Abeona Therapeutics Treats First Patient in Second Cohort of Phase 1/2 Clinical Trial for ABO-101 in Sanfilippo Syndrome Type...
May 14 2019 - 8:00AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that the Company has
completed dosing cohort 1 and treated the first patient in cohort 2
in an ongoing Phase 1/2 clinical trial evaluating ABO-101, its
novel one-time gene therapy for Sanfilippo syndrome type B (MPS
IIIB). The Company expects to report interim data from the trial in
the second half of 2019.
“We believe that ABO-101 can help address the
underlying lysosomal enzyme deficiency that ultimately results in
progressive cell damage and neurodevelopmental and physical decline
in children with MPS IIIB,” said João Siffert, M.D., Chief
Executive Officer. “The product safety profile to date and clear
evidence of biological activity provide support for advancing this
clinical trial to the second cohort. This multicenter study is made
possible by our close collaborations with expert academic
investigators and the participants who enrolled to date. We look
forward to presenting interim data in the second half of this
year.”
The Phase 1/2 trial is a two-year, open-label,
dose-escalation, global clinical trial in patients diagnosed with
MPS IIIB who are older than six months of age and have a minimum
cognitive Development Quotient of 60 or above. The study is
expected to include up to nine patients and is designed to evaluate
two doses of ABO-101 with cohort 1 receiving 2 X 1013 vg/kg and
cohort 2 receiving 5 X 1013 vg/kg. ABO-101 is delivered using
NAV® AAV9 technology via a one-time intravenous infusion. The
primary endpoint of the study is to assess neurodevelopment and
safety, with multiple secondary and exploratory endpoints including
neurocognitive and behavior evaluations, quality of life, enzyme
activity in cerebrospinal fluid (CSF) and plasma, biomarkers in
CSF, plasma and urine, and brain and liver volume.
Abeona is enrolling eligible patients with MPS
IIIB at sites in the U.S. and Spain and expects to activate
additional sites globally this year to accelerate enrollment.
Additional information about the study is available at
AbeonaTrials.com and ClinicalTrials.gov (NCT03315182).
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 ABO-101ABO-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 the NAV® 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 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 NAV® 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 StatementThis
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 regarding our pipeline including the
potential for ABO-101 in the treatment of Sanfilippo syndrome type
B, including the results of clinical studies, the ability to
present interim data, the ability to continue to enroll patients in
clinical studies, the ability to obtain regulatory marketing
approvals, and the company’s goals and objectives. We have
attempted to identify forward looking statements by such
terminology as “may,” will,” “anticipate,” “believe,” “estimate,”
“expect,” “intend,” and similar expressions.
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 initiate and enroll patients in clinical trials, 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 reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligation to revise
the forward-looking statements or 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
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Aug 2024 to Sep 2024
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Sep 2023 to Sep 2024