AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based
therapeutics, today announced positive safety results from the
first five cohorts of Phase I single ascending dose trials of
AVI-6002 and AVI-6003, AVI's lead drug candidates being evaluated
for the treatment of Ebola virus and Marburg virus, respectively.
Data from five cohorts of the AVI-6002 and AVI-6003 studies were
evaluated by an independent Data and Safety Monitoring Board
(DSMB), which issued recommendations for both studies to progress
as planned to the next highest dosing level after no safety
concerns were identified. The Phase I single ascending dose trials
are designed to characterize the safety, tolerability and
pharmacokinetics of each therapeutic candidate in healthy adult
volunteers.
"We are very encouraged that these two drugs, which use our
advanced PMOplus® chemistry, have demonstrated a favorable safety
profile through five cohorts in our dose-escalation studies. These
data also suggest that our backbone chemistry may be safe,
independent of the specific sequence that is being targeted, and
may support applications to other viral targets," said Chris
Garabedian, president and CEO of AVI BioPharma.
To date, 25 healthy human subjects (five per group) have been
enrolled into five sequential dose groups (0.01, 0.1, 1.0, 3.0 or
6.0 mg/kg) in each of the two studies. Within each group, four
subjects received the indicated dose of the therapeutic and one
subject received placebo. For each group, safety, clinical
laboratory and renal biomarker results through five days after
treatment were reviewed by a DSMB. Subjects enrolled in the sixth
group for the drug studies will receive 9.0 mg/kg of the
therapeutic or placebo. Final, unblinded safety and pharmacokinetic
results for all subjects will be available upon completion of the
trial.
AVI will present the data from the company's Marburg program at
the 49th Annual Meeting of the Infectious Diseases Society of
America in Boston, MA.
Tim Axtelle, senior director of clinical operations at AVI, will
deliver a poster presentation titled "A Phase 1, Single
Ascending-Dose Study of AVI-6003, a Combination of Two PMOplus®
Compounds with Activity against Marburg Virus" at 11:45 a.m. EDT on
Saturday, Oct. 22. The presentation will be given during poster
session 149, called "New Approaches to Anti-Viral Therapy."
The presentation will be posted on the AVI BioPharma website in
the "Events & Presentations" section after the session is
completed.
AVI-6002 and AVI-6003 are AVI's lead therapeutic candidates for
the Ebola and Marburg viruses, respectively. Both candidates employ
AVI's patented PMOplus® technology that selectively introduces
positive charges to its phosphorodiamidate morpholino oligomer
(PMO) backbone to improve interaction between the drug and its
target.
About Ebola and Marburg Viruses
Ebola hemorrhagic fever is a severe and often fatal disease in
humans. The disease was first recognized in 1976 and is one of two
members of a family of RNA viruses called Filoviridae. The disease
is generally understood to be endemic to parts of Africa. Onset of
illness from Ebola virus is abrupt with symptoms that include
fever, headache, muscle ache, vomiting and stomach pain. Internal
and external bleeding may also be observed in some patients. There
are currently no treatments for Ebola virus infection beyond
supportive care.
Marburg hemorrhagic fever is another severe and potentially
fatal disease in humans first recognized in 1967. It is also caused
by an RNA virus of the filovirus family and is understood to be
endemic to Africa. Onset of the disease is often sudden, and the
symptoms include fever, chills, nausea, vomiting, chest pain and
diarrhea. Increasingly severe symptoms may also include massive
hemorrhaging and multiple organ dysfunctions. There are currently
no treatments for Marburg virus infection beyond supportive
care.
About AVI's PMOplus® Chemistry
PMOplus® chemistry is an advanced generation of AVI's
phosphorodiamidate morpholino oligomer, or PMO, technology
pioneered by AVI. The PMO platform is designed to provide a stable
chemistry backbone with superior drug-like characteristics for
AVI's advanced RNA-based therapeutics. PMOplus® chemistry includes
specific molecular charges positionally inserted into the PMO's
inherent charge-neutral backbone. The PMOplus® modifications are
intended to specifically enhance drug performance characteristics
on two key parameters: targeted cell penetration and the
maintenance of antiviral performance in the presence of viral
mutation.
About JPM-TMT
The Joint Project Manager Transformational Medical Technologies
(JPM-TMT) Program supports the overall mission of the U.S.
Department of Defense (DoD) by protecting the Warfighter and the
nation from emerging, genetically engineered or unknown biothreats.
Chartered within the Joint Program Executive Office for Chemical
and Biological Defense (JPEO-CBD), JPM-TMT partners with the DoD,
other government agencies, academia, and industry for the advanced
development of adaptable platform technologies that can be rapidly
tailored and deployed to mitigate the effects of the unknown
threat, whether it be naturally occurring or man-made. Program
investments target the most difficult challenges of medical
capability development and fill gaps not currently addressed by the
biodefense community. For more information, visit
www.jpmtmt.mil.
About USAMRIID
U.S. Army Medical Research Institute of Infectious Diseases
(USAMRIID), located at Fort Detrick, Maryland, is the lead medical
research laboratory for the U.S. Department of Defense's Biological
Defense Research Program, and plays a key role in national defense
and in infectious disease research. The Institute conducts basic
and applied research on biological threats resulting in medical
solutions (such as vaccines, drugs and diagnostics) to protect the
Warfighter. While USAMRIID's primary mission is focused on the
military, its research often has applications that benefit society
as a whole. USAMRIID is a subordinate laboratory of the U.S. Army
Medical Research and Materiel Command. For more information, visit
www.usamriid.army.mil.
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, the Company is able to
target a broad range of diseases and disorders through distinct
RNA-based mechanisms of action. Unlike other RNA-based approaches,
AVI's 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 its highly differentiated
RNA-based technology platform, AVI has built a pipeline of
potentially transformative therapeutic agents, including eteplirsen
(the non-proprietary name assigned to AVI-4658), which is in
clinical development for the treatment of Duchenne muscular
dystrophy, and multiple drug candidates that are in clinical
development for the treatment of infectious diseases. For more
information, visit www.avibio.com.
Forward-Looking Statements and
Information
In order to provide AVI's investors with an understanding of its
current results and future prospects, this press release contains
statements that are forward-looking. Any statements contained in
this press release that are not statements of historical fact may
be deemed to be forward-looking statements. Words such as
"believes," "anticipates," "plans," "expects," "will," "intends,"
"potential," "possible" and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements include statements about the development of AVI's
product candidates, their efficacy, potency and utility in the
treatment of rare and infectious diseases and their 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: clinical trials may not demonstrate
safety and efficacy of any of AVI's drug candidates and/or AVI's
antisense-based technology platform; and any of AVI's 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.
Any of the foregoing risks could materially and adversely affect
AVI's business, results of operations and the trading price of
AVI's common stock. For a detailed description of risks and
uncertainties AVI faces, you are encouraged to review the official
corporate documents filed with the Securities and Exchange
Commission. AVI does not undertake any obligation to publicly
update its forward-looking statements based on events or
circumstances after the date hereof.
AVI Media and Investor Contact: Erin Cox Manager, Investor
Relations & Corporate Communications 425.354.5140 Email Contact
AVI Media Contact: David Schull Russo Partners 212.845.4271 Email
Contact
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