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news.Catabasis Pharmaceuticals, a clinical-stage drug development company built on a pathway pharmacology technology platform, announced that dosing of the first patient has been initiated in the MoveDMD trial, a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD).
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CAT-1004 is an oral small-molecule that the Company believes has the potential to be a disease-modifying therapy for DMD patients, regardless of the underlying dystrophin mutation, by inhibiting activated NF-kB.
NF-kB is a protein that plays an important role in regulating muscle health, and chronic activation of NF-kB has been reported in multiple skeletal muscle disorders, such as DMD. Preclinical studies have demonstrated that CAT-1004 can not only reduce muscle inflammation and degeneration, but also promote muscle regeneration.
DMD is a rare disease that involves progressive muscle degeneration that eventually leads to death and for which there are no approved therapies in the United States.
The MoveDMD trial will enroll approximately 18 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD, regardless of the specific dystrophin mutation. The enrolled boys will be steroid naive or have not used steroids for at least six months prior to the trial.
The trial will be conducted at three sites in the United States in two sequential parts, Part A and Part B. Part A of the study will assess the safety, tolerability and pharmacokinetics of CAT-1004 in patients at three dosing levels following seven days of dosing.
The primary endpoint for Part A is safety. Part B will be a randomized, double-blind, placebo-controlled trial. The primary efficacy endpoint in Part B will be change in muscle inflammation as measured by magnetic resonance imaging (MRI) of leg muscles. Additional measures of physical function and muscle strength will be collected.
CAT-1004 is an oral small-molecule 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. In skeletal muscle, activated NF-kB drives muscle degeneration and suppresses muscle regeneration.
In animal models of DMD, CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and degeneration and increased muscle regeneration. In Phase 1 clinical trials, CAT-1004 inhibited activated NF-kB and was well-tolerated with no observed safety concerns. The FDA has granted CAT-1004 orphan drug designation for the treatment of DMD.