Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that abstracts detailing
new interim results from its ABO-102 Phase 1/2 Transpher A study
for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB
have been accepted for platform oral presentations during the
late-breaking abstract session at the 17th Annual WORLDSymposium™
being held February 8-12, 2021.
“Children born with MPS IIIA and MPS IIIB
experience progressive neurodevelopmental decline and loss of motor
function that is life-threatening,” said Michael Amoroso, Chief
Operating Officer of Abeona. “We are excited to share new analyses
from the Transpher A study that will add to the understanding of
the potential for ABO-102 to help preserve neurocognitive
development in patients with MPS IIIA when they are treated at a
young age, and new results from the Transpher B study that will
provide insights into ABO-101’s biologic effect in patients with
MPS IIIB.”
Presentation Details
Title: Updated Results of
Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial
of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A
(Mucopolysaccharidosis IIIA)Abstract Number:
390Presenter: Kevin Flanigan, M.D., Center for
Gene Therapy at Nationwide Children’s
HospitalDate/Time: Friday, February 12, 2021, time
to be determined
Title: Updated Results of
Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial
of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B
(Mucopolysaccharidosis IIIB)Abstract Number:
407Presenter: Maria Jose de Castro, M.D., Hospital
Clínico Universitario Santiago de
CompostelaDate/Time: Friday, February 12, 2021,
time to be determined
About the Annual
WORLDSymposium™ The
WORLDSymposium™ is designed for basic, translational and clinical
researchers, patient advocacy groups, clinicians, and all others
who are interested in learning more about the latest discoveries
related to lysosomal diseases and the clinical investigation of
these advances. For additional information on the 17th Annual
WORLDSymposium™, please visit https://worldsymposia.org/.
About the Transpher A Study The
Transpher A Study (NCT02716246) is an ongoing, two-year,
open-label, dose-escalation, Phase 1/2 global clinical trial
assessing ABO-102 for the treatment of patients with Sanfilippo
syndrome type A (MPS IIIA). The study, also known as ABT-001, is
intended for patients from birth to 2 years of age, or patients
older than 2 years with a cognitive developmental quotient of 60%
or above. ABO-102 gene therapy is delivered using AAV9 technology
via a single-dose intravenous infusion. The study primary endpoints
are neurodevelopment changes and safety, with secondary endpoints
including behavior evaluations, quality of life, enzyme activity in
cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in
CSF, plasma and urine, and brain and liver volume.
About the Transpher B Study The
Transpher B Study (NCT03315182) is an ongoing, two-year,
open-label, dose-escalation, Phase 1/2 global clinical trial
assessing ABO-101 for the treatment of patients with Sanfilippo
syndrome type B (MPS IIIB). The study, also known as ABT-002, is
intended for patients from birth to 2 years of age, or patients
older than 2 years with a cognitive developmental quotient of 60%
or above. ABO-101 gene therapy is delivered using AAV9 technology
via a single-dose intravenous infusion. The study primary endpoints
are neurodevelopment changes and safety, with secondary endpoints
including behavior evaluations, quality of life, enzyme activity in
cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in
CSF, plasma and urine, and brain and liver volume.
About ABO-102 ABO-102 is a
novel gene therapy in Phase 1/2 development for Sanfilippo syndrome
type A (MPS IIIA), a rare lysosomal storage disease with no
approved treatment that primarily affects the central nervous
system (CNS). ABO-102 is dosed in a one-time intravenous infusion
using a self-complementary AAV9 vector to deliver a functional copy
of the SGSH gene to cells of the CNS and peripheral organs. The
therapy is designed to address the underlying SGSH enzyme
deficiency responsible for abnormal accumulation of
glycosaminoglycans in the brain and throughout the body that
results in progressive cell damage and neurodevelopmental and
physical decline. In the U.S., Abeona holds Regenerative Medicine
Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan
Drug designations for the ABO-102 clinical program. In the EU, the
Company holds PRIME and Orphan medicinal product designations.
About ABO-101 ABO-101 is a
novel gene therapy in Phase 1/2 development for Sanfilippo syndrome
type B (MPS IIIB), a rare lysosomal storage disease with no
approved therapy that primarily affects the central nervous system
(CNS). ABO-101 is dosed in a one-time intravenous infusion using a
self-complementary AAV9 vector to deliver a functional copy of the
NAGLU gene to cells of the CNS and peripheral tissues. The therapy
is designed to address the underlying NAGLU enzyme deficiency
responsible for abnormal accumulation of glycosaminoglycans in the
brain and throughout the body that results in progressive cell
damage and neurodevelopmental and physical decline. In the U.S.,
Abeona holds Fast Track and Rare Pediatric Disease designations for
ABO-101 and Orphan Drug designation in both the U.S. and EU.
