Abeona Therapeutics Announces Publication of Positive Long-Term Data from Phase 1/2a Clinical Trial Evaluating EB-101 Gene Th...
October 15 2019 - 9:15AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced positive long term
efficacy and safety results from its Phase 1/2a clinical trial
evaluating EB-101, a gene-corrected cell therapy for recessive
dystrophic epidermolysis bullosa (RDEB). Treatment with EB-101
resulted in sustained wound healing with a favorable safety profile
at three years post-treatment. These long-term follow-up data in
seven RDEB participants with 42 treated wounds were published in
the peer-reviewed journal JCI Insight. Full text of the publication
can be accessed here: https://insight.jci.org/articles/view/130554
“These results bolster our belief that EB-101 is
a safe and effective gene-corrected cell therapy capable of
providing durable, long-lasting healing for the most disabling
wounds in patients with RDEB,” said João Siffert, M.D., Chief
Executive Officer of Abeona. “These results are particularly
significant, as EB-101 treatment led to wound healing even in the
most challenging to treat large and painful chronic wounds. Given
the average RDEB chronic wound size is over 118 cm2, it is
essential that potential new treatments are capable of addressing
these wounds to improve quality of life. We thank our collaborators
at Stanford and the patients who volunteered to participate in this
study and look forward to building upon this strong clinical
foundation with the initiation of the pivotal Phase 3 VIITALTM
Study evaluating EB-101 for the treatment of RDEB.”
Key Study findings include:
- Wounds selected for treatment were present for a mean of 11.2
years (range 3-20 years)
- Three years after treatment with EB-101, a majority of RDEB
patients had sustained wound healing, with 80% (16/20) of wounds
achieving ³50% healing, and 70% (14/20) achieving ≥75%
- Two years after treatment, only 1 of 6 untreated (17%),
prospectively selected control wounds, had ≥50% healing
- 50% or greater wound healing was associated with no pain (0/16)
and no itch (0/16) at treated sites three years post-treatment,
compared with presence of pain in 53% (20/38) and itch in 61%
(23/38) of wound sites at baseline
- EB-101 was associated with long-term molecular expression of
type VII collagen protein, which plays an important role in
anchoring the dermal and epidermal layers of the skin
- No serious treatment-related adverse events were observed
during the three-year observation period
- No replication competent virus was present at any time
point
Researchers from Stanford University School of
Medicine conducted the Phase 1/2a single-center, open-label
clinical trial to evaluate the long-term wound healing and safety
of EB-101 in seven adult patients with severe generalized RDEB and
to assess patient-reported outcomes following treatment. Chronic
open wounds, defined as wounds present and unhealed for at least 12
weeks, with a total area of at least 100 cm2, were required for
enrollment. In the trial, gene-corrected EB-101 skin grafts (35 cm2
each) were transplanted onto six wound sites in each of the seven
adult participants (n= 42 sites total) and wounds selected for
treatment had been present for a mean of 11.2 years (range: 3-20
years). Participants were followed for two to five years after
transplantation of EB-101 and received standard of care therapies
including iron supplementation and esophageal dilations during the
study.
Abeona is currently continuing preparations for
the pivotal Phase 3 VIITALTM Study evaluating EB-101 for the
treatment of RDEB pending the anticipated receipt of Chemical,
Manufacturing and Controls (CMC) clearance from the U.S. Food and
Drug Administration expected in Q4 2019.
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 TherapeuticsAbeona
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 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 maybe 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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