- The FDA designates privately-held Catabasis Pharmaceuticals' investigational NF-kB inhibitor, CAT-1004, an Orphan Drug for the treatment of Duchenne muscular dystrophy (DMD).
- NF-kB is a key mediator of cellular injury. Evidence suggests that activated NF-kB contributes to the underlying pathology of DMD.
- In a Phase 1 trial, CAT-1004 was well-tolerated and demonstrated a significant reduction of activated NF-kB. The company plans to proceed to Phase 2 development in 1H 2015.
- Among the benefits of Orphan Drug designation is a seven-year period of market exclusivity if approved.
- DMD-related tickers: (SRPT +7.6%)(RNA +63.3%)(BMRN +1.7%)(PTCT +3.4%)(MRNA -3.3%)(ROSG-OLD +0.8%)
Catabasis Pharmaceuticals' CAT-1004 tagged an Orphan Drug for DMD
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