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.

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.

Donate