Abeona Therapeutics Provides Regulatory Update Ahead of Pivotal Phase 3 Clinical Trial for EB-101 in Recessive Dystrophic Epi...
September 23 2019 - 8:45AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that it has recently
received a clinical hold letter from the U.S. Food and Drug
Administration (FDA) clarifying that the FDA will not provide
approval for the Company to begin its planned Phase 3 clinical
trial for EB-101 until it submits to the FDA additional data points
on transport stability of EB-101 to clinical sites. Over the last
12 months, the Company has worked closely with the FDA to address
and narrow open Chemical, Manufacturing and Controls (CMC) items
and has been working to resolve this one item identified in the FDA
Clinical Hold Letter. The Company continues to anticipate receiving
CMC clearance for VIITAL™ trial in Q4 2019.
“Initiating the VIITAL™ pivotal Phase 3 trial
for EB-101 is the Company’s top priority,” said João Siffert, M.D.,
Chief Executive Officer of Abeona. “Efforts to gather supplemental
data points on transport stability of EB-101 are ongoing and we are
confident that the requested additional data will be submitted to
the FDA promptly. Looking ahead, we believe that completion of our
CMC work coupled with the durable safety and efficacy data, now out
to five years in some patients, will ultimately be critical to
support a future Biologics License Application. We remain deeply
committed to advancing EB-101 to provide a desperately needed
treatment for RDEB patients.”
The Company will hold a conference call
scheduled for Monday, September 23 at 09:30 a.m. ET. Interested
parties are invited to participate in the call by dialing
844-369-8770 (toll-free domestic) or 862-298-0840 (international)
or via webcast at
https://www.investornetwork.com/event/presentation/53728.
About EB-101EB-101 is an
investigational, autologous, gene-corrected cell therapy poised to
enter late-stage development for the treatment of recessive
dystrophic epidermolysis bullosa (RDEB), a rare connective tissue
disorder without an approved therapy. Treatment with EB-101
involves using gene transfer to deliver COL7A1 genes into a
patient’s own skin cells (keratinocytes) and transplanting them
back to the patient to enable normal Type VII collagen expression
and facilitate wound healing. In the U.S., Abeona holds
Regenerative Medicine Advanced Therapy, Breakthrough Therapy, and
Rare Pediatric designations for EB-101 and Orphan Drug designation
in both the U.S. and EU.
About Recessive Dystrophic Epidermolysis
BullosaRecessive dystrophic epidermolysis bullosa, or
RDEB, is a rare connective tissue disorder characterized by severe
skin wounds that cause pain and can lead to systemic complications
impacting the length and quality of life. People with RDEB have a
defect in the COL7A1 gene, leaving them unable to produce
functioning Type VII collagen which is necessary to anchor the
dermal and epidermal layers of the skin. There is currently no
approved treatment for RDEB.
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 about the timing for CMC clearance
for the VIITAL™ trial and the Company’s beliefs relating thereto,
the Company’s ability to provide additional transport stability
data points in response to the FDA clinical hold letter and the
timing thereof, the Company’s belief that completion of its CMC
work and the durable safety and efficacy data will ultimately be
critical to support a future Biologics License Application, the
ability of its management team to lead the Company and deliver on
key strategies, the market opportunities for the Company’s products
and product candidates, 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
(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 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 risk of whether or when the FDA will lift the
clinical hold respecting the Company’s planned Phase 3 clinical
trial for EB-101, 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 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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