Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial for MPS IIIB
December 20 2017 - 8:45AM
Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene and cell
therapies for life-threatening rare diseases, announced today that
the first patient in a Phase 1/2 clinical trial for ABO-101
(AAV-NAGLU), a single treatment gene therapy for patients with
Sanfilippo syndrome type B (MPS IIIB), has been dosed at Nationwide
Children’s Hospital, Columbus, Ohio.
“With the dosing of the first patient with
Sanfilippo syndrome type B, we are excited to begin enrolling our
third gene therapy clinical trial targeting rare diseases,” stated
Timothy J. Miller, Ph.D., President and CEO of Abeona. “Having
observed positive efficacy data and a strong safety profile in our
MPS IIIA program, we look forward to further validating the
clinical translation of this investigational gene therapy. This
trial is the culmination of many years of research and development,
and none of it would have been possible without continued support
from the Sanfilippo patient advocacy community.”
ABO-101 has been granted Orphan Product
Designation by the U.S. Food and Drug Administration (FDA) and
received Rare Pediatric Disease Designation as a pre-requisite
component of the FDA’s Priority Review Voucher process. The
ABO-101 clinical study is supported by a 25-subject MPS III Natural
History Study conducted at Nationwide Children’s Hospital.
“MPS IIIB is a devastating and deadly lysosomal
storage disease with no approved treatments available. Caused by a
single gene defect, the disease afflicts children, many of whom do
not reach adulthood. The ABO-101 gene therapy approach advances the
potential of AAV gene therapy as a treatment paradigm for
addressing this relentlessly progressive disease,” noted Kevin M.
Flanigan, M.D., Director and Principal Investigator with the Center
for Gene Therapy at Nationwide Children’s Hospital.
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 CNS and peripheral organs to
produce the missing NAGLU enzyme, which then restored the
underlying sugar (GAG) storage pathology to normal levels in cells.
Preclinical in vivo efficacy studies in Sanfilippo
syndrome animal model have 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 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 is developing
its proprietary vector platform, AIM™, for next generation product
candidates.
Investor Contact: Christine Silverstein Vice
President, Investor Relations Abeona Therapeutics Inc. +1
(212)786-6212 csilverstein@abeonatherapeutics.com
Media Contact: Lynn Granito Berry & Company
Public Relations +1 (212) 253-8881
lgranito@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 that involve risks and
uncertainties, including the statement that AAV treatment could
potentially benefit patients with Sanfilippo syndrome. These
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 develop our products and
technologies; the ability to obtain any necessary intellectual
property to commercialize any of our products; the ability to
achieve or obtain necessary regulatory approvals and licenses; the
impact of changes in the financial markets and global economic
conditions; and other risks as may be detailed from time to time in
the Company's Annual Reports on Form 10-K 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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