Catabasis Pharmaceuticals, Inc. announced top-line safety and efficacy results for Part B of the MoveDMD trial of edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD). The objective of Part B of the MoveDMD trial was to evaluate the effects of edasalonexent using magnetic resonance imaging (MRI) T2 as a biomarker at 12 weeks. The primary efficacy end point of average change from baseline to week 12 in the MRI T2 composite measure of lower leg muscles for the pooled edasalonexent treatment groups of 67 mg/kg/day and 100 mg/kg/day compared to placebo was not met (0.37 milliseconds for the pooled edasalonexent treatment groups versus 0.47 milliseconds for placebo, a smaller increase in MRI T2 correlating with less muscle inflammation). The edasalonexent 100 mg/kg/day treatment group consistently showed numerical improvement versus placebo across multiple measures although the changes were not statistically significant. The 67 mg/kg/day treatment group had mixed results compared with both the 100 mg/kg/day treatment group and placebo, which in each case were not statistically significant. Catabasis plans to complete a full analysis of the data from Part B of the MoveDMD trial and to submit the data for presentation at an upcoming scientific conference. The open-label extension portion (Part C) of the MoveDMD trial is ongoing. Catabasis intends to report the results from Part C in 2017, with an interim update in second quarter. Thirty-one boys enrolled in Part B and all completed the trial. Both dose levels of edasalonexent evaluated were well tolerated with no safety signals observed. The majority of adverse events were mild in nature and the most common treatment-related adverse events were gastrointestinal, primarily mild diarrhea and vomiting. There were no treatment-related serious adverse events, no drug discontinuations and no dose reductions. Edasalonexent plasma exposure in Part B of the MoveDMD trial was consistent with that observed in Part A. Additional efficacy end points included three age-appropriate timed function tests (10-meter walk/run, 4-stair climb and time to stand), the North Star Ambulatory Assessment (NSAA) and additional MRI measures. The trial was not powered to detect statistically significant changes in the timed function tests or NSAA and no significant changes were detected in these measures for those dosed with edasalonexent versus placebo. For all three timed function tests, the change in speed of performance from baseline to week 12 was numerically better for the 100 mg/kg/day treatment group compared to placebo. The change in NSAA was also numerically better for the 100 mg/kg/day treatment group compared to placebo. In addition to the lower leg, MRI T2 was also measured for the upper leg. The results in MRI T2 score for change from baseline to week 12 for the vastus lateralis (a large component of the quadriceps) were not statistically significant but showed a numerical improvement in the 100 mg/kg/day treatment group compared to placebo. Compared to the placebo group, patients in the edasalonexent 100 mg/kg/day group had characteristics of more advanced disease at baseline. Patients in this treatment group had been diagnosed at a younger age and, at baseline, did not perform as well on function tests.