– SMA Type 2 study to initiate in Q2 2017 ––
Streamlines development of Type 2 clinical data set –– Conference
call and webcast December 21, at 4:30 p.m. EST –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today reported
that, based on emerging data from its intended commercial Good
Manufacturing Practice (GMP) development work and ongoing
discussions with the U.S. Food and Drug Administration (FDA), the
company has made the strategic decision to use the intended
commercial GMP derived product in all future studies of the
company’s proprietary gene therapy candidate, AVXS-101, including
the planned spinal muscular atrophy (SMA) Type 2 trial.
The company had originally planned to use existing inventory for
its initial trial in SMA Type 2, and to then use its intended
commercial GMP derived product in later Type 2 trials. By using the
intended commercial GMP derived product for the duration of its
study of AVXS-101 in Type 2 patients, the need for a comparability
analysis later in program development is eliminated, potentially
accelerating the timeframe for gathering clinical data that may be
used as part of a data set to support a future SMA Type 2
indication.
“We believe this strategic decision has the potential to
streamline the development of the clinical data set for AVXS-101 in
SMA Type 2,” said Sean Nolan, President and Chief Executive Officer
of AveXis. “We are pleased with the advances we have seen from our
process development efforts and believe the optimal approach is to
use the scalable process in our clinical evaluation of AVXS-101 in
SMA Type 2 from the outset.”
The company now expects to initiate the planned study of
AVXS-101 in SMA Type 2 patients in Q2 2017, assuming a positive
outcome from its Type B manufacturing meeting with the FDA,
currently anticipated to occur in Q1 2017.
This approach is intended to potentially expedite the SMA Type 2
program and does not impact the development program for AVXS-101 in
SMA Type 1. AveXis expects to initiate a U.S. pivotal trial of
AVXS-101 in patients with SMA Type 1 in the first half of 2017, and
plans to use the same intended commercial GMP derived product for
that study.
Conference Call InformationAveXis will host a
conference call and webcast at 4:30 p.m. EST today, December 21,
2016, to discuss this clinical development update for AVXS-101.
Analysts and investors can participate in the conference call by
dialing (844) 889-6863 for domestic callers and (661) 378-9762 for
international callers, using the conference ID 43591277. The
webcast can be accessed live on the Events and Presentations page
in the Investors and Media section of the AveXis website,
www.AveXis.com. The webcast will be archived on the company’s
website for 30 days, and will be available for telephonic replay
for 14 days following the call by dialing (855) 859-2056 (Domestic)
or (404) 537-3406 (International), conference ID 43591277.
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. Approximately 30 percent of cases are expected
to be SMA Type 2. SMA Type 2 typically presents between six and 18
months of age. Affected patients will never walk without support,
and SMA Type 2 results in mortality for more than 30 percent of
patients by age 25.
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 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 children
with SMA Type 1, expectations regarding design and timing of the
SMA Type 2 trial of AVXS-101 and the overall clinical development
of AVXS-101 and AveXis’ manufacturing processes. 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" 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
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