CORVALLIS, OR and LONDON--(Marketwire - July 28, 2009) - AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, and Action Duchenne, a leading UK
charity dedicated to increasing awareness, engendering action and raising
funds to find a cure for Duchenne Muscular Dystrophy (DMD), today announced
a collaboration to support the acceleration of research and development for
AVI's exon skipping candidate drugs for the treatment of DMD.
"AVI has a new class of candidate drugs which are an important contribution
to Action Duchenne's search for a treatment for DMD. We have teamed up
with AVI to help accelerate these clinical programs as part of our
commitment to cure this devastating disease," said Nick Catlin, CEO of
Action Duchenne. "Our primary goal is to help provide treatment options for
the many children and adults afflicted with DMD. We believe we can provide
significant value to AVI's ground breaking efforts."
"AVI and Action Duchenne share a common goal to advance new therapeutics
and find treatment options for DMD patients," said Leslie Hudson, Ph.D.,
President and Chief Executive Officer of AVI BioPharma. "We believe this
collaboration has high potential and we are also very pleased to receive
the financial support from Action Duchenne which will help accelerate our
research and development efforts for new exon skipping therapeutics."
The agreement has a one-year term, with an option to extend for additional
years, and will provide approximately $1.2 million in support to AVI over
the initial term for advancement of research, regulatory efforts and
clinical trial recruitment.
AVI is currently conducting a dose-finding clinical trial 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 and at the Royal Victoria Infirmary,
Newcastle-Upon-Tyne, UK, which is the center for the European Treat
Neuromuscular Diseases (Treat-NMD) initiative. The clinical costs for the
trial are provided, in part, by the UK Medical Research Council.
AVI-4658 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 of 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 detected. The
clinical trial was conducted in collaboration with the MDEX Consortium in
London, UK. AVI is also developing AVI-5038, a new candidate drug based on
second-generation PPMO chemistry and designed to skip exon 50. The
preclinical work for AVI-5038 is funded in part by Charlie's Fund. The
Company is currently working to advance this new drug candidate into
clinical trials under an investigational new drug application (IND) in the
United States and an investigational medicinal product dossier (IMPD) in
Europe.
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 Action Duchenne
Action Duchenne (formally Parent Project UK) was set up by Duchenne
families in 2001 to promote new research for a cure for Duchenne. The
charity has a strong record in funding research and has to date funded 9
major projects costing over £1m and has been a leading partner in the £1.6m
DoH MDEX project. These projects have enabled much needed early work to be
completed on exon skipping and other therapeutic approaches.
Action Duchenne holds an international conference every year to bring
together researchers and families to exchange new research developments and
provide a vital meeting venue for scientists.
In 2005 Action Duchenne launched the Duchenne Registry, the first National
Duchenne database that holds gene information of people living with
Duchenne and can be used to speed up the recruitment of patients for
clinical trials. In 2006 Action Duchenne launched a comprehensive learning
and behaviour toolkit for use by parents and education professionals. For
more information please visit: www.actionduchenne.org
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.
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