AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing novel treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today reported
pulmonary results from an interim analysis of data as of April 1,
2016 from the ongoing Phase 1 trial of AVXS-101 for the treatment
of spinal muscular atrophy (SMA) Type 1. The data were presented by
Richard Shell, MD, member of the Section of Pulmonary Medicine at
Nationwide Children’s Hospital, at the International Conference of
the American Thoracic Society 2016 in San Francisco.
In the interim analysis presented at the meeting, gene therapy
appeared to reduce the need for ventilation support and allowed
patients to successfully recover from respiratory illnesses that
are often lethal to SMA Type 1 patients. The interim analysis found
that none of the patients in either dosing cohort required
permanent ventilation as of April 1, 2016. In addition, 8 of 10 (80
percent) patients on the proposed therapeutic dose of 2.0x1014
vg/kg who did not need biphasic/bi-level ventilation (BiPAP)
support prior to gene therapy continued with no ventilation
support. The two exceptions were children hospitalized in the first
quarter of 2016 for severe respiratory illness and placed on BiPAP
to aid recovery.
Dr. Shell said, “The progression of SMA Type 1 is such that
respiratory failure is the predominant cause of death. To see no
patients requiring permanent ventilatory support is encouraging. We
are pleased to see the two children hospitalized during a
challenging respiratory season in the first three months of 2016
recover from their illness, as children with SMA type 1 rarely
recover so quickly from such illnesses and often require permanent
ventilation support as a sequelae.” In addition, AVXS-101 continued
to demonstrate a favorable safety profile in patients studied as of
April 1, 2016, with no new treatment-related safety or tolerability
concerns identified. All patients in both the low-dose and proposed
therapeutic-dose cohorts remained without an “event,” defined as
death or until a patient requires at least 16 hours per day of
ventilation support for breathing for 14 consecutive days in the
absence of an acute reversible illness, or perioperatively.
The natural history of SMA Type 1 indicates that bulbar muscle
weakness, skeletal muscle weakness in the neck and intercostal
muscle weakness lead to respiratory impairment, poor clearance of
airway secretions, risk of aspiration and recurrent infections
leading to death or permanent ventilation. The median age to
permanent ventilation or death in a published natural history study
of SMA type 1 patients is 10.5 months (IQR 8.1-13.6 months), and by
13.6 months only 25 percent of SMA Type 1 patients are alive and
free of permanent ventilation1.
In the ongoing trial, as of April 1, 2016:
- No patient required permanent ventilation with the median age
of all 15 patients at 14.9 months.
- 8 of 10 (80%) patients that did not use biphasic/bi-level
ventilation (BiPAP) support before gene transfer continued without
any ventilation support (the two exceptions being after severe
illness/hospitalizations in the first quarter of 2016 to assist
recovery).
“The preliminary data appear to indicate AVXS-101 may have a
positive impact on the pulmonary outcomes of patients in the trial
suffering from SMA Type 1, which may be impacting the overall
survival benefit,” said Suku Nagendran, MD, Senior Vice President
and Chief Medical Officer, AveXis. “We look forward to reviewing
the ongoing data from this study over the coming year as we
continue the development of AVXS-101.”
Phase 1 Trial DesignThe Phase 1 open-label,
dose-escalation study is designed to evaluate safety and
preliminary indications of efficacy of AVXS-101 in patients
suffering from SMA Type 1. The primary outcome in the study is
safety and tolerability. The secondary outcome measure is efficacy
as defined by the time from birth to an “event,” with an event
defined as death or until a patient requires at least 16 hours per
day of required ventilation support for breathing for 14
consecutive days in the absence of an acute reversible illness or
perioperatively. Exploratory outcome measures include motor
function testing, measured by the Children’s Hospital of
Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND),
a test developed to measure motor skills of patients with SMA Type
1, and other motor milestone development surveys and tests.
The clinical protocol requires that each patient receive a
one-time dosage of AVXS-101, by intravenous injection over a
one-hour period. The patient remains at the clinical trial site for
48 hours after dosing for monitoring prior to discharge, and weekly
follow-up evaluations are conducted for one month after dosing.
After the first month, additional evaluations are conducted monthly
for 23 months.
The trial has fully-enrolled with a total of 15 patients who met
enrollment criteria of diagnosis of SMA Type 1 before six months of
age, with two copies of the SMN2 backup gene, as determined by
genetic testing. The trial includes two dosing cohorts:
- Cohort 1 (low dose) includes three patients dosed at (6.7 X1013
vg/kg), aged 5.9 to 7.2 months at time of dosing;
- Cohort 2 (proposed therapeutic dose) includes 12 patients dosed
at (2.0 X1014 vg/kg), aged 0.9 to 7.9 months at time of
dosing.
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.
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 1 is the leading genetic cause of
infant mortality.
About AVXS-101 AVXS-101 is a proprietary gene
therapy candidate of a one-time, intravenous treatment for SMA Type
1 and is the only clinical-stage gene therapy in development for
SMA. AVXS-101 is designed to address the monogenetic 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 crosses
the blood brain barrier allowing an intravenous (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.
1. Finkel, R. et al. Observational study of spinal
muscular atrophy type I and implications for clinical
trials. Neurology 83,810–817
(2014).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 expected timing for reporting
results from the ongoing Phase 1 clinical trial and the potential
for AVXS-101 to positively impact the pulmonary and nutritional
support of patients suffering from 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 United States and
foreign countries, as well as other factors discussed in the "Risk
Factors" and "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, 2015, filed
with the SEC on March 18, 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
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jun 2024 to Jul 2024
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jul 2023 to Jul 2024