AVI BioPharma's Drug for Duchenne Muscular Dystrophy Recommended for Orphan Drug Status in EU and Receiving Provisional GTAC App
October 14 2008 - 9:30AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced that the European Medicines Agency (EMEA) Committee
for Orphan Medicinal Products (COMP) adopted a positive opinion
recommending orphan medicinal product designation for AVI-4658 to
treat Duchenne muscular dystrophy (DMD). Additionally, the Company
received notification from the Gene Therapy Advisory Committee
(GTAC) in the UK granting provisional approval for the Company's
planned clinical trial for systemic delivery of AVI-4658 to treat
DMD. The conditions for final GTAC approval include certain wording
changes in the patient and parent information documents and
completion of normal site specific assessments. AVI expects to
comply with the conditions for final approval this quarter.
The Company believes the positive COMP opinion will serve as the
scientific basis for the European Commission to issue a European
Union orphan designation pursuant to Regulation (ED) 141/2000.
Orphan designation in the EU brings several benefits to the sponsor
including certain fee waivers, protocol assistance in product
development, and marketing exclusivity up to 10 years.
"The EMEA Committee's positive recommendation on orphan status
for AVI-4658 in DMD together with GTAC's favorable review of our
clinical trial protocol for the same drug candidate adds further
momentum to the development of AVI's portfolio of exon skipping
drugs to treat this devastating and debilitating disease," said Dr.
Leslie Hudson, President and Chief Executive Officer of AVI
BioPharma. "These positive events are evidence of the Company's
continuing commitment to advance potential new treatments for DMD.
We expect to see results from our ongoing intramuscular
administration trial shortly and to begin the intravascular
administration trial once all approvals are finalized. We also look
forward to advancing AVI-4658 towards the clinic in the US during
2009."
In June of 2008, the Medicine & Healthcare Product
Regulatory Agency (MHRA) in the UK gave clearance for the Company
to move forward with a Company-sponsored systemic clinical trial of
AVI-4658 in the United Kingdom. This trial, which will involve an
intravascular (IV) administration to sixteen ambulatory boys with
DMD, is expected to start in the current quarter once GTAC approval
is complete. The Company was granted an orphan drug designation for
AVI-4658 by the U.S. Food and Drug Administration (FDA) in November
of 2007.
AVI BioPharma is currently engaged in a clinical trial at the
Imperial College of London where patients with DMD are receiving a
single-dose, intramuscular (IM) administration of AVI-4658. This
study is being conducted in collaboration with the United
Kingdom-based MDEX Consortium. AVI-4658 is designed to skip exon 51
of the dystrophin gene, thus repairing the mutated reading frame in
the mRNA sequence coding for dystrophin, a vital protein which is
absent or virtually absent in boys with DMD. By skipping this exon,
a truncated, yet functional, form of the dystrophin protein is
produced and this could ameliorate the disease process, potentially
prolonging and improving the quality of life in these patients.
Leading experts specializing in the fields of genetic disease
and/or neuromuscular disorders believe that exon skipping is one of
the most exciting and promising approaches to treat a majority of
DMD patients. AVI has continued to demonstrate its scientific
leadership in the application of exon skipping to DMD through the
publication of preclinical results of studies demonstrating the
ability of AVI's new class of drug candidates -- termed PPMO-B --
to induce sustained expression of dystrophin in the mdx mouse model
of DMD. Treatment with this new class of AVI compound resulted in
the sustained production of functional dystrophin in numerous
tissues, including the heart, diaphragm and skeletal muscles. These
are key organs for the treatment of the disease. The papers were
published in peer-reviewed journals, Molecular Therapy, "Sustained
Dystrophin Expression Induced by Peptide-Conjugated Morpholino
Oligomers in the Muscles of mdx Mice," by Jearawiriyapaisarn N,
Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S, Iversen P,
Kole R and Proceedings of National Academy of Science "Effective
rescue of dystrophin improves cardiac function in
dystrophin-deficient mice by a modified morpholino oligomer" by Wu
B, Moulton HM, Iversen PL, Jiang J, Li J, Li J, Spurney CF, Sali A,
Guerron AD, Nagaraju K, Doran T, Lu P, Xiao X, Lu QL.
See:
http://www.nature.com/mt/journal/vaop/ncurrent/abs/mt2008120a.html
http://www.pnas.org/content/105/39/14814.full
About Orphan Drug Designation
In the United States, the Orphan Drug Act provides economic
incentives to encourage biotechnology and pharmaceutical companies
to develop drugs for rare diseases... those affecting fewer than
200,000 people in the U.S. Orphan drug designation entitles AVI to
7 years of market exclusivity for AVI-4658 for the treatment of
patients with DMD. Additional incentives include tax credits
related to development expenses, reduction in FDA user fees and FDA
assistance in clinical trial design. In the EU, Orphan Drug
designation is provided to encourage the development of products
that demonstrate promise for the diagnosis, prevention and/or
treatment of life-threatening or very serious conditions that are
rare and affect not more than 5 in 10,000 persons in the European
Union. The EU designation of orphan drug status provides many of
the same benefits as in the U.S.; however, the period of market
exclusivity for an approved drug is 10 years.
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 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, 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 AVI BioPharma
AVI BioPharma is focused on the discovery and development of
RNA-based drugs using the company's expanded portfolio of
proprietary antisense compounds (PMOs). The company's technology
applications leverage distinct mechanisms of action in a range of
genetic diseases, genetic disorders and the genetic code of
disease-causing organisms. The emerging field of directed
alternative RNA splicing represents AVI's newest and most exciting
application based on the company's core antisense technology.
Functional attributes of this approach may include correcting
genetic defects (RNA mutations; which AVI believes could produce
promising treatments for Duchenne muscular dystrophy), coding for
novel soluble receptors (an exciting and novel approach which could
have application in the treatment of inflammatory diseases such as
rheumatoid arthritis), and the reduction in activity of immune
modulators in disease states (currently being applied to IL-10).
AVI's RNA-based drug programs also include blocking mRNA
translation. In AVI's biodefense program, this application has been
successful against the single-stranded RNA viruses Ebola Zaire and
Marburg Musoke in non-human primates and may have value against
other viral targets such as HCV, Dengue, Junin, influenza and RSV
viruses. This application also will be evaluated in the clinic for
the treatment of cardiovascular restenosis by our partner Cook
Medical. More information about AVI is available at
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.
AVI Press and Investor Contact: Michael Hubbard Director of
Corporate Communications (503) 227-0554 Email Contact
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