Abeona Therapeutics Announces Authorization to Commence Phase 1/2 Gene Therapy Clinical Study for Patients with MPS IIIB in S...
September 12 2018 - 8:45AM
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 authorization to move forward with a Phase 1/2 clinical
trial in Spain for the Company’s gene therapy product ABO-101
(AAV-NAGLU) for patients with MPS IIIB (Sanfilippo syndrome type
B). The clinical study was approved by the Agencia Espanola de
Medicamentos y Productos Sanitarios and is being conducted at
Hospital Clinico Universitario of Santiago de Compostela, Spain.
This will be the Company’s second clinical trial conducted in
Europe, alongside the ongoing Phase 1/2 clinical trial for patients
with MPS IIIA (Sanfilippo syndrome type A). Abeona first initiated
this trial in the United States. Abeona plans to add clinical sites
for the trial in three European countries including France, Germany
and the United Kingdom.
“The authorization of our ABO-101 trial in Spain
is a significant milestone for children suffering from MPS IIIB, a
devastating and deadly disease with no approved treatment options,”
stated Carsten Thiel, PhD, CEO. “We are encouraged by the
preliminary results observed in our US trial to date, both in
clinically relevant biomarkers and in the ongoing safety profile
and are excited to bring this therapy to patients in Europe.”
Subjects in the Phase 1/2 trial receive a
single, intravenous infusion of ABO-101, which uses an AAV vector
to introduce the functional NAGLU gene to treat patients with MPS
IIIB disease. Subjects will be evaluated at multiple time points
post-injection for safety assessments and efficacy
parameters. The clinical program is supported by a Natural
History Study which included potential efficacy assessments
consisting of neurocognitive evaluations, biochemical assays and
MRI data generated over one year of follow-up assessments.
ABO-101 has been granted Rare Pediatric Disease
Designation in the U.S., and Orphan Product Designation in both the
U.S. and the European Union.
About ABO-101 (AAV-NAGLU):
ABO-101 is Abeona’s first-in-human, adeno-associated viral
(AAV)-based gene therapy for MPS III (Sanfilippo syndrome).
Treatment involves a one-time intravenous delivery of a functioning
copy of the N-acetyl-α-D-glucosaminidase (NAGLU) gene to cells of
the central nervous system (CNS) and peripheral organs, with the
aim of correcting the effects that result from the genetic
aberrations that are the root cause of the disease. Following
administration of a single dose in Sanfilippo preclinical animal
models, ABO-101 induced cells in the Central Nervous System and
peripheral organs to produce the missing NAGLU enzyme, which then
restored underlying sugar (glycosaminoglycan or GAG) storage
pathology to normal levels in cells. In preclinical in vivo
efficacy studies in Sanfilippo syndrome animal model, ABO-101
demonstrated functional benefits that continue for months after
treatment. A single dose of ABO-101 significantly restored normal
cell and organ function, corrected cognitive defects, increased
neuromuscular function and normalized the lifespan of animals with
MPS IIIB after treatment compared to untreated control animals.
These results are consistent with studies from several laboratories
suggesting AAV treatment could potentially benefit patients with
Sanfilippo syndrome. Safety and efficacy studies of AAV gene
therapy treatments for Sanfilippo syndrome have recently been
published in several peer-reviewed scientific journals.
About MPS IIIB: (also known as
Sanfilippo syndrome type B) is a genetic, progressive, and
devastating rare lysosomal storage disease. In patients with MPS
IIIB, genetic mutations result in a marked decrease in NAGLU enzyme
activity, which leads to accumulation of heparan sulfate (HS) in
the brain and other organs as well as progressive brain atrophy
with cortical gray matter volume loss. The accumulation of abnormal
HS results in neurocognitive decline, behavioral disturbances,
speech loss, increasing loss of mobility, and premature death. MPS
IIIB typically presents in children during the first few years of
life, and 70% of patients do not reach 18 years of age. There are
no approved treatments for MPS IIIB.
About Abeona: 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 skin grafts) 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 juvenile batten
disease, ABO-202 (AAV-CLN1) for treatment of infantile batten
disease, EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC)
for Fanconi anemia (FA) disorder and ABO-302 using a novel
CRISPR/Cas9-based gene editing approach to gene therapy for rare
blood diseases. In addition, Abeona is developing a proprietary
vector platform, AIM™, for next generation product candidates. For
more information, visit www.abeonatherapeutics.com.
Investor Contact:Christine SilversteinSVP,
Finance & Investor Relations Abeona Therapeutics Inc.+1 (646)
813-4707csilverstein@abeonatherapeutics.com
Media Contact:Lynn GranitoBerry & Company
Public Relations+1 (212) 253-8881lgranito@berrypr.com
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. Such statements include that we are encouraged
by the results of the US trial, and that we plan to add clinical
sites for the trial in three European countries. 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 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 obligations to make any
revisions to the forward-looking statements contained in this
release or to update them to reflect events or circumstances
occurring after the date of this release, whether as a result of
new information, future developments or otherwise.
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