|Indication||Compound||Discovery||Pre-clinical||Phase I/II||Phase III|
|Duchenne Muscular Dystrophy (DMD)||PRO044|
Prosensa’s second product in development, PRO044, induces exon 44 skipping in the dystrophin gene and is intended for approximately 6% of all Duchenne Muscular Dystrophy (DMD) patients, including those with deletions of exon 43, exon 45, exons 38-43, exons 40-43, exons 42-43, and exons 45-54. The underlying chemistry and mechanism of PRO045 are similar to drisapersen. PRO044 is highly sequence specific minimizing the risk for off-target effects.
PRO044 addresses a separate sub-population of DMD patients with a mutation around location 44 in the DNA of the dystrophin protein. PRO044 has recently completed a phase I/II dose-escalation study in Europe, assessing six doses in 18 DMD patients. The extension study for participants of this dose-escalation study is anticipated to start in the fourth quarter of 2013. A pivotal study is anticipated to start in the fourth quarter of 2014. PRO044 has been extensively tested in a series of cultured muscle cells from patients with different relevant mutations, and in the hDMD mouse model.
Details of the clinical trials, such as inclusion/exclusion criteria and trial sites, are posted on the website www.clinicaltrials.gov and all required approvals of authorities and ethics committees are granted. PRO044 has been granted orphan drug status in the European Union and the United States.