Therapy Areas: Cardiovascular
Catabasis Pharmaceuticals Presents Edasalonexent, a Potential Foundational Treatment for Duchenne Muscular Dystrophy
9 October 2019 - - US-based clinical-stage biopharmaceutical company Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB) has presented the findings from the MoveDMD trial of edasalonexent, the company said.

In the Phase 2 MoveDMD trial and open-label extension, edasalonexent slowed disease progression compared to the off-treatment control period and was well tolerated through more than 55 cumulative patient years of exposure in boys affected by Duchenne muscular dystrophy.

These data were presented by Dr. Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children's Health System, and Principal Investigator for the Phase 2 MoveDMD and Phase 3 PolarisDMD studies of edasalonexent in DMD at the 24th International Annual Congress of the World Muscle Society.

In the MoveDMD trial and open-label extension, edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in the off-treatment control period, significantly improved biomarkers of muscle health and inflammation and was safe and well-tolerated.

In more than 55 cumulative patient years of exposure, the majority of adverse events were mild in nature, and the most common treatment-related adverse event was diarrhea, generally mild and transient.

There were no serious adverse events observed on treatment, and no adverse trends in chemistry, hematology, or measures of adrenal function.

Edasalonexent is not a steroid and has not shown the known side effects of corticosteroids.

Edasalonexent is an investigational oral small molecule designed to inhibit NF-kB. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression.

Edasalonexent is currently being studied in the Phase 3 PolarisDMD trial, which has fully enrolled 130 boys with DMD, ages 4 to 7 (up to 8th birthday) with any mutation type and who had not been on steroids for the past 6 months.

After the completion of 52 weeks of treatment, all boys and their eligible siblings are expected to have the option to enroll in GalaxyDMD, an open-label extension study designed to assess the long-term safety of edasalonexent.

Top-line results from the Phase 3 PolarisDMD trial are expected in the fourth quarter of 2020, and the trial is anticipated to support an NDA filing in 2021.

Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation.

In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression.

The company's ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes.

Edasalonexent is also being dosed in the open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial and open-label extension, we observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation.

The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD.
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