Axovant Announces Dosing of First Patient in Clinical Program for AXO-AAV-GM1, a Novel Gene Therapy for GM1 Gangliosidosis
May 16 2019 - 4:05PM
Axovant Gene Therapies Ltd. (Nasdaq: AXGT), a clinical-stage
company developing innovative gene therapies, today announced
dosing of the first patient in a clinical study of AXO-AAV-GM1
(also known as AAV9-GLB1), an investigational gene therapy for the
treatment of GM1 gangliosidosis. To date, the patient has
experienced no complications related to the intravenous (IV)
administration of the vector and continues to be followed by
physicians. GM1 gangliosidosis is a progressive and fatal pediatric
lysosomal storage disorder caused by mutations in the GLB1 gene
leading to impaired production of the beta-galactosidase enzyme.
There are currently no approved treatments for GM1 gangliosidosis.
Axovant expects initial data from the AXO-AAV-GM1 clinical program
in the second half of 2019, as well as continued enrollment of
patients in this program throughout 2019.
The clinical study of AXO-AAV-GM1 is being
conducted at the National Institutes of Health (NIH), by Cynthia
Tifft, M.D., Ph.D., Deputy Clinical Director at the National Human
Genome Research Institute, and a leading expert in ganglioside
storage disorders. A three-way Cooperative Research and Development
Agreement (CRADA) between Axovant, the NIH, and the University of
Massachusetts (UMass) Medical School has been established to
support the conduct of the clinical program.
“GM1 gangliosidosis is a devastating disease in
young children, for which there are no currently approved treatment
options. The development of a safe and effective gene therapy for
these patients would be a welcome advancement in the field of
pediatric lysosomal storage disorders affecting the brain,” said
Dr. Tifft.
The endpoints of the clinical study include safety, biomarker,
neurodevelopment, and Magnetic Resonance Imaging (MRI) and Magnetic
Resonance Spectroscopy (MRS) measures.
AXO-AAV-GM1 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.
About Axovant Gene
Therapies
Axovant, 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
neurological and neuromuscular diseases. The company’s current
pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2
gangliosidosis (including Tay-Sachs disease and Sandhoff disease),
Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD),
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.
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.
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
Information
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 “may,” “will,” “expect,”
“would,” “intend,” “future,” and other similar expressions are
intended to identify forward-looking statements. 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. In addition, Axovant’s business is subject to additional
risks and uncertainties, including among others, the initiation and
conduct of preclinical studies and clinical trials; the timing and
availability of data from clinical trials; the expectations for
regulatory submissions and approvals; the potential safety concerns
or profile of Axovant’s product candidates; and the availability or
commercial potential of 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 for the quarterly period ended December 31, 2018, filed with
the Securities and Exchange Commission on February 7, 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:
MediaMike BeyerSam Brown Inc.
(312) 961-2502mikebeyer@sambrown.commedia@axovant.com
InvestorsTricia
TruehartAxovant(631) 892-7014investors@axovant.com
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