Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Dis...
February 03 2021 - 7:00AM
Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company
focused on developing gene therapies to radically transform the
lives of patients with neurodegenerative diseases, today announced
that the first patient with infantile Tay-Sachs disease has been
dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an
investigational gene therapy for the treatment of GM2
gangliosidosis, also known as Tay-Sachs or Sandhoff disease.
“We are proud to bring the first potentially disease-modifying
treatment for GM2 gangliosidosis to the clinic, which is a
milestone for Sio, for patients, and for the field of gene
therapy,” said Gavin Corcoran, M.D., Chief R&D Officer of Sio.
“By restoring lysosomal enzyme activity where it is essential,
AXO-AAV-GM2 has the potential to change the course of this disease
and help affected children attain and retain important
neuro-developmental milestones. The prior expanded access study of
AXO-AAV-GM2 provided important proof-of-concept data and we look
forward to the results of the first stage of our study as we strive
to develop a treatment for children suffering from this rapidly
progressive and fatal disease.”
Florian Eichler, M.D., Director of the Leukodystrophy Service of
the Center for Rare Neurological Diseases at Massachusetts General
Hospital, and principal investigator, added, “To date, the current
GM2 treatment landscape is limited to supportive care, underscoring
the significant need for new treatment options to address this
devastating pediatric neurodegenerative disease. AXO-AAV-GM2 has
significant potential to address the clinical manifestations of
both Tay Sachs and Sandhoff diseases, and as a result, the dosing
of this patient represents a major step forward for this therapy.
We look forward to evaluating the results of this study and
advancing the first potentially disease-modifying treatment option
for patients with GM2.”
The Phase 1/2 study (NCT04669535) is an open-label, two-stage
clinical trial designed to evaluate safety and dose-escalation
(Stage 1) and safety and efficacy (Stage 2) of surgical delivery of
AXO-AAV-GM2 directly to the brain and spinal cord of pediatric
participants with both infantile and juvenile GM2 gangliosidosis.
AXO-AAV-GM2 has been granted Orphan Drug and Rare Pediatric Disease
Designation by the FDA and is the first investigational gene
therapy to enter clinical trials for GM2 gangliosidosis. In 2019,
clinical evidence from two patients under an expanded access IND
found that treatment with AXO-AAV-GM2 was generally well-tolerated
and associated with improved bioactivity outcomes.
“The families of children with Sandhoff and Tay-Sachs diseases
show incredible bravery in choosing to participate in
investigational studies of novel therapeutics like AXO-AAV-GM2. We
share their hope that this treatment can halt or reverse the
otherwise inexorable course of these tragic diseases,” said Terence
R. Flotte, MD, Professor of Pediatrics and Dean at the University
of Massachusetts Medical School and principal investigator of the
trial.
GM2 gangliosidosis is a set of rare, monogenic neurodegenerative
lysosomal storage disorders caused by mutations in the genes that
encode the enzyme β-Hexosaminidase A. It can be categorized into
two distinct diseases, Tay-Sachs disease, which results from a
mutation in the gene encoding the alpha subunit of the
β-Hexosaminidase A enzyme (HEXA), and Sandhoff disease, which
results from a mutation in the gene encoding the beta subunit of
the β-Hexosaminidase A enzyme (HEXB). Children affected by GM2
gangliosidosis suffer from a progressively debilitating disease
course and reduced life expectancy.
Sue Kahn, Executive Director of National Tay-Sachs & Allied
Diseases Association (NTSAD), added, “This news represents the
culmination of many years of work to advance this research and
immense support from the GM2 community, and it underscores the dire
need for new treatment options capable of providing meaningful
benefits to patients and families. We are extremely excited by the
progress Sio has made and the hope it brings to our community.”
Sio aims to advance the program through strategic partnerships
with leading research organizations. The Company has 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, Sio has begun patient
identification and screening activities for the ongoing clinical
study.
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 Sio Gene TherapiesSio Gene Therapies
combines cutting-edge science with bold imagination to develop
genetic medicines that aim to radically improve the lives of
patients. Our current pipeline of clinical-stage candidates
includes the first potentially curative AAV-based gene therapies
for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are
rare and uniformly fatal pediatric conditions caused by single gene
deficiencies. We are also expanding the reach of gene therapy to
highly prevalent conditions such as Parkinson’s disease, which
affects millions of patients globally. Led by an experienced team
of gene therapy development experts, and supported by
collaborations with premier academic, industry, and patient
advocacy organizations, Sio is focused on accelerating its
candidates through clinical trials to liberate patients with
debilitating diseases through the transformational power of gene
therapies. For more information, visit www.siogtx.com.
In 2018, Sio 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 “expect” “potentially,” and “potential,”
and other similar expressions are intended to identify
forward-looking statements. For example, all statements Sio makes
regarding costs associated with its operating activities are
forward-looking. All forward-looking statements are based on
estimates and assumptions by Sio’s management that, although Sio
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
Sio 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 for AXO-Lenti-PD; the
scaling up of manufacturing, the expectations for regulatory
submissions and approvals; the continued development of our gene
therapy product candidates and platforms; Sio’s scientific approach
and general development progress; and the availability or
commercial potential of Sio’s product candidates. These statements
are also subject to a number of material risks and uncertainties
that are described in Sio’s most recent Quarterly Report on Form
10-Q filed with the Securities and Exchange Commission on November
13, 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. Sio 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
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.com info@siogtx.com
Investors and Analysts
Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief
Commercial Officer investors@siogtx.com
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