AVI BioPharma Inc. (AVII), a developer of RNA-based therapeutics, reported that treatment with eteplirsen met the primary efficacy endpoint in a randomized, double-blind, placebo-controlled Phase IIb study in boys with Duchenne muscular dystrophy or DMD. Eteplirsen administered once weekly at 30mg/kg over 24 weeks resulted in a statistically significant increase in novel dystrophin compared to no increase in the placebo group.
In the study, a shorter duration of eteplirsen treatment, 12 weeks, did not show a significant increase in novel dystrophin, despite administration of the drug at a higher dose, the company added. This finding suggests that a longer duration of dosing is required before meaningful levels of dystrophin are produced.
Eteplirsen is AVI's lead drug candidate that is systemically delivered for the treatment of a substantial subgroup of patients with DMD.
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