Catabasis announced positive biomarker data from Part A of the MoveDMD trial, as well as initiation of dosing for the first patient for Part B (Phase 2) - a 12-week trial to assess the efficacy of CAT-1004 in Duchenne. We look forward to continued success from our partners at Catabasis and thank you to all the families participating in this trial!
Read the Press Release:
Catabasis Pharmaceuticals Initiates Part B of the MoveDMDSM Trial of CAT-1004 for the Treatment of Duchenne Muscular Dystrophy
- Positive NF-kB Biomarker Data from Part A of the MoveDMD Trial in Patients
- CAMBRIDGE, MA, April 12, 2016 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that dosing of the first patient has been initiated in Part B of the MoveDMD trial, a 12-week trial to assess the efficacy of CAT-1004 in Duchenne muscular dystrophy (DMD). Catabasis also announced positive biomarker results from Part A of the MoveDMD trial, demonstrating successful NF-kB target engagement. The biomarker assay piloted in boys affected by DMD showed a statistically significant decrease in NF-kB gene expression markers compared to baseline at the 67 mg/kg and 100 mg/kg per day doses as well as a statistically significant dose response in the gene expression data measured from whole blood samples. Catabasis has previously reported positive safety, tolerability and pharmacokinetics results from Part A of the trial. “We are very glad to advance CAT-1004 into Phase 2 with the initiation of Part B of the MoveDMD trial in boys affected by DMD,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis.
“We are pleased with the positive NF-kB biomarker results in Part A of the trial demonstrating target engagement as well as the 5 - 10 fold higher CAT-1004 concentration that we have seen in muscle compared to plasma in pre-clinical models.”
“I am glad to see the advancement of this novel potential therapy in boys affected by Duchenne,” said H. Lee Sweeney, Ph.D., Professor, Director, Myology Institute at the University of Florida. “Therapies that have the potential to make a meaningful difference are needed to address the profound unmet medical need in DMD.”
CAT-1004 is an oral small-molecule that the Company believes has the potential to be a diseasemodifying therapy for the treatment of DMD, 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 and rare diseases. 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 had 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 100 mg/kg/day) during seven days of dosing. Part B is a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of CAT-1004 in DMD over a 12-week period at 5 clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day. The boys that participated in Part A of the MoveDMD trial are asked to participate in Part B and additional participants are expected to be enrolled for a total of approximately 30 boys. We are currently identifying additional patients who are interested in participating in Part B of the trial. Entry criteria are similar to those in Part A. The Parent Project Muscular Dystrophy and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.
More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on ClinicalTrials.gov under trial identifier NCT02439216.