AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology
19 Aprile 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 initial results from the pivotal study (STR1VE) of AveXis’
proprietary gene therapy, AVXS-101, for the treatment of spinal
muscular atrophy (SMA) Type 1, and 24-month follow-up data from the
Phase 1 trial, will be presented at the 2018 Annual Meeting of
the American Academy of Neurology (AAN) taking
place April 21-27 in Los Angeles.
“AVXS-101 Gene Replacement Therapy Pivotal Trial in SMA Type 1:
Pivotal Study (STR1VE) Update,” will be presented during the
Emerging Science Poster Session IV on April 25 at 11:30 a.m. –
7:00 p.m. PDT. John W. Day, MD, PhD, Professor, Neurology &
Neurological Sciences Director, Stanford Neuromuscular Disorders
Program, Stanford University Medical Center, will be available
starting at 5:30 p.m. PDT.
Four platform presentations will be delivered consecutively
during S29: Child Neurology and Developmental Neurology I on April
25 from 1:00 – 1:48 p.m. PDT:
- “AVXS‐101 Phase 1 Gene Replacement Therapy Clinical
Trial in SMA Type 1: Event Free Survival and Achievement of
Developmental Milestones after 24-Months Post-Dosing,” by Samiah
Al-Zaidy, MD, Co-Investigator for the study.
- “AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in
SMA Type 1: Patients Treated Early with the Proposed Therapeutic
Dose Were Able to Sit Unassisted at a Younger Age (24-Month
Follow-Up Data Set),” by Linda Lowes, PhD, Director of Clinical
Therapies Research and a member of the Center for Gene Therapy at
the Research Institute of Nationwide Children’s Hospital.
- “AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in
SMA Type 1: Continued Independence from Nutritional and Ventilatory
Support in Patients Dosed Early in Disease Progression After
24-Months Post-Dosing,” by Richard Shell, MD, member of the Section
of Pulmonary Medicine at Nationwide Children’s Hospital.
- “AVXS-101 Trial Experience: CHOP-INTEND Effectively
Demonstrates Marked, Early, Rapid, and Sustained Improvements
Following Treatment and Precedes Major Motor Milestone Achievement
(24-Month Follow-Up Data Set),” by Dr. Linda Lowes.
Abstracts can be accessed through the AAN website
at www.aan.com.
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-101AveXis’ initial product
candidate, AVXS-101, is its proprietary gene therapy currently in
development for the one-time treatment of 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 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 Type 2. 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 Type 1 and Type 2,
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.
Media Inquiries:Lauren BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
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