Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome Type A
(Thomson Reuters ONE) -
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(at)abeonatherapeutics.com
Media Contact:
Andrea Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)786-6208
alucca(at)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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