Our hearts are heavy when the news reached us that the Phase 3 PolarisDMD trial with edasalonexent did not meet its primary or secondary endpoints. Families involved in the PolarisDMD and GalaxyDMD trials who have questions are encouraged to contact their study site directly. All in the Duchenne community are welcome to join the webinar with PPMD on Tuesday, October 27 at 1pm ET, 6pm CET that will share more details provides the opportunity questions about the trial and the results.
The trial’s primary endpoint was to measure the change in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent treatment, compared to a placebo. The secondary endpoints were the timed function test (time to stand, 10-meter walk/run, and 4-stair climb) did not show statistically significant improvements. Muscle enzymes and heart rate did not show significant differences between edasalonexent and placebo.
Edasalonexent was well-tolerated, which is consistent with the safety profile that is seen to date. The majority of adverse events were mild in nature, and the most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash. There were no treatment-related serious adverse events and no dose reductions. There were no adverse trends in blood tests and growth was maintained.