Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM2 for Tay-Sachs and Sandhoff Disease
13 Octobre 2020 - 1:00PM
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-GM2, a one-time gene therapy delivered directly
to the central nervous system that is in development for GM2
gangliosidosis, also known as Tay-Sachs and Sandhoff disease. In
addition to the Rare Pediatric Disease designation, AXO-AAV-GM2 has
Orphan Drug Designation (ODD) and is the first gene therapy that
has been administered to children with Tay-Sachs disease.
“We are thrilled to bring AXO-AAV-GM2 one step closer to
patients in need through this Rare Pediatric Disease designation.
AXO-AAV-GM2 has the potential to be the first treatment approved
for Tay-Sachs and Sandhoff disease, rare and fatal pediatric
diseases with no current treatment options,” said Sean O’Bryan,
Senior Vice President, Regulatory Affairs & Quality.
Axovant expects to evaluate AXO-AAV-GM2 in a
registrational clinical trial which consists of a Stage 1
dose-ranging study and a Stage 2 efficacy study. Previously,
Axovant reported the first evidence for potential disease
modification in Tay-Sachs disease from an expanded access study
administering investigational AXO-AAV-GM2 gene therapy in two
patients with infantile (Type I) Tay-Sachs disease. AXO-AAV-GM2 was
successfully administered in both patients and has been generally
well-tolerated to date, with no serious adverse events or
clinically relevant laboratory abnormalities related to
therapy.
GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff
disease, is a rare and fatal pediatric neurodegenerative lysosomal
storage disorder (LSD) resulting from deficiencies in
beta-hexosaminidase, a key enzyme in the lysosome. These genetic
defects lead to the toxic accumulation of gangliosides, resulting
in neurodegeneration and life expectancy shortened to just two to
four years of age.
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-GM2
AXO-AAV-GM2 is an investigational gene therapy for Tay-Sachs and
Sandhoff disease, which rare and fatal pediatric neurodegenerative
genetic disorders within the GM2 gangliosidosis family, caused by
defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading
to Sandhoff disease) genes that encode the two subunits of the
β-hexosaminidase A (HexA) enzyme. Both forms of GM2 gangliosidosis
are caused by overwhelming storage of GM2 ganglioside within
neurons throughout the central nervous system), which is normally
degraded in the lysosome by the isozyme HexA. These genetic defects
lead to progressive neurodegeneration and shortened life
expectancy. AXO-AAV-GM2 aims to restore HexA levels by introducing
a functional copy of the HEXA and HEXB genes via delivery of two
co-administered AAVrh8 vectors.
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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