Updated Data Released Regarding Use of Edasalonexent in DMD Patients


Updated data on MoveDMD, a phase 2, open-label extension trial of edasalonexent in boys diagnosed with Duchenne muscular dystrophy (DMD), reports positive results.

This week, at the New Directions in Biology and Disease of Skeletal Muscle Conference in New Orleans, Louisiana, Catabasis Pharmaceuticals Inc. released data from its MoveDMD phase 2, open-label, extension trial of assessing edasalonexent in boys diagnosed with Duchenne muscular dystrophy (DMD).

Edasalonexent is an oral small molecule inhibitor of NF-kB that helps improve skeletal, diaphragm, and cardiac disease as observed in DMD mouse and dog models.

The NF-kB pathway is the critical link between loss of dystrophin and disease manifestation and progression in those with DMD. The updated data included reports regarding new NF-kB inhibition biomarker results, which further support the significant NF-kB biomarker results observed in phase 1 of the MoveDMD trial as they are consistent with significantly decreased C-reactive protein (CRP) with edasalonexent treatment.

“We are pleased to see additional clinical evidence demonstrating the activity of edasalonexent in inhibiting NF-kB in boys affected by Duchenne, consistent with the observed substantial slowing of Duchenne disease progression in the assessments of muscle function and magnetic resonance,” Andrew Nichols, PhD, chief scientific officer of Catabasis, said in a recent statement.

For the trial, investigators set out to assess the safety and efficacy of edasalonexent in boys between the ages of 4 and 7 with DMD—regardless of mutation type—who were not on steroids.

Decreased NF-kB-regulated transcripts with edasalonexent treatment proved to be consistent with biomarker results which showed that C-reactive protein (CRP) was significantly decreased with edasalonexent at weeks 12, 24, 36, and 48, compared with baseline in the 100 mg/kg treatment group (p≤0.001).

“CRP is a well-characterized blood marker that provides a global assessment of inflammation, and CRP is elevated in boys affected by DMD,” according to a recent press release.

The investigators noted a significant decrease in CRP with edasalonexent treatment, which further supports the biological activity of NF-kB inhibition by treatment with the drug decreasing inflammation. Furthermore, compared with the rate of change in the off-treatment control period, edasalonexent substantially slowed DMD disease progression in boys on 100 mg/kg through more than a year of treatment. Consistent improvements were also observed across all assessments of muscle function in the rate of decline after 12, 24, 36, 48 and 60 weeks with oral 100 mg/kg edasalonexent treatment.

Overall, edasalonexent was observed to be well-tolerated by trial participants; no safety signals were reported.

Looking forward, Dr Nichols expressed his excitement for a future phase 3 trial. “Building on the results previously reported for edasalonexent treatment in patients in the MoveDMD trial, these new biomarker data further support the consistent positive edasalonexent results,” he said. “We look forward to advancing edasalonexent in a single global phase 3 trial this year with the goal of improving the quality and length of life for those affected by Duchenne.”

DMD is a rare form of muscular dystrophy characterized by progressive weakness and atrophy in the skeletal and cardiac muscles. A lack of dystrophin is the primary cause of DMD, which causes mechanical stress to activate NF-kB, thus promoting inflammation and fibrosis, suppression of muscle regeneration, and muscle degeneration.

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