Abeona Therapeutics Announces Positive Interim Data from the ABO-102 Phase 1/2 Gene Therapy Clinical Trial in MPS IIIA
July 25 2019 - 8:15AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced positive data from its
ongoing Phase 1/2 clinical trial evaluating ABO-102, the Company’s
investigational one-time, adeno-associated virus 9 (AAV9) gene
therapy for Sanfilippo syndrome type A (MPS IIIA). These new
results showed that treatment of the youngest patients with
ABO-102, all enrolled in the high-dose cohort 3, resulted in
preservation of neurocognitive development 12-18 months post
treatment. Robust and sustained improvement observed in biomarkers
confers additional evidence of a clear biological effect following
ABO-102 administration. In addition, longer-term safety remained
favorable eight months to two years after treatment. Abeona is
enrolling eligible patients at sites in the U.S., Spain, and
Australia. Additional information about the trial, also known as
The Transpher A Study (ABT-001), is available at AbeonaTrials.com
and ClinicalTrials.gov (NCT02716246).
“We are very encouraged by these most recent
results supporting the potential of a single intravenous
administration of ABO-102 to treat children with MPS IIIA, a
devastating lysosomal storage disease without an approved therapy.
These data showed that treating younger children during early
stages of their disease confers the greatest chance of
neurocognitive benefits,” said João Siffert, M.D., Chief Executive
Officer. “The longer-term data continue to demonstrate that ABO-102
has a clear biological effect and a favorable safety profile.”
Summary of Interim Data
- The three youngest patients enrolled in cohort 3 – ages 26
months, 19 months, and 14 months at dosing – continued to track
within normal age equivalent development 12-18 months post
treatment.
- Dose dependent and sustained reductions in CSF heparan sulfate
were observed in all three cohorts; levels reached lower limit of
quantitation in all eight patients treated with higher ABO-102 dose
in cohort 3.
- No product-related serious adverse events were reported to
date.
As part of the ABO-102 program’s Regenerative
Medicine Advanced Therapy (RMAT) designation, Abeona is pursuing a
meeting with the U.S. Food and Drug Administration in the second
half of 2019 to assess the next steps in the program’s development
pathway. About ABO-102ABO-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 an 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, and Rare Pediatric Disease
designations for ABO-102 and Orphan Drug designation in both the
U.S. and EU.
About The Transpher A StudyThe
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. The study has enrolled 14 patients to date across three
dose-escalating cohorts (N=3, N=3, N=8) and remains open for
enrollment. The gene therapy ABO-102 is delivered using AAV9
technology via a one-time 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.
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 central nervous system 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 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 IIIA is a deficiency in
the SGSH 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.
The Company’s clinical programs include EB-101, its autologous,
gene-corrected cell therapy for recessive dystrophic epidermolysis
bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene
therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS
IIIB), respectively. The Company’s portfolio of AAV9-based gene
therapies also features ABO-202 and ABO-201 for CLN1 disease and
CLN3 disease, respectively. Its preclinical assets include ABO-401,
which uses the novel AIM™ AAV vector platform to address all
mutations of cystic fibrosis. Abeona has received numerous
regulatory designations from the FDA and EMA for its pipeline
candidates and is the only company with Regenerative Medicine
Advanced Therapy designation for two candidates (EB-101 and
ABO-102). For more information, visit
www.abeonatherapeutics.com.
Forward-Looking Statements This
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’s clinical trials
and its products and product candidates, future regulatory
interactions with regulatory authorities, as well as the Company’s
goals and objectives. 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 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
as may be detailed from time to time in the Company’s Annual
Reports on Form 10-K and quarterly reports on Form 10-Q and other
periodic reports filed by the Company 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
presentation, whether as a result of new information, future
developments or otherwise, except as required by the federal
securities laws.
Investor Contact:Sofia Warner Senior Director,
Investor Relations Abeona Therapeutics +1 (646) 813-4710
swarner@abeonatherapeutics.com
Media Contact:Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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