– The FDA notified AveXis it may initiate the
Phase 1 trial in SMA Type 2 based on a review of data provided by
the company; trial to commence immediately –
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, the company may initiate
its planned Phase 1 clinical trial of AVXS-101 for patients with
spinal muscular atrophy (SMA) Type 2 via the intrathecal (IT) route
of administration, using material produced by the company’s Good
Manufacturing Practice (GMP) commercial manufacturing process at
the AveXis manufacturing facility. The company plans to initiate
this trial immediately.
“We are quite pleased to initiate our first trial of AVXS-101 in
patients with SMA Type 2,” said Sean Nolan, President and Chief
Executive Officer of AveXis. “Our goal has been to expand the study
of gene therapy beyond Type 1 infants to address the urgent medical
needs of children with SMA Type 2, and we look forward to
understanding the potential clinical impact of AVXS-101 in these
patients who, left untreated, will never walk on their own and most
will never stand without assistance.”
U.S. Phase 1 Trial in SMA Type 2 (STRONG)The
open-label, dose-comparison, multi-center Phase 1 trial – known as
STRONG – is designed to evaluate the safety, optimal dosing, and
proof of concept for efficacy of AVXS-101 in two distinct age
groups of patients with SMA Type 2, utilizing a one-time IT route
of administration. The trial will enroll 27 infants and children
with a genetic diagnosis consistent with SMA, including the
bi-allelic deletion of SMN1 and three copies of SMN2 without the
SMN2 genetic modifier, who are able to sit but have no historical
or current ability to stand or walk.
Two dosage strengths will be evaluated and patients will be
stratified into two age groups: patients less than 24 months, and
patients at least 24 months but less than 60 months. There will be
at least a four-week interval between the dosing of the first three
patients for each dose being studied and, based on the available
safety data, a decision will be made whether to proceed.
- Cohort 1 (Dose A) will receive a dose of 6.0 x 1013 vg of
AVXS-101 and enroll three patients less than 24 months of age.
- If safety is established according to the Data Safety
Monitoring Board (DSMB), the study will proceed to Cohort 2.
- Cohort 2 (Dose B) will receive a dose of 1.2 X 1014 vg of
AVXS-101 and enroll three patients less than 60 months of age.
- If safety is established according to the DSMB, an additional
21 patients will be enrolled until there are a total of 12 patients
less than 24 months, and 12 patients at least 24 months but less
than 60 months of age, who have received Dose B.
According to the well-characterized natural history of the
disease by the Pediatric Neuromuscular Clinical Research Network,
100 percent of children with SMA Type 2 will never walk without
support, 95 percent of children will never stand without assistance
and more than 30 percent will die by 25 years of age. Additionally,
children with SMA Type 2 experienced a mean decrease of - 0.33
points on the Hammersmith Function Motor Scale Expanded over a
12-month period.
Outcome Measures for Patients Less than 24 Months of
Age
- The primary outcome measure for patients less than 24 months of
age at the time of dosing is the achievement of the ability to
stand without support for at least three seconds.
- The secondary outcome measure is the proportion of patients who
achieve the ability to walk without assistance, defined as taking
at least five steps independently while displaying coordination and
balance.
- Developmental abilities, including motor function, will be
evaluated as exploratory objectives.
Outcome Measures for Patients Between 24 and 60 Months
of Age
- The primary outcome measure for patients between 24 months and
60 months of age at the time of dosing is the achievement of change
in Hammersmith Functional Motor Scale Expanded from baseline.
- The secondary outcome measure is the proportion of patients who
achieve the ability to walk without assistance, defined as taking
at least five steps independently displaying coordination and
balance.
- Developmental abilities, including motor function, will be
evaluated as exploratory objectives
The trial is projected to be conducted at 11 sites in the United
States, including: Ann and Robert H. Lurie Children's Hospital of
Chicago, Boston Children's Hospital, Children's Hospital of
Philadelphia, David Geffen School of Medicine at UCLA, Johns
Hopkins Pediatric Neurology, Nationwide Children's Hospital,
Stanford University Medical Center, University of Central Florida
College of Medicine, University of Texas Southwestern Medical
Center, University of Utah and Washington University School of
Medicine.
For more information about these clinical trials, please visit
clinicaltrials.gov.
“This Phase 1 trial in children with SMA Type 2 will allow us to
evaluate safety, optimal dosing and proof-of-concept for efficacy
of AVXS-101 compared to the well-characterized natural history
using the one-time intrathecal route of administration,” said Dr.
Sukumar Nagendran, Chief Medical Officer of AveXis. “Because
AVXS-101 targets the root cause of SMA, we are optimistic that we
will observe a similar preclinical to clinical translation in this
Type 2 trial as was seen in the SMA Type 1 study using intravenous
administration.”
SMA Type 1 UpdateOn December 5, 2017, the
company had an end-of-Phase 1 meeting with FDA with respect to
AVXS-101 for SMA Type 1. The company anticipates providing an
update on feedback from FDA following the receipt of the final
meeting minutes in early January.
Today’s Conference Call InformationAveXis will
host a conference call and webcast at 4:30 p.m. EST today, December
13, 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
2945908. 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
2945908.
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 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 trial in SMA Type 2, ability
to enroll for, and the results of, AveXis’ planned clinical trials
in SMA Type 1 and SMA Type 2, the overall clinical development of
AVXS-101, if approved, AveXis’ research, development and regulatory
plans for AVXS-101, including AveXis’ commercial manufacturing
process, timing of feedback from FDA on AVXS-101, 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 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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