– FDA notified AveXis it may initiate pivotal trial
based on a review of data provided by the company following CMC
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 the
U.S. Food and Drug Administration (FDA) has notified the company
that based on review of data submitted, including the potency
assay, it may initiate its planned pivotal trial of AVXS-101 for
patients with spinal muscular atrophy (SMA) Type 1 using the
intravenous (IV) formulation produced by the company’s Good
Manufacturing Practice (GMP) commercial manufacturing process. The
company plans to initiate this trial immediately.
“We are pleased to reach this outcome following a thorough
review by the FDA of the voluminous information we supplied to
address the commitments made during the
Chemistry, Manufacturing, and Controls Type B meeting in May,
and are eager to initiate our pivotal trial of AVXS-101 in SMA Type
1 in the U.S. using product from our GMP process,” said Sean Nolan,
President and Chief Executive Officer of AveXis. “Moving AVXS-101
back into the clinic, as planned, with product from our GMP process
is a significant milestone, not only for AveXis but also for the
patients we hope to serve.”
The AveXis facility is the production site to supply the pivotal
and future trials and, should AVXS-101 be approved for marketing,
to meet projected commercial demand.
With the pivotal trial now starting, AveXis and the FDA are
continuing discussions on key topics, including dosing, for
intrathecal administration of AVXS-101 for the planned clinical
trial in patients with SMA Type 2. An update on this program will
be provided in the fourth quarter of 2017.
U.S. Pivotal Trial in SMA Type 1 (STR1VE)The
open-label, single-arm, single-dose, multi-center trial – known as
STR1VE – is designed to evaluate the efficacy and safety of a
one-time IV infusion of AVXS-101 of 1.1 x 1014 vg/kg, which is
equivalent to the proposed therapeutic dose received by the second
dosing cohort in the Phase 1 trial, in patients with SMA Type 1.
Based on the data derived from the company’s new analytical methods
that were submitted and reviewed by FDA, it has been determined
through direct test with the improved PCR method that the dose used
in AveXis’ Phase 1 trial of AVXS-101 in SMA Type 1 was 1.1 x 1014
vg/kg. Additionally, extensive testing in the SMN delta 7 mouse
potency assay has demonstrated the equivalence of dose response
between the products produced by the Phase 1 and Phase 3
manufacturing process.
The trial will enroll a minimum of 15 patients with SMA Type 1
who are less than six months of age at the time of gene therapy,
and who have one or two copies of the SMN2 backup gene as
determined by genetic testing and bi-allelic SMN1 gene deletion or
point mutations. There will be at least a four-week dosing interval
between dosing of the first three patients to allow review of the
safety analysis from six time points (days one, two, seven, 14, 21
and 30), as well as early signals of efficacy, prior to dosing of
the next patient.
The intent-to-treat population is defined as patients who are
less than six months of age and symptomatic at the time of gene
therapy, with two copies of the SMN2 gene as determined by genetic
testing, bi-allelic SMN1 gene deletion and no c.859G>C mutation
in SMN2.
The co-primary efficacy outcome measures of the trial will
include:
- The achievement of the developmental milestone of independent
sitting for at least 30 seconds at 18 months of age; and,
- Event-free survival at 14 months of age, with an event 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.
Co-secondary outcome measures will include:
- The ability to thrive, defined as the ability to: remain
independent from feeding support, tolerate thin liquids and
maintain weight; and,
- The ability to remain independent of ventilatory support at 18
months of age.
The trial is projected to be conducted at 16 sites in the United
States, including: Ann and Robert H. Lurie Children's Hospital of
Chicago, Boston Children's Hospital, Children's Hospital Colorado,
Children's Hospital of Philadelphia, Columbia University, David
Geffen School of Medicine at UCLA, Duke University, Johns Hopkins
Pediatric Neurology, Nationwide Children's Hospital, Oregon Health
and Science University, Stanford University Medical Center,
University of Central Florida College of Medicine, University of
Texas Southwestern Medical Center, University of Utah, University
of Wisconsin, and Washington University School of Medicine.
“We are appreciative of the detailed reviews and timely feedback
we have received from the FDA,” said James L’Italien, PhD, Chief
Regulatory and Quality Officer for AveXis. “We look forward to our
end-of-Phase 1 meeting, which has been scheduled for late in the
fourth quarter, to discuss next steps in the regulatory process for
AVXS-101.”
Today’s Conference Call InformationAveXis will
host a conference call and webcast at 8:00 a.m. EDT today,
September 29, 2017. 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
93365683. 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
93365683.
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.
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,
will soon be in the pivotal phase of study for the treatment of SMA
Type 1, and a Phase 1/2a 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 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, the expected timing of the
initiation of AveXis’ planned clinical trials in SMA Type 1 and SMA
Type 2 and the results of these trials the overall clinical
development of AVXS-101, if approved, AveXis’ research, development
and regulatory plans for AVXS-101, including AveXis’ commercial
manufacturing process, AveXis’ ability to meet future commercial
demand for AVXS-101 through its manufacturing facility 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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