20 February 2019 - - US-based clinical-stage biopharmaceutical company Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB) has released additional clinical results from the MoveDMD trial of edasalonexent, the company said.

In the Phase 2 MoveDMD trial and open-label extension, boys with Duchenne muscular dystrophy treated with edasalonexent on average grew in line with the growth of unaffected boys in the same age range.

These data were presented on Sunday, February 17, 2019, at the XVII International Conference on Duchenne and Becker Muscular Dystrophy in Rome, Italy.

In the MoveDMD trial, boys treated with edasalonexent grew an average of 2.1 inches taller per year and gained 2.9 pounds per year, and their overall body mass index decreased from 70th percentile of unaffected boys to the 55th percentile over 72 weeks of treatment, approaching the average body mass index for unaffected boys.

Boys treated with corticosteroids, the standard of care in DMD, typically experience excess weight gain and curtailed growth.

Edasalonexent is being studied in the Phase 3 PolarisDMD trial, which is actively enrolling patients. The PolarisDMD trial is evaluating the efficacy and safety of edasalonexent in patients with DMD and is intended to support an application for commercial registration of edasalonexent.

A total of 17 PolarisDMD clinical trial sites are now open for enrollment across the United States, Canada and Australia. Additional PolarisDMD clinical trial sites are expected to open in the United States, Canada, Europe, Australia and Israel in the next couple of months.

In total, the PolarisDMD trial will include approximately 40 clinical trial sites globally with enrollment expected to be completed in 2019.

The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months.

Boys on a stable dose of eteplirsen may be eligible to enroll. The primary efficacy endpoint is change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo.

Key secondary endpoints include the age-appropriate timed function tests: time to stand, 4-stair climb and 10-meter walk/run.

Assessments of growth, cardiac and bone health are also included as important potential areas of differentiation. Two boys will receive 100 mg/kg/day of edasalonexent for each boy that receives placebo, and, after 12 months, all boys are expected to receive edasalonexent in an open-label extension study.

The PolarisDMD trial design was informed by discussions with regulators as well as input from treating physicians, patient organizations and families of boys affected by Duchenne.

The Phase 3 PolarisDMD trial is designed to further validate the positive efficacy seen in the MoveDMD Phase 2 trial and open-label extension evaluating edasalonexent.

In the MoveDMD trial, the company observed that edasalonexent preserved muscle function and substantially slowed DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to stand, 4-stair climb and 10-meter walk/run) through 72 weeks of treatment compared to an off-treatment control period.

Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been well tolerated through more than 55 patient-years of treatment with no safety signals observed.

Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential new standard of care for all patients affected by DMD, regardless of their underlying mutation.

Edasalonexent inhibits NF-kB, which is a key link between loss of dystrophin and disease progression in DMD. NF-kB has a fundamental role in skeletal and cardiac muscle disease in DMD.

Catabasis Pharmaceuticals'lead programme is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy.

The company's global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne.