19 December 2019 - - US-based clinical-stage biopharmaceutical company Catabasis Pharmaceuticals, Inc. (NASDAQ: CATB) has released results from an analysis of the baseline characteristics of the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy, the company said.

The analysis shows that overall the patients enrolled in the Phase 3 trial have similar characteristics to the patients that enrolled in the previous Phase 2 MoveDMD trial.

Top-line results from the Phase 3 PolarisDMD trial are expected in 4Q20 and the trial is anticipated to support an NDA filing in 2021.

Both the Phase 3 PolarisDMD trial and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4 to 7 (up to 8th birthday) with any mutation type whom had not been on steroids for the previous 6 months.

The Phase 3 trial enrolled 131 boys at 37 sites in the United States, Canada, Europe, Israel and Australia and 98% were steroid-naïve. The Phase 2 trial enrolled 31 boys in the United States, all of whom were steroid-naïve.

A comparison was made between the populations at the baseline of each trial.

Baseline age, North Star Ambulatory Assessment score and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run) were similar in both trials; there were no significant differences in these baseline characteristics between the two trials.

Distribution of baseline NSAA and timed function tests was less variable in the Phase 3 trial than in the Phase 2 trial. These findings support the assumptions on which the Phase 3 trial was powered.

Boys in the Phase 3 trial had an elevated heart rate and elevated muscle enzyme levels at baseline, which was also consistent with the Phase 2 trial population.

In the earlier 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 well-tolerated.

In more than 60 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 (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 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, the company 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.

The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of edasalonexent in patients with DMD. The trial enrolled patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who had not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen were also 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.

For each boy that receives placebo, two boys are receiving 100 mg/kg/day of edasalonexent and after 12 months, all boys are expected to receive edasalonexent in the open-label extension study GalaxyDMD.

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.

Top-line results from the Phase 3 PolarisDMD trial are expected in 4Q20.

At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families.

Its lead programme is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy.