AVI BioPharma Announces Commencement of Dosing in Clinical Trial for Duchenne Muscular Dystrophy
December 21 2007 - 3:08PM
Business Wire
AVI BioPharma, Inc. (Nasdaq:AVII), today announced dosing of the
first patient in a proof-of-principle clinical trial using
AVI-4658, AVI�s lead drug candidate for Duchenne muscular dystrophy
(DMD), based on the company�s proprietary ESPRIT (Exon Skipping
Pre-RNA Interference Technology) drug platform. AVI-4658 is
designed to skip exon 51 of the dystrophin gene, and thus benefit
DMD patients with certain types of mutations that impair the
function of dystrophin, a key protein in muscle cells. The trial is
being conducted by research teams at the Imperial College London,
in collaboration with the United Kingdom-based MDEX Consortium. The
trial will include up to nine boys with DMD, each of whom will
receive a single intramuscular (IM) administration of the drug. Two
to three weeks following the injection, the muscle will be biopsied
and examined for molecular evidence of corrected dystrophin
production. Preclinical studies have demonstrated that AVI-4658
restores proper RNA reading frame and production of dystrophin in
cells from patients with certain types of deletions in the gene
that codes for dystrophin production. This trial will be the first
to assess the drug�s effect in patients. The principal investigator
for the U.K. study is Professor Francesco Muntoni, Department of
Paediatrics, Hammersmith Hospital Campus, Imperial College, London.
The coordinating investigator of the project is Professor Dominic
Wells, M.A., VetMB, Ph.D., MRCVS, Department of Cellular and
Molecular Neuroscience, Imperial College Faculty of Medicine.
Imperial College will serve as the sponsor for the trial, with AVI
BioPharma serving as its clinical development collaborator. �We are
pleased that this clinical trial in DMD is now moving forward. We
are simultaneously moving toward initiating clinical trials
evaluating longer-term systemic administration of AVI-4658,� said
K. Michael Forrest, interim chief executive officer of AVI. AVI
recently announced it has received a translational research grant
of $2.45 million from Charley�s Fund to support the selection and
development of a lead molecule designed to skip exon 50 and restore
production of functional dystrophin. This therapeutic approach is
similar to that of AVI-4658, but targets patients with a different
set of mutations. In addition, in November, AVI announced that the
U.S. Food and Drug Administration (FDA) granted orphan drug
designation for AVI-4658 for treatment of DMD. Earlier this month,
the FDA also granted Fast Track status to the same product
candidate. In parallel with the U.K. study, AVI is moving toward
initiating clinical trials evaluating longer-term systemic
administration of AVI-4658. AVI�s DMD research and development
programs are being conducted in conjunction with the company�s DMD
cross-licensing and development partner, Ercole Biotech Inc. About
ESPRIT Technology In normal genetic function, gene transcription
produces a full-length pre-RNA that is then processed to a much
shorter and functional messenger RNA. The mRNA is the template for
creating a protein. During pre-RNA processing, packets of useful
genetic information, called exons, are snipped out of the
full-length RNA and spliced together to make the functional mRNA
template. AVI�s proprietary third-generation NEUGENE� chemistry can
be used to target splice-joining sites in the pre-RNA, thus forcing
the cell machinery to skip over targeted exons, providing altered
mRNA, which in turn produces altered proteins. In some diseases,
such as DMD, skipping an exon can restore a proper RNA reading
frame and restore at least partial function of the protein to
overcome the devastating clinical consequences of the mutation.
About Duchenne Muscular Dystrophy DMD is the most common fatal
genetic disorder to affect children around the world. It is a
devastating and incurable muscle-wasting disease associated with
specific inborn errors in the gene that expresses dystrophin, a
protein that is an essential component for striated muscle
function. When dystrophin is missing or nonfunctional due to a
mutation in coding of the dystrophin gene, as it is in DMD, the
result is membrane leakage and fiber damage, ultimately leading to
degeneration and death of the muscle fiber. There is no cure or
effective treatment for DMD. Approximately one in 3,500 boys is
born with DMD, and an estimated 15,000 to 20,000 children are
afflicted in the United States alone. About AVI BioPharma AVI
BioPharma develops therapeutic products for the treatment of
life�threatening diseases using third�generation NeuGene antisense
drugs and ESPRIT exon skipping technology. AVI's ESPRIT technology
is initially being applied to potential treatments for Duchenne
muscular dystrophy. AVI's NeuGene compounds are also designed to
treat cardiovascular restenosis, and aid in Coronary Artery Bypass
Graft (CABG) procedures. In addition to targeting specific genes in
the body, AVI's antiviral program uses NeuGene antisense compounds
to combat disease by targeting single�stranded RNA viruses,
including Marburg virus, Ebola Zaire virus, and H5N1 avian
influenza virus. More information about AVI is available on the
company's Web site at www.avibio.com. �Safe Harbor� Statement under
the Private Securities Litigation Reform Act of 1995: The
statements that are not historical facts contained in this release
are forward-looking statements that involve risks and
uncertainties, including, but not limited to, the results of
research and development efforts, the results of preclinical and
clinical testing, the effect of regulation by the FDA and other
agencies, the impact of competitive products, product development,
commercialization and technological difficulties, and other risks
detailed in the company�s Securities and Exchange Commission
filings.
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