Leiden, The Netherlands, 17 November 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.