AVI BioPharma Initiates Dosing in Phase 2 Study of Eteplirsen in Duchenne Muscular Dystrophy Patients

AVI BioPharma, Inc. AVII today announced that it has initiated dosing in a Phase 2 study of eteplirsen, the Company's lead exon-skipping therapeutic candidate for the treatment of Duchenne muscular dystrophy. The placebo-controlled study of twelve patients, which will be conducted at Nationwide Children's Hospital in Columbus, Ohio, is designed to evaluate the efficacy and safety of eteplirsen in DMD patients over 24 weeks of dosing. Patients enrolled in the study will receive once weekly intravenous infusions of either 50mg/kg of eteplirsen, 30mg/kg of eteplirsen or placebo, and will be evaluated on a number of safety and efficacy endpoints. The efficacy endpoints will include biochemical markers in muscle biopsies, such as the production of the dystrophin protein and markers of immune-inflammatory response, as well as clinical outcomes to measure muscle strength, function and degree of ambulation.