AveXis Announces Single-Arm Design for European Pivotal Study of AVXS-101 in SMA Type 1 Patients
February 06 2017 - 8:00AM
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today announced
that the planned pivotal study of AVXS-101 in spinal muscular
atrophy (SMA) Type 1 in the European Union (EU) will reflect a
single-arm design, using natural history of the disease as a
comparator, and will enroll approximately 30 patients. This update
is based on the receipt of the Scientific Advice response from the
Scientific Advice Working Party within the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency
(EMA).
In addition to evaluating safety the planned pivotal trial is
expected to evaluate achievement of motor milestones, specifically
patients’ ability to sit unassisted, as well as an efficacy measure
defined by the time from birth to an “event,” defined as death or
requiring at least 16 hours per day of ventilation support for
breathing for greater than two weeks in the absence of an acute
reversible illness, or perioperatively.
“The scientific advice process has been quite productive, with
the EMA providing constructive guidance on a pivotal study design
most appropriate for EU product registration,” said James
L’Italien, Ph.D., Senior Vice President, Chief Regulatory and
Quality Officer of AveXis. “We will utilize the enhanced
communication pathways afforded to us via the PRIority MEdicines
program and work in collaboration with the EMA to generate the
high-quality data needed to support the most rapid submission that
meets the requirements for marketing authorization in the EU.” The
CHMP additionally recommended AveXis discuss the potential for
Conditional Marketing Authorization in a future meeting with EMA.
More specific clinical trial details will be made available at the
time the study is initiated, which is expected in the second half
of 2017.
The company expects to initiate a separate pivotal clinical
trial of AVXS-101 in SMA Type 1 in the United States in the first
half of 2017.
About SMASMA is a severe neuromuscular disease
characterized by the loss of motor neurons leading to progressive
muscle weakness and paralysis. SMA is caused by a genetic defect in
the SMN1 gene that codes SMN, a protein necessary for survival of
motor neurons. The incidence of SMA is approximately one in 10,000
live births. SMA is the leading genetic cause of infant
mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder
characterized by motor neuron loss and associated muscle
deterioration, which results in mortality or the need for permanent
ventilation support before the age of two for greater than 90
percent of patients.
About AVXS-101AVXS-101 is a proprietary gene
therapy candidate of a one-time treatment for SMA Type 1 and is
designed to address the monogenic root cause of SMA and prevent
further muscle degeneration by addressing the defective and/or loss
of the primary SMN1 gene. AVXS-101 also targets motor neurons
providing rapid onset of effect, and crosses the blood brain
barrier allowing an IV dosing route and effective targeting of both
central and systemic features.
About AveXis, Inc.AveXis is a clinical-stage
gene therapy company developing treatments for patients suffering
from rare and life-threatening neurological genetic diseases. The
company’s initial proprietary gene therapy candidate, AVXS-101, is
in an ongoing Phase 1 clinical trial for the treatment of SMA Type
1. For additional information, please visit www.avexis.com.
Forward-Looking StatementsThis press release
contains "forward-looking statements," within the meaning of the
Private Securities Litigation Reform Act of 1995, regarding, among
other things, AveXis’ research, development and regulatory plans
for AVXS-101, including the planned design of our SMA Type 1
pivotal study in the EU, potential of AVXS-101 to benefit patients
with SMA Type 1, the potential expediting and streamlining of the
European regulatory approval process for AVXS-101 resulting from
the acceptance of AVXS-101 into the EMA’s PRIority MEdicines
(PRIME) program and based upon recommendations from the CHMP,
expectations regarding timing of the planned US and European Union
pivotal trials of AVXS-101 in patients with SMA Type 1, potential
for Conditional Marketing Authorization in the EU, and the overall
clinical development of AVXS-101. Such forward-looking statements
are based on current expectations and involve inherent risks and
uncertainties, including factors that could delay, divert or change
any of them, and could cause actual results to differ materially
from those projected in its forward-looking statements. Meaningful
factors which could cause actual results to differ include, but are
not limited to, the scope, progress, expansion, and costs of
developing and commercializing AveXis’ product candidates;
regulatory developments in the European Union, as well as other
factors discussed in the "Risk Factors" included as Exhibit 99.1 to
the Company’s Current Report on Form 8-K filed with the Securities
and Exchange Commission on September 7, 2016 and the "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" section of AveXis’ Quarterly Report on Form 10-Q for
the quarter ended September 30, 2016, filed with the SEC on
November 10, 2016. In addition to the risks described above and in
the Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q,
Current Reports on Form 8-K and other filings with the SEC, other
unknown or unpredictable factors also could affect AveXis’ results.
There can be no assurance that the actual results or developments
anticipated by AveXis will be realized or, even if substantially
realized, that they will have the expected consequences to, or
effects on, AveXis. Therefore, no assurance can be given that the
outcomes stated in such forward-looking statements and estimates
will be achieved.
All forward-looking statements contained in this press release
are expressly qualified by the cautionary statements contained or
referred to herein. AveXis cautions investors not to rely too
heavily on the forward-looking statements AveXis makes or that are
made on its behalf. These forward-looking statements speak only as
of the date of this press release (unless another date is
indicated). AveXis undertakes no obligation, and specifically
declines any obligation, to publicly update or revise any such
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by law.
Media Inquiries:
Lauren Barbiero
W2O Group
646-564-2156
lbarbiero@w2ogroup.com
Investor Inquiries:
Jim Goff
AveXis, Inc.
650-862-4134
jgoff@avexis.com
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