Catabasis Pharmaceuticals and the Muscular Dystrophy Association (MDA) have announced a partnership that will support Part B of the MoveDMD clinical trial, currently evaluating the effectiveness and safety of Duchenne muscular dystrophy (DMD) treatment candidate CAT-1004 in children.
CAT-1004, believed to be a disease modulator regardless of mutation, is an oral small molecule that inhibits activated NF-kB, a protein that, in skeletal muscle, is involved in muscle degeneration and suppression of muscle regeneration. CAT-1004 has been granted Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA and the Orphan Medicinal Product designation in Europe for the treatment of DMD. The drug has proven, in animal studies, to decrease inflammation and muscle degeneration and increase regeneration. In Phase 1 clinical trials, CAT-1004 was shown to inhibit activated NF-kB and was well tolerated, with no safety concerns, in adults.
The MoveDMD clinical trial (NCT02439216) is a Phase 1/2 study, divided in two parts, of CAT-1004 in DMD-affected boys, from ages 4 to 7, with any confirmed mutation. Part A of the trial, evaluating the safety, tolerability, and pharmacokinetics of CAT-1004, was completed with successful results. Part B, expected to begin in the first half of 2016, will include eligible boys from the study’s part A and is currently recruiting more patients to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week period.
The MDA will provide the funds for the transportation of DMD selected patients that need to travel to the sites of the study. “The DMD community desperately needs and deserves treatment options that will help families by allowing for everyday freedoms that many take for granted such as fastening a button without assistance, moving into a more comfortable position independently, or just being able to hug the people you love,” said Valerie Cwik, M.D., MDA executive vice president and chief medical and scientific officer, in a press release. “We are proud to partner with Catabasis and look forward to what is sure to be an exciting partnership that will work toward bringing urgently needed treatments to our families.”