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news.AVI BioPharma, a developer of RNA-based drugs, has announced positive results from a Phase I trial of its drug candidate AVI-4658 for the treatment of Duchenne muscular dystrophy by exon skipping.
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Biopsy data showed that injection of the drug into the muscles of a series of Duchenne muscular dystrophy (DMD) patients successfully induced dystrophin production in each patient.
The proof of principle, single dose escalation study tested the effect of an intramuscular injection of AVI-4658 in boys with DMD. Each patient received an injection of 0.09 or 0.9mg of AVI-4658 into the exterior digitorum brevis muscle of one foot and an injection of saline as placebo into the corresponding muscle of the opposite foot to provide an internal treatment comparison.
Approximately three to four weeks later, each injected muscle was biopsied and examined for evidence of dystrophin production. Results demonstrated that injection of AVI-4658 elicited dystrophin production in a dose dependent manner in all treated patients. Further, the drug was well tolerated, with no significant detectable drug-related adverse events, the company said.
Leslie Hudson, president and CEO of AVI BioPharma, said: These are promising data which support the continued development of AVI-4658 as a potential exon skipping treatment for DMD and are highly competitive with data disclosed by other companies working in this field.