Catabasis, MDA partner to develop novel oral disease-modifying treatment for DMD

The parties will work on the part B portion of the MoveDMD clinical trial of CAT-1004. MDA will provide funding required to transport patients who need to travel to participate in the study.

Catabasis reported positive top-line results for safety, tolerability and pharmacokineticsin in the recently completed part A of the MoveDMD trial.

The company plans to start part B of the trial in the first half of this year.

The boys in part A of the study will be asked to take part in part B and additional participants will also be enrolled.

Catabasis is in the process of searching for more patients who are interested to take part in part B of the trial.

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 a key role in muscle health.

Activated NF-kB drives muscle degeneration and suppresses muscle regeneration in skeletal muscle.

In animal models of DMD, CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and degeneration and increased muscle regeneration.

Catabasis said in phase 1 clinical studies, CAT-1004 inhibited activated NF-kB and was well tolerated with no observed safety concerns in adults.

The US Food and Drug Administration granted CAT-1004 orphan drug, fast track and rare pediatric disease designations to treat DMD.

CAT-1004 has also secured orphan medicinal product designation from the European Commission for DMD.

Catabasis is presently undertaking the MoveDMD Phase 1 / 2 trial of CAT-1004 in 4-7 year-old boys with DMD.

Image: Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: courtesy of Dr. Edwin P. Ewing, Jr.