Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced positive
topline data from its pivotal Phase 3 VIITAL study assessing the
safety and efficacy of EB-101 for the treatment of patients with
recessive dystrophic epidermolysis bullosa (RDEB). The VIITAL study
met its two co-primary efficacy endpoints demonstrating
statistically significant, clinically meaningful improvements in
wound healing and pain reduction in large chronic RDEB wounds.
“We are very pleased with the topline results
from our pivotal VIITAL study, which reinforce the strong value
proposition of EB-101 as a potential one-time therapy to both
significantly improve wound healing and reduce pain for the most
disabling, challenging to treat wounds in patients with RDEB,” said
Vish Seshadri, Chief Executive Officer of Abeona. “The VIITAL study
is differentiated from any other pivotal study in RDEB by the
co-primary endpoint measuring patient-reported pain. We believe the
significant result in this endpoint supports EB-101’s potential for
improving the daily life of RDEB patients. Based on the efficacy
and safety profile of EB-101 in VIITAL, we are looking forward to
sharing the VIITAL study results with the FDA and progressing
toward submission of a BLA. We are grateful to the patients, their
families, caregivers, and the patient advocacy groups for their
support of this study, and are also thankful for the clinical
investigators, study site personnel, and the entire Abeona team who
collectively contributed to this milestone achievement.”
Summary of Topline Results: All
Evaluated Endpoints Successfully Achieved
The pivotal Phase 3 VIITAL study evaluated the
efficacy, safety and tolerability of EB-101 in 43 large chronic
wound pairs in 11 subjects with RDEB. The large chronic wounds
randomized and treated in VIITAL measured greater than 20 cm2 of
surface area and had remained open for a minimum of six months and
a maximum of 21 years (mean 6.2 years). The co-primary endpoints of
the study were: 1) the proportion of RDEB wound sites with greater
than or equal to 50% healing from baseline, comparing randomized
treated with matched untreated (control) wound sites at the
six-month timepoint, 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 randomized treated and matched untreated
(control) wounds at the six-month timepoint. The study allowed for
wounds not included in the randomized primary efficacy analysis to
receive EB-101 treatment (n=14 non-randomized wounds). The tables
below summarize the topline primary efficacy results:
- Wound Healing
Endpoints: EB-101 significantly improved wound healing vs.
control at six months
Wound Healing Level from Baseline (investigator
assessed) |
% Randomized
TreatedWounds(n=43) |
% Randomized Untreated Control
Wounds(n=43) |
p-value* |
50% or greater (co-primary endpoint) |
81.4% |
16.3% |
<0.0001 |
* Two-sided p-value calculated from permutation
test using randomized wound pairs (n=43 pairs).
Other VIITAL study endpoints measuring proportion of wounds
achieving 75% or greater wound healingand complete wound healing at
six months also achieved statistical significance.
- Pain Endpoint:
EB-101 showed significant pain reduction associated with wound
dressing changes vs. control at six months
|
Randomized
TreatedWounds(n=43) |
Randomized Untreated Control
Wounds(n=42) |
p-value** |
Mean pain reduction from baseline*(co-primary endpoint) |
3.07 |
0.90 |
0.0002 |
* Based on patient reported outcomes assessing
pain severity on 0-10 scale in increments of 2 (i.e., 0, 2, 4, 6,
8, 10). ** Two-sided p-value calculated from permutation test using
randomized wound pairs (n=42 pairs).
- Post-Hoc Analysis of Pain
Data
In addition to meeting the co-primary pain
endpoint, in a post-hoc analysis of the EB-101 treated severe
wounds (baseline pain score of 6 or greater), including randomized
and non-randomized (n=27), a mean pain reduction from baseline at
six months of 5.70 was observed, as compared to a mean pain
reduction of 3.51 for all treated randomized and non-randomized
wounds for which pain was evaluated (n=53).
