Arrowhead Pharmaceuticals Files for Regulatory Clearance to Initiate a Phase 1/2 Study of ARO-DUX4 for Facioscapulohumeral Muscular Dystrophy
“FSHD is a debilitating disease caused by the aberrant expression of the DUX4 gene in skeletal muscle. ARO-DUX4 engages the RNA interference pathway to selectively inhibit DUX4 expression and is a promising strategy to reverse the downstream myotoxicity of DUX4 and potentially enable stabilization or improvement in muscle function in patients. We believe ARO-DUX4 has the potential to be an impactful new medicine for patients,” said
An application for approval of the clinical trial was submitted to the
About Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is a debilitating, muscle-wasting disease characterized by progressive asymmetrical weakness and fatty infiltration of skeletal muscles in the face, shoulders, upper arms, abdomen, or lower leg and is the third most common form of muscular dystrophy. There is currently no approved therapy for FSHD.
ARO-DUX4 is an investigational RNAi therapeutic designed to reduce expression of the gene that encodes the human double homeobox 4 (DUX4) protein as a potential treatment for FSHD. FSHD is an autosomal dominant disease associated with the failure to maintain complete epigenetic suppression of DUX4 expression in differentiated skeletal muscle, leading to overexpression of DUX4, which is myotoxic and can lead to muscle degeneration. Expression of downstream DUX4 associated genes over time leads to muscle fatty infiltration and progressive muscle weakness. As DUX4 overexpression is the cause of muscle pathology in FSHD patients, it is expected that the selective knockdown of DUX4 messenger RNA transcripts using RNA interference will inhibit DUX4 translation, thereby preventing or reversing downstream myotoxicity and allowing muscle repair and partial or complete return of normal muscle function in patients.
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