AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3
25 Avril 2018 - 2:00PM
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 first patient has been dosed in a Phase 3 trial evaluating
AVXS-101 in pre-symptomatic patients with spinal muscular atrophy
(SMA) Types 1, 2 and 3 (SPRINT).
“Treating SMA as early as possible is critically
important in order to rescue motor neurons before they are
permanently lost. SPRINT enables us to understand how intervening
in pre-symptomatic infants with AVXS-101 may impact clinical
outcomes, including milestone development such as functional
sitting, standing without support and walking,” said Dr. Sukumar
Nagendran, Chief Medical Officer of AveXis. “In addition to our
ongoing studies in SMA Types 1 and 2, SPRINT adds to our evaluation
of AVXS-101 in multiple SMA sub-types, including Type 3. We are
excited by the progress made across our clinical development
program as we continue toward the goal of making AVXS-101 available
to the SMA community.”
SPRINT is a multi-cohort, multi-national trial
expected to enroll approximately 44 pre-symptomatic patients with a
bi-allelic deletion of SMN1; two, three or four copies
of SMN2; and who are less than six weeks of age at the time of
gene therapy. The trial is designed to evaluate appropriate
clinical endpoints, including developmental milestones, survival,
bulbar function and safety of a one-time intravenous infusion of
AVXS-101 of 1.1 x 1014 vg/kg.
2 Copy SMN2 Cohort
- Primary Outcome: proportion of patients who achieve functional
independent sitting for at least 30 seconds, up to 18 months
of age
- Secondary Outcomes: event-free survival at 14 months of age;
and, ability to maintain weight at or above the third percentile
without need for non-oral/mechanical feeding support up to 18
months of age— An event is 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
3 Copy SMN2 Cohort
- Primary Outcome: proportion of patients who achieve the ability
to stand without support for at least three seconds, up to 24
months of age
- Secondary Outcome: ability to walk without assistance by 24
months of age, defined as the ability to take at least five steps
independently displaying coordination and balance
4 Copy SMN2 Cohort
- Primary Outcome: proportion of patients who do not manifest
symptoms consistent with SMA Type 3 based on a scaled score on
Bayley V.3 Gross and Fine Motor Subtests within 1.5 standard
deviations of chronological development reference standard, as
assessed at 36 months of age
The trial will be conducted at 26 sites in 13
countries.
- U.S. sites include: Center for Rare Neurological Diseases;
Clinic for Special Children; Columbia University; David Geffen
School of Medicine at UCLA, Department of Neurology; Duke
University; Massachusetts General; Nationwide Children's Hospital;
Nemours Children’s Hospital; Stanford University Medical Center;
University of Texas Southwestern Medical Center, Children's Medical
Center Ambulatory Care Pavilion in Dallas; University of Utah, Utah
Program For Inherited Neuromuscular Disorders; University of
Wisconsin, Madison; and, Washington University School of
Medicine.
- Sites outside of the U.S. include: Agostino Gemelli; Area
Genetica Clínica y Molecular; Belgium (Liege); Canada Children’s
Hospital of Eastern Ontario; Dr. Von Haunersches Kinderspital;
Fondazione Policlinico Universitario; Great Ormond Street Hospital
for Children; Hospital Valle Hebron, Barcelona; Israel, Schneider
Children's Medical Center of Israel, Institute of Child Neurology;
National Taiwan University Hospital; Pusan National University
Children's Hospital; Sydney Children’s Hospital; and, UMC Utrecht,
Wilhelmina Kinderziekenhuis.
For more information about SPRINT, please visit
clinicaltrials.gov.
About SMA
SMA 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. SMA Type 3 typically presents in early childhood to
early adulthood, and those affected may lose the ability to walk
over time. The incidence per live births among subtypes is
approximately 60, 27 and 13 percent for SMA Type 1, Type 2 and Type
3, respectively.
About AVXS-101
AveXis’ initial product candidate, AVXS-101, is its
proprietary gene therapy currently in development for the one-time
treatment of SMA Types 1, 2 and 3, 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 crossing the blood brain barrier to allow effective
targeting of both central and systemic features.
About AveXis, Inc.
AveXis, Inc. is a clinical-stage gene therapy
company, dedicated to developing and commercializing novel
treatments for patients suffering from rare and life-threatening
neurological genetic diseases. Our initial product candidate,
AVXS-101, is our proprietary gene therapy currently in development
for the treatment of spinal muscular atrophy, or SMA, Type 1, the
leading genetic cause of infant mortality, and SMA Types 2 and 3.
The U.S. Food and Drug Administration, or FDA, has granted AVXS-101
Orphan Drug Designation for the treatment of all types of SMA and
Breakthrough Therapy Designation, as well as Fast Track Designation
for the treatment of SMA Type 1. In addition to developing AVXS-101
to treat SMA, we also plan to develop other novel treatments for
rare neurological diseases, including Rett syndrome and a genetic
form of amyotrophic lateral sclerosis 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’ continued enrollment of patients in the SPRINT trial and
AveXis’ research, development and regulatory plans for 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 and regulatory
developments in the U.S., EU and Japan, 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, 2017, filed with the SEC on February 28, 2018. 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.
Media Inquiries:Lauren BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com
Investor Inquiries:Jim GoffAveXis,
Inc.650-862-4134jgoff@avexis.com
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