Abeona Therapeutics Announces Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission
30 August 2023 - 9:30PM
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the
Company’s intention to proceed with the submission of a Biologics
License Application (BLA) to the U.S. Food and Drug Administration
(FDA) for EB-101, its investigational autologous, engineered cell
therapy, for patients with recessive dystrophic epidermolysis
bullosa (RDEB) after the recent completion of a pre-BLA meeting
with the FDA.
At the meeting, Abeona reached alignment with
the FDA that the EB-101 clinical efficacy and safety data appear
adequate to support a BLA submission. The Agency also agreed that
retroviral vector manufactured at Abeona and Indiana University
appear comparable based on the data that Abeona provided in its
briefing book. The FDA requested that Abeona include within its BLA
submission additional background and data supporting the scientific
rationale underlying its EB-101 potency and identity assays so that
they can be fully evaluated by the Agency post-submission. The
Agency also requested that supplemental data pertaining to certain
chemistry, manufacturing, and controls and clinical topics be
included in the BLA package. Abeona believes that it has the
necessary supporting data in-hand to generate these additional
reports, including those regarding potency and identity, to address
the Agency’s requests.
“We are pleased with the outcome of the pre-BLA
meeting for EB-101 and believe that we have aligned with the FDA on
what is needed for our upcoming BLA submission,” said Vish
Seshadri, Chief Executive Officer of Abeona. “We are focused on
gathering and packaging the existing data over the coming weeks to
meet the Agency’s expectations. With the constructive feedback from
the FDA now in-hand, we are proceeding on a clear regulatory path
leading to the planned BLA submission for EB-101 early this
Fall.”
EB-101 has been granted Regenerative Medicine
Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare
Pediatric Disease designations by the FDA.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing cell and gene therapies for serious diseases.
Abeona’s lead clinical program is EB-101, its investigational
autologous, engineered cell therapy currently in development for
recessive dystrophic epidermolysis bullosa. The Company’s
development portfolio also features AAV-based gene therapies for
ophthalmic diseases with high unmet medical need. Abeona’s novel,
next-generation AAV capsids are being evaluated to improve tropism
profiles for a variety of devastating diseases. Abeona’s fully
integrated cell and gene therapy cGMP manufacturing facility
produced EB-101 for the pivotal Phase 3 VIITAL™ study and is
capable of clinical and potential commercial production of
AAV-based gene therapies. 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. We have
attempted to identify forward-looking statements by such
terminology as “may,” “will,” “believe,” “anticipate,” “expect,”
“intend,” 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, the timing and outcome of our
Biologics License Application submission to the FDA for EB-101;
continued interest in our rare disease portfolio; our ability to
enroll patients in clinical trials; the outcome of future meetings
with the FDA or other regulatory agencies, including those relating
to preclinical programs; the ability to achieve or obtain necessary
regulatory approvals; the impact of any changes in the financial
markets and global economic conditions; risks associated with data
analysis and reporting; and other risks disclosed in the Company’s
most recent Annual Report on Form 10-K and subsequent periodic
reports filed 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 press release, whether as a result
of new information, future developments or otherwise, except as
required by the federal securities laws.
Investor and Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
ir@abeonatherapeutics.com
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