PORTLAND, OR--(Marketwire - May 06, 2009) -
AVI BioPharma, Inc. (NASDAQ: AVII
developer of RNA-based drugs, today announced a $2.5 million contract with
Children's National Medical Center in Washington, D.C. to support
preclinical studies in the development of AVI-4658 for treatment of
Duchenne muscular dystrophy. The work will be conducted with Children's
National collaborators Eric Hoffman, Ph.D., an authority on DMD and
Professor of Pediatrics, and Edward Connor, M.D., Director, Office of
Investigational Therapeutics and Professor of Pediatrics. AVI will serve
as a subcontractor to a grant awarded to Children's National by the U.S.
Department of Defense.
"We are pleased to collaborate with Dr. Hoffman, a distinguished researcher
and expert in the field of DMD, and advance AVI's efforts toward the
development of therapeutics utilizing exon skipping for the treatment of
DMD," said Steve Shrewsbury, M.D., Chief Medical Officer and Senior Vice
President of Clinical and Regulatory Affairs of AVI BioPharma. "Through
this collaboration, AVI hopes to provide the additional data as requested
by the FDA to allow clinical studies with AVI-4658 to begin in the U.S."
The collaboration will support a series of GLP toxicology studies for AVI's
exon skipping drug candidates based on phosphorodiamidate morpholino
oligomers (PMO) chemistry. The funding is part of the Department of
Defense's Congressionally Directed Medical Research Program to identify and
pursue research with the most promise for treatment of DMD.
"Exon skipping represents one of the most promising investigational
approaches for the treatment of Duchenne muscular dystrophy, and it is
imperative to accelerate the clinical development of therapeutics that
could improve the care and quality of life for boys with this disease,'
said Dr. Hoffman. "We are excited to be collaborating with AVI, both a
pioneer and a leading developer of RNA-based drugs."
"A strong pre-clinical GLP toxicology package is a critical part of a
robust drug development program, and this new grant is designed to help
enable both intravenous and subcutaneous clinical trials in DMD patients in
the U.S.," said Dr. Connor.
AVI is currently evaluating AVI-4658 in human clinical trials in Europe.
The drug is designed to skip exon 51 of the dystrophin gene, allowing for
restoration of the reading frame in the mRNA sequence. By skipping this
exon, a truncated, yet potentially functional form of the dystrophin
protein is produced, which could ameliorate the disease process and
possibly prolong and improve the quality of life in these patients. Results
from a Phase 1 proof-of-concept trial showed that injection of the drug
into the muscles of a series of DMD boys successfully induced dystrophin
production in a dose-responsive manner. Further, the drug was well
tolerated, with no significant drug-related adverse events reported. The
clinical trial was conducted in collaboration with the MDEX Consortium in
London UK. AVI is currently sponsoring an ongoing clinical trial in the UK
evaluating the systemic delivery of AVI-4658. This is an open label, 12
week safety trial, which includes measures of drug efficacy and
pharmacokinetics, being conducted in London, UK at the UCL Institute of
Child Health / Great Ormond Street Hospital NHS Trust facilities by members
of the MDEX Consortium.
About Duchenne Muscular Dystrophy (DMD)
DMD is the most common fatal genetic disorder 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 six. Progressive muscle weakness
of the legs and pelvis eventually spreads to the arms, neck, and other
areas. By age 10, braces may be required for walking, and most patients are
confined to a wheelchair by age 12. Eventually, this progresses to
complete paralysis and 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.
There is currently no cure for DMD, but for the first time in decades,
there are promising therapies in or moving into 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 therapeutic approaches,
AVI's antisense technology has 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 Children's National Medical Center/Children's Research Institute
Children's National Medical Center, located in Washington, D.C., is a
proven leader in the development of innovative new treatments for childhood
illness and injury. Children's has been serving the nation's children for
more than 135 years. Children's National is proudly ranked among the best
pediatric hospitals in America by US News & World Report and the Leapfrog
Group. For more information, visit www.childrensnational.org. Children's
Research Institute, the academic arm of Children's National Medical Center,
encompasses the translational, clinical, and community research efforts of
the institution. Learn more about our research programs at
www.childrensnational.org/research and www.wickerproject.org.
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