Catabasis Pharmaceuticals starts MoveDMD part B trial of CAT-1004

12th April 2016 (Last Updated April 12th, 2016 18:30)

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.

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.

"Catabasis has received positive biomarker results from part A of the MoveDMD trial, showing successful NF-kB target engagement."

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.