NF-kB has a role in potentiating muscle degeneration in Duchenne, and plays an important role in inflammation. Mouse models of DMD have shown that disease severity is reduced when NF-kB is inhibited.
Recent results of the phase 2 Move-DMD trial revealed that Catabasis Pharmaceuticals drug candidate Edasalonexent (CAAT-1004) slowed the rate of functional decline in boys aged 4-7 with DMD. Part C of the trial is now underway to determine the long-term effects of the drug, with results being expected by the end of 2017.
Edasalonexent is an inhibitor of NF-KB. In the mdx mouse model of DMD, activation of NF-KB has been detected even before the onset of dystrophic damage and degeneration. Therapies which target specific mutations only appeal to a subset of patients, whereas this drug could prove effective for patients regardless of specific mutation.
