Prosensa Updates on Patent Position
November 17, 2011
For immediate release
Leiden, November 17, 2011 – Prosensa, the Dutch company focusing on RNA modulating therapeutics for rare diseases with unmet medical needs, has been granted two new patents for exon-skipping by the Japanese Patent Office and the United States Patent and Trademark Office, and has confirmed its strong position in Europe during an opposition procedure which was concluded yesterday.
The Japanese patent (no. 2002-529499), granted in October 2011, describes methods of inducing exon skipping in the messenger RNA of eukaryotic cells by the use of compounds called antisense oligonucleotides. It covers the broad portfolio of Duchenne muscular dystrophy (DMD) compounds currently in development by Prosensa, whereas the US patent (no. 7,973,015), granted in July 2011, describes a method for multiple exon skipping. These patents further strengthen Prosensa’s intellectual property position.
Prosensa also announces the outcome of the Opposition Division of the European Patent Office (EPO) ruling regarding its European Patent EP1619249. The EPO Opposition Division, presiding in oral proceedings on 16 November 2011 in Munich, ruled that the patent was allowable in an amended form. The allowed patent describes, amongst others, the skipping of exon 51 in the DMD gene using a 14- to 40-mer antisense oligonucleotide as a potential therapy to treat DMD. The amendment is the result of an opposition proceeding brought by the US based company AVI BioPharma. With this ruling, the EPO confirmed the strong IP position that Prosensa has built in the field of exon skipping in DMD. The patent directly provides a strong protection for Prosensa’s lead product PRO051/GSK2402968.
Welcoming the decision to uphold the most important claims in this Prosensa patent, Hans Schikan, CEO of Prosensa commented: “This is a positive endorsement of the quality of our patents. Prosensa has a strong intellectual property estate in the area of exon skipping and RNA modulation more widely. We are firmly committed to bringing innovative treatments to patients suffering from rare diseases, and advancing our compounds for Duchenne muscular dystrophy patients.”
Prosensa has the broadest and most advanced exon skipping portfolio of compounds in development for DMD. PRO051/GSK2402968, developed in collaboration with GlaxoSmithKline and designed for the skipping of exon 51 in specific populations of DMD patients, is currently in phase III clinical trials. Moreover, Prosensa has 5 additional exon skipping programmes in clinical and preclinical development (targeting exons 44, 45, 53, 52 and 55). PRO051/GSK2402968 and PRO044 have been granted Orphan Drug status in Europe and the US, which provides additional protection.
About DMD
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. Patients suffer from progressive loss of muscle strength due to the absence or defect of the dystrophin protein, often making 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 lead to the manufacture of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the rest of the exons 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 dystrophin protein.
About Prosensa
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics correcting gene expression in diseases with large unmet medical needs, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy (DM1) 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, AGF Private Equity, GIMV, LSP and MedSciences Capital. For more information about Prosensa, please visit www.prosensa.com
Prosensa recently received the award of “Most Innovative European Biotech SME 2011” by EuropaBio.
Press contacts for this press release
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Luc Dochez - Chief Business Officer - Prosensa
languages: English, Dutch, French
Timezone: Europe/Amsterdam
Phonetel phone_1: +31 (0) 71 33 22 085
Phonetel phone_2: +31 (0) 71 33 22 100
E-mail: l.dochez@prosensa.nl
