AVI BioPharma Presents Updated Safety Data From Ongoing Systemic Trial of AVI-4658 at 7th Annual Action Duchenne International C
October 26 2009 - 8:00AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
presented an update on preliminary safety data from its ongoing
systemic Phase 1b/2 clinical trial of exon skipping AVI-4658 in
patients with Duchenne muscular dystrophy (DMD) at the 7th Annual
Action Duchenne Conference in London, UK.
The most recent data from the ongoing Phase 1b/2 trial at two
MDEX sites in the UK demonstrate that AVI-4658 was well tolerated
by DMD patients in a dose escalation study that is now up to the
fifth cohort (10 mg/kg). Results from the 12 week dosing periods of
the first four completed cohorts (0.5 mg/kg, 1.0 mg/kg, 2.0 mg/kg
and 4.0 mg/kg) have been reviewed with data from the ongoing cohort
5 (10 mg/kg) and have demonstrated the drug to be well tolerated.
There have been no safety issues identified, although one Serious
Adverse Event was reported due to an anaesthetic-induced
post-treatment biopsy procedure at 14 weeks, 2 weeks after last
dose, causing nausea and vomiting.
The maximum cumulative dose administered to date is 2797 mg and
the maximum single dose is 300 mg with no signs of intolerance, in
either case. Blood tests, including measures of coagulation, have
remained satisfactory, with the elevated levels of muscle enzyme,
creatine kinase tending to fall in most boys during treatment. Lung
function, vital signs and examinations have also remained stable.
In addition, no significant side effects from the treatment have
been reported for any of the 16 boys treated to date.
In each cohort, including the final cohort of 20 mg/kg, data for
the clinical effects of the treatment will be collected for 26
weeks from first dose.
"We are pleased with the continued, encouraging safety profile
and believe this bodes well for our ability to progress to the
final cohort at 20 mg/kg," said Steve Shrewsbury, M.D. Chief
Medical Officer and Senior Vice President of Preclinical, Clinical
and Regulatory Affairs. "We look forward to the continued
advancement of this trial and to the potential that exon skipping
may hold as a first disease modifying therapy for DMD
patients."
Also presenting at the Action Duchenne Annual Conference was AVI
collaborator Steve Wilton, Ph.D. Professor at the Center for
Neuromuscular and Neurological Disorders, University of Western
Australia, Perth, Western Australia, Australia. Wilton presented an
overview of exon skipping and clinical trials and also chaired a
workshop on exon skipping.
"The Action Duchenne Annual Conference brought together leaders
in the research and treatment of Duchenne muscular dystrophy from
around the world. It is exciting to hear about AVI's
ground-breaking progress in its trial of AVI-4658 and the potential
for exon skipping drugs to help the thousands of DMD patients who
do not have an effective therapy for this disease," said Nick
Catlin, Chief Executive Officer of Action Duchenne.
The open label dose-finding clinical trial is evaluating the
systemic delivery of AVI-4658 once per week for 12 weeks by
intravenous infusion. Although the study is primarily a safety
trial, it includes measures of drug efficacy and pharmacokinetics
and is 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 led by Professor Francesco Muntoni
and by Professor Kate Bushby at the Royal Victoria Infirmary,
Newcastle-Upon-Tyne, UK, which is the coordinating 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.
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 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 the MDEX Consortium
The MDEX consortium led by Professor Francesco 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), 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 www.mdex.org.uk.
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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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
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other risks detailed in the company's Securities and Exchange
Commission filings.
AVI Press and Investor Contact: Julie Rathbun Investor Relations
(541) 224-2575 Investorrelations@avibio.com
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