AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs,
today announced positive results from two preclinical studies
evaluating the therapeutic potential of AVI-7100 against a fully
virulent pandemic H1N1 virus, also known as swine flu or swine
origin influenza virus. An analysis of the data from the studies
demonstrated statistically significant reductions in average viral
titer versus a saline control and a control with Tamiflu®, a
standard of care drug. The studies of AVI-7100, which used AVI's
proprietary PMOplus™ chemistry, were supported by the
Transformational Medical Technologies program (TMT) of the U.S.
Department of Defense to identify RNA-based drug candidates against
pandemic H1N1 virus. The studies were undertaken as part of a rapid
response exercise demonstrating TMT's ability, in partnership with
AVI, to rapidly respond to a real-world emerging viral threat.
"These H1N1 results build on the significant success we've had
applying our RNA-based technologies and advanced proprietary
chemistries, such as PMOplus™, to anti-infective therapeutic
candidates," said J. David Boyle II, interim President and CEO of
AVI BioPharma. "Based on these results, TMT continues to support
AVI-7100, funding an accelerated IND enabling program and Phase 1
study, as well as expanded preclinical evaluation that explores
AVI-7100's potential as a broad spectrum influenza therapeutic. We
look forward to continuing to work closely with TMT to support our
national preparedness against biological threats."
Study Results An analysis of the results
from the two preclinical studies evaluating AVI-7100 against a
fully virulent strain of pandemic H1N1 virus in a ferret model
demonstrated potent, statistically significant reductions in the
average viral titer in the upper respiratory region versus saline
and Tamiflu controls. In the analysis of these two studies,
AVI-7100 administered intraperitoneally at 10 mg/kg resulted in
statistically significant reductions in the combined daily average
viral titer through peak viral load (days 1-3) versus saline
control (p=0.0012) and Tamiflu control (p=0.0103) by up to 3.9 log.
Maximum reductions in the cumulative average viral titer of 5.1 log
versus saline control and 4.52 log versus Tamiflu control were
observed through day 5, but statistical analysis at this time point
was not possible due to limited animal numbers.
Microscopic examination of the lungs of the infected ferrets
seven days after infection evaluated the extent of regional tissue
damage. Pathology scores in each study revealed only mild damage in
the AVI-7100 treated ferrets versus severe damage in both saline
controls and Tamiflu controls. The gross examination of the lung
and spleens of the AVI-7100 treated ferrets appear normal, but
obvious damage was observed in these tissues in the saline and
Tamiflu controls. Finally, the number of infiltrating macrophages
in the bronchiolar space was reduced in the AVI-7100 treated
ferrets compared to the saline or the Tamiflu controls.
Presentation of the final data from these studies is planned for
a medical conference in 2010.
About The TMT/AVI H1N1 Rapid Response
Exercise As part of its ongoing evaluation of programs being
conducted in cooperation with AVI, the U.S. Department of Defense
Transformational Medical Technologies program established a
contract with AVI to conduct a rapid response exercise against a
real-world emerging threat, the pandemic H1N1 virus. The intent of
the exercise was to demonstrate the capability of AVI to
efficiently respond to a real-world emerging viral threat by
rapidly designing and producing multiple therapeutic candidates and
evaluating preclinical efficacy.
Initially the exercise involved identifying target sequences
against H1N1, designing several drug candidates utilizing
proprietary derivatives of AVI's antisense phosphorodiamidate
morpholino oligomers (PMO) chemistry, and then manufacturing the
candidates in sufficient quantity for preclinical testing. This was
successfully accomplished in approximately one week, demonstrating
AVI's ability to rapidly respond to a real-world viral threat
utilizing the AVI's RNA based therapeutics platform.
Subsequently, AVI evaluated its RNA-based drug candidates in
preclinical studies using a mouse model of seasonal flu and
identified two lead candidates, including AVI-7100. The two lead
candidates were tested in the more advanced ferret model utilizing
a fully virulent human pandemic H1N1 virus. The ferret studies
included various treatment groups employing the lead candidates
administered via intraperitoneal and intranasal dosing routes, a
saline control group, a scrambled RNA sequence control group and a
control group dosed with Tamiflu, a standard of care drug. While
both lead candidates and routes of administration were indicative
of activity versus all controls, AVI-7100 administered
intraperitoneally demonstrated overall superiority.
About AVI-7100 AVI-7100 employs AVI's
patented PMOplus™ technology that selectively introduces positive
charges to its phosphordiamidate morpholino oligomer (PMO) backbone
to improve selective interaction between the drug and its target.
The PMOplus™ chemistry platform previously generated two
Investigational New Drugs (INDs) for AVI-6002 and AVI-6003, AVI's
lead hemorrhagic fever virus therapeutic candidates for Ebola and
Marburg viruses, respectively. These hemorrhagic fever virus
therapeutic candidates are being developed under a July 2010
contract awarded through the U.S. Department of Defense
Transformational Medical Technologies program (TMT). This contract
provides funding to AVI of up to approximately $291 million.
