Avidity eyes registrational path for rare muscle disease drug after early Phase 1/2 data

Avidity Biosciences’ antibody oligonucleotide conjugate has shown signs of efficacy in an early-stage test in patients with a rare muscle-wasting disease.

Just last month, Leerink analysts were cautious about the path forward for the treatment, dubbed del-brax, noting there are “gaps in knowledge” for facioscapulohumeral muscular dystrophy (FSHD). But with the preliminary data, Avidity is now expediting plans for registrational development.

Early data shows del-brax achieved more than 50% mean reductions in DUX4-regulated gene expression in the muscles of 12 FSHD patients. Signs of functional improvement were also observed, including improved strength in the upper and lower limb muscles, although the study is not statistically powered to assess functional benefit, according to Avidity.

The placebo-controlled Phase 1/2 FORTITUDE trial tested two doses of del-brax. The condition is caused by an abnormal expression of the DUX4 gene and leads to lifelong loss of muscle function, pain and…
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