– All patients were alive and event-free at 20
months of age as of the August 7, 2017 data cut-off –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, will report
top-line data as of August 7, 2017, from the Phase 1 trial of
AVXS-101 in patients with spinal muscular atrophy (SMA) Type 1 in a
late-breaking breaking poster authored by Jerry Mendell, M.D.,
principal investigator in the trial and Curran-Peters Chair of
Pediatric Research, Professor of Pediatrics and Neurology at the
Research Institute at Nationwide Children’s Hospital and The Ohio
State University, Columbus, Ohio, during the 22nd International
Annual Congress of the World Muscle Society (WMS) in Saint Malo,
France.
The Phase 1, open-label, dose-escalating study was designed to
evaluate the safety and tolerability of AVXS-101 in patients with
SMA Type 1. The key measures of efficacy were the time from birth
to an “event,” which was defined as either death or at least 16
hours per day of required ventilation support for breathing for 14
consecutive days in the absence of acute reversible illness or
perioperatively, and video confirmed achievement of ability to sit
unassisted. Additionally, several exploratory objective measures
were assessed, including a standard motor milestone development
survey and Children’s Hospital of Philadelphia Infant Test of
Neuromuscular Disorders (CHOP INTEND).
Data as of August 7, 2017, showed all patients (100%) were alive
and event-free at 20 months of age. Patients in Cohort 2 continued
to demonstrate improvements in motor milestones, including:
- Nine of 12 patients (75%) could sit unassisted for 30 seconds
or more.
- Eleven of 12 (92%) patients have sustained CHOP-INTEND scores
more than 40 for a mean of 18.8 months.
- All previous motor milestone achievements reported at the
American Academy of Neurology (AAN) meeting in April 2017 were
validated and re-confirmed at each patient’s last visit by the
independent, external reviewer. New milestones reported at AAN that
had not been validated by the external reviewer at that time were
confirmed and validated during the August 2017 review.
Additionally, patients who were free of respiratory or
nutritional support at January 20, 2017, continue without the need
for supportive care.
There were no new treatment-related adverse events
identified.
A manuscript regarding these data has been accepted for
publication in a major medical journal. In keeping with the embargo
policy of that journal, AveXis will not report additional trial
data until after the publication.
About SMA
SMA 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.
About AVXS-101
AVXS-101 is a proprietary gene therapy candidate of a one-time
treatment for SMA Type 1, 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 also
targets motor neurons, providing rapid onset of effect and crossing
the blood brain barrier to allow 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 the pivotal phase of study
for the treatment of SMA Type 1. The company also intends to expand
the study of gene therapy into SMA Type 2 and 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 the potential of AVXS-101 to positively
impact quality of life and alter the course of disease in patients
with SMA Type 1 and SMA Type 2 and expectations regarding AveXis’
research, development and regulatory plans for its programs for
treatment of RTT and genetic ALS. 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
June 30, 2017, filed with the SEC on August 10, 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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