AVI BioPharma to Present Additional Data From the Phase IIb Study of Eteplirsen for the Treatment of Duchenne Muscular Dystro...
2012年4月25日 - 9:30PM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based
therapeutics, announced that data from a Phase IIb study evaluating
eteplirsen as a treatment for boys with Duchenne muscular dystrophy
(DMD) will be presented today at the American Academy of Neurology
64th Annual Meeting in New Orleans, Louisiana. Principal
investigator, Jerry R. Mendell, M.D. of Nationwide Children's
Hospital, will describe the data via a brief oral presentation of
the abstract titled "A Phase IIb Placebo-Controlled Study of the
Exon-Skipping Drug Eteplirsen in Subjects with Duchenne Muscular
Dystrophy" during the AAN Emerging Science Session (abstract #004
at 5:54 pm CDT), followed by a more detailed poster presentation
(6:30 to 7:00 pm CDT).
The abstract will describe new and previously reported efficacy
and safety data from the Phase IIb study examining 24 weeks of
treatment with eteplirsen in boys with DMD. Results from the
randomized, double-blind, placebo-controlled study confirm that the
trial met its primary endpoint, demonstrating a significant
increase in dystrophin at 24 weeks compared to placebo. Data showed
that eteplirsen, administered once weekly at 30mg/kg over 24 weeks,
resulted in a statistically significant (p≤0.002) increase in novel
dystrophin (22.5% dystrophin-positive fibers as a percentage of
normal) compared to no increase in the placebo group.
Additional data to be presented today include:
- Individual patient data on the primary endpoint of change in
dystrophin-positive fibers from baseline;
- Biochemical findings across the 24-week treatment cohort (30
mg/kg/wk dose) including RT-PCR and western blot images for each
individual patient in this cohort;
- An exploratory analysis of performance on the 6-minute walk
test comparing eteplirsen-treated patients compared to placebo. Two
patients in the 30 mg/kg cohort showed rapidly progressive decline
on the 6-minute walk test and were excluded from the analysis. Of
the remaining patients, the eteplirsen-treated patients
demonstrated a 17.8 meter benefit versus placebo over 24 weeks
(-3.2 meter decline from baseline vs. -21.0 decline, respectively).
This was an exploratory analysis and not a statistically
significant finding.
- A summary of treatment-emergent adverse events comparing
eteplirsen-treated patients versus placebo, which demonstrated that
eteplirsen was well tolerated through 24 weeks of treatment. No
treatment-related adverse events, serious adverse events, and
treatment discontinuations related to eteplirsen were observed. In
addition, no treatment related changes were detected on any safety
laboratory parameters, including several biomarkers for renal
function.
Dr. Mendell's presentation will be posted on the AVI BioPharma
web site in the "Events & Presentations" section after the
session is completed.
About Duchenne Muscular Dystrophy
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 affected with DMD. A devastating and incurable
muscle-wasting disease, DMD is associated with specific inborn
errors in the gene that codes for dystrophin, a protein that plays
a key structural role in muscle fiber function. Progressive muscle
weakness eventually spreads to the arms, neck and other areas.
Eventually, this progresses to complete paralysis and increasing
difficulty in breathing due to respiratory muscle dysfunction
requiring ventilatory support as well as cardiac muscle dysfunction
leading to heart failure. The condition is terminal, and death
usually occurs before the age of 30.
About Study 201 (Eteplirsen Phase IIb
Study)
Study 4658-US-201 was conducted at Nationwide Children's
Hospital in Columbus, Ohio. Twelve boys meeting the inclusion
criteria being between 7 and 13 years of age with appropriate
deletions of the dystrophin gene that confirm eligibility for
treatment with an exon-51 skipping drug received double-blind IV
infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50
mg/kg of eteplirsen once weekly for 24 weeks (n=4). Muscle biopsies
for evaluation of dystrophin were obtained at baseline for all
subjects and after 12 weeks for patients in the 50 mg/kg cohort and
after 24 weeks for patients in the 30 mg/kg cohort. Two placebo
patients were randomized to the 30 mg/kg cohort and two placebo
patients were randomized to the 50 mg/kg cohort. This study design
allowed AVI to investigate the relationship of dose and duration of
eteplirsen treatment on the production of dystrophin over the
course of the 24 week study.
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.
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 its 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 disease.
For more information, please 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 eteplirsen
and its efficacy, potency and utility in the treatment of DMD.
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 eteplirsen and/or AVI's antisense-based
technology platform; and any of AVI's drug candidates, including
eteplirsen, 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 425.354.5140 Email
Contact AVI Media Contact: David Schull 212.845.4271 Email
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