Baseline Characteristics of Patients Enrolled in PolarisDMD, A Phase 3 Trial of Edasalonexent for Duchenne Muscular Dystrophy

Topic:

Clinical Trials

Poster Number: 48

Author(s):

Richard Finkel, MD, Craig McDonald, MD, Lee Sweeney, PhD, Kathryn Wagner, MD, Erin Neil, MD, Maria Mancini, MHP, James MacDougall, PhD, Joanne Donovan, MD, PhD

Institutions:

1. Nemours Children’s Hospital, University of Central Florida College of Medicine, 2. UC Davis, 3. University of Florida, 4. Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA, 5. University of Michigan, 6. Catabasis Pharmaceuticals, 7. Catabasis Pharmaceuticals, 8. Catabasis Pharmaceuticals

Background:
Edasalonexent is an oral NF-kB inhibitor and is in development to treat Duchenne muscular dystrophy, regardless of the underlying mutation. The Phase 2 MoveDMD trial and open-label extension showed slowing of disease progression on MRI and functional measures and supported design of a pivotal Phase 3 trial, PolarisDMD, in a similar population.

Objectives:
The Phase 3 trial is a one-year placebo-controlled study to assess edasalonexent efficacy and safety and is fully enrolled. This analysis compared the baseline demographics and functional characteristics of the two trials.

Methods:
Both studies enrolled 4 to 7 year-old boys (<8th birthday) with confirmed DMD not on corticosteroids for at least 6 months. Phase 2 (n=31) was only in the US while Phase 3 (n=131) is global. Both trials enrolled boys able to complete all timed function tests (TFT) at baseline, but the Phase 3 trial excluded boys unable to stand from supine in <10 sec. Comparison was made between populations at the baseline of the Phase 2 trial and the baseline of the Phase 3 trial.

Results:
In the Phase 3 trial, boys enrolled at 37 sites in the US (n=84), Canada (10), Europe and Israel (31), and Australia (6). Age, baseline North Star Ambulatory Assessment (NSAA), and TFT (time to stand, 4-stair climb, and 10-meter walk/run) velocities were similar in both trials with no significant differences in mean values or population distribution. As expected with the entry criteria, mean time to stand was slightly faster in the Phase 3 trial. Baseline plasma muscle enzymes and heart rate were also similar. Distribution of baseline NSAA and TFT was less variable in the Phase 3 trial than in the Phase 2 trial.

Conclusions:
Baseline demographic and functional characteristics of patients enrolled in the PolarisDMD Phase 3 trial are similar to patients enrolled in the Phase 2 MoveDMD edasalonexent trial.