AVI BioPharma Announces Successful Clinical Trial of AVI-4658 for Treatment of Duchenne Muscular Dystrophy by Exon Skipping

[Chrissy]

AVI BioPharma Announces Successful Clinical Trial of AVI-4658 for Treatment of Duchenne Muscular Dystrophy by Exon Skipping

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AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced results from a Phase 1 trial of its drug candidate AVI-4658 for the treatment of Duchenne muscular dystrophy (DMD) by exon skipping. Biopsy data showed that injection of the drug into the muscles of a series of DMD patients successfully induced dystrophin production in each patient.

The proof of principle, single dose escalation study tested the effect of an intramuscular (IM) injection of AVI-4658 in boys with DMD. Each patient received an injection of 0.09 or 0.9 mg 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. 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 clinical study was performed in the UK by members of the MDEX Consortium led by Professor Francesco Muntoni. Professor Muntoni commented, "As a clinician and scientist, I am very pleased by these findings and the prospects they offer for the potential treatment of this serious, life threatening condition. Biopsies from muscles injected with the higher dose of test drug showed an unequivocal, widespread and robust response in terms of number of dystrophin positive muscle fibers. We will publish these exciting data in a peer-reviewed journal in due course."

Patients with DMD have a very low capacity to make dystrophin. In general, and in this study, DMD patients have less than 5% dystrophin positive muscle fibers prior to treatment.

"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," said Leslie Hudson, Ph.D., President and Chief Executive Officer of AVI BioPharma. "A multi-dose, dose escalation trial to examine the efficacy of the drug candidate following systemic administration (IV) -- also in collaboration with the MDEX Consortium -- was opened in December 2008 and our collaborators have begun the work up of the first cohort of DMD patients prior to dosing."

DMD is an incurable muscle-wasting disease associated with errors in the gene that makes dystrophin, a protein that plays a key structural role in muscle fiber function. The drug candidate AVI-4658 is designed to skip exon 51 of the dystrophin gene, allowing for restoration of the reading frame in the mRNA sequence. Restoration of dystrophin production achieved by skipping this exon may improve or significantly slow the disease process, thus prolonging and improving the quality of life for the affected patient population. It is important to note that different mutations in the dystrophin gene require different oligonucleotide drugs. In principle, approximately 80% of all DMD patients could be treated with exon-skipping drugs. AVI-4658, and the four related exon-skipping drugs under development in AVI BioPharma could be used to treat more than half of this population -- if they prove to be effective -- with a potential market value of approximately $2.0 billion. AVI BioPharma was granted orphan drug designation for AVI-4658 by the U.S. Food and Drug Administration in November 2007 and by the European Medicines Agency (EMEA) in December 2008.

The IM injection trial was funded by the Department of Health (UK) and conducted by the members of the MDEX Consortium, led by Professor Muntoni at Imperial College Healthcare NHS Trust facilities. Imperial College London is the sponsor for the trial, with AVI BioPharma serving as its clinical development collaborator.

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[WCGuy]

nucleic acid drugs are pretty difficult to roll out successfully.

While the concept is good, I wouldn't expect a lot of good outcomes.