Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome Type A
August 16 2017 - 9:38AM
NEW YORK and CLEVELAND, Aug. 16, 2017 (GLOBE
NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading
clinical-stage biopharmaceutical company focused on developing
novel gene therapies for life-threatening rare diseases, today
announced the pivotal expansion of its gene therapy clinical trials
for patients with MPS IIIA in the USA, Europe and Australia.
"We have completed the necessary regulatory and
ethical committee approvals and site initiations in Europe and
Australia in order to accelerate enrollment," stated Juan Ruiz,
M.D., Ph.D., Abeona's Chief Medical Officer. "We remain very
encouraged by the improvements observed in clinically relevant
biomarkers post-dosing of ABO-102, including durable reductions in
heparan sulfate measured in the CNS, reduction of organ disease
pathology, and signals of CNS improvement or stabilization at
one-year follow-up in Cohort 1 subjects, and look forward to
providing a more fulsome clinical update at important clinical
conferences, including ESGCT, this fall," he continued.
The planned expansion will enroll an additional
eight to ten MPS IIIA subjects, with total enrollment of fourteen
to sixteen subjects to be completed by 1Q2018. Per the design of
the clinical trial, subjects will receive a single, intravenous
injection of ABO-102 to deliver the AAV viral vector systemically
throughout the body to introduce a corrective copy of the gene that
underlies the cause of the MPS IIIA disease. Subjects are evaluated
at multiple time points post-injection for safety assessments and
initial signals of biopotency and clinical activity, which suggest
that ABO-102 successfully reached target tissues throughout the
body, including the central nervous system.
Sanfilippo syndromes (or
mucopolysaccharidosis (MPS) type III): a group of four inherited
genetic diseases each caused by a single gene defect, described as
type A, B, C or D, which cause enzyme deficiencies that result in
the abnormal accumulation of glycosaminoglycans (GAGs, or sugars)
in body tissues. MPS III is a lysosomal storage disease, a group of
rare inborn errors of metabolism resulting from deficiency in
normal lysosomal function. The incidence of MPS III (all four types
combined) is estimated to be 1 in 70,000 births.
Mucopolysaccharides are long chains of sugar molecule used in the
building of connective tissues in the body. There is a continuous
process in the body of replacing used materials and breaking them
down for disposal. Children with MPS III are missing an enzyme
which is essential in breaking down the used mucopolysaccharides
called heparan sulfate. The partially broken down
mucopolysaccharides remain stored in cells in the body causing
progressive damage. In MPS III, the predominant symptoms occur due
to accumulation within the central nervous system (CNS), including
the brain and spinal cord, resulting in cognitive decline, motor
dysfunction, and eventual death. Importantly, there is no cure for
MPS III and treatments are largely supportive care.
About Abeona: Abeona
Therapeutics Inc. is a clinical-stage biopharmaceutical company
developing gene therapies for life-threatening rare genetic
diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts)
for recessive dystrophic epidermolysis bullosa (RDEB). Abeona
is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type
B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile
Batten disease (INCL), 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 has a
proprietary vector platform, AIM(TM), for next generation product
candidates. For more information,
visit www.abeonatherapeutics.com.
Investor Contact:
Christine Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc.
+1 (212)786-6212
csilverstein@abeonatherapeutics.com
Media
Contact:
Andrea Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)786-6208
alucca@abeonatherapeutics.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 that
involve risks and uncertainties. These statements include, without
limitation, our plans for continued development and
internationalization of our clinical programs, that patients will
continue to be identified, enrolled, treated and monitored in the
EB-101 clinical trial, and that studies will continue to indicate
that EB-101 is well-tolerated and may offer significant
improvements in wound healing; the addition of two additional
global clinical sites will accelerate our ability to enroll and
evaluate ABO-102 as a potential treatment for patients with
Sanfilippo syndrome type A, or MPS IIIA. Such statements are
subject to 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;
our belief that initial signals of biopotency and clinical
activity, which suggest that ABO-102 successfully reached target
tissues throughout the body, including the central nervous system
and the increased reductions in CNS GAG support our approach for
intravenous delivery for subjects with Sanfilippo syndromes, 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.
This
announcement is distributed by Nasdaq Corporate Solutions on behalf
of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: Abeona Therapeutics Inc via Globenewswire
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