AVI BioPharma Announces Eteplirsen Meets Primary Endpoint, Demonstrating a Significant Increase in Dystrophin at 24 Weeks Com...
02 Abril 2012 - 6:00AM
Marketwired
AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based
therapeutics, today announced that treatment with eteplirsen met
the primary efficacy endpoint in a randomized, double-blind,
placebo-controlled Phase IIb study in boys with Duchenne muscular
dystrophy (DMD). 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.
"This study represents a major advance in the field of DMD
research as the results indicate that eteplirsen is producing
consistent levels of dystrophin, which is the essential protein
that these patients need," said Jerry Mendell, M.D., Director of
the Centers for Gene Therapy and Muscular Dystrophy at Nationwide
Children's Hospital and principal investigator of the Phase IIb
study. Dr. Mendell added, "We anticipate that these levels of
dystrophin could lead to significant clinical benefit if maintained
over a longer course of treatment."
In the study, a shorter duration of eteplirsen treatment, 12
weeks, did not show a significant increase in novel dystrophin
(0.79% dystrophin-positive fibers as a percentage of normal;
p-value NS), despite administration of the drug at a higher dose
(50mg/kg once weekly). This finding suggests that a longer duration
of dosing is required before meaningful levels of dystrophin are
produced. There were no significant improvements in clinical
outcomes in the treated groups compared to placebo. Performance on
the 6-minute walk test and other outcome measures were generally
stable across most of the patients, including the placebo patients,
suggesting that a longer period of observation will be required to
demonstrate clinical effects of eteplirsen versus a placebo
control.
Eteplirsen was well tolerated at both dose levels through 24
weeks of treatment. There were no treatment-related adverse events,
no serious adverse events, and no treatment discontinuations
related to eteplirsen. Furthermore, no treatment related changes
were detected on any safety laboratory parameters, including
several biomarkers for renal function.
"We are very encouraged by the results of this first
placebo-controlled study investigating exon-skipping technology in
DMD," said Chris Garabedian, President and CEO of AVI BioPharma.
"Eteplirsen represents the first drug candidate for DMD to
demonstrate the production of novel dystrophin in a robust and
consistent manner and these study results support advancing
eteplirsen into a pivotal study."
Conference Call AVI BioPharma, Inc. will
hold a conference call to discuss these results today at 8:00 a.m.
EDT (5:00 a.m. PDT). The conference call may be accessed by dialing
800.561.2718 for domestic callers and 617.614.3525 for
international callers. The passcode for the call is 99858553.
Please specify to the operator that you would like to join the "AVI
BioPharma Phase IIb Top-Line Data Results Call." The conference
call will be webcast live under the events section of AVI's website
at www.avibio.com and will be archived there following the call for
90 days. Please connect to AVI's website several minutes prior to
the start of the broadcast to ensure adequate time for any software
download that may be necessary. An audio replay will be available
through April 9, 2012 by calling 888.286.8010 or 617.801.6888 and
entering access code 16040637.
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 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 eteplirsen
and its efficacy, potency and utility in the treatment of DMD and
the potential for the creation of novel dystrophin to lead to
significant clinical benefit over a longer course of treatment.
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
the safety and efficacy of eteplirsen and/or AVI's antisense-based
technology platform; treatment of patients with DMD using
eteplirsen over a longer duration may not lead to significant
clinical benefit; 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 Investor and Media Contact: Erin Cox 425.354.5140 Email
Contact AVI Media Contact: David Schull 858.717.2310 or
212.845.4271 Email Contact
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