Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM2 Gene Therapy in Patients with GM2 Gangliosidosis
01 Novembre 2021 - 12:00PM
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-GM2, an investigational gene therapy
for the treatment of early infantile, late infantile, and
juvenile-onset Tay-Sachs and Sandhoff disease. The Fast Track
designation is intended to facilitate the development and review of
drugs to treat serious conditions and fill an unmet medical need.
“The FDA’s decision to grant AXO-AAV-GM2 gene therapy Fast Track
designation signifies an important milestone towards developing a
safe and effective treatment for Tay-Sachs and Sandhoff diseases,
both rare and fatal pediatric diseases with no approved
treatments,” said Pavan Cheruvu, M.D., Chief Executive Officer of
Sio Gene Therapies. “This designation complements the previously
granted Rare Pediatric Disease and Orphan Drug designations for
AXO-AAV-GM2, along with similar designations for AXO-AAV-GM1 for
GM1 gangliosidosis, and we look forward to working closely with the
FDA as we continue enrollment in our ongoing, registration-enabling
trial in Tay-Sachs and Sandhoff diseases to bring AXO-AAV-GM2 to
patients and families.”
The current 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 (also known as Tay-Sachs or Sandhoff disease).
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. Currently, there are no
FDA-approved treatment options for GM2 gangliosidosis.
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.
AXO-AAV-GM2 has received Orphan Drug Designation, Rare Pediatric
Disease Designation and Fast Track Designation from the Food
and Drug Administration and is the only gene therapy in
clinical development for all pediatric forms of GM2
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.cominfo@siogtx.com
Investors and Analysts
Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief
Commercial Officerinvestors@siogtx.com
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