LEIDEN, The Netherlands – June 27, 2014 – Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that Dr. Judith van Deutekom, Prosensa’s Vice President of Drug Discovery, has been awarded a $200,000 research grant from Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne Muscular Dystrophy (DMD), to fund the development of a multi exon-skipping strategy for exons 10-30 of the dystrophin gene.
The project titled “Single AON-induced multiple exon skipping in the mdx mouse model” will focus on obtaining proof-of-concept in the mdx mouse model. The studies are expected to be completed in the second half of 2015.
“We are delighted with PPMD's funding of our project that aims at the pre-clinical development of single antisense oligonucleotides capable of inducing multiple exon skipping in the exon 10 to 30 region of the DMD gene,” commented Dr. van Deutekom. “This would apply to DMD patients with more rare mutations upstream of the deletion hot spot (exons 45-55), which could address up to 13% of DMD patients.”
“Recognizing the need to bring this promising technology to as many with Duchenne as possible, in August 2013 we announced a request for applications focused on the development of antisense oligonucleotides for mutations in the dystrophin gene that were not already in clinical development. Our Scientific Advisory Committee, composed of leading experts in the field, selected the proposal from Prosensa as one of the applications to be funded,” said Sharon Hesterlee, PPMD’s Vice President of Research. “We very much look forward to working with Prosensa to support important clinical research initiatives that could have a significant impact on the health and lives of young boys diagnosed with this rare and devastating genetic disease,” she added.
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, Myotonic dystrophy and Huntington’s disease.
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.
Parent Project Muscular Dystrophy (PPMD) is a not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization’s mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. Only this comprehensive approach will lead to the day that 100% of those diagnosed can turn to a treatment that will end Duchenne. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information, visit www.parentprojectmd.org.
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 multi-exon skipping program, PROSPECT, and grant funding around this program. 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 containing this press release and certain sections of the Company’s Registration Statement 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.
Issued for and on behalf of Prosensa by Instinctif Partners.
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