About Sanfilippo Syndrome Type A (MPS
IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare,
fatal lysosomal storage disease with no approved treatment that
primarily affects the CNS and is characterized by rapid
neurodevelopmental and physical decline. Children with MPS IIIA
present with progressive language and cognitive decline and
behavioral abnormalities. Other symptoms include sleep problems and
frequent ear infections. Additionally, distinctive facial features
with thick eyebrows or a unibrow, full lips and excessive body hair
for one’s age, and liver/spleen enlargement are also present in
early childhood. MPS IIIA is caused by genetic mutations that lead
to a deficiency in the SGSH enzyme responsible for breaking down
glycosaminoglycans, which accumulate in cells throughout the body
resulting in rapid health decline associated with the disorder.
About Sanfilippo syndrome type B (MPS
IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and
fatal lysosomal storage disease with no approved therapy that
primarily affects the central nervous system and is characterized
by rapid neurodevelopmental and physical decline. Children with MPS
IIIB present with progressive language and cognitive decline and
behavioral abnormalities. Other symptoms include sleep problems and
frequent ear infections. Additionally, distinctive signs such as
facial features with thick eyebrows or a unibrow, full lips and
excessive body hair for one’s age and liver/spleen enlargement are
also present. The underlying cause of MPS IIIB is a deficiency in
the NAGLU enzyme responsible for breaking down glycosaminoglycans,
which accumulate throughout the body resulting in rapid decline
associated with the disorder.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing gene and cell therapies for serious diseases.
Abeona’s clinical programs include EB-101, its autologous,
gene-corrected cell therapy for recessive dystrophic epidermolysis
bullosa in Phase 3 development, as well as ABO-102 and ABO-101,
novel AAV-based gene therapies for Sanfilippo syndrome types A and
B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development.
The Company’s portfolio also features AAV-based gene therapies for
ophthalmic diseases with high unmet medical needs. Abeona’s novel,
next-generation AIM™ capsids have shown potential to improve
tropism profiles for a variety of devastating diseases. Abeona’s
fully functional, gene and cell therapy GMP manufacturing facility
produces EB-101 for the pivotal Phase 3 VIITAL™ study and is
capable of clinical and commercial production of AAV-based gene
therapies. For more information, visit
www.abeonatherapeutics.com.
Forward-Looking StatementsThis
press release contains certain statements that are forward-looking
within the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended, and that involve risks and uncertainties. These statements
include statements about the Company exploring all strategic
options, including the sale of some or all of its assets or sale of
the Company. We have attempted to identify forward-looking
statements by such terminology as “may,” “will,” “believe,”
“estimate,” “expect,” and similar expressions (as well as other
words or expressions referencing future events, conditions or
circumstances), which constitute and are intended to identify
forward-looking statements. Actual results may differ materially
from those indicated by such forward-looking statements as a result
of various important factors, numerous risks and uncertainties,
including but not limited to the potential impacts of the COVID-19
pandemic on our business, operations, and financial condition, the
outcome of the strategic review, continued interest in our rare
disease portfolio, our ability to enroll patients in clinical
trials, the outcome of any future meetings with the U.S. Food and
Drug Administration or other regulatory agencies, the impact of
competition, the ability to secure licenses for any technology that
may be necessary to commercialize our products, the ability to
achieve or obtain necessary regulatory approvals, the impact of
changes in the financial markets and global economic conditions,
risks associated with data analysis and reporting, and other risks
disclosed in the Company’s most recent Annual Report on Form 10-K
and subsequent quarterly reports on Form 10-Q and other periodic
reports filed with the Securities and Exchange Commission. The
Company undertakes no obligation to revise the forward-looking
statements or to update them to reflect events or circumstances
occurring after the date of this press release, whether as a result
of new information, future developments or otherwise, except as
required by the federal securities laws.
Investor and Media Contact:
Greg Gin
VP, Investor Relations
Abeona Therapeutics
+1 (646) 813-4709
ggin@abeonatherapeutics.com
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