Abeona Therapeutics Announces Successful Type B Meeting with FDA for Pivotal Phase 3 VIITAL™ Study of EB-101 in Recessive D...
January 25 2021 - 7:30AM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that the company held a
successful Type B meeting with the U.S. Food and Drug
Administration (FDA) to align with the Agency on the company’s
proposal regarding co-primary endpoints for the pivotal Phase 3
VIITAL™ study of EB-101 in recessive dystrophic epidermolysis
bullosa (RDEB). Following the meeting, Abeona is proceeding with
its plan to enroll between 10 to 15 patients with RDEB, comprising
approximately 35 large chronic wound sites treated in total.
“We appreciate the clarity provided by the FDA
and we are pleased to be aligned with the Agency on the co-primary
endpoints for the Phase 3 VIITAL™ study,” said Michael Amoroso,
Principal Executive and Chief Operating Officer of Abeona.
“Following the successful completion of the FDA meeting, we
continue with all necessary steps to enroll our next patient in the
VIITAL™ study and aim to complete enrollment in 2021.”
The co-primary endpoints of the study are: 1)
the proportion of RDEB wound sites with greater than or equal to
50% healing from baseline, comparing treated with untreated wound
sites at Week 24 (Month 6) as determined by direct investigator
assessment; and 2) pain reduction associated with wound dressing
change assessed by the mean differences in scores of the Wong-Baker
FACES scale between treated and untreated wounds at Week 24 (Month
6).
As previously announced, data from a Phase 1/2a
clinical trial presented at the 2020 Society for Pediatric
Dermatology Annual Meeting showed that wound healing of 50% or
greater following EB-101 treatment in patients with RDEB was
associated with no pain at treated sites at three-, four- and
five-years post-treatment, compared with presence of pain in 53% of
wound sites at baseline.
Jodie Gillon, Vice President and Chief Patient
Officer of Abeona commented, “We greatly appreciate the level of
clarity we received from the FDA as we continue to work with our
clinical study partners at Stanford University Medical Center and
patient advocacy groups to enroll additional patients in the
VIITAL™ study.”
Investigators at Stanford University Medical
Center are currently enrolling eligible patients into the VIITAL™
study. Additional information about the trial, including
eligibility criteria, is available at
https://www.abeonatherapeutics.com/clinical-trials/rdeb and
https://clinicaltrials.gov/ (Identifier: NCT04227106).
About Recessive Dystrophic Epidermolysis
Bullosa Recessive dystrophic epidermolysis bullosa (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 EB-101 EB-101 is an
autologous, gene-corrected cell therapy currently being
investigated in the pivotal Phase 3 VIITAL™ study for the treatment
of recessive dystrophic epidermolysis bullosa (RDEB), a rare
connective tissue disorder without an approved therapy. The EB-101
VIITAL™ study is a randomized clinical trial enrolling 10 to 15
RDEB patients with approximately 30 large, chronic wound sites
treated in total. Treatment with EB-101 involves using gene
transfer to deliver COL7A1 genes into a patient’s own skin cells
(keratinocytes and its progenitors) and transplanting them back to
the patient to enable normal Type VII collagen expression and
facilitate wound healing. Abeona produces EB-101 for the VIITAL™
study at its fully-functional gene and cell therapy manufacturing
facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101
provided durable wound healing for RDEB patients lasting 2+ to 5+
years, including for the largest, most challenging wounds that
affect the majority of the RDEB population.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing gene and cell therapies for serious diseases.
Abeona’s clinical programs include EB-101, its autologous,
gene-corrected cell therapy for recessive dystrophic epidermolysis
bullosa in Phase 3 development, as well as ABO-102 and ABO-101,
novel AAV-based gene therapies for Sanfilippo syndrome types A and
B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development.
The Company’s portfolio also features AAV-based gene therapies for
ophthalmic diseases with high unmet medical needs. Abeona’s novel,
next-generation AIM™ capsids have shown potential to improve
tropism profiles for a variety of devastating diseases. Abeona’s
fully functional, gene and cell therapy GMP manufacturing facility
produces EB-101 for the pivotal Phase 3 VIITAL™ study and is
capable of clinical and commercial production of AAV-based gene
therapies. For more information, visit
www.abeonatherapeutics.com.
Forward-Looking StatementsThis
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 Company’s aim to complete enrollment
of patients in our VIITAL study in 2021. We have attempted to
identify forward-looking statements by such terminology as “may,”
“will,” “believe,” “estimate,” “expect,” 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 potential impacts
of the COVID-19 pandemic on our business, operations, and financial
condition, the outcome of our announced strategic review, continued
interest in our rare disease portfolio, our ability to enroll
patients in clinical trials, the outcome of any future meetings
with the U.S. Food and Drug Administration or other regulatory
agencies, 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 disclosed in the Company’s most recent
Annual Report on Form 10-K and subsequent quarterly reports on Form
10-Q and other 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
+1 (646) 813-4709
ggin@abeonatherapeutics.com
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