Full Press Release Details
Positive Phase 1/2 Interim Data Presented at WORLDSymposium Shows Neurocognitive Development of Young MPS IIIA Patients
Preserved up to Three Years Following Treatment with Abeona's ABO-102 Gene Therapy
addition to preservation of neurocognitive development with ABO-102 in MPS IIIA, new clinical results of ABO-102 in MPS IIIA and
ABO-101 in MPS IIIB continue to show dose-dependent and sustained reductions in disease-specific biomarkers, denoting clear biologic
addition, ABO-102 and ABO-101 continue to show favorable safety profile in ongoing studies
to host investor webinar on Tuesday, February 16, 2021 at 1:00 p.m. EST
YORK and CLEVELAND, Feb. 12, 2021 - Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell
therapy, today announced new positive data from two ongoing Phase 1/2 clinical trials of the company's investigational AAV-based
gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The interim data was presented in late-breaking platform
oral presentations at the 17th Annual WORLDSymposium . The presentation slides are available on the company's
website at www.abeonatherapeutics.com.
Amoroso, Principal Executive and Chief Operating Officer of Abeona, stated, "We are excited to share updated positive efficacy
and safety results that continue to suggest ABO-102 has the potential to be a life-altering treatment option for children with
MPS IIIA, a rare, debilitating condition with no approved treatment that leads to progressive neurodevelopmental and physical
decline, and often results in death early in life. We have requested a meeting with the FDA later this quarter to discuss the
ABO-102 data and the potential path towards a Biologics License Application filing for ABO-102. In addition, the new results from
the Transpher B study continue to support ABO-101's biologic activity in patients with MPS IIIB."
updated results from the Transpher A study evaluating ABO-102 in Sanfilippo syndrome type A (MPS IIIA) demonstrated that neurocognitive
development was preserved within normal range of a non-afflicted child for 2.5 years to 3 years (the latest time point measured)
after treatment with ABO-102 (3x1013 vg/kg) in three young patients in the high-dose cohort 3. The three young patients
were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages ranging from 3.5 years to 5+ years,
the timepoint at which patients with MPS IIIA have already started to experience neurocognitive decline based on the natural history
of disease progression. Dose-dependent and statistically significant reductions in cerebrospinal fluid heparan sulfate, denoting
enzyme activity in the central nervous system (CNS), and liver volume were sustained for two years after treatment. ABO-102 has
been well-tolerated with long-term safety remaining favorable 24-55 months following treatment. There have been no treatment-related
severe adverse events and no clinically significant adverse events reported.
Flanigan, M.D., Director, Center for Gene Therapy at AWRI at Nationwide Children's and Transpher A study principal investigator,
said, "The results presented today show a single intravenous dose of ABO-102 can help preserve neurocognitive development
for up to 3 years following treatment in young MPS IIIA patients during early stages of their disease. The data shows ABO-102's
ability to deliver a functional copy of the disease-causing SGSH gene to cells of the CNS and peripheral organs, as evidenced
by the clinical benefits in neurocognition and biophysical measures and improvements in disease-specific biomarkers."
from the Transpher B study evaluating ABO-101 in Sanfilippo syndrome type B (MPS IIIB) showed that treatment with ABO-101 is associated
with a dose-dependent and sustained improvement in central nervous system and systemic biomarkers, indicating the potent biologic
effect of ABO-101 in patients with MPS IIIB. ABO-101 has been well-tolerated with no infusion related or early acute reactions
and no clinically significant adverse events or laboratory abnormalities.
Jose de Castro, M.D., Hospital Cl nico Universitario Santiago de Compostela and Transpher B study investigator, said, "The
results from the Transpher B study provide evidence of ABO-101's impact on disease biomarkers and potential to break down
the accumulation of glycosaminoglycans that underlie MPS IIIB pathology. We look forward to continued follow-up to assess ABO-101's
potential to preserve neurocognitive development in patients with MPS IIIB."
Webinar on MPS III Gene Therapy Programs
management along with Dr. Flanigan and Dr. de Castro will host an investor webinar on February 16, 2021 at 1:00 p.m. EST. The
live webinar, including audio and presentation slides, will be accessible at https://investors.abeonatherapeutics.com/events
at the time of the meeting. To register in advance for the live webinar, click here.
archived replay of the webinar will be available after the conclusion of the live event at https://investors.abeonatherapeutics.com/events.
the Annual WORLDSymposium
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/.
the Transpher A Study
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 6 months 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 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.
the Transpher B Study
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 6 months 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 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.
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.
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.
Sanfilippo Syndrome Type A (MPS IIIA)
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.
Sanfilippo syndrome type B (MPS IIIB)
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
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.
press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of