Axovant Provides Clinical Program Update for AXO-AAV-GM1, a Novel Investigational Gene Therapy for GM1 Gangliosidosis
December 19 2019 - 5:41PM
Axovant Gene Therapies Ltd. (NASDAQ: AXGT) today announced
preliminary findings from an expanded access treatment in which a
single patient with GM1 gangliosidosis was administered
investigational AXO-AAV-GM1 gene therapy.
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. AXO-AAV-GM1 is an investigational gene therapy that
delivers a functional copy of the GLB1 gene via an adeno-associated
viral (AAV9) vector, with the goal of restoring β-galactosidase
enzyme activity. The natural history of GM1 gangliosidosis
demonstrates progressive neurological decline including worsening
of swallowing ability, which impacts nutritional status, and
impaired motor function.
“GM1 gangliosidosis is a devastating pediatric
disease for which there are no currently approved treatment
options,” said Dr. Cynthia Tifft, Deputy Clinical Director at the
National Human Genome Research Institute (NHGRI), part of the
National Institutes of Health (NIH), and a leading expert in
ganglioside storage disorders. “These preliminary findings suggest
safety, tolerability, and clinical improvement in the first child
dosed with gene therapy for GM1 gangliosidosis. I am particularly
encouraged that this child has regained her ability to swallow more
effectively and has demonstrated meaningful weight gain during the
6 months following gene therapy. We are hoping to complete
enrollment in Part A of the ongoing registrational study in early
2020.”
According to Dr. Tifft, from baseline gene
transfer to 6-month follow-up, the subject was observed to have
clinically significant improvements based on neurological exam, the
Vineland-3 scale, Clinical Global Impression (CGI) assessments, and
nutritional status. The Vineland-3 scale is an individually
administered measure of adaptive behavior that is widely used to
assess individuals with intellectual, developmental, and other
disabilities.
In addition, Dr. Tifft observed that AXO-AAV-GM1
was generally well tolerated with no reports of serious adverse
events related to the investigational gene therapy or intravenous
(IV) administration of the vector.
A separate registrational study of AXO-AAV-GM1
is also being conducted at the NIH Clinical Center by Dr.
Tifft in collaboration with Axovant Gene Therapies. Part A of
the registrational study of AXO-AAV-GM1 (n=5) evaluates safety,
tolerability, and exploratory measures of efficacy.
AXO-AAV-GM1 was granted orphan drug designation
(ODD) by the U.S. Food and Drug Administration (FDA) in November
2019.
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. A three-way Cooperative Research and Development
Agreement (CRADA) between Axovant, the NHGRI, and the University of
Massachusetts was established earlier this year to support the
conduct of the clinical program.
About Axovant Gene
Therapies
Axovant Gene Therapies, part of the Roivant
family of companies, 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 (including 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 (UMMS)
for the development and commercialization of gene therapy programs
for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs
and Sandhoff diseases.
About Roivant
Roivant Sciences aims to improve health by rapidly delivering
innovative medicines and technologies to patients. It does this by
building Vants – nimble, entrepreneurial biotech and healthcare
technology companies with a unique approach to sourcing talent,
aligning incentives, and deploying technology to drive greater
efficiency in R&D and commercialization. For more information,
please visit www.roivant.com.
Forward Looking Statements and
InformationThis 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 “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 the
initiation, timing, progress, and reporting of results of clinical
trials and research and development programs; its ability to
advance its gene therapy product candidates into and successfully
initiate, enroll, and complete clinical trials; the potential
clinical utility of its product candidates; its ability to continue
to develop its gene therapy platforms; 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 initiation and conduct of preclinical studies and clinical
trials; the availability of data from clinical trials; the
expectations for regulatory submissions and approvals; the
continued development of product candidates; 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 November 8, 2019, 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 and Investors
Parag V. Meswani, PharmD Axovant Gene Therapies (212) 547-2523
investors@axovant.com media@axovant.com
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