Prosensa Advances Three Exon Skipping Candidates for Duchenne Muscular Dystrophy into the Next Development Stage - Prosensa to receive up to £27M in development and milestone payments from GSK
September 12, 2011
For immediate release
Leiden, September 12, 2011 – Prosensa, the Dutch biopharmaceutical company focusing on rare diseases with an unmet medical need, announced today that they have agreed with GlaxoSmithKline (GSK) to advance three further exon skipping compounds (PRO044, PRO045 and PRO053) into the next development stage under their ongoing collaboration relationship in Duchenne Muscular Dystrophy (DMD).
The two companies have agreed upon the development terms for the exon 44 skipping programme, which sees GSK providing additional support for PRO044, currently in Phase I/II. Prosensa and GSK will also work together on a preclinical and clinical development program for PRO045 and PRO053, which are expected to enter clinical trials in the first half of 2012. The three candidates PRO044, PRO045 and PRO053 are compounds designed to skip exons 44, 45 and 53 respectively, in the dystrophin gene, each targeting different subpopulations of DMD patients.
Under the collaboration agreement, Prosensa is eligible for milestone payments and development funding payments which could total up to £27M, depending upon the course and outcome of the clinical development for these programs. Prosensa and GSK will also embark on a natural history study that will provide a better understanding of the disease course of DMD.
Under the terms of the alliance established in 2009, GSK has an exclusive option to licence PRO044 and an option to licence one of either PRO045 or PRO053 for later stage development and commercialisation, with Prosensa retaining certain limited commercialisation rights. For the compound not selected by GSK, Prosensa will retain full development and commercialisation rights.
“In addition to the progress made with our lead compound targeting exon 51 (GSK2402968/PRO051) which is currently being investigated in a global phase III study, the advancement of these three additional candidates underlines the commitment of both parties to develop safe and effective compounds for the treatment of DMD,” commented Hans Schikan, Chief Executive Officer of Prosensa, adding, “The advancement of compounds targeting exons 44, 45 and 53 will allow us to use our exon skipping approach for the benefit of additional patients affected by this rare, progressive disease. It illustrates our ambition to help as many patients as soon as possible.”
Giles Campion, Prosensa’s Chief Medical Officer, said: “Prosensa has developed an ambitious plan to accelerate the development of a portfolio of therapeutic agents for rare diseases with high unmet medical need. Our collaboration with GSK allows us to now faster investigate additional treatments for more DMD patients.”
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 rare diseases with a high unmet medical need. Prosensa’s focus is on developing a treatment for DMD. Prosensa’s technology is based on a collaboration with Leiden University Medical Center. 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. The early development of PRO044 was supported in part by Cure Duchenne. 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 was recently shortlisted for the Biotech Company of the Year category at the 2011 Scrip Awards.
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
