AVI BioPharma Initiates Dosing in Phase 2 Study of Eteplirsen in Duchenne Muscular Dystrophy Patients
August 15 2011 - 4:05PM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based
therapeutics, today announced that it has initiated dosing in a
Phase 2 study of eteplirsen, the Company's lead exon-skipping
therapeutic candidate for the treatment of Duchenne muscular
dystrophy (DMD).
The placebo-controlled study of twelve patients, which will be
conducted at Nationwide Children's Hospital in Columbus, Ohio, is
designed to evaluate the efficacy and safety of eteplirsen in DMD
patients over 24 weeks of dosing. Patients enrolled in the study
will receive once weekly intravenous infusions of either 50mg/kg of
eteplirsen, 30mg/kg of eteplirsen or placebo, and will be evaluated
on a number of safety and efficacy endpoints. The efficacy
endpoints will include biochemical markers in muscle biopsies, such
as the production of the dystrophin protein and markers of
immune-inflammatory response, as well as clinical outcomes to
measure muscle strength, function and degree of ambulation.
"In this Phase 2 study we will evaluate eteplirsen at higher
doses and over a longer duration of treatment, which will help us
understand the potential disease-modifying effects and safety of
eteplirsen as chronically-administered therapy," said Chris
Garabedian, AVI's CEO and president. "We expect data from this
study around the end of the second quarter of 2012, which will
guide our design for a pivotal study."
Jerry R. Mendell, M.D., of Nationwide Children's Hospital and
principal investigator of the study added, "Despite many years of
awareness and investment in therapeutic development, we only have
supportive treatments available for DMD patients today. We have
seen tremendous promise for eteplirsen to potentially modify the
progression of DMD in patients and we look forward to further
understanding its potential through this longer study."
The travel costs for the patients participating in the Phase 2
clinical study are supported in part by grants from Parent Project
Muscular Dystrophy and Muscular Dystrophy Association.
About Eteplirsen
Eteplirsen is AVI's lead drug candidate that is systemically
delivered for the treatment of a substantial subgroup of patients
with DMD. Data from clinical studies of eteplirsen in DMD patients
have demonstrated a broadly favorable safety and tolerability
profile and restoration of dystrophin protein expression.
Eteplirsen uses AVI's novel phosphorodiamidate morpholino
oligomer (PMO)-based chemistry and proprietary exon-skipping
technology to skip exon 51 of the dystrophin gene. By skipping exon
51, eteplirsen may restore the gene's ability to make a shorter,
but still functional, form of dystrophin from mRNA. Promoting the
synthesis of a truncated dystrophin protein is intended to improve,
stabilize or significantly slow the disease process and prolong and
improve the quality of life for patients with DMD. The results of
an AVI sponsored UK based trial were recently published in The
Lancet. The abstract and full paper can be accessed at
http://www.thelancet.com/journals/lancet/article/PIIS0140-6736%2811%2960756-3/abstract.
AVI is also developing other PMO-based exon-skipping drug
candidates intended to treat additional patients with DMD.
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 a highly differentiated
RNA-based technology platform, AVI has built a pipeline of
potentially transformative therapeutic agents, including
eteplirsen, 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 and their efficacy, potency and utility in the
treatment of rare and infectious diseases and the availability of
study results from the Phase 2 study of eteplirsen around the end
of the second quarter of 2012.
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 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.
"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 Investor and Media Contact: David Schull Russo Partners
858.717.2310 or 212.845.4271 Email Contact
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