Axovant Sciences (NASDAQ: AXON), a clinical-stage company focused
on innovative gene therapies for neurological and neuromuscular
diseases, today announced that it has licensed exclusive worldwide
rights for the development and commercialization of two novel gene
therapy programs to address GM1 gangliosidosis and GM2
gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) from
the University of Massachusetts (UMass) Medical School.
GM1 gangliosidosis, Tay-Sachs and Sandhoff
diseases are rapidly progressive and fatal pediatric lysosomal
storage disorders that reduce life expectancy to less than two to
four years of age in the severe forms of the diseases. GM1
gangliosidosis has an incidence of approximately one out of 100,000
live births worldwide, and Tay-Sachs and Sandhoff diseases have an
incidence of approximately one out of 180,000 live births
worldwide. GM1 gangliosidosis is caused by defects in the GLB1 gene
and GM2 gangliosidosis is caused by defects in the HEXA (leading to
Tay-Sachs disease) and HEXB (leading to Sandhoff disease) genes,
resulting in impaired enzyme function and the accumulation of toxic
gangliosides primarily in the central nervous system.
AXO-AAV-GM1 and AXO-AAV-GM2 are each designed to
introduce functional copies of the respective genes encoding the
critical enzymes impacted in these diseases, with an aim to improve
survival and enable children to reach key developmental milestones.
In prior animal studies conducted with these gene therapies,
dose-dependent increases in enzyme activity, reductions in
accumulated gangliosides and prolonged survival have been
observed.
AXO-AAV-GM1 will be evaluated in an
investigator-initiated clinical program conducted at the National
Institutes of Health (NIH), with the first patient expected to be
dosed in the first half of 2019. The NIH has assembled one of the
largest natural history databases of patients with GM1
gangliosidosis, Tay-Sachs and Sandhoff diseases that documents the
clinical progression of the disease in affected patients, which
could enable a historical control group for registrational studies.
We expect initial data from this clinical program in the second
half of 2019 and expect continued enrollment of patients in this
program throughout 2019.
The AXO-AAV-GM2 clinical program is ongoing with
the first subject having been dosed with the therapy. Initial data
from this program are expected in first quarter 2019 and we expect
patients to be enrolled in a multi-subject clinical trial in
2019.
“Diseases like Tay-Sachs are attractive targets
for the transformative possibilities of gene therapy because we
have been able to identify the underlying genetic cause of the
disease and now have well-understood methods of delivering the
corrective genes,” said Miguel Sena-Esteves, Ph.D., associate
professor of neurology at UMass Medical School and a principal
scientist of the AXO-AAV-GM1 and AXO-AAV-GM2 programs. “Axovant’s
expertise in the development and manufacturing of investigational
gene therapies and their focus on execution on behalf of patients
makes them a strong partner to translate the impressive preclinical
results for AXO-AAV-GM1 and AXO-AAV-GM2 into the clinic.”
“We are excited to add these potentially
life-saving gene therapy programs for GM1 gangliosidosis, Tay-Sachs
and Sandhoff diseases to our growing pipeline of innovative gene
therapy product candidates. The devastating nature of these
disorders creates an urgent need to pursue opportunities that may
offer hope to these children and their families,” said Pavan
Cheruvu, M.D., chief executive officer of Axovant. “We look forward
to working with world-recognized pioneers in gene therapies at the
University of Massachusetts Medical School and the National
Institutes of Health to bring these treatments to patients. We are
also inspired by and anticipate working closely with affected
patient communities through the National Tay-Sachs & Allied
Diseases Association and the Cure Tay-Sachs Foundation.”
“We have lost too many children to these
devastating diseases. Patients and their families deserve the hope
that these potentially life-saving gene therapies could provide,”
said Sue Kahn, executive director of National Tay-Sachs &
Allied Diseases Association. “The families of these children have
been waiting for treatment options for too long and we are excited
to see Axovant accelerate these gene therapies into the
clinic.”
In exchange for these exclusive worldwide
licenses for the gene therapy programs for GM1 and GM2
gangliosidoses, Axovant will be making payments to UMass Medical
School tied to development, regulatory and commercial
milestones.
About the Collaboration with University
of Massachusetts Medical School
Research into the causes and potential therapies
for lysosomal storage diseases such as Tay-Sachs, Sandhoff diseases
and GM1 gangliosidosis at UMass Medical School has led to
significant advances in the field, including research and
development of the gene therapy vector used to deliver functioning
copies of the defective genes that cause disease. The AXO-AAV-GM1
and AXO-AAV-GM2 programs were developed by a team of researchers at
UMass Medical School, including Miguel Sena-Esteves, Ph.D., Heather
Gray-Edwards, Ph.D., D.V.M., and dean of the School of Medicine,
Terence Flotte, M.D.
