AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy
January 16 2018 - 7:20AM
– Company to expand study of AVXS-101 into
additional SMA populations including pre-symptomatic, older
pediatric Type 2 and Type 3 SMA patients –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today provided an
overview of the expanded clinical development program for the
company’s initial gene therapy candidate, AVXS-101, for the
treatment of spinal muscular atrophy (SMA). In addition to the
ongoing pivotal trial in SMA Type 1 (STR1VE) and the ongoing Phase
1 trial in SMA Type 2 (STRONG), the company plans to initiate three
studies to further evaluate AVXS-101, including in new SMA patient
populations. Additionally, the company announced the first patient
has been dosed in the Phase 1 trial of AVXS-101 in SMA Type 2.
“Our focus has always been to serve the SMA community, and our
expanded clinical development program is designed to evaluate the
impact of AVXS-101 in a broader set of SMA patients,” said Dr.
Sukumar Nagendran, Chief Medical Officer of AveXis. “We believe the
year ahead has the potential to be one of significant clinical
progress as we continue toward our ultimate goal of bringing
AVXS-101 to the patients and families devastated by SMA.”
Clinical Development Program Overview of AVXS-101 for
the Treatment of SMA
Ongoing Clinical Trials
- Pivotal Trial of AVXS-101 in SMA Type 1
(STR1VE): The ongoing, open-label, single-arm,
single-dose, multi-center trial is designed to evaluate the
efficacy and safety of a one-time IV infusion of AVXS-101 in
patients with SMA Type 1. The trial is expected to 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. Three patients have been
dosed to date.
- Phase 1 Trial of AVXS-101 in SMA Type 2
(STRONG): The on-going, open-label, dose-comparison,
multi-center Phase 1 trial is designed to evaluate the safety,
optimal dosing, and proof of concept for efficacy of AVXS-101 in
two distinct age groups of patients with SMA Type 2, utilizing a
one-time intrathecal (IT) route of administration. The trial is
expected to enroll 27 infants and children who are symptomatic with
a genetic diagnosis consistent with SMA, including the bi-allelic
deletion of SMN1 and three copies of SMN2 without the SMN2 genetic
modifier, who are able to sit but have no historical or current
ability to stand or walk. One patient has been dosed to date.
Planned Trials in SMA
- Pivotal Trial of AVXS-101 in SMA Type 1 in Europe
(STR1VE EU): The planned trial is expected to reflect a
single-arm design, using natural history of the disease as a
comparator, and is expected to enroll approximately 30 patients
with SMA Type 1 who are less than six months of age at the time of
gene therapy. The trial is designed to evaluate safety and efficacy
of a one-time IV dose of AVXS-101, including 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. AveXis
incorporated scientific advice from the European Medicines Agency
into the protocol design, and expects to initiate the trial in the
first half of 2018.
- Pre-Symptomatic SMA Types 1, 2, 3 (SPRINT):
The planned multi-national trial is expected to enroll
approximately 44 patients with two, three and four copies of SMN2
who are less than six weeks of age and pre-symptomatic at the time
of gene therapy. The trial is designed to evaluate appropriate
clinical endpoints, including developmental milestones, survival,
bulbar function and safety, of a one-time IV infusion of AVXS-101.
AveXis expects to initiate the trial in the first half of 2018, and
will provide more design details at the time of initiation.
- Pediatric “All Comers” with SMA Types 1, 2, 3
(REACH): The planned multi-national trial is expected to
enroll approximately 50 patients between approximately six months
and 18 years of age who do not qualify for other AVXS-101 trials at
the time of gene therapy. The trial is designed to evaluate a
one-time IT dose of AVXS-101. AveXis expects to initiate the trial
in late Q4 2018 or early 2019, and will provide more trial design
details at the time of initiation.
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’ research, development and regulatory plans
for AVXS-101, including expected trial design, planned enrollment
and timing of anticipated clinical trials in additional SMA patient
populations, and the potential of AVXS-101 to positively impact
quality of life and alter the course of disease in patients with
SMA Type 1. 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
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