In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline. Below you can find an update about the current state of development of these compounds.
Prosensa is currently conducting a clinical study to investigate the effect of PRO044 in boys with Duchenne muscular dystrophy, who have a dystrophin gene mutation amenable to an exon 44 skip. This study is a phase I/IIa study that will assess the safety, tolerability, efficacy and pharmacokinetics of PRO044. The inclusion/exclusion criteria, as well as the endpoints and investigator locations are available on www.clinicaltrials.gov (search terms ‘Duchenne and Prosensa’ or ‘PRO044’).
Prosensa has four additional compounds in development that target exons 45, 53, 52 and 55 (PRO045, PRO053, PRO052 and PRO055). These programs currently are in the pre-clinical development phase. It is anticipated that the company will be able to progress PRO045 and PRO053 into the clinic in the second half of 2012. Discussions are on-going with the regulatory authorities to ensure optimum development. Each trial will be posted at the website www.clinicaltrials.gov as soon as the study design is finalized and all the required approvals of authorities and ethics committees are granted.
It is always difficult to predict timelines for drug development. Therefore, please keep in mind that expected start and end dates are based on predictions and could be delayed if unforeseen events occur.
Prosensa is very committed to developing safe and effective treatments and we will do our utmost to succeed herein. Our goal is to develop compounds for as many patients as feasible who may benefit from the exon skipping platform.