This morning, AVI BioPharma released information discussing their findings thus far in their Phase IIb study of Eteplirsen.
Eteplirsen uses AVI’s phosphorodiamidate morpholino oligomer (PMO)-based chemistry to skip exon 51 of the dystrophin gene. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, functional form of dystrophin.
The Eteplirsen Phase IIb Study is being conducted at Nationwide Children's Hospital in Columbus, Ohio. Twelve boys between 7 and 13 years of age eligible for treatment with an exon-51 skipping drug received either IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg ofeteplirsen once weekly for 24 weeks (n=4). Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects, after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort. Following the placebo trial, all patients were placed into the open label study.
In the open label study, two placebo patients were randomized to the 30 mg/kg cohort (now n=6) and two placebo patients were randomized to the 50 mg/kg cohort. The open label trial extension trial is ongoing. An extension has just been added to the study, which includes muscle biopsies at 24 weeks and 40 weeks in both the 30 mg and 50 mg cohorts.
Results have demonstrated the following:
These findings show that:
AVI is to be commended for their dedication to Duchenne research and to be congratulated on their current findings. We are pleased to be partners in their continued research. Eteplirsen is the first DMD specific drug to demonstrate production of new, novel dystrophin consistently throughout the muscle. This extremely important finding supports the need for a pivotal trial. AVI is in the process of producing a brief for the FDA, collating the remainder of their data and presenting the need for next steps. A comprehensive summary of their findings will also be presented at the annual American Association of Neurology meeting in New Orleans, April 25, 2012, during the Emerging Science session.
Kathi Kinnett, Director of Clinical Care
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