Clinical-stage drug development firm Catabasis Pharmaceuticals has received fast-track designation from the US Food and Drug Administration (FDA) for its CAT-1004 to treat Duchenne muscular dystrophy (DMD).
DMD is a rare disease, which involves progressive muscle degeneration that can lead to death, while there are currently no approved therapies for the disease in the US.
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 crucial role in muscle health.
Catabasis co-founder and CEO Dr Jill Milne said: "Fast-track designation for CAT-1004 highlights its potential to treat a serious, life-threatening disease with few treatment options for these young patients.
"By targeting activated NF-kB in pre-clinical studies, CAT-1004 has demonstrated disease-modifying potential for this devastating condition."
Activated NF-kB promotes muscle degeneration and suppresses muscle regeneration in skeletal muscle.
The CAT-1004 inhibited activated NF-kB, reduced muscle inflammation and degeneration and increased muscle regeneration in animal models of DMD.
In Phase I clinical studies, CAT-1004 suppressed activated NF-kB and was well-tolerated with no observed safety concerns, said the company.
The company previously received FDA orphan drug designation for CAT-1004 to treat DMD.
Catabasis develops and commercialises novel therapeutics through using its proprietary safely metabolised and rationally targeted (SMART) linker technology platform.
The platform is based on the concept of treating diseases by simultaneously modulating multiple targets in one or more related disease pathways.
Image: Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: courtesy of Dr Edwin P Ewing Jr.