Move DMD Trial Update

The MoveDMD trial with edasalonexent (CAT-1004) in Duchenne muscular dystrophy (DMD) has just completed Part B, and Catabasis has released the top-line results.

Summary:

• The top-line results that we are reporting are for Part B of a 3-part study in young boys with DMD. Positive results were reported for Part A in 2016. Part C is ongoing.
• In Part B of the study we were using MRI as an early, innovative biomarker for the primary end point to look for safety and efficacy for two 2 doses of edasalonexent
• We had used the MRI end point because steroids which show effects in DMD have shown changes in MRI in about 12 weeks, and we were pioneering a path to enable efficient trials in DMD
• In our top-line results, we reported that we did not meet the MRI end point at 12 weeks
• The higher dose treatment group of edasalonexent, 100 mg/kg/day, consistently showed numerical improvement vs. placebo across multiple measures although the changes were not statistically significant
• The lower dose treatment group of edasalonexent, 67 mg/kg/day, had mixed results compared with both the 100 mg/kg/day treatment group and placebo, neither of which was statistically significant
• No safety signals were seen and edasalonexent was well tolerated. There were no dose reductions or discontinuations
• We are continuing to assess effects in patients on edasalonexent over a longer time in the ongoing open-label extension (Part C) using conventional end points in DMD such as timed function tests and NSAA in addition to MRI

For more information, please review the Catabasis quarterly newsletter.