Prosensa, a Netherlands-based biopharmaceutical company focusing on RNA modulating therapeutics, announces the start of a phase I/II clinical study for PRO044 in patients with Duchenne Muscular Dystrophy.
The objective of this open label, dose-escalating study is to evaluate the safety and tolerability of the systemic delivery of PRO044 in twelve Duchenne Muscular Dystrophy (DMD) patients receiving weekly subcutaneous injections for a period of five weeks. The effect of PRO044 will be assessed at the RNA level, to demonstrate specific exon 44 skipping, and at the protein level, to demonstrate novel dystrophin expression in muscle biopsies.
The first patients have been enrolled at the University Hospital Leuven in Belgium by principal investigator Dr. Nathalie Goemans, department of Pediatric Neurology. Prosensa also received approval from the regulatory authorities in Sweden, where patient screening has been initiated, and Italy. Participating centers are the Queen Silvia Children’s Hospital in Gothenburg, Sweden and the University of Ferrara in Italy.
PRO044 is Prosensa’s second RNA based therapeutic candidate for the treatment of DMD. Prosensa’s lead DMD compound, PRO051/GSK2402968, is close to entering phase III clinical development and was recently partnered with GlaxoSmithKline (GSK). GSK also has an exclusive option on PRO044, which will be triggered upon successful completion of this phase I/II study.
“We are delighted to advance a second promising candidate for the treatment of DMD into clinical development. With the addition of PRO044 to our drug candidate portfolio, we will be able to address a broader patient population upon successful completion of these clinical development programs.” commented Dr. Giles Campion, Chief Medical Officer of Prosensa. “This important clinical development step confirms Prosensa’s commitment to the treatment of neuromuscular disorders.”
Date: March 30, 2010
Filed Under: Drug Discovery