LEIDEN, The Netherlands – 9 September 2014 – Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that it will be presenting 12 abstracts at the 19th International Congress of the World Muscle Society (WMS), to be held in Berlin, Germany from October 7-11, 2014.
Dr. Giles Campion, Prosensa’s Chief Medical Officer and co-author of several presentations, said “The wide range and high quality data reported across twelve presentations at WMS, including biomarkers to measure disease progression, clinical results, and our pre-clinical work on multi-exon skipping, which could address the rarer mutations in the dystrophin gene, highlights the scientific rigor and pioneering spirit of our outstanding scientists and collaborators. We will continue to work together to deliver much needed therapies as quickly and efficiently for boys with DMD.”
A schedule highlighting Prosensa’s abstracts at WMS 2014 follows:
Oral Presentations
In the session “Muscular Dystrophies” on Thursday 9 October from 11:00:
• G.O.10 “Skeletal muscle, cardiac, and pulmonary imaging biomarkers of disease activity in boys with Duchenne muscular dystrophy”
Authors: A. Mankodi; R. Janiczek; M. Froeling; N. Azzabou; L. Gaur; D. Stock; R. Evers; C. Bishop; L. Yao; C. Grunseich; A. Arai; P. Carlier
• G.O.11 “Longitudinal quantitative muscle MRI in 5 Duchenne boys treated with exon 51 skipping – a pilot study”
Authors: M.T. Hooijmans; B. HA. Wokke; N. Goemans; G. Campion; J.G.M. Verschuuren; EH. Niks; H.E. Kan
In the session ”Treatment approaches in the clinic” on Saturday 11 October from 9:00:
• G.O.23 “Drisapersen: An overview of the clinical programme to date in Duchenne Muscular Dystrophy (DMD)”
Authors: CM. McDonald; E. Mercuri; N. Goemans; T. Voit; R. Wilson; C. Wardell; G. Campion
Poster Presentations
In the session “DMD 1 experimental and animal approaches” on Wednesday 8 October from 14:30:
• G.P.86 “Digital droplet RT-PCR for the absolute quantification of exon skipping induced by antisense oligonucleotides in (pre-)clinical development for Duchenne muscular dystrophy”
Authors: I.G.M. Kolfschoten; R.C. Verheu; J.C.T. van Deutekom; N.A. Datson
• G.P.87 “Prospects for single antisense oligonucleotide-induced multiple exon skipping for rare non-hotspot mutations in Duchenne muscular dystrophy”
Authors: J.A.M. Janson, I.G.M. Kolfschoten, R.E.Y. van den Eijnde, R. Weij, R.C. Verheul, A. Baghat, M.M. Plug, P.C. de visser, J.C.T. van Deutekom
In the session ‘DMD 2 Therapeutic evaluations and approaches’ on Wednesday 8 October from 16:00:
• G.P.107 “Complementary human skin models as a tool to study oligonucleotide-induced injection site reactions”
Authors: C. den Besten; T. Steevels; P. Ekhart; S. Jones; S. Gibbs; G. Campion
• G.P.108 “Transient proteinuria with oligonucleotide therapy due to interference with tubular protein reabsorption”
Authors: G. Campion; C. den Besten; S. Jones; M. Wilme; R. Masereeuw
• G.P.113 “Drisapersen (DRIS) treatment for Duchenne muscular dystrophy (DMD): results of up to 188 weeks’ follow-up of an open-label extension study”
Authors: N. Goemans; M. Tulinius, R. Wilson; C. Wardell; P Bedwell; G. Campion
• G.P.114 “Evaluation of efficacy and safety baseline parameters in patients with Duchenne muscular dystrophy (DMD) from three placebo-controlled studies of drisapersen (DRIS)”
Authors: E. Mercuri; T. Voit; N. Goemans; CM. McDonald; R. Wilson; C. Wardel; G. Campion
• G.P. 115 “Pooled analyses of efficacy parameters in patients with Duchenne muscular dystrophy (DMD): results from the drisapersen (DRIS) clinical trial”
Authors: N. Goemans; T. Voit; CM. McDonald; E. Mercuri; R. Wilson, C. Wardell; G. Campion
• G.P.116 “SCOPE – DMD, an EU FP7 funded consortium for skipping trial across Europe in Duchenne muscular dystrophy”
Authors: O. Veldhuizen; G. Campion; A. Morgan; H. Aygun; S. Wojczewski; T. Voit; P. Carlier; J. Verschuuren; A. Aartsma – Rus; V. Straub
In the session ‘DM1 + Myasthenia + Channel diseases’ on Wednesday 8 October from 16:00:
• G.P.132 “Pre-clinical development of peptide-conjugated antisense oligonucleotides for myotonic dystrophy type 1 (DM1)”
Authors: J.C.T. van Deutekom; S.A.M. Mulders; B. Aguilera; A. Gonzalez-Barriga; J. van de Giesse; W.J.A. van den Broe; D.G. Wansink; N.A. Datson
- ENDS - About Prosensa Holding N.V.
Prosensa (NASDAQ: RNA) is a 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. www.prosensa.com
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.
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 pipeline of DMD compounds in addition to our Huntington’s disease and Myotonic Dystrophy 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’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.
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