Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM1 for GM1 Gangliosidosis
09 Outubro 2020 - 8:00AM
Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage
company developing innovative gene therapies for neurological
diseases, today announced that it has received Rare Pediatric
Disease Designation from the U.S. Food and Drug Administration
(FDA) for AXO-AAV-GM1, an AAV9-based gene therapy delivered via a
single intravenous administration that is in Phase 1/2 development
for GM1 gangliosidosis. In addition to the Rare Pediatric Disease
designation, AXO-AAV-GM1 has Orphan Drug designation and is the
only gene therapy in clinical development for both infantile (Type
I) and juvenile (Type II) GM1 gangliosidosis.
“Receiving Rare Pediatric Disease Designation in addition to the
previously granted Orphan Drug Designation for AXO-AAV-GM1
highlights the importance of expediting a therapy for GM1
Gangliosidosis, a progressive, fatal, pediatric disease with no
approved treatment options,” said Sean O’Bryan, Senior Vice
President, Regulatory Affairs & Quality. “AXO-AAV-GM1 is the
first gene therapy to enter clinical trials for GM1 gangliosidosis
and has the potential to provide meaningful clinical benefit to
both Type I and Type II patients.”
Axovant is on-track to report 6-month Stage 1
data from the low dose juvenile cohort (Type II) by the fourth
quarter of 2020, and expects to initiate the high dose cohort which
includes infantile (Type I) and juvenile (Type II) patients in the
second half of 2020.
GM1 Gangliosidosis is a progressive and fatal pediatric
lysosomal storage disorder caused by mutations in the GLB1 gene
leading to impaired production of the β-galactosidase enzyme.
Currently, there are no approved treatment options for GM1
Gangliosidosis.
The FDA defines a rare pediatric disease as a serious or
life-threatening disease in which the disease manifestations
primarily affect individuals aged from birth to 18 years. Pediatric
diseases recognized as “rare” affect under 200,000 people in the
United States.
About AXO-AAV-GM1
AXO-AAV-GM1 is an investigational gene therapy that delivers a
functional copy of the GLB1 gene via an adeno-associated viral
(AAV) vector, with the goal of restoring β-galactosidase enzyme
activity for the treatment of GM1 gangliosidosis. The gene therapy
is delivered intravenously, which has the potential to broadly
transduce the central nervous system and treat peripheral
manifestations of the disease as well. Preclinical studies in
murine and a naturally-occurring feline model of GM1 gangliosidosis
have supported AXO-AAV-GM1’s ability to improve β-galactosidase
enzyme activity, reduce GM1 ganglioside accumulation, improve
neuromuscular function, and extend survival.
In 2018, Axovant licensed exclusive worldwide rights from the
University of Massachusetts Medical School for the development and
commercialization of gene therapy programs for GM1 gangliosidosis
and GM2 gangliosidosis, including Tay-Sachs and Sandhoff
diseases.
About
Axovant Gene
Therapies
Axovant Gene Therapies is a clinical-stage gene
therapy company focused on developing a pipeline of innovative
product candidates for debilitating neurodegenerative diseases. Our
current pipeline of gene therapy candidates targets GM1
gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease
and Sandhoff disease), and Parkinson’s disease. Axovant is focused
on accelerating product candidates into and through clinical trials
with a team of experts in gene therapy development and through
external partnerships with leading gene therapy organizations. For
more information, visit www.axovant.com.
Forward-Looking Statements
This press release contains forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
The use of words such as “intended”, "may," "might," "will,"
"would," "should," "expect," "believe," "estimate," and other
similar expressions are intended to identify forward-looking
statements. For example, all statements Axovant makes regarding
costs associated with its operating activities are forward-looking.
All forward-looking statements are based on estimates and
assumptions by Axovant’s management that, although Axovant believes
to be reasonable, are inherently uncertain. All forward-looking
statements are subject to risks and uncertainties that may cause
actual results to differ materially from those that Axovant
expected. Such risks and uncertainties include, among others, the
impact of the Covid-19 pandemic on our operations, the initiation
and conduct of preclinical studies and clinical trials; the
availability of data from clinical trials; the scaling up of
manufacturing, the expectations for regulatory submissions and
approvals; the continued development of our gene therapy product
candidates and platforms; Axovant’s scientific approach and general
development progress; and the availability or commercial potential
of Axovant’s product candidates. These statements are also subject
to a number of material risks and uncertainties that are described
in Axovant’s most recent Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission on August 11, 2020, as
updated by its subsequent filings with the Securities and Exchange
Commission. Any forward-looking statement speaks only as of the
date on which it was made. Axovant undertakes no obligation to
publicly update or revise any forward-looking statement, whether as
a result of new information, future events or otherwise.
Contacts:
Media & Investors
Josephine Belluardo, Ph.D.LifeSci
Communications646-751-4361jo@lifescicomms.commedia@axovant.com
Parag MeswaniAxovant Gene Therapies Ltd.(212)
547-2523investors@axovant.com
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