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AVI BioPharma Announces Clinical Development Initiative for Muscular Dystrophy
Posted on 2006-12-07 08:50:00
AVI BioPharma, Inc. announced the initiation of a clinical program for the treatment of Duchenne Muscular Dystrophy (DMD) using its ESPRIT (Exon Skipping Pre-RNA Interference Technology) exon-skipping technology. The clinical program is based on positive preclinical data amassed over the past two years, including collaborator studies published in the October 2006 issue of Neuromuscular Disorders and the February 2006 issue of Nature Medicine. This new technology application is designed to delete disease-causing gene sequences in patients with certain genetic diseases, including DMD.
"AVI introduced this new approach to treating genetic disorders in fall 2005," said Denis R. Burger, Ph.D., chief executive officer of AVI. "ESPRIT therapeutics enable the body to bypass defective genetic information at the RNA processing level, providing a new and very potent tool for altering many disease mechanisms."
AVI's clinical program will start in collaboration with the United Kingdom-based MDEX Consortium, which was established and funded to conduct clinical trials in DMD. In this proof-of-principle, controlled, dose-escalating trial, up to nine young boys with DMD will receive a single, intramuscular administration of AVI-4658, which targets exon 51. After four to six weeks, the muscle will be biopsied and examined for molecular evidence of dystrophin production, representing a positive end point. Positive results will support the expansion of AVI-4658 clinical development to systemic administration. In collaborative preclinical studies in the mdx mouse model, dystrophin was produced for at least 16 weeks following initial systemic dosing.
The principal investigator for this study is professor Francesco Muntoni, Department of Paediatrics, Hammersmith Hospital Campus, Imperial College, London. The coordinating investigator of the project is professor Dominic Wells, MA, VetMB, Ph.D., MRCVS, Department of Cellular and Molecular Neuroscience, Imperial College Faculty of Medicine, London. Imperial College will serve as the sponsor for the trial, with AVI BioPharma serving as its clinical development collaborator. The study received a favorable review by the U.K.'s Gene Therapy Advisory Committee (GTAC) in September 2006.
In addition, AVI plans to file an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) in 2007 for a company-sponsored, multicenter DMD clinical study in the United States. This trial will eventually expand beyond exon 51 to other exons implicated in DMD.
As part of this initiative, AVI is planning to host a clinical investigator meeting in the first quarter of 2007. DMD-experienced clinical investigators who are interested in participating should contact Peter O'Hanley, Ph.D., M.D., senior vice president, Clinical Development and Regulatory Affairs, AVI, for more information.
Finally, AVI is exploring sponsorship and support for future DMD clinical trials in Australia and elsewhere with its collaborators.
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