Marathon preps deflazacort NDA to treat DMD

Marathon Pharmaceuticals LLC (Northbrook, Ill.) plans to submit an NDA to FDA next month for deflazacort ( MP-104) to treat Duchenne muscular dystrophy. On Tuesday, Marathon presented results of a Phase III study in which the compound met primary and secondary endpoints to treat DMD. Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties.

At the American Academy of Neurology meeting in Vancouver, B.C., on Tuesday, Marathon said two doses of deflazacort improved average muscle strength over baseline after 12 weeks vs. placebo, meeting the study's primary endpoint (p=0.0173 for 0.9 mg/kg/day, p=0.0003 for 1.2 mg/kg/day). Both doses also improved performance on timed functional tests, including time from lying down to standing, time to climb four stairs and time to run or walk 30 feet (p<0.002). ...