Authored by Karl Simpson
As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are leading rare disease research. In the ninth in the series we look at Dutch company Prosensa.
Genzyme has seen many of its past executives and talented professionals leave with their excellent knowledge of orphan drugs and rare diseases to go and help build new biotech ventures. Prosensa is one such company. Led by ex-Genzyme VP Global Marketing and Strategic Development for their rare genetic disease franchise, Hans Schikan, Prosensa in a biopharmaceutical company based in Lieden, Netherlands who are gaining a lot of traction in the DMD disease area, a seriously debilitating and fatal disease which affects 1 in 3500 males.
Prosensa is focused on applying its technology platform, developed jointly with the Leiden University Medical Center, to the correction of mutated mRNA initially in finding therapies for the life threatening disease Duchenne Muscular Dystropy, as well as beginning to develop therapeutic products in Myotonic Dystrophy and Huntington’s Disease. This single-stranded RNA-based antisense oligonucleotides to correct mutated mRNA. This allows Prosensa combines this approach with their exon skipping technology to modify the disease symptoms.
Prosensa was founded in 2002 and now employs in excess of 80 people, having also garnered investment from a range of prominent life science ventures capitalists, including Life Science Partners, Abingworth and New Enterprise Associates (NEA), who led a E23m round last January, bringing David Mott, General Partner of NEA and formerly a key executive with MedImmune to the Supervisory Board. Also added to this Supervisory Board in recent months has been Henri Termeer, former Chairman and CEO of Genzyme for some three decades.
One of the pivotal dates in Prosena’s emergence as a rare disease company was October 2009, when the company announced a worldwide partnership with GSK (GlaxoSmithKline). GSK’s Centre of Excellence for External Drug Discovery formed an alliance with Prosensa to develop four RNA-based products for patients affected with DMD. This alliance gives GSK exclusive rights to develop and license the Prosensa’s lead product drisapersen, PRO051/GSK2402968, intended to treat DMD by skipping exon 51 for the dystrophin gene. GSK also obtained rights to the follow on compounds , including PRO044 which targets exon 44.
This partnership delivered upfront payments and triggers milestone payments, one of which was received in October 2012 to the value of £10m. Prosensa now has a portfolio of six compounds in DMD, all of which now have orphan drug status, giving Prosensa a portfolio capable of treating in excess of 40% of all DMD patients. With recent candidates PRO052 and PRO055 entering pre-clinical, and PRO045 and PRO053 reaching the important milestone of orphan drug status, Prosensa can progress them through to clinical trials in coming months. While GSK has rights to one of these products, Prosensa retains rights to the other, which will give the company the strategic opportunity to take a product through the clinical trials and into commercialisation themselves.
Drisapersen, Prosensa’s lead candidate co-developed with GSK, is a fully enrolled late stage Phase III trial of 186 patients, in 47 trial sites in 20 countries. In results of a Phase I-IIa study of PRO051 announced in The New England Journal of Medicine, Prosensa showed the new dystrophin expression was observed between approximately 60% and 100% of muscle fibers in 10 of the 12 patients.
Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter