EDASALONEXENT (CAT-1004) - Catabasis’ MoveDMD Trial

 

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • Currently, the Phase 2 part of the MoveDMD® trial is ongoing.  MoveDMD is a Phase 1/2 clinical trial in boys with Duchenne Muscular Dystrophy. The first part of the trial (Part A) has completed and the results were positive: edasalonexent was generally well tolerated, there were no safety issues, the pharmacokinetics were in line with what was expected, and biomarkers showed an effect on target genes.  The second part of the trial (Part B) is complete. Although the magnetic resonance imaging (MRI) T2 composite primary endpoint at 12 weeks was not achieved, all 6 function-associated exploratory endpoints assessed in Part B showed numerical improvement in the higher dose group compared to placebo, although not statistically significant. This includes the 4-stair climb, 10-meter walk/run, time to stand and the North Star Ambulatory Assessment as well as pediatric outcomes data collection instrument (PODCI) and muscle strength. No safety signals were observed in Part B and Glossary Link plasma exposure in Part B of the trial was consistent with that observed in Part A. The third part of the MoveDMD trial (Part C) is ongoing. In Part C, patients receive open-label edasalonexent for 60 weeks. Catabasis is expecting to report results from Part C periodically throughout the year.

 

Where is this research being done and who is funding this research?

  • This research is being done by Catabasis, a clinical-stage biopharmaceutical company with a mission to bring hope and life-changing therapies to patients and their families. Catabasis is focused on the treatment of rare diseases, including Duchenne. Both PPMD and MDA have supported patient travel for the MoveDMD trial.

 

What is the goal or purpose of this research?

  • Edasalonexent is an oral small Glossary Link molecule investigational drug candidate that has been observed to inhibit NF-kB. Dystrophin is a Glossary Link protein that keeps muscles healthy by maintaining the structure of muscle cells. In boys with Duchenne, the absence of dystrophin combined with mechanical stress in muscle leads to activation of NF-kB, which in turn drives muscle damage and prevents muscle regeneration. By inhibiting NF-kB, we believe that edasalonexent may reduce muscle damage and regenerate muscle in boys with Duchenne.

 

Where does the clinical trial take place?

  • Participating sites for the MoveDMD trial: Shriners Hospitals for Children, Portland, OR.The Children's Hospital of Philadelphia, Philadelphia, PA; University of Florida, Gainesville, FL; Nemours Children's Hospital, Orlando, FL; and University of California Los Angeles, Los Angeles, CA

 

Who is eligible to participate in a clinical trial?

  • Althouth enrollment is closed, requirements for participating in this clinical trial include:
    • Boys with a diagnosis of DMD (any confirmed Glossary Link mutation) between the ages 4 and 7 years
    • Ability to walk independently
    • No corticosteroid use within the past 6 months and no plans to start corticosteroids in the next 6 months
  • The key inclusion and exclusion criteria and additional details about this clinical trial are available at ClinicalTrials.gov. 

 

What will participants do in Part C of this trial?

  • Participants will receive edasalonexent for up to 60 weeks in Part C. Data on MRI of the leg muscles, physical function assessments and muscle strength will be collected at ~12 week intervals throughout the study.

 

Where can I learn more about this research?

  • You can learn more at www.catabasis.com and ClinicalTrials.gov (NCT02439216).
  • You can contact Catabasis directly with any questions at This email address is being protected from spambots. You need JavaScript enabled to view it.

 

(Updated 04/28/2017)