US biopharma company Catabasis Pharmaceuticals has wrapped up enrollment for the phase 3 PolarisDMD clinical trial designed to evaluate edasalonexent, an NF-kB inhibitor, in Duchenne muscular dystrophy (DMD).

The biopharma company said that the target enrollment of 125 boys was surpassed owing to strong interest from its 40 clinical sites across eight countries along with the backing of patient advocacy organizations.

Catabasis Pharmaceuticals expects to release the top-line results from the PolarisDMD clinical trial in the fourth quarter of next year. The late-stage clinical trial for edasalonexent is expected to support an NDA filing in 2021.

Edasalonexent is an investigational oral small molecule, designed to inhibit NF-kB. The small molecule is being developed by Catabasis Pharmaceuticals as a potential foundational therapy for all patients suffering from Duchenne muscular dystrophy, irrespective of their underlying mutation.

In Duchenne muscular dystrophy, the loss of dystrophin results in chronic activation of NF-kB, which is a crucial driver of skeletal and cardiac muscle disease progression.

The PolarisDMD clinical trial enrolled 130 boys aged 4 to 7 (up to eighth birthday) with any mutation type and who had not been on steroids for the last six months.

The phase 3 clinical trial is a randomized, double-blind, placebo-controlled study with 2:1 randomization. Upon the completion of 52 weeks of treatment, all boys and their eligible siblings are expected to be given the option to take part in GalaxyDMD, an open-label extension trial designed to evaluate the long-term safety of edasalonexent.

The participating boys can start or continue treatment with an approved exon skipping therapy in the GalaxyDMD trial, which has an optimized schedule with visits to the clinical trial sites every six months, said Catabasis Pharmaceuticals.

Commenting on the PolarisDMD clinical trial, Joanne Donovan – Chief Medical Officer of Catabasis Pharmaceuticals, said: “We are thrilled to reach this important milestone. The interest and feedback from families and trial sites has been overwhelmingly positive. At a time when there are multiple trials for Duchenne, we are very pleased that physicians and families chose the Phase 3 PolarisDMD trial for edasalonexent.

“Edasalonexent has the potential to be a foundational therapy, providing benefit to boys, regardless of their underlying mutation, with the potential to benefit muscle function, as well as cardiac function and bone health. We look forward to completing the trial next year and are working diligently toward the goal of making edasalonexent available to patients.”

  Catabasis Pharmaceuticals, Duchenne muscular dystrophy, GalaxyDMD trial, Joanne Donovan, NF-kB inhibitor, PolarisDMD trial, USA