Activities to develop AVI-7100 as a medical countermeasure
against the pandemic H1N1 influenza virus are being funded under a
June 2010 contract awarded to AVI through the TMT program. The
contract provides for funding of up to $18 million to advance the
development of AVI-7100, and includes studies enabling an
Investigational New Drug (IND) application with the U.S. Food and
Drug Administration, the study of an intranasal delivery
formulation, and the funding of a Phase 1 clinical trial to obtain
human safety data to support potential use under an Emergency Use
Authorization.
Additional funding under an earlier contract awarded to AVI via
the TMT program is supporting continued preclinical evaluation of
AVI-7100 against H1N1 as well as expanded preclinical evaluation
against H5N1 (avian flu) and drug resistant H1N1 and H3N2 flu
strains. Funding from this earlier contract also supported the
rapid response exercise and is valued at up to $8.1 million.
About Pandemic H1N1 Influenza On June 11,
2009 the World Health Organization declared a pandemic of H1N1
influenza. The virus was first detected in people in the U.S. in
April 2009 and was referred to as "swine flu" because many of the
genes in the virus were very similar to those found in flu viruses
that circulate in pigs (swine). Illness with the 2009 H1N1 virus
has ranged from mild to severe. Symptoms include fever, cough,
runny nose, headache, chills and fatigue. Many people infected with
H1N1 also have respiratory symptoms without a fever. Severe illness
and deaths have occurred as a result of illness associated with the
virus. The Centers for Disease Control and Prevention (CDC)
estimated that between April 2009 and April 2010 there were up to
89 million cases of H1N1 infection in the U.S. The CDC also
estimated that there were up to 403,000 H1N1-related
hospitalizations in the U.S. during the same time period.
About the Defense Threat Reduction Agency
The Defense Threat Reduction Agency (DTRA) was founded in 1998 to
integrate and focus the capabilities of the Department of Defense
(DoD) that address the threat by weapons of mass destruction (WMD).
DTRA's mission is to safeguard the United States and its allies
from chemical, biological, radiological, nuclear and high-yield
explosive WMDs by providing capabilities to reduce, eliminate and
count the threat and mitigate its effects. DTRA combines DoD
resources, expertise, and capabilities to ensure the United States
remains ready and able to address the present and future WMD
threats. For more information on DTRA, visit www.dtra.mil.
About the Transformational Medical Technologies
Program (TMT) The TMT program was created by the U.S.
Department of Defense to protect the Warfighter from emerging and
genetically altered biological threats by discovering and
developing a wide range of medical countermeasures through enhanced
medical research, development, test and evaluation programs. The
TMT Program Office is matrixed from the Joint Science and
Technology Office -- DTRA and Joint Program Executive Office --
Chemical and Biological Defense, with oversight from the Office of
the Secretary of Defense. For more information on TMT, visit
http://www.tmti-cbdefense.org.
About AVI BioPharma AVI BioPharma is
focused on the discovery and development of novel RNA-based
therapeutics for rare and infectious diseases, as well as other
select disease targets. Applying pioneering technologies developed
and optimized by AVI, we are able to target a broad range of
diseases and disorders through distinct RNA-based mechanisms of
action. Unlike other RNA-based approaches, our technologies can be
used to directly target both messenger RNA (mRNA) and precursor
messenger RNA (pre-mRNA) to either down-regulate (inhibit) or
up-regulate (promote) the expression of targeted genes or proteins.
By leveraging our highly differentiated RNA antisense-based
technology platform, we have built a pipeline of potentially
transformative therapeutic agents, including a clinical stage
Duchenne muscular dystrophy candidate and anti-infective candidates
for influenza and hemorrhagic fever viruses. For more information,
visit www.avibio.com.
Forward-Looking Statements and
Information
This press release contains statements that are forward-looking,
including statements about the amount and timing of potential
funding; the development of AVI 7100, AVI 6002 and AVI 6003,
including preclinical development, filing of an IND application,
completion of a Phase 1 human safety clinical trial, clinical
development and FDA approval; AVI's PMOplus™ chemistry and other
antisense-based technology and its ability to protect against the
H1N1 virus, as well as its efficacy, potency and utility in the
treatment of infectious diseases, and its potential to treat a
broad number of human diseases. These forward-looking statements
involve risks and uncertainties, many of which are beyond AVI's
control. Known risk factors include, among others: development of
any of AVI 7100 , AVI 6002 and/or AVI 6003 may not result in
funding from the TMT in the anticipated amounts or on a timely
basis, if at all; clinical trials may not demonstrate safety and
efficacy of any of our drug candidates and/or our antisense-based
technology platform; any of our drug candidates may fail in
development, may not receive required regulatory approvals, or be
delayed to a point where they do not become commercially viable.
The results and analysis of the AVI 7100 studies described in this
press release are subject to interpretation, and may not be
repeated in later preclinical and clinical trials. The AVI 7100
study described in this press release is subject to interpretation
and included small numbers of animal subjects which may limit the
utility of the statistical analysis conducted in predicting future
results. Further, the AVI 7100 study results described in this
press release is not predictive of future outcomes and may not be
repeated in later preclinical and clinical trials.
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