Eteplirsen Shows Efficacy for Duchenne Muscular Dystrophy

AVI BioPharma has reported positive Phase Ib/II study results of eteplirsen, an exon-skipping therapy for the treatment of Duchenne muscular dystrophy. The genetic muscle-wasting disease is caused by the absence of functional dystrophin, an essential muscle protein.The 19-patient, 12-week, six-dose cohort study assessed eteplirsen’s safety, tolerability, pharmacokinetic profile and ability to restore dystrophin expression.

The study also showed that eteplirsen induced exon 51 skipping in all cohorts, and dystrophin protein expression was observed in a dose-dependent manner. Francesco Muntoni, head of the Dubowitz Neuromuscular Centre at UCL Institute of Child Health in London, said that the significant and dose-dependent improvements in dystrophin expression and other associated biochemical markers suggest that eteplirsen has the potential to reduce muscle damage in Duchenne muscular dystrophy patients and positively modify the severe progressive nature of the disease.