AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks Compared to Placebo in Phase IIb Trial for the Treatment of Duchenne Muscular Dystrophy
BOTHELL, WA, Apr 02, 2012
There were no significant improvements in clinical outcomes in the treated groups compared to placebo. Performance on the 6-minute walk test and other outcome measures were generally stable across most of the patients, including the placebo patients.
" After 74 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts who were able to perform the 6MWT (modified Intent-to-Treat or mITT population; n=6) showed a statistically significant treatment benefit of 65.2 meters (p ≤ 0.004) when compared to the placebo/delayed-treatment cohort (n=4). The eteplirsen-treated patients in the mITT population demonstrated less than a 5 percent decline (13.4 meters) from baseline in walking ability."
or did you mean
"We are encouraged to see a continued stabilization of walking ability in patients treated with eteplirsen for nearly one and a half years," said Sarepta CEO Chris Garabedian, in a statement. "These data are particularly compelling when viewed in the context of published natural history studies, which showed substantial declines on the 6-minute walk test over this timeframe in a similar ambulatory DMD population.
"Stable" is also the operative word for the four DMD boys who began the study on a placebo and were switched over to eteplirsen after 24 weeks. On the same six-minute walk test conducted after 74 weeks, these four boys walked 64.6 meters less, on average, than they did when the study began. When the same test was performed at 62 weeks, the decrease in walking distance was 63.8 meters.
Like the boys treated with eteplirsen from the beginning of the study, the four boys in the "control" arm demonstrated stable walking ability from 62 weeks through 74 weeks "
The study was only placebo controlled in the first 24 weeks. At 24 weeks there was no difference between active and placebo in terms of walk distance. Sorry this is the data. Any open label data after 24 weeks will be disregarded by the FDA. Specifically considering there was no improvement in walk with 50mg as compared to 30mg. No dose response. No relationship between dystrophin levels and walk. Only 8 patients in the study, two non responder patients eliminated from the analysis, all data coming from only one center. ALL BIG NO NO'S FOR THE FDA.