AVI BioPharma Opens Investigational New Drug (IND) Application for AVI-4658 in Duchenne Muscular Dystrophy
July 07 2010 - 8:00AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based
therapeutics, today announced that following review by the U.S.
Food and Drug Administration the Company's Investigational New Drug
(IND) application for AVI-4658 is open. AVI-4658 is AVI's lead drug
candidate being developed as a systemically administered treatment
for a substantial subgroup of patients with Duchenne muscular
dystrophy (DMD), a genetic muscle wasting disease caused by failure
to produce dystrophin. AVI plans to initiate a Phase 1b/2 clinical
trial in DMD in the U.S. this year.
The intended site for the planned U.S. based study is Nationwide
Children's Hospital in Columbus, Ohio, with Jerry R. Mendell, M.D.
as the Principal Investigator. The clinical program design is being
reviewed in consultation with Dr. Mendell, co-investigator Kevin
Flanigan, M.D., and other DMD key opinion leaders. It is
anticipated that future clinical evaluation will explore increasing
doses of AVI-4658 considering the generally well tolerated nature
of the drug candidate as exhibited in the clinical and preclinical
studies to date, and the substantial, but variable, increases in
dystrophin measurements demonstrated in patients with DMD in the
U.K. based Phase 1b/2 clinical trial.
"We are actively working with scientific and medical experts and
regulatory authorities to finalize plans for our U.S. based Phase
1b/2 study as we complete the collection and analysis of clinical
data from the recent U.K. trial of AVI-4658," stated Stephen B.
Shrewsbury, M.D., Senior Vice President and Chief Medical Officer,
AVI BioPharma, Inc. "The results we have reported to date are very
promising and suggest an overall very favorable safety profile. As
we continue the clinical evaluation of systemically administered
AVI-4658, I remain optimistic about its potential to induce
consistent, substantial novel dystrophin protein expression in
patients with DMD."
AVI-4658 is an RNA-based therapeutic employing AVI's novel
phosphorodiamidate morpholino oligomer (PMO) based chemistry and
exon skipping technologies. It is being developed as a systemic
treatment for patients with DMD.
About Duchenne Muscular Dystrophy
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 affected with
DMD. Girls are rarely affected by the disorder. DMD 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 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 require full-time use of a wheelchair by
age 12. Eventually, this progresses to complete paralysis and
increasing difficulty in breathing due to respiratory muscle
dysfunction requiring ventilatory support, and cardiac muscle
dysfunction leading to heart failure. The condition is terminal and
death usually occurs before the age of 30. The outpatient cost of
care for a non-ambulatory DMD patient is very high. There is
currently no cure for DMD, but for the first time ever there are
promising therapies in, or moving into, development.
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.
"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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