Duchenne MD Therapy Showing Potential in Initial Clinical Trial

Catabasis Pharmaceuticals, Inc., recently announced that the first part of its MoveDMD clinical trial, a Phase 1/2 study of CAT-1004 in the treatment of Duchenne muscular dystrophy (DMD), has received positive top-line results, and it will soon begin the study’s second phase.

“We are pleased with these results and believe that they support the initiation of Part B of the MoveDMD trial in the first half of 2016” Catabasis’ CEO, Jill Milne, PhD, said in a press release. “We sincerely thank the boys, their families, the investigators and the study site staff who participated in the trial as well as the advocacy organizations for their support of the CAT-1004 program.”

CAT-1004 is an inhibitor of NF-kB, a protein that is chronically activated in DMD and in multiple other skeletal muscle disorders. The drug is an oral small molecule, and Catabasis believes it has the potential to be a disease-modifying therapy for the treatment of DMD regardless of the underlying dystrophin mutation. In animal models of DMD, CAT-1004 was seen to inhibit NFkB, and reduce muscle degeneration and increase regeneration.

The MoveDMD study has two sequential parts, A and B. Part A recruited 17 ambulatory boys, between 4 and 7 years old, with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations to receive CAT-1004. Three sites in the United States participated in the trial, assessing the safety, tolerability, and pharmacokinetics of CAT-1004 given in three daily doses — 100mg/kg, 67mg/kg, and 33mg/kg — for seven days.

CAT-1004 was generally well-tolerated in all three doses, with no serious adverse effects or treatment discontinuation, Catabasis reported. Pharmacokinetics results demonstrated CAT-1004 average plasma exposure levels consistent with those previously observed in adults with an NF-kB inhibition. Reported mild adverse effects were commonly gastrointestinal, primarily diarrhea. The company plans to present trial data from Part A at an upcoming medical conference.

Part B will be a randomized, placebo-controlled, double-blind trial to evaluate the treatment’s safety and efficacy over a 12-week period (84 days). Study participants from Part A will be asked to enroll in Part B, and additional DMD patients who meet the initial entry criteria are being identified. More information on Part B of the MoveDMD trial, including participation, is available through a Catabasis website and through clinical trials.gov (NCT02439216).

“The unmet medical need in Duchenne is profound and potential therapies that could make a meaningful difference are needed,” said Richard Finkel, MD, chief of the Division of Neurology at Nemours Children’s Health System. “Showing positive safety, tolerability and pharmacokinetics results is an important milestone in the development of CAT-1004. I look forward to the advancement of this novel potential therapy.”