Catabasis Pharmaceuticals has initiated an open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial investigating edasalonexent (CAT-1004) in the treatment of boys with Duchenne muscular dystrophy (DMD).
Edasalonexent is a non-corticosteroid, oral small molecule investigational drug that inhibits activated NF-ĸB. In boys with Duchenne MD, the absence of dystrophin combined with mechanical stress in the muscle leads to a robust activation of NF-ĸB. Activated NF-ĸB drives muscle damage and prevents muscle regeneration.
The Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy (MoveDMD) is a three-part, multi-site trial evaluating the efficacy, safety, pharmacodynamics (PD) and pharmacokinetics (PK) of CAT-1004 in boys with DMD. Overall, 36 ambulatory boys ages 4 to 8 were enrolled. The trial’s primary endpoint is change in magnetic resonance imaging (MRI).
Part A of MoveDMD started in April 2015 and is now complete. All the doses of CAT-1004 examined were well tolerated with no major safety events observed. The majority of adverse events were mild and related to gastrointestinal events, mostly diarrhea.
Part B is a randomized, double-blind, placebo-controlled trial that evaluated the safety, efficacy, pharmacokinetics, and pharmacodynamics of edasalonexent over 12 weeks at two dosing levels: 67 mg/kg a day and 100 mg/kg a day.
Patients who participated in Part A could also participate in Part B, along with newly enrolled male patients. After completing Part B, all patients will receive CAT-1004 for 36 weeks in Part C, the open-label portion of the CAT-1004-201 study.
In the open-label extension, all patients will be treated with edasalonexent for 36 weeks beyond the 12-week placebo-controlled period. Safety will be monitored and other evaluations, including MRI, muscle strength measures, timed function tests, the North Star Ambulatory Assessment, and the pediatric outcomes data collection instrument (PODCI), will be performed.
This study is currently recruiting participants. Catabasis expects to report top-line data later this year.
“We are pleased to extend edasalonexent dosing in the MoveDMD trial based on the acceptable safety and tolerability data seen to date,” Catabasis Chief Medical Officer Joanne Donovan, MD, PhD, said in a press release.
“The MoveDMD open-label extension is expected to inform on safety and efficacy of edasalonexent when administered for up to 48 weeks, and is important to our overall development strategy,” she said, adding that Catabasis appreciates the support it has received from the DMD community for the clinicial trial.
Erika Finanger, MD, a principal investigator in the MoveDMD trial, said she is pleased with both the progress of the study and to offer additional dosing of CAT-1004 to Duchenne MD boys when they complete the 12-week portion of the trial.
“We need therapies that have the potential to make a meaningful difference in boys affected by DMD regardless of the underlying mutation,” said Finanger, assistant professor for pediatrics and neurology at Oregon Health & Sciences University and a consulting neurologist at Shriners Hospitals for Children.