EB-101 was shown to be well-tolerated with no
serious treatment-related adverse events observed, consistent with
past clinical experience. There were no deaths or instances of
positive replication-competent retrovirus (RCR) results, and no
systemic immunologic responses were reported during the study, as
well as no squamous cell carcinoma (SCC) at treatment sites after
application of EB-101. Two subjects reported at least one serious
adverse event (SAE) unrelated to EB-101. Four subjects reported
related treatment emergent adverse events (TEAEs), including
procedural pain, muscle spasms and pruritis. Infections unrelated
to EB-101 were observed in eight patients.
Abeona anticipates submitting results from this
study, including further details with additional exploratory
endpoints and the Week 12 results, for presentation at future
medical meetings and for publication in a peer-reviewed journal.
The Week 12 results are similar to Week 24 results, achieving
statistical significance for pain reduction and wound healing at
all levels.
Jean Tang, M.D., Ph.D., Professor of
Dermatology, Stanford University School of Medicine and Principal
Investigator of the EB-101 pivotal Phase 3 VIITAL study said,
“Large chronic RDEB wounds are the toughest to treat and often
associated with intense chronic pain that significantly impacts the
quality of life of RDEB patients, necessitating frequent use of
opioids. In the Phase 3 VIITAL study, EB-101 has been shown to both
heal such large chronic wounds and significantly reduce pain. And
we continue to see durable clinical benefit of EB-101 with up to
eight years of follow-up in our Phase 1/2a study.”
Brett Kopelan, Executive Director, debra of
America, and father to Rafi, a 15-year-old with RDEB, said, “I am
incredibly enthused to see new clinical evidence of EB-101’s
potential to treat the more difficult chronic and large wounds. Our
patient community needs options to address not only the healing of
wounds but also the chronic pain and the acute treatment related
pain of daily wound care associated with these wounds. Today’s
standard of care comprises hours of brutal and painful wound care,
and EB-101’s promise to be a transformational option for RDEB
patients is truly exciting.”
Next Steps
Based on the positive topline results, Abeona
intends to submit a Biologics License Application (BLA) for EB-101
to the U.S. Food and Drug Administration (FDA) in the second
quarter of 2023. EB-101 has been granted Orphan Drug and Rare
Pediatric Disease (RPD) designations by the FDA. Among the benefits
of Orphan Drug designation are seven years of market exclusivity
following FDA approval, potentially preventing FDA approval of
another product deemed to be the same as the approved product for
the same indication, waiver of application fees, and tax credits
for clinical testing expenses conducted after orphan designation is
received. A sponsor who receives an approval for a BLA with RPD
designation may qualify for a Priority Review Voucher (PRV),
subject to final determination by the FDA. The PRV can be used to
receive an expedited review process of a subsequent marketing
application for a different product or sold to another company.
Conference Call Details
Abeona Therapeutics will host a conference call
and webcast on Thursday, November 3, 2022, at 8:30 a.m. EDT, to
discuss the positive topline results from the VIITAL study. To
access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011
(international) and Entry Code: 844393 five minutes prior to the
start of the call. A live, listen-only webcast and archived replay
of the call can be accessed on the Investors & Media section of
Abeona’s website at www.abeonatherapeutics.com. The archived
webcast replay will be available for 30 days following the
call.
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, engineered cell therapy currently being developed 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 with target
enrollment of at least 10 to 15 RDEB patients with approximately 36
large, chronic wound sites treated in total. Treatment with EB-101
involves using gene transfer to deliver the COL7A1 gene into a
patient’s own skin cells (keratinocytes and its progenitors) and
transplanting those cells back to the patient. EB-101 is being
investigated for its ability to enable normal Type VII collagen
expression and to facilitate wound healing. The U.S. FDA has
granted Rare Pediatric Disease designation for EB-101. Abeona
produces EB-101 for the VIITAL study at its fully integrated gene
and cell therapy manufacturing facility in Cleveland, Ohio. EB-101
is an investigational product not yet approved 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
produces 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, our ability to continue as a going
concern; 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 any future meetings with the FDA or
other regulatory agencies; 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 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
ir@abeonatherapeutics.com
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