Catabasis Pharmaceuticals announced top-line results from the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy (DMD). The trial did not meet the primary endpoint, which was a change from baseline in the North Star Ambulatory Assessment (NSAA) over one year of edasalonexent compared to placebo. The secondary endpoint timed function tests (time to stand, 10-meter walk/run and 4-stair climb) also did not show statistically significant improvements. Edasalonexent was generally safe and well-tolerated in this trial. Catabasis is halting the development of edasalonexent, including the ongoing GalaxyDMD open-label extension trial. Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published.
PolarisDMD was a one-year placebo-controlled trial designed to evaluate the safety and efficacy of edasalonexent in boys ages 4-7 with DMD. The global trial enrolled 131 boys across eight countries, with any mutation type, who were not on steroids. Edasalonexent was well-tolerated. The majority of adverse events were mild in nature and the most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash.
Catabasis plans to work with external advisors to explore and evaluate strategic options going forward.
Edasalonexent inhibits NF-kB, a protein which plays a fundamental role in skeletal and muscle disease in Duchenne. By inhibiting NF-kB, edasalonexent had the potential to decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression.
The FDA granted edasalonexent Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of DMD. The European Commission granted Orphan Medicinal Product Designation for edasalonexent for the treatment of DMD.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a genetic condition that affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and loss (atrophy) of skeletal and heart muscles. Early signs of DMD may include delayed ability to sit, stand, or walk and difficulties learning to speak. Muscle weakness is usually noticeable in early childhood. Most children with DMD use a wheelchair by their early teens. Heart and breathing problems also begin in the teen years and lead to serious, life threatening complications. DMD is caused by genetic changes (DNA variants) in the DMD gene. DMD is inherited in an X-linked recessive pattern and may occur in people who do not have a family history of DMD. While there is no known cure for DMD, there are treatments that can help control symptoms.