Catabasis Pharmaceuticals, Inc. announced positive top-line results from Part A of the MoveDMD trial, a Phase 1 /2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD or Duchenne). All three doses of CAT-1004 tested were generally well tolerated with no safety signals observed. The majority of adverse events were mild in nature, and the most common adverse events were gastrointestinal, primarily diarrhea.

There were no serious adverse events and no drug discontinuations. Pharmacokinetics results demonstrated CAT-1004 average plasma exposure levels consistent with those previously observed in adults at which inhibition of NF-kB was observed. Based on these results, Catabasis plans to initiate Part B of the MoveDMD trial in the first half of 2016.

Catabasis plans to submit the data from Part A of the trial for presentation at an upcoming medical meeting. CAT-1004 is an oral small-molecule that the Company believes has the potential to be a disease-modifying therapy for the treatment of Duchenne, regardless of the underlying dystrophin mutation. CAT-1004 is an inhibitor of NF-kB, a protein that is chronically activated in DMD as well as multiple other skeletal muscle disorders.

In animal models of DMD, CAT-1004 inhibited NF-kB, reduced muscle degeneration and increased muscle regeneration. The MoveDMD trial is being conducted in two sequential parts, Part A and Part B. In Part A of the MoveDMD trial, 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received CAT-1004. The boys were steroid naive or have not used steroids for at least six months prior to the trial.

Part A of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100mg/kg/day) during seven days of dosing. Part B will be a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week period. The boys in the first part of the trial will be asked to participate in the second part of the trial, and additional participants will also be enrolled.