Duchenne Muscular Dystrophy Therapy Designated Orphan Drug

Catabasis announced that the FDA has granted Orphan Drug designation to CAT-1004 for the treatment of Duchenne Muscular Dystrophy (DMD).

CAT-1004 is a new chemical entity that inhibits activated NF-κB. CAT-1004 is designed to reduce muscle inflammation and subsequent degeneration, and increase regenerating muscle cells. Catabasis’ proprietary SMART (Safely Metabolized And Rationally Targeted) Linker technology enables selective intracellular delivery and synergistic activity of CAT-1004, which is a conjugate of salicylate and the omega 3 fatty acid docosahexaenoic acid (DHA).

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Catabasis plans to initiate a Phase 2 clinical trial for CAT-1004 in the first half of 2015.

For more information visit Catabasis.com.