AVI BioPharma Announces Successful Clinical Trial of AVI-4658 for Treatment of Duchenne Muscular Dystrophy by Exon Skipping
January 21 2009 - 8:00AM
Marketwired
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.
Conference Call Details
AVI BioPharma, Inc. will hold a conference call today,
Wednesday, January 21, 2009, at 9:00 a.m. Eastern time (6:00 a.m.
Pacific) to provide an update on the Company's Duchenne muscular
dystrophy program.
Individuals interested in listening to the live conference call
may do so by dialing 866-303-7746 toll free within the United
States and Canada, or 416-641-6139 for international callers.
A replay of the call will be available by dialing 800-408-3053
toll free within the U.S. and Canada or 416-695-5800. The passcode
for the replay is 3281420. In addition, a recording of the call
will be available within approximately 24 hours at
www.avibio.com.
About Duchenne Muscular Dystrophy (DMD)
DMD is one of the most common fatal genetic disorders to affect
children around the world. Approximately one in every 3,500 boys
worldwide is afflicted with Duchenne muscular dystrophy with 20,000
new cases reported each year. It is a devastating and incurable
muscle-wasting disease associated with specific inborn errors in
the gene that codes for dystrophin, a protein that plays a key
structural role in muscle fiber function. Symptoms usually appear
in male children before age 6. Progressive muscle weakness of the
legs and pelvis eventually spreads to the arms, neck, and
respiratory muscles. By age 12, patients are confined to a
wheelchair. Eventually, the condition progresses to complete
paralysis with increasing difficulty in breathing. The condition is
terminal and death usually occurs before the age of 30. The
outpatient cost of care for a non-ambulatory DMD boy is among the
highest of any disease and has been estimated at between
$400-500,000 per annum. There is currently no cure for DMD, but for
the first time in decades, there are promising therapies in or
moving into clinical development.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based drugs utilizing proprietary derivatives of its antisense
chemistry (morpholino-modified phosphorodiamidate oligomers or
PMOs) that can be applied to a wide range of diseases and genetic
disorders through several distinct mechanisms of action. Unlike
other RNA-based therapeutic approaches, AVI's antisense technology
have been used to directly target both messenger RNA (mRNA) and its
precursor (pre-mRNA), allowing for both up- and down-regulation of
targeted genes and proteins. AVI's RNA-based drug programs are
being evaluated for the treatment of Duchenne muscular dystrophy as
well as for the treatment of cardiovascular restenosis through our
partner Global Therapeutics, a Cook Group Company. AVI's antiviral
programs have demonstrated promising outcomes in Ebola Zaire and
Marburg Musoke virus infections and may prove applicable to other
viral targets such as HCV or Dengue viruses. For more information,
visit www.avibio.com.
About the MDEX Consortium
The MDEX consortium led by Professor Muntoni, is a
mutlidisciplinary enterprise to promote translational research into
muscular dystrophies, and is formed by the clinical groups of
Professor Francesco Muntoni (Imperial College London and UCL
Institute of Child Health) and Professor Kate Bushby and Professor
Volker Straub (Newcastle University), and scientists from Imperial
College London (Professor Dominic Wells), UCL Institute of Child
Health (Dr Jennifer Morgan), Royal Holloway University of London
(Professor George Dickson and Dr Ian Graham), Oxford University (Dr
Matthew Wood) and University of Western Australia (Prof Steve
Wilton). In addition, the charities Muscular Dystrophy Campaign
(MDC), Action Duchenne and Duchenne Family Support Group also
participate in the Consortium. For more information visit
www.mdex.org.uk.
"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.
AVI Press and Investor Contact: Julie Rathbun Investor Relations
(541) 224-2575 Investorrelations@avibio.com
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