Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced recent updates on its
clinical programs, highlighted by the restart of patient enrollment
in the Company’s pivotal Phase 3 VIITAL™ study of EB-101, its
autologous, gene-corrected cell therapy, for the treatment of
recessive dystrophic epidermolysis bullosa (RDEB). Enrollment in
the VIITAL™ study at Stanford University Medical Center was paused
in March 2020 in order to redirect healthcare resources to COVID-19
patients and to ensure the safety of study participants and site
staff.
“RDEB is a debilitating and life-threatening rare genetic
disorder without an approved treatment option. Reinitiating
enrollment in the VIITAL study is important for patients with RDEB
and brings us a step closer toward concluding the clinical
development of EB-101,” said João Siffert, M.D., Chief Executive
Officer of Abeona. “The unprecedented COVID-19 global health crisis
has had a broad impact across our industry, and we will continue to
monitor the pandemic to ensure the safety of trial participants,
healthcare professionals and our employees.”
Abeona’s MPS III Phase 1/2 Clinical Trials Continue
Despite COVID-19 Pandemic
Abeona also announced that two ongoing MPS III Phase 1/2
clinical trials of its investigational adeno-associated virus
(AAV)-based gene therapies, ABO-102 and ABO-101, have continued to
treat patients, with additional enrollment expected in the programs
in the coming weeks. In June 2020, the 10th patient was dosed in
cohort 3 of the ABO-102 Transpher A study for MPS IIIA (Sanfilippo
syndrome type A), bringing the total to 16 patients. The Company
previously reported in May 2020 that additional patients were
treated in dose cohort 3 of the Transpher A study and the ABO-101
Transpher B study for MPS IIIB (Sanfilippo syndrome type B),
bringing the total enrollment in the Transpher B study to 9
children to date.
Dr. Siffert added, “We have worked closely with sites in our MPS
III clinical studies to continue to enroll patients given the
urgent need to treat children with Sanfilippo syndrome as early as
possible. We are encouraged by the number of new pre-screened
patients that may meet enrollment criteria and could allow dosing
in the coming weeks. We also hope to resume on-site follow-up study
visits that include neurocognitive assessments of already enrolled
patients.”
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 VIITALTM study for the treatment of recessive
dystrophic epidermolysis bullosa (RDEB), a rare connective tissue
disorder without an approved therapy. The EB-101 VIITALTM study is
a multi-center, 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 VIITALTM 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. More information on the clinical
trials of EB-101 can be found at
https://www.abeonatherapeutics.com/clinical-trials/rdeb and
ClinicalTrials.gov (Identifier: NCT04227106).
About the VIITAL™ Phase 3 Study The VIITAL™
Phase 3 study is a multi-center, randomized clinical trial
assessing EB-101 in up to 15 RDEB patients, with approximately 30
large, chronic wound sites treated in total. The primary outcome
measure is wound healing, comparing treated with untreated wound
sites on the same patient. Secondary endpoints include the
assessments of pain, as well as other patient reported outcomes.
Investigators at Stanford University Medical Center are currently
enrolling eligible patients into the VIITALTM study. Additional
information about the trial is available at
https://www.abeonatherapeutics.com/clinical-trials/rdeb and
ClinicalTrials.gov (Identifier: NCT04227106).
About The Transpher A Study The Transpher A
Study (NCT02716246) is an ongoing, two-year, open-label,
dose-escalation, Phase 1/2 global clinical trial assessing ABO-102
for the treatment of patients with Sanfilippo syndrome type A (MPS
IIIA). The study, also known as ABT-001, is intended for patients 6
months to 2 years of age, or patients older than 2 years with a
cognitive Developmental Quotient of 60% or above. The gene therapy
ABO-102 is delivered using AAV9 technology via a single-dose
intravenous infusion. The study primary endpoints are
neurodevelopment and safety, with secondary endpoints including
behavior evaluations, quality of life, enzyme activity in
cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in
CSF, plasma and urine, and brain and liver volume.
About The Transpher B Study The Transpher B
Study (NCT03315182) is an ongoing, two-year, open-label,
dose-escalation, Phase 1/2 global clinical trial assessing ABO-101
for the treatment of patients with Sanfilippo syndrome type B (MPS
IIIB). The study, also known as ABT-002, is intended for patients
from birth to 2 years of age, or patients older than 2 years with a
cognitive Developmental Quotient of 60% or above. The gene therapy
ABO-101 is delivered using AAV9 technology via a single-dose
intravenous infusion. The study primary endpoints are
neurodevelopment and safety, with secondary endpoints including
behavior evaluations, quality of life, enzyme activity in
cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in
CSF, plasma and urine, and brain and liver volume.
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 of
AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1
disease and CLN3 disease, respectively. Abeona’s library of 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 VIITALTM study and is
capable of clinical and 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. These statements
include statements about the Company’s clinical trials and its
products and product candidates, future regulatory interactions
with regulatory authorities, as well as the Company’s goals and
objectives. 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,
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 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 periodic 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:Greg Gin VP, Investor
Relations Abeona Therapeutics +1 (646)
813-4709ggin@abeonatherapeutics.com
Media Contact:Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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