Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangl...
02 Dezembro 2020 - 9:00AM
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 has been dosed in the high-dose cohort of
the Phase 1/2 (“Stage 1”) study for Type I (infantile) and Type II
(late infantile and juvenile onset) GM1 gangliosidosis.
“AXO-AAV-GM1 is the only gene therapy in the clinic targeting
patients with Type I and Type II GM1 gangliosidosis, a devastating
and fatal pediatric disease,” said Gavin Corcoran, M.D., Chief
R&D Officer of Sio. “The initiation of the high-dose cohort
builds on evidence of extension of survival in naturally-occurring
GM1 disease animal models and encouraging clinical data from an
expanded access study conducted by a National Human Genome Research
Institute (NHGRI) team led by our principal investigator, Dr.
Cynthia Tifft at the National Institutes of Health’s (NIH) Clinical
Center. Our team and academic partners are dedicated to improving
the lives of children affected by this devastating disease, and we
look forward to reporting topline data from the low-dose cohort
before year end.”
The Phase 1/2 study (NCT03952637) is designed to evaluate the
safety, tolerability, and potential efficacy of AXO-AAV-GM1
delivered intravenously in patients with Type I and Type II GM1
gangliosidosis.
- The low-dose cohort evaluated 1.5x1013 vg/kg AXO-AAV-GM1 gene
therapy in a total of five Type II (juvenile) patients. Six-month
follow-up data from the low-dose cohort are expected by the end of
2020.
- The high-dose cohort is evaluating a dose of 4.5x1013 vg/kg
AXO-AAV-GM1 gene therapy.
AXO-AAV-GM1 has received both Orphan Drug Designation and Rare
Pediatric Disease Designation and is the only gene therapy in
clinical development for both Type I and Type II GM1
gangliosidosis.
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 approved treatment options for GM1
gangliosidosis. In 2019, Sio reported clinically meaningful
improvements from baseline to six-month follow-up for the first GM1
Type II child dosed with low-dose AXO-AAV-GM1 gene therapy under an
expanded access protocol.
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.
About Sio Gene Therapies
Sio 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 “will,” “expect,” “believe,” “estimate,”
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.cominfo@siogtx.com
Investors and Analysts
David NassifSio Gene Therapies Inc.Chief Financial Officer and
General Counsel(646) 677-6770investors@siogtx.com
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