EB-101 pivotal trial for Recessive Dystrophic
Epidermolysis Bullosa planned for mid-2019 enrollment
Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel cell and gene
therapies for life-threatening rare genetic diseases, today
announced key pipeline updates during the Company’s 2018 R&D
Day.
“The important clinical and preclinical updates we shared today
further establish Abeona’s pathway to bring long-term value to our
shareholders and hope to our patients,” said João Siffert, M.D.,
interim Chief Executive Officer, Chief Medical Officer and Head of
R&D. “We are very pleased to share next steps for our lead
programs and to unveil the potential of the novel AIMTM AAV vector
platform that could be a catalyst for the next generation of gene
therapy.”
EB-101 for Recessive Dystrophic Epidermolysis Bullosa
(RDEB)Abeona is developing gene-corrected cell therapy
EB-101 for the treatment of RDEB, a skin disease characterized by
chronic epidermal wounds in which patients suffer from pain,
itching, and widespread complications impacting quality-of-life and
life expectancy.
The Company expects to initiate a pivotal clinical trial
evaluating the potential of EB-101 for the treatment of RDEB in the
middle of 2019. The VITAL Study will be a multicenter, randomized,
Phase III clinical trial assessing 10-15 patients treated with
EB-101 compared to intra-patient untreated wounds. The primary
outcome measure of the study will be the proportion of treated
wounds with >50% healing at three months, with secondary
endpoints of investigator global assessment of wounds and changes
in pain and itch from baseline.
The Company also reported that it has established GMP
manufacturing capability for EB-101 at its gene therapy
manufacturing facility in Cleveland. The facility, known as the
Elisa Linton Center for Rare Disease Therapies, can produce
clinical product, and has scalable capacity to support the
potential commercial launch of EB-101.
“We believe that we are strongly positioned to initiate a
pivotal trial evaluating EB-101 by mid-2019 thanks to the important
CMC work undertaken by colleagues at our gene therapy manufacturing
facility in Cleveland, which also addressed guidance received
through frequent regulatory interactions afforded by the
Regenerative Medicine Advanced Therapy and other designations we
hold. We believe this work is critical for our path towards BLA
filing,” added Dr. Siffert.
ABO-102 for Sanfilippo Syndrome Type A (MPS
IIIA)Abeona is developing novel gene therapy ABO-102 for
the treatment of MPS IIIA, a lysosomal storage disease with no
approved treatment that is characterized by neurodevelopmental
decline, behavior abnormalities, seizures, loss of speech or
vision, an inability to sleep, and premature death.
The Company plans to amend its ongoing Phase I/II trial
evaluating ABO-102 for MPS IIIA to enroll patients at earlier
stages of disease. ABO-102 has been well tolerated to date
with no serious drug-related adverse events. The study has also
demonstrated a substantial, dose-related improvement in biomarkers,
including reductions in cerebrospinal fluid heparan sulfate levels
and liver volume in patients treated with ABO-102. Investigators
also observed encouraging neurocognitive signals in younger, higher
functioning patients enrolled in the higher dose of Cohort 3.
Patients unable to participate in the modified Phase I/II study may
be eligible to enroll in other studies within our MPS IIIA
program.
“The encouraging data generated to date and our interactions
with the FDA and EMA have informed the advancement of our Phase
I/II trial, which will seek to enroll patients likely to receive
the most benefit from treatment,” added Dr. Siffert.
ABO-401 for Cystic FibrosisAbeona presented new
pre-clinical data today from the Company’s first program produced
by the novel AIMTM vector platform for gene therapy delivery. The
data suggest that ABO-401, based on the vector AAV204, efficiently
targets lung cells and that ABO-401 corrects the underlying cystic
fibrosis (CF) chloride channel deficit, regardless of underlying
mutations of the CF transmembrane conductance regulators, including
the most common CF mutation, delta-F508.
AIMTM Vector Platform Targeting the EyeThe
Company presented non-human primate data suggesting that
next-generation AIMTM AAV vectors can efficiently target the
retinal epithelium after intravitreal injection, creating the
potential for new pipeline candidates that can address multiple eye
disorders. Also presented were data showing that certain AIMTM
capsids with high tropisms for central nervous system tissue
can evade neutralizing antibodies against naturally occurring AAV
serotype, and potentially enable redosing in patients that have
previously received an AAV injection.
“Our AIMTM vector platform enables the potential for gene
therapy for patients living with cystic fibrosis, regardless of
mutation, which could change the landscape of treatment and alter
the course of this progressive, genetic disease,” said Timothy J.
Miller, Ph.D., co-Founder, President, and Chief Scientific Officer.
“We are very encouraged by the preclinical data presented today
demonstrating delivery and correction of the underlying genetic
deficit in CF patient cells. Furthermore, we are very excited to
show the capability of the AIMTM vectors for delivering genes to
the eye and are excited about their potential as the next
generation of gene therapy across tissue types.”
ABO-202 for Infantile Batten Disease
(CLN1)Abeona is developing ABO-202 for the treatment of
CLN1, a rare and fatal autosomal recessive genetic disorder with no
approved treatment, which is characterized by vision impairment and
rapid neurological regression.
The Company presented new preclinical data today that will
inform the submission of an investigational new drug application
(IND) for ABO-202 in the first quarter of 2019. Findings from a
combination pre-clinical, dose-escalation study suggest that
ABO-202 may have a favorable safety profile, with no significant
toxicology findings. Other IND-enabling studies also demonstrated
normalized survival, improvement of motor function and cognition in
affected mice treated with ABO-202, and that combination dosing of
intravenous and intrathecal administrations may enhance the
therapeutic potential of ABO-202.
Abeona has received numerous regulatory designations from the
FDA and EMA for its pipeline candidates and is the only company
with RMAT designation for two investigational therapies (EB-101 and
ABO-102).
About Abeona Therapeutics Abeona Therapeutics
Inc. is a clinical-stage biopharmaceutical company developing cell
and gene therapies for life-threatening rare genetic diseases.
Abeona's lead programs include EB-101 (gene-corrected cell therapy)
for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102
(AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for
Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type B (MPS IIIB). Abeona is also developing ABO-201
(AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for
treatment of CLN1 disease, EB-201 for epidermolysis bullosa (EB),
ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder ABO-302 using
a novel CRISPR/Cas9-based gene editing approach to gene therapy for
rare blood diseases, and ABO-401 using a novel AAV vector platform,
AIM™ with CFTR minigene addresses 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
RMAT designation for two investigational therapies (EB-101 and
ABO-102). 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 regarding our pipeline and
product portfolio, approval by regulatory agencies relative to
amendments to INDs and to protocol amendments including related to
ABO-102, timelines for initiation of further clinical studies
including the pivotal study for EB-101, the establishment and
scalability of manufacturing capabilities, the capabilities of the
novel AIM™ vector platform in development; the market opportunities
for the Company’s products and product candidates, the ability to
generate long term shareholder value, meet patient expectations,
and achieve 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.
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 submit protocols and protocol amendments to regulatory
agencies, our ability to initiate and enroll patients in clinical
trials, the adequacy of manufacturing capabilities, the impact of
competition, the ability to secure licenses or establish
intellectual property rights for any technology that may be
necessary to continue to develop and 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 reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligation to revise the
forward-looking statements or 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:Christine SilversteinSVP,
Finance & Investor RelationsAbeona Therapeutics Inc.+1 (646)
813-4707csilverstein@abeonatherapeutics.com
Media Contact:Scott SantiamoDirector, Corporate
CommunicationsAbeona Therapeutics Inc.+1 (718)
344-5843ssantiamo@abeonatherapeutics.com
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