Treatment Of First Patient In Systemic Clinical Trial Of AVI-4658 For Treatment Of Duchenne Muscular Dystrophy

February 23, 2009 at 11:00 am Leave a comment

AVI BioPharma, Inc.
(NASDAQ: AVII), a developer of RNA-based drugs, today announced
treatment of the first patient in a clinical trial evaluating the
systemic delivery of AVI-4658 for the treatment of Duchenne muscular
dystrophy (DMD).

“We are very pleased to begin the systemic evaluation of our exon
skipping drug — AVI-4658 — for the treatment of DMD,” said Stephen
Shrewsbury, M.D., Chief Medical Officer and Senior Vice President,
Clinical and Regulatory Affairs of AVI BioPharma. “We believe that
this trial will build significantly on the data generated by the
successful recent trial evaluating intramuscular administration of
the same drug in DMD boys.”

The trial will enroll 16 ambulatory boys with DMD and initially
evaluate multiple intravenous doses of AVI-4658 between 0.5 – 4.0
mg/kg. This is an open label, 12 week safety trial, which includes
measures of drug efficacy and pharmacokinetics. The clinical study
started in London, UK at the UCL Institute of Child Health / Great
Ormond Street Hospital NHS Trust facilities by members of the MDEX
Consortium led by Professor Francesco Muntoni and will shortly start
to recruit patients in Newcastle Upon Tyne. AVI BioPharma is the
sponsor for the trial and Professor Muntoni has been awarded funding
support of $1.3 million from the UK Medical Research Council to
offset some of the clinical costs of the trial.

In January 2009, AVI announced results from a Phase 1 trial
evaluating the intramuscular administration of AVI-4658 for the
treatment of DMD, also performed in collaboration with the MDEX
Consortium. Biopsy data showed that injection of the drug into the
muscles of a series of DMD patients successfully induced dystrophin
production in a dose responsive manner. Further, the drug was well
tolerated, with no significant drug-related adverse events. The
Company was granted an orphan drug designation for AVI-4658 by the
U.S. Food and Drug Administration in November 2007 and by the
European Medicines Agency in December 2008.

DMD is an incurable muscle-wasting disease associated with errors in
the gene that codes for dystrophin, a protein that plays a key
structural role in muscle fiber function. AVI-4658 is designed to
skip exon 51 of the dystrophin gene, allowing for restoration of the
reading frame in the mRNA sequence. Based on its pre-clinical
research and the Phase 1 trial results, AVI believes that, by
skipping this exon, a truncated but functional form of the dystrophin
protein is produced to ameliorate the disease process, potentially
prolonging and improving the quality of life in these patients.

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 other areas. By age
10, braces may be required for walking, and most patients are
confined to a wheelchair by age 12. Eventually, the disease
progresses to a 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 and Marburg virus infections
(both of which have product candidates heading into clinical programs
under US INDs) and may prove applicable to other viral targets such
as HCV or Dengue viruses. For more information, visit

About the MDEX Consortium

The MDEX consortium led by Professor Muntoni, is a multidisciplinary
enterprise to promote translational research into muscular
dystrophies, and is formed by the clinical groups of Professor
Francesco Muntoni (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 (Professor 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

“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.

MDEX Consortium



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