– Company provides update following receipt
of FDA minutes from Type B meeting –
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today announced
that the planned pivotal study of AVXS-101 in spinal muscular
atrophy (SMA) Type 1 will reflect a single-arm design, using
natural history of the disease as a comparator, and enroll
approximately 20 patients. This update is based on the receipt of
the minutes following the Type B meeting with the U.S. Food and
Drug Administration (FDA) held on September 30, 2016.
In addition to evaluating safety, the planned program is
expected to evaluate 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.
At the Type B meeting and in the meeting minutes, the FDA
acknowledged the company’s rationale for a single-arm pivotal study
and provided a number of constructive suggestions to help optimize
such a trial design. The FDA also indicated its preference for a
design with co-primary endpoints consisting of a measure of
developmental milestone achievement (such as sitting unassisted)
along with a clinically meaningful measure of survival (such as
time to an “event” as described above). Based on FDA’s suggestions
as well as other expert input, AveXis continues to evaluate a
number of the details of the trial design. More specific
information will be made available at the time the study is
initiated, which is expected in the first half of 2017.
“We believe the Type B meeting had a positive tone, with FDA
offering a number of constructive suggestions which we believe will
better enable implementation of a pivotal study design that is most
appropriate for the patients suffering from this devastating
disease,” said Sean Nolan, President and Chief Executive Officer of
AveXis. “With the feedback needed from the FDA to move forward with
our pivotal trial, we plan to proceed as expeditiously as possible
to begin the study in the first half of 2017.”
With regard to the ongoing Phase 1 trial of AVXS-101, the FDA
stated the following in the meeting minutes: “We strongly recommend
that at the completion of the study, you request an end-of-Phase 1
meeting to evaluate the adequacy of data to support future product
development, including a discussion of whether the data from the
Phase 1 study might provide the substantial evidence necessary to
support a marketing application.”
The company’s strategy with the SMA Type 1 program is to
complete the ongoing Phase 1 trial and, in parallel, execute on the
single-arm pivotal trial, while continuing collaborative
discussions with the FDA regarding the most expeditious pathways
for FDA approval of AVXS-101.
Conference Call InformationAveXis will host a
conference call and webcast at 4:30 p.m. EDT today, November
1, 2016, to discuss the clinical development pathway for AVXS-101
in SMA Type 1.
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 12184733. 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 12184733.
This call is in lieu of the previously announced conference call
scheduled for Thursday, November 10, 2016 at 4:30 p.m. EST. AveXis
will report financial and operating results for the third quarter
ended September 30, 2016 on Thursday, November 10 after the close
of U.S.-based financial markets, but will no longer host a
conference call and webcast.
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 the 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-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 SMN 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 StatementsThis 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
pivotal trial of AVXS-101 as well as the overall clinical
development of AVXS-101. 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’ Annual
Report on Form 10-Q for the quarter ended June 30, 2016, filed with
the SEC on August 12, 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
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