--Positive dose response in central nervous system with 60.7%
+/- 8.8% reduction of disease-causing heparan sulfate GAG observed
in Cohort 2--Reduction of disease manifestation observed in
decreased liver volume of 14.81% (+/- 1.2%)--ABO-102 well-tolerated
in six subjects through 1100 days follow up with no Serious Adverse
Events--Cohort 1 demonstrated stabilized or improved Leiter
Nonverbal IQ scores at six months--Conference call today at 10:00am
EDT; 877-269-7756 for domestic callers and 201-689-7817 for
international callers
Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare diseases, announced updated
data from the ongoing gene therapy clinical trial for Sanfilippo
syndrome Type A (MPS IIIA) at the American Society Gene and Cell
Therapy (ASGCT) 20th Annual Meeting. The ongoing Phase 1/2 trial
for ABO-102 (AAV-SGSH) is a first-in-man clinical trial utilizing a
single intravenous injection of AAV gene therapy for subjects with
Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive
lysosomal storage disease.
“Abeona continues to advance gene therapy for
MPS IIIA patients and we are excited about the positive dose
response in the CNS seen in Cohort 2. The observation of a dose
response supports our clinical approach, and we are encouraged to
observe further reductions in central nervous system (CNS) heparan
sulfate, reduction in liver volume, and preliminary evidence of
slowed neurocognitive decline, are very encouraging. We look
forward to accelerating enrollment with the recently initiated
global sites (Spain and Australia) and reporting additional
clinical data in the ABO-102 global MPS IIIA trial later this
year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona
Therapeutics.
Per the design of the clinical trial, subjects
received a single, intravenous injection of ABO-102 to deliver the
AAV viral vector systematically 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. Highlights reported data on five (n=3
Cohort 1, n=2 Cohort 2) out of the six patients treated to date in
the gene therapy trial included:
Biopotency: positive dose response observed in Cohort 2.--At 30
days post-injection, two patients in the Cohort 2 demonstrated
60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan
sulfate (HS).
Hepatosplenomegaly: consistent reduction in liver volume
observed.--At 30 days post-injection, Cohort 2 subjects
demonstrated reductions in liver volumes of 14.81% (+/- 1.2%).--The
natural history study in 25 subjects with MPS III (Truxal et. al.,
2016, Mol. Genet. Metab.) demonstrated that subjects had increased
liver volumes averaging 116% at baseline that did not change over a
year of follow-up.
Cognitive Assessments: evidence of cognitive stabilization at
six months in Cohort 1. --Cognitive assessments, taken at baseline,
at the six-month timepoint for the Cohort 1 (n=3), subjects showed
evidence of stabilization or improvement in the Leiter-R non-verbal
IQ and Vineland (adaptive behavior) scales.--Cognitive assessments
are taken at six-month and twelve-month follow-up visits.--Leiter
Nonverbal IQ assessments in Cohort 1 subjects demonstrated
stabilized or improved scores at six-months post-injection.
Notably, one subject improved +10 (+/-6) points, while age-matched
controls in the Natural History study would have predicted a
decrease of -11.1 (+/-2.7) points over 6 months.--Vineland
assessments in Cohort 1 at six months post-injection suggest
stabilization in adaptive behavior scores.
Safety: well-tolerated in all subjects through 1100 days
cumulative post-injection.--No serious adverse events (SAEs)
reported in subjects in either cohort receiving ABO-102
(Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg).
“We remain encouraged by signs of tolerability
and biological effects that we have observed in Cohort 1 and in the
initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D.,
principal investigator, Director of the Center for Gene Therapy at
Nationwide Children’s Hospital and Professor of Pediatrics and
Neurology at The Ohio State University College of Medicine. “We are
pleased to see decreases in CSF HS compared to the Cohort 1 at 30
days post-injection, and we look forward to enrolling
additional high-dose patients.”
The ongoing Phase 1/2 clinical trial, which has
received FastTrack designation, Orphan Product Designation, and
Rare Pediatric Disease designation by the FDA, is designed to
evaluate safety and efficacy of ABO-102 in patients with MPS
IIIA. The global clinical study is supported by a 25-subject
MPS III 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.
Company Conference Call
Details: Abeona will host a live conference call
briefing today at 10:00am EDT. Analysts and investors can
participate in the conference call by dialing 877-269-7756 for
domestic callers and 201-689-7817 for international callers.
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
plasma-based protein therapy pipeline, including SDF Alpha™
(alpha-1 protease inhibitor) for inherited COPD, using its
proprietary SDF™ (Salt Diafiltration) ethanol-free process. 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:Andre’a Lucca Vice President,
Communications & Operations Abeona Therapeutics Inc. +1
(212)-786-6208alucca@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 the
statement that the addition of two additional global clinical site
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 develop
our products and technologies; 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.
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