AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced that CureDuchenne and the Foundation to Eradicate
Duchenne (FED), each awarded grants of $250,000 to AVI BioPharma to
support continued research and development of the Company's exon
skipping drug candidates for the treatment of Duchenne Muscular
Dystrophy (DMD), a genetic muscle wasting disease caused by failure
to produce dystrophin. Cure Duchenne and FED are US not-for-profit
foundations fully dedicated to supporting the research and
development of a cure for DMD.
"AVI shares a commitment with the Foundation to Eradicate
Duchenne and CureDuchenne to advance the research and development
of disease-modifying drugs, treat DMD, and significantly help
patients," said Leslie Hudson, Ph.D., President and Chief Executive
Officer of AVI BioPharma. "We are grateful for the generous
financial support of both organizations. This funding will help us
continue to advance our drug candidates, including our lead drug
candidate, AVI-4658, and move them closer to becoming new treatment
options for patients."
"Exon skipping holds promise as a treatment for Duchenne
muscular dystrophy. CureDuchenne is very happy to support AVI
BioPharma as it advances these treatments to boys with DMD as soon
as possible," stated Debra Miller, President and Founder,
CureDuchenne. "As the parent of a 13-year-old boy afflicted with
DMD, I shall be very pleased to see AVI's programs progress as
quickly as possible."
"The exon-skipping strategies being developed by AVI offer the
greatest prospect for meaningful clinical therapies for the
majority of boys and young men afflicted with this cruel disorder.
We are gratified by the partnership with CureDuchenne, Children's
National Medical Center and AVI," commented Joel Wood, President
and Founder of the Foundation to Eradicate Duchenne. "Speaking as
the parent of a 12-year-old with DMD, I'm tremendously optimistic
that we can punch through the remaining hurdles in time for this
generation of DMD kids. This is an anxious and exciting time in the
history of this disorder."
AVI-4658 Study 28 Overview
AVI is currently conducting a dose-finding clinical trial
evaluating the systemic delivery of AVI-4658. Known as Study 28,
this ongoing Phase 1b/2 open label clinical trial is assessing the
safety, tolerability, pharmacokinetics and exploratory efficacy of
AVI-4658 in ambulatory DMD boys between the ages of 5 and 15 years
of age who have an error in the gene coding for dystrophin that
could be treated by skipping exon 51. Patients are dosed once per
week for 12 weeks by intravenous infusion. Nineteen patients were
enrolled in total and assigned to one of six dose cohorts: 0.5,
1.0, 2.0, 4.0, 10.0 or 20.0 mg/kg. After completion of dosing,
patients are followed for a further 14 weeks. The primary objective
of the trial is to assess the safety of AVI-4658 at these doses
over the 26-week duration of the trial. The trial is fully enrolled
and the final cohort is being dosed.
Data from patients dosed to date demonstrate that AVI-4658
continues to be generally well tolerated. Adverse events reported
to date are mostly mild, unrelated to drug treatment and transient.
In the patients who completed dosing, two serious adverse events,
both deemed unrelated to AVI-4658, were reported in different
patients after they completed their 12-week treatment period and
during the 14-week follow-up period.
In Study 28, efficacy data from patients in the first four dose
cohorts who completed 12 weeks of treatment demonstrates that all
patients in the 2 and 4 mg/kg cohorts (3 of 3 in total) showed
correctly spliced mRNA for the dystrophin protein. One of these
patients, a boy in the 2mg/kg cohort, showed a robust treatment
response: a fivefold increase in dystrophin expression (from 0.9%
to 5.3% of normal) on a western blot analysis, and an increase from
1% pre-treatment to 21%, in the percentage of dystrophin positive
muscle fibers in patient muscle biopsies as measured by
immunofluorescence analysis. After completing treatment, no RNA or
protein expression signal was detected in patients in the lowest
dose cohorts, 0.5 mg/kg or 1.0 mg/kg. Restoration of functional
dystrophin expression is considered critical for successful
treatment of DMD.
Studies Towards US IND
AVI has completed a series of 12-week preclinical studies of
AVI-4658 under Good Laboratory Practice (GLP) conditions required
to open an Investigational New Drug (IND) application in the US.
The studies tested doses up to 960 mg/kg in both mdx and wild type
mice, and up to 320 mg/kg in non-human primates, both doses being
the maximum feasible single doses in these animals. In all cases
the PMO was well tolerated at doses equivalent to 80 mg/kg and 110
mg/kg in humans respectively (based on standard allometric
scaling), suggesting the potential for a wide therapeutic index.
These studies were conducted by AVI in cooperation with Eric
Hoffman, Ph.D., of the Children's National Medical Center,
Washington DC, and supported by a U.S. Defense Department
grant.
An additional GLP study of AVI-4225 PMO, to skip exon 23, in the
mdx mouse has also been completed, with similar encouraging reports
of good tolerability. The histopathology is currently being
reviewed but initial reports suggest that the muscles of treated
mice show improvement over the 12 weeks of study.
About Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (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
DMD 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 by age three. 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 requiring ventilatory support. 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 ever, there are promising therapies in
or moving into development.
About CureDuchenne
CureDuchenne is a nonprofit organization that raises awareness
and funds research specifically aimed at taking on Duchenne
Muscular Dystrophy (DMD). By working closely with the world's
leading DMD scientists CureDuchenne works to determine the most
viable research projects that will accelerate the clinical trial
process and bring potential life saving drugs to help this
generation of young boys living with the deadly disease. Our vision
is our name...to Cure Duchenne. Learn more at:
www.cureduchenne.org.
About The Foundation to Eradicate Duchenne
The Foundation to Eradicate Duchenne is a 501c3 charitable
organization established in 2001 to pursue therapeutics for
Duchenne Muscular Dystrophy. It is headquartered in Alexandria, VA.
Since its inception, the FED has funded millions of dollars in
aggressive research and is a principal funder of the Cooperative
International Neuromuscular Research Group, an international
clinical trials network founded at Children's National Medical
Center in Washington, DC.
About Children's National Medical Center/Children's Research
Institute
Children's National Medical Center, located in Washington, DC,
is a 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
consistently ranked among the best pediatric hospitals by U.S.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 Children's Research Institute at
www.childrensnational.org/research.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based medicines 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, including an ongoing systemic Phase 1b/2
clinical trial of exon skipping with AVI-4658. 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 Junín, influenza, HCV or Dengue viruses. For
more information, visit www.avibio.com.
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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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