Sio Gene Therapies Announces Dosing of First GM1 Gangliosidosis Early Infantile (Type I) Patient in Ongoing Phase 1/2 Study of AXO-AAV-GM1 Gene Therapy
September 09 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
dosing of the first Type I, or early infantile, patient in the
low-dose cohort of its ongoing Phase 1/2 study of AXO-AAV-GM1, its
adeno-associated viral vector (AAV)9-based gene therapy candidate
for the treatment of GM1 gangliosidosis. No serious adverse events
considered by the investigators to be related to AXO-AAV-GM1 have
been observed following the administration of GM1 gene therapy, and
additional screening and enrollment of Type I patients is ongoing.
“Dosing the first Type I patient represents a major milestone
for the AXO-AAV-GM1 program and for the GM1 gangliosidosis
community. The efforts by both Sio and our trial partners at the
NIH are the culmination of our dedication to patients,” said Gavin
Corcoran, M.D., Chief R&D Officer of Sio Gene Therapies. “We
are excited to expand the age range of patients that have been
dosed to address this more severe form of GM1 gangliosidosis, and
we expect to report 12-month topline safety, biomarker, and
clinical outcomes data from the Type II low-dose cohort of
AXO-AAV-GM1 in October 2021.”
Dr. Cynthia Tifft, Deputy Clinical Director of the National
Human Genome Research Institute (NHGRI) and study Principal
Investigator added, “GM1 gangliosidosis patients and their families
are faced with the diagnosis of a terminal illness with no
available treatments. This milestone brings patients, now including
those suffering from Type I early infantile disease, one step
closer to the first approved gene therapy for this devastating
pediatric lysosomal storage disorder.”
The clinical study (NCT03952637) is designed to evaluate the
safety, tolerability, and potential efficacy of AXO-AAV-GM1
delivered intravenously in children with early infantile, or Type
I, and late infantile and juvenile, or Type II, GM1 gangliosidosis.
Stage 1 is a dose-escalation study in which the low-dose cohort is
evaluating 1.5x1013 vg/kg and the high-dose cohort is evaluating a
dose of 4.5x1013 vg/kg in both disease sub-types. Target enrollment
of eight late infantile and juvenile-onset (Type II) patients
across the low- and high-dose cohorts was recently completed and up
to three Type I patients each in the low-dose and high dose cohorts
are expected to be enrolled.
GM1 gangliosidosis is a progressive and fatal pediatric
lysosomal storage disorder caused by mutations in the GLB1 gene
that cause impaired production of the β-galactosidase enzyme.
Currently, there are no FDA-approved treatment options for GM1
gangliosidosis.
About AXO-AAV-GM1
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.
AXO-AAV-GM1 has received both Orphan Drug Designation and Rare
Pediatric Disease Designation from the Food and Drug
Administration and is the only gene therapy in clinical
development for all pediatric forms of GM1 gangliosidosis.
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.
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.
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," "estimate," "may" and other
similar expressions are intended to identify forward-looking
statements. For example, all statements Sio makes regarding costs
associated with its operating activities, funding requirements
and/or runway to meet its upcoming clinical milestones, and timing
and outcome of its upcoming clinical and manufacturing milestones
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 actual
funds and/or runway required for our clinical and product
development activities and anticipated upcoming milestones; actual
costs related to our clinical and product development activities
and our need to access additional capital resources prior to
achieving any upcoming milestones; 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 August 12, 2021, 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, except as required
by law.
Contacts:
Media
Josephine Belluardo, Ph.D. LifeSci Communications(646)
751-4361jo@lifescicomms.cominfo@siogtx.com
Investors and Analysts
Parag V. MeswaniSio Gene Therapies Inc.Chief Commercial
Officerinvestors@siogtx.com
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