Catabasis Wins With Early-Stage Duchenne Muscular Dystrophy Study

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Catabasis Pharmaceuticals Inc. (NASDAQ: CATB) shares made a handy gain on Tuesday after the company announced early stage data from its Duchenne muscular dystrophy (DMD) study.

The company announced that data from the Phase 1 MoveDMD clinical trial of edasalonexent were published in the Journal of Neuromuscular Diseases.

Overall edasalonexent was well tolerated in pediatric patients and the data demonstrated that edasalonexent inhibited NF-kB in pediatric patients with DMD. NF-kB is a key link between loss of dystrophin and disease progression in DMD.

MoveDMD is a multipart trial including Phase 1, Phase 2 and an ongoing open-label extension. Catabasis is currently enrolling the Phase 3 PolarisDMD trial for edasalonexent.

The Phase 1 MoveDMD results demonstrated:

  • Edasalonexent was well tolerated, with all patients completing the 1-week study without serious adverse events or dose reductions and with no safety signals
  • Edasalonexent was rapidly absorbed, with peak levels observed 2-6 hours after dosing and exposure was dose-dependent
  • After 7 days of treatment, NF-kB-regulated genes were significantly decreased in a dose-dependent manner, confirming that edasalonexent inhibited NF-kB in boys with DMD

Erika Finanger, M.D., Associate Professor of Pediatrics, Division of Neurology, School of Medicine at Oregon Health & Science University and principal investigator for both the MoveDMD and PolarisDMD trials, commented:

The data from the Phase 1 MoveDMD trial reinforce the good tolerability and safety profile of edasalonexent that we have now also observed in the Phase 2 trial and open-label extension. I am pleased to continue to evaluate edasalonexent as a potential novel therapy for those affected by Duchenne, and I am excited to participate in the Phase 3 PolarisDMD study.

The primary objective of the Phase 1 portion of the MoveDMD trial was to assess the safety and tolerability of edasalonexent, with the secondary objective to assess the PK and target engagement of edasalonexent in pediatric patients with DMD to provide guidance in the Phase 2 portion of the study.

Shares of Catabasis were last seen up about 4% at $0.60, in a 52-week range of $0.53 to $2.38. The stock has a consensus analyst price target of $3.83.