Phase III study of deflazacort shows efficacy in Duchenne Muscular Dystrophy- Marathon Pharmaceuticals

Marathon Pharmaceuticals has announced presentations of analyses of a pivotal Phase III study of deflazacort, an investigational glucocorticoid, in patients with Duchenne Muscular Dystrophy (DMD). The AAN presentations provide further detail on the previously presented primary endpoint of the pivotal study, which showed deflazacort improved muscle strength in DMD patients versus placebo at 12 weeks. Results of timed functional tests (TFT), a secondary endpoint being presented at AAN, show deflazacort patients at both doses had significant improvement from baseline to Week 12 versus placebo in the ability to perform three tests: time from lying down to standing, time to climb four stairs, and time to run or walk 30 feet.

The most common adverse events were cushingoid appearance, hirsutism, weight gain, central obesity and increased appetite. In addition, Marathon is presenting post-hoc analyses showing the effects of deflazacort on muscle strength and pulmonary function in the subset of DMD patients who had lost the ability to walk. The most common adverse events in the deflazacort treatment arms for the post-hoc analyses of non-ambulatory patients were cushingoid appearance, erythema, central obesity, weight gain and headache. Results are being shared at the 68th American Academy of Neurology (AAN) Annual Meeting.