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news.AVI BioPharma has released positive Phase 1b/2 study results evaluating company's exon-skipping therapy, eteplirsen as a treatment for Duchenne muscular dystrophy (DMD).
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The results of the trial suggested that the drug was well tolerated and improved dystrophin expression in patients.
The 19-patient, 12-week, six dose cohort study investigated the safety, pharmacokinetic, tolerability, profile and ability of eteplirsen to restore dystrophin expression.
The study also demonstrated that Eteplirsen induced exon 51 skipping in all cohorts, and dystrophin protein expression was observed in a dose-dependent manner.
UCL Institute of Child Health Dubowitz Neuromuscular Centre head Francesco Muntoni said 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 DMD patients and positively modify the severe progressive nature of the disease.
”we are eager to continue the investigation of eteplirsen in placebo-controlled trials to evaluate biochemical markers and clinical endpoints over a longer treatment duration," Muntoni said.