Abstract
We evaluate the efficacy of losmapimod in inhibiting the aberrant expression of DUX4, the root cause of FSHD. Secondary objectives are to evaluate safety, tolerability, pharmacokinetic (PK), and target engagement (TE) in blood and muscle. FSHD is caused by aberrant expression of the transcription factor DUX4 in skeletal muscle. DUX4 activates a downstream transcriptional program resulting in skeletal muscle loss and motor disability. Losmapimod is a small molecule inhibitor of p38α/β. Pre-clinical studies demonstrated treatment with losmapimod resulted in dose-dependent reduction of DUX4 protein, DUX4 transcriptional program and skeletal muscle cell death in FSHD myotubes across all genotypes tested.
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