments for boys diagnosed with this rare and devastating genetic disease,” said Debra Miller, CEO & Founder of CureDuchenne. “This partnership underscores the important role that patient groups play in accelerating the research and development for Duchenne and other diseases,” she added.
In June Prosensa received positive feedback from the United States Food and Drug Administration (FDA) enabling it to pursue an NDA filing for its lead DMD therapy, drisapersen, under an accelerated approval pathway based on existing data. The FDA’s guidance also requested that the company commence a confirmatory randomized, placebo controlled study in a follow-on exon skipping drug with a similar mechanism of action prior to a potential approval for drisapersen. The placebo controlled trial with PRO044 anticipated to start in the first half of 2015 may serve this purpose.

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