The MoveDMD trial (NCT02439216) is investigating the safety and efficacy of edasalonexent in boys ages 4-7 at enrollment with any DMD-related mutation. The double-blind Phase 2 part included 31 ambulatory boys.
A total of 16 boys started taking edasalonexent either at the beginning of Phase 2 or at the open-label extension stage. All 13 boys who continued to participate at the time of this analysis received oral edasalonexent treatment (100 mg/kg per day) for at least 48 weeks. Eight boys already reached 60 weeks of treatment.
The positive results are from the ongoing extension phase. Treatment with edasalonexent was compared to a control period before edasalonexent treatment (average duration of 39 weeks). The results build on earlier data of treatment up to 36 weeks.
Treatment with edasalonexent improved muscle function in timed function tests — the 10-meter walk/run; four-stair climb; time to stand; and the North Star Ambulatory Assessment (NSAA) test, an integrated evaluation of muscle function.
Additional changes in non-effort measures of muscle health were also observed, including long-term reductions in muscle enzymes and in C-reactive protein (CRP) — a blood biomarker for inflammation that is elevated in DMD — after 12, 24, 36, 48, and 60 weeks of treatment.
Edasalonexent continues to be well-tolerated. Most adverse events (AEs) have been mild, and no serious AEs have been reported.
Of note, boys treated with edasalonexent showed a reduction in their heart rate (a higher heart rate is a typical complication in DMD) compared to normal values.
Catabasis will present these results on Feb. 17, 2018, at the XVI International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy.
“We are thrilled to see this preservation of muscle function and substantial slowing of disease progression in boys following more than a year of edasalonexent treatment. This effect has the potential to be extremely impactful for boys affected by Duchenne,” Jill C. Milne, PhD, CEO of Catabasis, said in a press release.
Richard Finkel, MD, one of the study’s principal investigators, said it is “compelling that all of the assessments of muscle function are demonstrating stabilization at an age when boys with DMD have a predictable decline. Clinically meaningful changes are observed here. The coherence of these data is very promising.”
Catabasis plans to initiate a global Phase 3 study evaluating the safety and effectiveness of edasalonexent in patients with DMD regardless of mutation type in the first half of 2018.
Like MoveDMD, the company expects to enroll about 125 patients ages 4-7 who have not been on steroids for at least six months. Treatment with edasalonexent will be compared to placebo.
The primary endpoint will be the change in the North Star Ambulatory Assessment score after 12 months of treatment. Function tests, as well as assessments of growth, and cardiac and bone health, are also planned. The company anticipates top-line results in 2020.
Edasalonexent inhibits the protein NF-kB, which is activated in DMD. NF-kB has a pro-inflammatory and pro-fibrotic effect, leading to muscle degeneration and suppressing muscle regeneration.
The DMD treatment candidate was granted orphan drug, fast track, and rare pediatric disease designations by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Commission.