Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases
09 Novembre 2020 - 1:00PM
Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage
company developing innovative gene therapies, today announced that
the U.S. Food and Drug Administration (FDA) has lifted its clinical
hold and cleared the Investigational New Drug (IND) Application to
initiate a registrational study of AXO-AAV-GM2 gene therapy to
treat patients with Tay-Sachs disease and Sandhoff disease.
AXO-AAV-GM2 is the first investigational gene therapy to achieve
IND clearance for Tay-Sachs and Sandhoff diseases. The Company
received a letter from the FDA indicating that it has
satisfactorily addressed all issues related to the clinical hold.
“The IND clearance of AXO-AAV-GM2, the first
potentially curative treatment for Tay-Sachs and Sandhoff diseases
to enter the clinic, marks a seminal moment for the entire GM2
gangliosidosis community. Along with AXO-AAV-GM1 for GM1
gangliosidosis, today’s announcement represents the second IND
clearance for Axovant’s gene therapy pipeline in the last 12
months,” said Gavin Corcoran, M.D., Chief R&D Officer of
Axovant. “AXO-AAV-GM1 and AXO-AAV-GM2 are the first gene therapies
to enter active clinical development in their indications. Each
program captures the essence of our mission – to liberate patients
from debilitating neurological diseases by rapidly advancing these
gene therapy programs into clinical development. We look forward to
working with our study investigators, academic partners, the
patient community, and families to begin enrollment in the study
promptly.”
Sue Kahn, Executive Director of the National
Tay-Sachs & Allied Diseases Association (NTSAD), added, “This
is a big step toward a potential therapy for the GM2 community. It
has been a long journey since NTSAD started supporting this
promising gene therapy research in 2006. We are thrilled that it is
now entering the clinic, bringing tremendous hope to families and
the entire NTSAD community.”
Axovant aims to advance the program through
strategic partnerships with leading research organizations. The
Company recently announced a partnership with Viralgen, an AskBio
subsidiary, to support AAV-based vector manufacturing of clinical
trial material for the registrational study. Additionally, through
an existing genetic testing collaboration with Invitae, ongoing
partnership with GM2 gangliosidosis patient groups, and
collaboration with leading academic researchers at the University
of Massachusetts Medical School and Massachusetts General Hospital,
Axovant expects to begin patient identification and site startup
activities in preparation for dosing children in the planned
clinical study.
AXO-AAV-GM2 is an investigational gene therapy
for Tay-Sachs and Sandhoff diseases, which are rare, monogenic
neurodegenerative lysosomal storage disorders caused by mutations
in the genes that encode β-Hexosaminidase A, HEXA and HEXB.
Children affected by Tay-Sachs and Sandhoff diseases suffer from a
progressively debilitating disease course and reduced life
expectancy. AXO-AAV-GM2 delivers two vectors encoding the HEXA and
HEXB genes directly to the central nervous system to produce a
fully functional β-Hexosaminidase A enzyme. In 2019, clinical
evidence from two patients under an investigator-initiated study
found that treatment with AXO-AAV-GM2 was generally well-tolerated
and associated with improved bioactivity outcomes. In addition, the
data demonstrated the attainment of normal neurodevelopmental
milestones and improvement in myelination. AXO-AAV-GM2 has been
granted Orphan Drug and Rare Pediatric Disease Designation by the
FDA.
The study will enroll both infantile and
juvenile subjects with GM2 gangliosidosis in the U.S. The two-part
trial, sponsored by Axovant, will consist of (1) a dose ranging
cohort evaluating the safe and efficacious dose of the gene
therapy, followed by (2) an efficacy cohort, both of which form the
basis of the registrational program. Terence R. Flotte, M.D.,
Professor of Pediatrics and Dean at the University of Massachusetts
Medical School, will serve as principal investigator on the
clinical trial.
About AXO-AAV-GM2
AXO-AAV-GM2 is an investigational gene therapy
for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff
diseases), a set of rare and fatal pediatric neurodegenerative
genetic disorders 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. These genetic defects lead to progressive
neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims
to restore HexA function by introducing a functional copy of
the HEXA and HEXB genes via delivery of two
co-administered AAVrh8 vectors.
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 target 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.
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.
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 "will," "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 development of a
suspension-based manufacturing process; 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:
Investors
Parag MeswaniAxovant Gene Therapies Ltd.(212)
547-2523investors@axovant.com
Media
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.commedia@axovant.com
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