Catabasis Pharmaceuticals has dosed the first patient as part of a part B of the MoveDMD trial, which is a 12-week study to assess the efficacy of CAT-1004 in Duchenne muscular dystrophy (DMD) disorder.
CAT-1004 is an inhibitor of NF-kB and an oral small-molecule. It could be a disease-modifying therapy for treatment of DMD, regardless of the underlying dystrophin mutation.
In animal models of DMD, CAT-1004 inhibited NF-kB, which is a protein activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration.
Catabasis is conducting the MoveDMD trial in two chronological parts, part A and part B.
During part A of the MoveDMD trial, 17 ambulatory boys between the age of four and seven with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received CAT-1004.
The boys were steroid naive or had not used steroids for at least six months prior to the trial. Part A was conducted at three sites in the US.
The trial assessed the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels, which include 33mg/kg/day, 67mg/kg/day and 100mg/kg/day, during seven days of dosing.
Catabasis has received positive biomarker results from part A of the MoveDMD trial, showing successful NF-kB target engagement.
The biomarker assay piloted in boys affected by DMD demonstrated a statistical decrease in NF-kB gene expression markers compared to baseline at the 67mg/kg and 100mg/kg per day doses, as well as a statistically significant dose response in the gene expression data measured from whole blood samples.
Catabasis previously announced positive safety, tolerability and pharmacokinetics results from part A of the trial.
Part B the MoveDMD trial is a randomised, double-blind, placebo-controlled trial that will evaluate the safety and efficacy of CAT-1004 in DMD over a period of 12 weeks at five clinical trial sites in the US at two dosing levels, including 67mg/kg/day and 100mg/kg/day.
The boys who participated in part A of the MoveDMD trial are also expected to be involved in part B, with more participants expected to be enrolled for a total of around 30 boys.
Catabasis is currently identifying patients who are interested in participating in part B of the trial.