“We are enthusiastic to partner with Axovant and
its experienced team in the treatment of GM1 gangliosidosis,
Tay-Sachs and Sandhoff diseases,” said Heather Gray-Edwards, Ph.D.,
D.V.M., an assistant professor of radiology at UMass Medical
School. “The work of Dr. Esteves, Dr. Gray-Edwards and their
collaborators is a wonderful example of UMass Medical School
scientists and physicians bringing the power of gene therapy to
bear on a medical condition that can be truly tragic for families
with affected babies,” said Terence R. Flotte, M.D., dean of the
School of Medicine, professor of pediatrics at UMass Medical School
and clinical principal investigator for the investigator-initiated
protocol. “Bringing hope to families is what translational research
is all about. Tay-Sachs’ families have waited an incredibly long
time for this hope to be offered.”
About GM1 Gangliosidosis, Tay-Sachs and
Sandhoff Diseases
GM1 gangliosidosis, Tay-Sachs and Sandhoff
diseases are a set of rare and fatal neurodegenerative genetic
disorders caused by impaired β-galactosidase (β-gal) and
β-hexosaminidase A (Hex A) enzyme activity, respectively. GM1
gangliosidosis is caused by defects in the GLB1 gene, which encodes
the β-gal enzyme. GM2 gangliosidosis, including Tay-Sachs and
Sandhoff diseases, is caused by defects in the HEXA (leading to
Tay-Sachs disease) and HEXB (leading to Sandhoff disease) genes
that encode the two subunits of the Hex A enzyme. Defects in these
genes cause impaired enzyme activity leading to the toxic
accumulation of gangliosides, resulting in neurodegeneration that
presents as cognitive impairment, paralysis and early death. There
are currently no disease-modifying treatments for these diseases
and children born with these disorders mostly have a life
expectancy shortened to two to four years of age.
About the AXO-AAV-GM1
Program
AXO-AAV-GM1 delivers a functional copy of the
GLB1 gene via an adeno-associated viral (AAV) vector, AAV9, which
is effective in crossing the blood-brain barrier and transducing
neurons, with the goal of restoring β-gal 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. In preclinical studies, AXO-AAV-GM1 was shown to improve
β-gal enzyme activity, reduce GM1 ganglioside accumulation, improve
neuromuscular function, and extend survival. Magnetic resonance
imaging (MRI) of felines with GM1 gangliosidosis treated with GM1
gene therapy showed normal brain architecture through at least two
years of age.
About the AXO-AAV-GM2
Program
AXO-AAV-GM2 delivers functional copies of the
HEXA and HEXB genes via two, co-administered AAVrh8 vectors
delivered directly to the central nervous system with the goal of
restoring Hex A enzyme activity to address both Tay-Sachs and
Sandhoff diseases. The preclinical data for AXO-AAV-GM2 in murine
models showed dose-dependent increases in Hex A enzyme activity,
reductions of GM2 gangliosides in the brain and prolonged survival
rates. A next-generation gene therapy for Tay-Sachs and Sandhoff
diseases aimed at enabling systemic intravenous administration is
in earlier-stage development.
About Axovant Sciences
Axovant is a clinical-stage gene therapy company focused on
developing a pipeline of innovative product candidates for
debilitating neurological diseases such as Parkinson's disease, GM1
gangliosidosis, Tay-Sachs and Sandhoff diseases, oculopharyngeal
muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS),
frontotemporal dementia, and other indications. For more
information, visit www.axovant.com
About the University of Massachusetts Medical
School
The University of Massachusetts Medical School, one of five
campuses of the University system, comprises the School of
Medicine, the Graduate School of Biomedical Sciences, the Graduate
School of Nursing, a thriving research enterprise and an innovative
public service initiative, Commonwealth Medicine. Its mission is to
advance the health of the people of the commonwealth through
pioneering education, research, public service and health care
delivery with its clinical partner, UMass Memorial Health Care. In
doing so, it has built a reputation as a world-class research
institution and as a leader in primary care education. The Medical
School attracts more than $264 million annually in research
funding, placing it among the top 50 medical schools in the nation.
In 2006, UMass Medical School’s Craig C. Mello, PhD, Howard Hughes
Medical Institute Investigator and the Blais University Chair in
Molecular Medicine, was awarded the Nobel Prize in Physiology or
Medicine, along with colleague Andrew Z. Fire, PhD, of Stanford
University, for their discoveries related to RNA interference. For
more information, visit www.umassmed.edu
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,” “might,” “will,”
“expect,” “plan,” “anticipate,” “believe,” “intend,” “future,” or
“continue” and other similar expressions are intended to identify
forward-looking statements. For example, all statements Axovant
makes regarding the potential efficacy of its product candidates;
initiation, timing, progress, and reporting of results of its
preclinical programs, clinical trials, and research and development
programs; its ability to advance its product candidates into and
successfully initiate, enroll, and complete clinical trials; and
the timing or likelihood of its regulatory filings and approvals,
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 its 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 for the quarterly period
ended September 30, 2018, filed with the Securities and Exchange
Commission on November 7, 2018, 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, except as required by
law.
Contacts:
Media
Lara Yuan(646) 802-3585media@axovant.com
Investors
Tricia Truehart(631)
892-7014investors@axovant.com
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