Abeona Therapeutics Receives FDA Fast Track Designation for ABO-202 AAV9 Gene Therapy in CLN1 Disease
June 18 2019 - 8:29AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that the U.S. Food and
Drug Administration (FDA) has granted Fast Track designation to its
ABO-202 program. ABO-202, the Company’s novel one-time AAV9 gene
therapy for CLN1 disease, is designed to deliver a functional copy
of the PPT1 gene to the central nervous system and peripheral
organs using a combination of intravenous and intrathecal
administrations. Abeona is preparing to initiate a Phase 1/2
clinical trial evaluating ABO-202 in patients with CLN1 disease and
will provide guidance on the timing of the trial later this year.
“Receiving Fast Track designation acknowledges
the urgency for developing a therapy for children suffering from
this rapidly-progressing and fatal disease and highlights the
significant potential of ABO-202 to address this unmet need,” said
João Siffert, M.D., Chief Executive Officer.
ABO-202 is administered as a one-time
adeno-associated virus 9 (AAV9) gene therapy that delivers a
functional copy of the PPT1 gene to cells of the central nervous
system and peripheral organs. This enables cells to produce a
functioning PPT1 enzyme, which is critical for proper metabolism in
lysosomes. The absence of this enzyme in patients with CLN1
disease results in malfunctioning cells, including brain cells,
neuroinflammation, and neurodegeneration. In preclinical studies,
ABO-202 normalized survival and improved neurological function in
CLN1 mice. These studies also showed that a combination of
intravenous and intrathecal administrations of ABO-202 improved
efficacy over either delivery route alone, and that early treatment
significantly improved outcomes.
Fast Track designation, granted by the FDA, is a
process designed to facilitate the development and expedite the
review of drugs to treat serious conditions and fill an unmet
medical need. The purpose is to get important new drugs to the
patient earlier through more frequent interactions with FDA,
potential eligibility for accelerated approval, priority regulatory
review, and rolling BLA review. ABO-202 has also received
Orphan Drug designations in the U.S. and EU, and Rare Pediatric
Disease designation from the FDA.
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 progresses to blindness, motor and cognitive
decline, seizures and ultimately early death. The underlying cause
of the disorder is mutations in the PPT1 gene that encodes the
enzyme of the same name, resulting in lysosome dysfunction that
leads to cellular dysfunction, 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 disease.
About ABO-202ABO-202 is a
novel, one-time AAV9 gene therapy for patients with CLN1 disease, a
rapidly-progressing rare lysosomal storage disease with no approved
therapy. ABO-202 is administered through intravenous and
intrathecal infusions using an AAV9 vector to deliver a functional
copy of the PPT1 gene to cells of the central nervous system and
peripheral organs. In preclinical studies, ABO-202 normalized
survival and improved neurological function in CLN1 mice. These
studies also showed that a combination of intravenous and
intrathecal administrations of ABO-202 improved efficacy over
either delivery route alone, and that early treatment significantly
improved outcomes.
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 Statement 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 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) 813-4707
swarner@abeonatherapeutics.com
Media Contact:Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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