AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Prelimina...
January 30 2018 - 4:01PM
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, following review of safety data and early signals of efficacy
from the first three patients dosed in its pivotal trial of
AVXS-101 for spinal muscular atrophy (SMA) Type 1, the company,
with agreement from the U.S. Food and Drug Administration, will
initiate screening for the remaining patients in the trial as per
the protocol.
The open-label, single-arm, multi-center trial (STR1VE) is
designed to evaluate the efficacy and safety of a one-time
intravenous (IV) infusion of AVXS-101. There was at least a
four-week interval scheduled between dosing the first three
patients to allow review of the safety analysis as well as early
signals of
efficacy.
“We are pleased to share that we will be initiating screening for
the remaining patients to be enrolled in STR1VE as planned,
following review of safety data and early signals of efficacy from
the first three patients who received a one-time IV infusion of
AVXS-101,” said Dr. Sukumar Nagendran, Chief Medical Officer of
AveXis. “We know there is an urgent need for new treatment
approaches, and we are eager to rapidly enroll the remaining
patients in the pivotal trial as we further our clinical
understanding of AVXS-101.”
The trial will enroll a minimum of 15 patients with SMA Type 1
who are less than six months of age at the time of gene therapy,
and who have one or two copies of the SMN2 backup gene as
determined by genetic testing and bi-allelic SMN1 gene deletion or
point mutations. The intent-to-treat population is defined as
patients who are less than six months of age and symptomatic at the
time of gene therapy, with two copies of the SMN2 gene as
determined by genetic testing, bi-allelic SMN1 gene deletion and no
c.859G>C mutation in SMN2.
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 and 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. SMA Type 2 typically presents between six and
18 months of age, and those affected will never walk without
support and most will never stand without support. SMA Type 2
results in mortality in more than 30 percent of patients by the age
of 25.
About AVXS-101AVXS-101 is a proprietary gene
therapy candidate of a one-time treatment for SMA Types 1 and 2,
designed to address the monogenic root cause of SMA and prevent
further muscle degeneration by addressing the defective and/or loss
of the primary SMN gene. AVXS-101 is also designed to target motor
neurons, providing rapid onset of effect and crossing the blood
brain barrier to allow 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 the pivotal phase of study for the treatment of SMA Type 1, and
a Phase 1 trial for SMA Type 2. The company also intends to expand
its development of gene therapy into two additional rare
neurological monogenic disorders: Rett syndrome (RTT) and a genetic
form of amyotrophic lateral sclerosis (ALS) caused by mutations in
the superoxide dismutase 1 (SOD1) gene.
For additional information, please visit www.avexis.com.
Forward-Looking Statements This press release
contains "forward-looking statements," within the meaning of the
Private Securities Litigation Reform Act of 1995, regarding, among
other things, AveXis’ continued enrollment of patients in the
STR1VE trial, its research, development and regulatory plans for
AVXS-101, and the planned expansion of the company’s development of
gene therapy into additional disorders. 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 U.S. and EU, as well as
other factors discussed in the "Risk Factors" and the "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of AveXis’ Annual Report on Form 10-K for the
year ended December 31, 2016, filed with the SEC on March 16, 2017,
and AveXis’ Quarterly Report on Form 10-Q for the quarter ended
September 30, 2017, filed with the SEC on November 9, 2017. 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 BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jul 2024 to Aug 2024
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Aug 2023 to Aug 2024