View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. News
April 12, 2016

Catabasis Pharmaceuticals starts MoveDMD part B trial of CAT-1004

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.

Related Companies

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena