AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1
January 04 2018 - 4:00PM
– Company to submit information requested by FDA
to the IND on an on-going basis – – AveXis plans to request a
pre-BLA meeting in Q2 2018 –
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
update following the receipt of minutes from the end-of-Phase 1
meeting with the U.S. Food and Drug Administration (FDA) conducted
on December 5, 2017, regarding the company’s primary gene therapy
candidate, AVXS-101, for the treatment of spinal muscular atrophy
(SMA) Type 1.
The goal of the end-of-Phase 1 meeting was to review the
non-clinical, clinical and Chemistry, Manufacturing and Controls
(CMC) data that has been generated by AveXis to date, and to align
with the FDA on next steps leading to a Biologics License
Application (BLA) submission. The FDA provided detailed information
requests in each of the areas discussed, which the company plans to
address by submitting the requested information to the
investigational new drug (IND) application on an on-going
basis. AveXis has been working on many of these areas of focus in
anticipation of the requests at some point during the review
process. AveXis also plans to provide available data from its
on-going pivotal trial of AVXS-101 in SMA Type 1 (STR1VE) prior to
the pre-BLA meeting.
“We are very pleased that the constructive and collaborative
discussion during the end-of-Phase 1 meeting resulted in the
identification of the specific next steps we must take on our path
to a BLA submission for AVXS-101 in SMA Type
1,” said Sean Nolan, President and Chief Executive Officer of
AveXis. “We greatly appreciate the level of clarity we
received from the FDA and will provide our
responses on an on-going basis through a series of
submissions to the IND, with the expectation that we
will request a pre-BLA meeting in the second quarter of
2018.”
The general purpose of the pre-BLA meeting is to outline what
information is to be submitted in the BLA and how that information
will be submitted. AveXis intends to make the requested data
submissions to the IND in advance of the pre-BLA meeting, which may
allow the meeting itself to focus on how the BLA and supportive
information will be submitted.
Today’s Conference Call InformationAveXis will
host a conference call and webcast at 4:30 pm EST today, January 4,
2018. 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 8188476. 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 90 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 8188476.
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 also targets 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
the study 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 the expected timing of future interactions
with the FDA, including AveXis’ expected timing to request a
pre-BLA meeting with the FDA and the submission of requested
information to the FDA 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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