AVI BioPharma, Inc., has 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 (DMD).
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.
"In this Phase 2 study we will evaluate eteplirsen at higher doses and over a longer duration of treatment, which will help us understand the potential disease-modifying effects and safety of eteplirsen as chronically-administered therapy," said Chris Garabedian, AVI's CEO and president.
Garabedian continued, "We expect data from this study around the end of the second quarter of 2012, which will guide our design for a pivotal study."
Jerry R. Mendell, M.D., of Nationwide Children's Hospital and principal investigator of the study added, "Despite many years of awareness and investment in therapeutic development, we only have supportive treatments available for DMD patients today. We have seen tremendous promise for eteplirsen to potentially modify the progression of DMD in patients and we look forward to further understanding its potential through this longer study."
The travel costs for the patients participating in the Phase 2 clinical study are supported in part by grants from Parent Project Muscular Dystrophy and Muscular Dystrophy Association.