According to a press release, the presentation, “MoveDMD: Phase 1/2 trial of Edasalonexent, an NF-kB Inhibitor, in 4- to 7-Year-Old Patients with Duchenne Muscular Dystrophy,” will be delivered next week by Joanne Donovan, MD, PhD, chief medical officer of Catabasis, during the 2017 Muscular Dystrophy Association Scientific Conference March 19-22 in Arlington, Virginia.
MoveDMD is a three-part Phase 1/2 clinical trial (NCT02439216) that is examining the safety, effectiveness, pharmacokinetics (PK, how the drug moves in the body) and pharmacodynamics (PD, how the body reacts to the drug) of edasalonexent in boys ages 4 to 7 with DMD.
Part A of the study is now finished. All dose levels of CAT-1004 tested were well-tolerated with no major safety signals reported.
Part B evaluated the safety, effectiveness, PK and PD of edasalonexent in 31 boys with DMD over 12 weeks at two dosing levels: 67 mg per kg of body weight a day and 100 mg/kg a day, compared to a placebo.
In February, Catabasis reported that the Part B trial had not met its primary endpoint. At 12 weeks of treatment, there were no statistically significant alterations between edasalonexent treatment groups (67 mg/kg/day and 100 mg/kg/day) and a placebo on lower-leg muscle function as measured by MRI.
Patients treated with edasalonexent at 100 mg/kg/day showed improvements across multiple measures compared to placebo-treated patients. But these differences did not reach statistical significance.
Also, the 67 mg/kg/day treatment group had mixed results compared with both the 100 mg/kg/day groups and the placebo, also not achieving statistical significance.
Part B also examined CAT-1004’s effectiveness in age-appropriate timed function tests, which served as secondary endpoints. These included a four-stair climb, the 10-meter walk/run, the time it takes boys to stand, and the North Star Ambulatory Assessment (NSAA).
Catabasis also reported there were no statistically significant changes in the clinical outcomes of these measures between edasalonexent-treated and placebo-treated patients.
Catabasis initiated Part C, an open-label extension of MoveDMD, that is examining the long-term safety and effectiveness of CAT-1004 in July 2016. The boys taking part in Part C will receive the drug for 36 weeks beyond the 12-week placebo-controlled portion of the study. All primary and secondary endpoints will be monitored.
Edasalonexent is an oral drug that researchers hope will revolutionize DMD treatment. Edasalonexent targets NF-kB, a protein activated in DMD. Inhibition of NF-kB could slow muscle degeneration and enhance muscle regeneration.
Edasalonexent received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). The European Commission has also granted the drug Orphan Medicinal Product status for the treatment of DMD.
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