Prosensa wins “Emerging Star Award” at the European Mediscience Awards
June 22, 2012
For immediate release
Leiden, The Netherlands, June 22, 2012 – Prosensa, the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, has won the Emerging Star accolade at last night’s European Mediscience Awards.
The European Mediscience Awards is the largest annual gathering of private and publicly quoted healthcare, biotech and life science companies in Europe recognising achievements in the life sciences industry. Supported by Peel Hunt, the inaugural Emerging Star Award celebrates the achievements of a private or young public mediscience company that has demonstrable credibility in terms of business model/strategy, management team and financing. The winner's focus on innovation is also key, with its technology/IP capable of significant market impact or disruption.
Collecting the award, Dr.Giles Campion, Prosensa’s Chief Medical Officer and SVP Research & Development said: “We are very pleased to be recognised by the European Mediscience judging panel. This prestigious award is an additional motivation in our efforts to help patients and their families affected by rare, progressively debilitating diseases.”
Prosensa has the most advanced portfolio of drug candidates for the treatment of Duchenne muscular dystrophy (DMD), with two compounds in clinical trials in partnership with GlaxoSmithKline and four additional compounds in late stage pre-clinical development. Prosensa’s DMD compounds are based on its proprietary exon-skipping technology that uses antisense oligonucleotides to restore expression of a functional dystrophin protein and to provide potential treatment for patients affected by this progressively debilitating neuromuscular disease. The company recently raised €23 million in a private equity financing round and is supported by a consortium of investors including Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates.
The other companies shortlisted for the Mediscience “Emerging Star Award” were BioCartis NV, Oxford Nanopore Technologies Ltd and Sphere Medical Ltd.
Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 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 sequences 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 read 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 a defective exon and thereby correct the reading frame, enabling the production of a novel, functional dystrophin protein.
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy and Huntington’s disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound (GSK2402968/ PRO051), being developed by GSK, entered Phase III clinical trials in January 2011. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit www.prosensa.com.