Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM1 (AAV9-GLB1) Gene Therapy in Patients with GM1 Gangliosidosis
October 21 2021 - 8:00AM
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 U.S. Food and Drug Administration (FDA) has granted Fast
Track Designation to AXO-AAV-GM1, its adeno-associated viral vector
(AAV)9-based gene therapy candidate for the treatment of Type I
(early infantile-onset) and Type II (late infantile-onset and
juvenile-onset) GM1 gangliosidosis. The Fast Track process is
designed to facilitate the development and expedite the review of
drugs to treat serious conditions and fill an unmet medical need.
“Receiving Fast Track Designation is a critical step in our
mission to develop the first potential treatment for all pediatric
forms of this rare, terminal disease. This designation joins both
the Orphan Drug Designation and Rare Pediatric Disease Designation
assigned to AXO-AAV-GM1 by the FDA, which we believe further
demonstrates the potential impact of this work on the patient
community,” said Pavan Cheruvu, M.D., Chief Executive Officer of
Sio Gene Therapies. “Building on the recently presented data at
ESGCT demonstrating normalization of key disease biomarkers in the
high-dose cohort with no serious adverse events attributed to
AXO-AAV-GM1, this designation will help us accelerate clinical
development of this promising investigational therapy for children
and families.”
The current Phase 1/2 study (NCT03952637) is designed to
evaluate the safety, tolerability, and potential efficacy of
AXO-AAV-GM1 gene therapy delivered intravenously in children with
early infantile, or Type I, and late infantile and juvenile, or
Type II, GM1 gangliosidosis. Stage 1 of the study 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. Stage 2 of the trial will then evaluate the
efficacy and safety of the optimal dose identified in Stage 1.
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 achieve a broad central
and peripheral biodistribution. Preclinical studies in murine and a
naturally-occurring feline model of GM1 gangliosidosis have
supported AXO-AAV-GM1’s ability to increase 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 FDA and is the
only gene therapy in clinical development for all pediatric forms
of GM1 gangliosidosis.
In 2018, Sio licensed exclusive worldwide rights from UMass Chan
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 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.
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 occurrence of adverse safety events
during our current and future trials; the development of a
suspension-based manufacturing process for AXO-Lenti-PD; the
scaling up of manufacturing; the outcome of interactions with
regulatory agencies and 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.com info@siogtx.com
Investors and Analysts
Parag V. MeswaniSio Gene Therapies Inc.Chief Commercial
Officerinvestors@siogtx.com
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