Massachusetts based Catabasis Pharmaceuticals, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics based on their proprietary pathway pharmacology technology platform, recently announced that they have begun treating patients in their MoveDMD trial, a Phase 1/2 trial of CAT- 1004 for the treatment of Duchenne muscular dystrophy (DMD), with the first patient receiving their initial dose on June 17th.
This announcement was made shortly after Joanne Donovan, M.D., Ph.D., chief medical officer of Catabasis, gave a presentation entitled, “CAT-1004, an Oral Agent Targeting NF-kB in Development for Treatment of Duchenne Muscular Dystrophy: Design of MoveDMD, a Phase 1/2 Trial” at the Parent Project Muscular Dystrophy’s (PPMD) Annual Connect Conference held June 18-21, 2015, in Washington, DC. In a personal note to parents, Dr. Donovan made it known that, “Catabasis understands that the decision to involve your child in a clinical trial is significant. We have looked for ways to make the trial easier on everyone in your family including travel support. No website can answer all of the questions that you will have so free to contact me with any questions.”
The trial is expected to enroll at least 18 boys between the ages of 4 and 7 who meet the following study criteria:
- Confirmed diagnosis of DMD, regardless of the specific dystrophin mutation.
- Do not have a history of being treated with steroids or have not used steroids for at least six months prior to the trial.
- Have the ability to walk independently (assistive devices are permitted)
- Have an adequate immunization history as defined by an immunization for influenza within 1 year AND either full immunization (series of 2 vaccinations) or natural acquired history of varicella infection with confirmation of development of varicella antibodies
- Be able to participate in 1 of the 3 study locations: Gainesville, Florida; Portland, Oregon; Philadelphia, Pennsylvania
The purpose of the trial is to assess the safety, tolerability and pharmacokinetics of CAT- 1004 in patients at three dosing levels following seven days of dosing, as well as, the change in muscle inflammation as measured by magnetic resonance imaging (MRI) of leg muscles, along with additional measures of physical function and muscle strength.
For more information on study enrollment please click here.
CAT-1004 is an investigational drug that inhibits activated NF-kB, a protein that coordinates cellular response to muscular damage, stress and inflammation and plays an important role in muscle health.