Abeona Therapeutics Receives FDA Fast Track Designation for ABO-101 for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
April 04 2019 - 8:25AM
Abeona Therapeutics Receives FDA Fast Track Designation for ABO-101
for Treatment of Sanfilippo Syndrome Type B (MPS IIIB)
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in cell and gene therapy development, today announced that the U.S.
Food and Drug Administration (FDA) has granted Fast Track
designation to ABO-101, the Company’s novel one-time gene therapy
for Sanfilippo syndrome type B (MPS IIIB). ABO-101 is designed to
deliver a functional copy of the NAGLU gene to the central nervous
system and peripheral tissues. Abeona is enrolling eligible
patients with MPS IIIB at sites in the U.S. and Spain and expects
to activate additional sites globally this year to accelerate
enrollment. Information about the study is available at
AbeonaTrials.com and ClinicalTrials.gov (NCT03315182).
“Receiving Fast Track designation is recognition
by FDA that ABO-101 shows promise in treating a serious
life-threatening disease and may address the significant unmet
medical need of children with MPS IIIB,” said João Siffert, M.D.,
Chief Executive Officer. “We will continue to interact with global
regulatory authorities about the ABO-101 clinical program to
facilitate development of this one-time AAV9 mediated gene
therapy.”
Fast Track designation is granted by the FDA to
facilitate the development and expedite review of investigational
therapies intended to treat serious or life-threatening diseases
that show potential for addressing significant unmet medical needs.
Clinical development of drug products receiving the designation
benefit from frequent FDA interactions during all aspects of
development and if specific criteria are met, they are eligible for
priority review and accelerated approval. ABO-101 has also received
Orphan Drug designations in the U.S. and EU, and Rare Pediatric
Disease designation from the FDA.
ABO-101 is part of the Company’s portfolio of
novel, one-time, AAV9-based gene therapies for rare lysosomal
storage diseases. Pre-clinical data demonstrated that ABO-101
delivers a functional copy of the NAGLU gene to the central nervous
system and peripheral tissues through a single intravenous
infusion. The therapy is designed to address the underlying
lysosomal enzyme deficiency responsible for abnormal accumulation
of glycosaminoglycans in the brain and throughout the body that
result in progressive cell damage and neurodevelopmental and
physical decline.
About Sanfilippo
SyndromeSanfilippo syndrome, or MPS type III, is a group
of devastating rare genetic lysosomal storage diseases with no
approved treatments. MPS III is characterized by developmental
decline, including loss of ability to communicate, cognitive
impairment, hearing loss, behavioral disturbances, seizures, as
well as difficulties with sleep. Systemic manifestations include
coarsening of facial features and enlargement of organs such as the
liver. An estimated 70% of children with MPS III do not reach age
18. The underlying cause of the syndrome is a missing enzyme in the
lysosome that is essential to breaking down heparan sulfate. As a
result, partially degraded heparan sulfate accumulates inside the
cells, including the brain and spinal cord, causing progressive
damage. MPS III is caused by single gene defects unique to each
type of the syndrome - A, B, C or D. The cause of MPS IIIA is a
deficiency in the SGSH enzyme, while MPS IIIB is distinguished by a
marked decrease in NAGLU enzyme activity.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing cell and gene therapies for life-threatening
rare genetic diseases. The Company’s lead programs include EB-101,
its gene-corrected cell therapy for recessive dystrophic
epidermolysis bullosa, and ABO-102, a novel AAV9-based gene therapy
for Sanfilippo syndrome type A (MPS IIIA). The Company’s portfolio
of AAV9-based gene therapies also features ABO-101 for Sanfilippo
syndrome type B (MPS IIIB), and ABO-202 and ABO-201 for CLN1
disease and CLN3 disease, respectively. Its preclinical assets
include ABO-401, which uses the novel AIM™ AAV vector platform to
address 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 Regenerative
Medicine Advanced Therapy designation for two investigational
therapies (EB-101 and ABO-102). For more information, visit
www.abeonatherapeutics.com.
Forward Looking StatementThis
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 the
potential for ABO-101 for the treatment of Sanfilippo syndrome,
status of the clinical program including ongoing Phase 1/2 studies,
pending INDs and feedback from regulatory agencies, timelines for
initiation of further clinical studies, and 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 initiate and 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 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:Sofia Warner Senior Director,
Investor Relations Abeona Therapeutics +1 (646) 813-4707
swarner@abeonatherapeutics.com
Media Contact:Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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