Prosensa and CureDuchenne Strengthen Long-term Collaboration with an Additional [Eur]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.

About Prosensa Holding N.V.

Prosensa (NASDAQ: RNA) is a Dutch biotechnology company engaged in the discovery and development of RNA-modulating therapeutics for the treatment of genetic disorders. Its primary focus is on rare neuromuscular and neurodegenerative disorders with a large unmet medical need, including Duchenne muscular dystrophy (DMD), myotonic dystrophy and Huntington's 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 use an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients.

About CureDuchenne

CureDuchenne is a US national nonprofit organization located in Newport Beach, California, dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide.

CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne's distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have made their way into human clinical trials - a unique accomplishment as few health-related nonprofits have been as successful in being a catalyst for human clinical trials.

About DMD

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects up to 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small segments of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot be processed past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to, or containing, the fault and thereby correct the RNA processing, enabling the production of a novel, largely functional dystrophin protein. Prosensa's exon skipping technology was licensed from Leiden University Medical Center.

About PRO044

PRO044 induces exon 44 skipping in the dystrophin gene and is intended for up to approximately 6% of all DMD patients, including those with deletions of exon 43, exon 45, exons 38-43, exons 40-43, exons 42-43, and exons 45-54. PRO044 has been granted orphan drug status in the European Union and the United States.

PRO044 has completed a Phase I/II dose-escalation study in Europe. Data from this study were presented at the World Muscle Society Congress in October 2013. PRO044 was generally well-tolerated up to dose levels of 12mg/kg for five weeks by subcutaneous or intravenous administration. Safety findings of the study are consistent with the known class safety profile and no drug related serious adverse events were reported.

About the notes

A total of €5.0 million of notes may be issued under a note purchase agreement executed on August 11, 2014. A €0.5 million note will be issued initially, and the remaining €4.5 million of notes will be issued if and when specified milestones are met. The notes bear interest at a below-market interest rate that accumulates to the principal amount of the note. The notes must be repaid on fixed terms on the earliest to occur of a change of control, twelve months from when the Company obtains regulatory approval of its first product candidate and June 30, 2019. CureDuchenne has the option, under specified conditions, to convert the notes into the Company's ordinary shares prior to maturity at a conversion price based on the Company's share price at the time of conversion.

Forward Looking Statement

This press release contains certain forward-looking statements. All statements, other than statements of historical facts, contained in this press release, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements around our exon skipping programs, drisapersen, PRO044, PRO045, PRO053, funding around these programs and the regulatory review of our product candidates. Actual results may differ materially from those projected or implied in such forward-looking statements. Such forward-looking information involves risks and uncertainties that could significantly affect expected results. These risks and uncertainties are discussed in the Company's SEC filings, including, but not limited to, the Company's Form 6-K's and the Company's Annual Report on Form 20-F. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.

CONTACT: Prosensa Holding N.V. Celia Economides, Senior Director IR & Corporate Communications Phone: +1 917 941 9059 Email:

Source:Prosensa Holding N.V.