Abeona Therapeutics Announces FDA Clearance of Investigational New Drug Application for ABO-202 Gene Therapy in CLN1 Disease
May 21 2019 - 8:00AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that the Company is
cleared to begin a Phase 1/2 clinical trial evaluating its novel,
one-time gene therapy ABO-202 for the treatment of CLN1 disease,
following acceptance of its Investigational New Drug (IND)
application by the U.S. Food and Drug Administration (FDA). ABO-202
is designed to deliver a functional copy of the PPT1 gene to cells
of the central nervous system and peripheral organs using a
combined intravenous and intrathecal delivery via the AAV9 vector.
The Company will provide guidance on the timing of the clinical
trial later this year.
“This significant step brings hope to people
impacted by this devastating disease and was achieved in
partnership with Dr. Steven Gray and Taylor’s Tale,” said Timothy
J. Miller, Ph.D., Co-Founder, President & Chief Scientific
Officer. “We are very encouraged that ABO-202 was well-tolerated
and demonstrated significant efficacy in preclinical studies. These
results are consistent with findings in our other pre-clinical
studies for AAV9-based programs for lysosomal storages diseases,
MPS IIIA and MPS IIIB.”
In preclinical studies, ABO-202 effectively
delivered a functional copy of the PPT1 gene to the central nervous
system and peripheral organs. IND-enabling studies in a CLN1 animal
model demonstrated that ABO-202 normalized survival and led to
improvement of neurological function in affected mice. These
studies also showed that combination intravenous and intrathecal
dosing provided incremental efficacy over either delivery route
alone and thus may enhance the therapeutic potential of this gene
therapy.
ABO-202 is a one-time AAV gene therapy designed
to enable cells to produce the normal PPT1 enzyme, which is
critical for proper lysosomal function. Lack of this enzyme in
patients with CLN1 disease results in neuroinflammation and
neurodegeneration. ABO-202 has been granted Orphan Drug and Rare
Pediatric Disease designations by the FDA and has received Orphan
Medicinal Product designation in the EU. The global trial will be
conducted at centers of excellence in CLN1 research, including the
University of Rochester Medical Center in the U.S. and the
University of Hamburg-Eppendorf in Germany.
“ABO-202 is a promising AAV9 gene therapy that
extended survival and improved neurological function in the animal
model of CLN1 disease. Importantly, the combined intravenous and
intrathecal administration approach showed additional benefits
compared to a single route of delivery, providing a new treatment
paradigm for patients with devastating neurological diseases,” said
Steven J. Gray, Ph.D., Batten disease researcher and Associate
Professor, Pediatrics, UT Southwestern Medical Center.
About CLN1 disease (Infantile Batten
disease)CLN1 disease, also known as Infantile Neuronal
Ceroid Lipofuscinosis or infantile Batten disease, is a
rapidly-progressing rare lysosomal storage disease with no approved
treatment. It primarily affects the central nervous system and
typically manifests during the first year of life with vision
impairment that can progress to blindness, progressive motor and
cognitive decline, seizures and ultimately early death. The
underlying cause of the disorder is a defect in the PPT1 gene that
encodes the enzyme of the same name, resulting in the accumulation
of lipopigments within cells, leading to neuroinflammation and
neurodegeneration. Some patients with CLN1 disease develop symptoms
later in childhood or in adulthood; these variants are called
late-infantile, juvenile, or adult-onset CLN1.
About ABO-202ABO-202 is a novel, one-time gene
therapy for CLN1 disease, a rapidly-progressing rare lysosomal
storage disease with no approved therapy. ABO-202 is dosed through
intravenous and intrathecal infusions using the AAV9 vector to
deliver a functional copy of the PPT1 gene to cells of the central
nervous system and peripheral organs. Preclinical data demonstrated
that combination intravenous and intrathecal dosing may enhance the
therapeutic potential of ABO-202. The therapy is designed to
address the underlying gene and enzyme deficiency that leads to
abnormal buildup of lipopigments and results in neuroinflammation
and neurodegeneration.
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 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
therapeutic potential for ABO-202 in the treatment of CLN1,
including the ability to effectively treat CLN1 disease in human
patients, 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
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