Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at the 13th Annual WORLDSymposium™ 2017
February 17 2017 - 7:45AM
Abeona Therapeutics Inc. (Nasdaq:ABEO):
- ABO-102 gene therapy well-tolerated in 4 subjects (N=3 low
dose, N=1 high dose) through 650 days follow up with no Serious
Adverse Events
- 63% +/- 0.5% central nervous system reduction of heparan
sulfate GAG 6 months post-injection (N=2)
- Continued evidence of biopotency including reduced liver and
spleen volumes and decreased urinary GAGs
- Two subjects assessed at the 6-month timepoint showed evidence
for stabilization or improvement (average 60% over 2 subjects) in
several Mullen subdomains
- Adaptive behavior ratings on the Vineland stabilized
- Subjects showed improved ability to complete individual items
on the Leiter-R non-verbal IQ assessment resulting in improved raw
scores
Abeona Therapeutics Inc. (NASDAQ:ABEO), a
leading clinical-stage biopharmaceutical company focused on
developing therapies for life-threatening rare genetic diseases,
announced updated data from the ongoing gene therapy clinical trial
for Sanfilippo syndrome Type A (MPS IIIA), at the 13th Annual
WORLDSymposium™ 2017 lysosomal storage disorders conference in San
Diego, CA. 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 MPS IIIA, a rare
autosomal recessive disease affecting every cell and organ in the
body, which results in neurocognitive decline, speech loss, loss of
mobility, and premature death in children.
“We remain encouraged by continued signs of
tolerability and biopotency in the low-dose cohort, and enrollment
of the high-dose cohort is underway,” stated Kevin M. Flanigan,
M.D., principal investigator with the Center for Gene Therapy at
Nationwide Children’s Hospital and Professor of Pediatrics and
Neurology at The Ohio State University College of Medicine.
“Additionally, we are pleased to see further decreases in CSF GAG
measurements, as well as preliminary evidence for stabilization or
improvement of some cognitive functions, at six months
post-dosing.”
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. Observations reported at the
WORLDSymposium™ conference included:
- Safety: ABO-102 is well-tolerated in subjects injected
with the low dose of 5E13 vg/kg ABO-102, with no treatment related
adverse events or serious adverse events (SAEs) through over 650
days cumulative post-injection. Enrollment in the high dose cohort
has commenced with no Serious Adverse Events (SAEs) reported to
date.
- Biopotency: As reflected in published natural history
studies evaluating MPS III subjects, cerebral spinal fluid (CSF)
and urine GAG (heparan sulfate or “HS”) are significantly elevated
in the subject population as a symptom of disease pathology. As
announced previously, all subjects in the low-dose cohort
experienced reductions from baseline in CSF HS of 25.6% +/- 0.8%,
suggesting ABO-102 crossed the blood brain barrier after
intravenous administration. At the six-month follow-up (n=2),
CSF HS continued to decrease to 63.1% +/- 0.5% of baseline values,
suggesting further improvement in the elimination of the storage
pathology. Data presented showed reduction in urinary heparan
sulfate and urinary total GAG fragments.
- Hepatosplenomegaly: The natural history study in 25
subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.)
demonstrated that subjects had increased liver and spleen volumes
averaging 116% and 88%, respectively at baseline that did not
change over a year of follow-up. All three subjects demonstrated
significant reductions in liver volumes at 30-days post injection
(17.1% +/- 1.9%). At the six-month follow-up in low dose subjects
(n=2), this effect was sustained, with a liver volume further
decreased by 29.7 – 30.3% and spleen volume by 2.2 – 12.9% from
baseline.
- Cognitive Assessments: The clinical trial utilizes three
validated neurocognitive and behavioral assessment tools, including
the Leiter International Performance Scale Third Edition
(Leiter-3), the Vineland Adaptive Behavior Scale, Second Edition
(Vineland-II) and the Mullen Scale of Early Learning. Cognitive
assessments are taken at baseline, and have been taken at the
six-month (n=2) and will be taken at the twelve-month follow-up
visits. These assessments provide the opportunity to measure
several sub-domains, such as fine motor, visual acuity, expressive
language, receptive language, among others. Assessments at
six-month for the first two lose-dose patients provided early
evidence of cognitive stabilization. The two subjects assessed at
the 6-month timepoint showed evidence for stabilization or
improvement of scores (average of 60% across 2 subjects) in several
Mullen subdomains. Adaptive behavior ratings on the Vineland also
stabilized. Both subjects showed improved ability to complete
individual items on the Leiter-R non-verbal IQ assessment resulting
in improved raw scores.
“The data demonstrate an early and robust
systemic delivery of ABO-102, and the increased reductions in CNS
HS GAG support our approach for intravenous ABO-102 delivery for
subjects with Sanfilippo syndromes,” stated Timothy J. Miller,
Ph.D., President and CEO of Abeona Therapeutics. “We are excited
about continued biomarker signals in this trial, as well as early
positive signals in the neurocognitive assessments. While we are
still very early in the trial, we are extremely encouraged by these
early results and look forward to expanding enrollment in this
clinical trial with enrollments accelerating at two additional
international clinical sites.”
Abeona’s MPS IIIA program, ABO-102, has been
granted Orphan Product Designation in the USA and in the European
Union, has received the Rare Pediatric Disease Designation in the
US, and recently received Fast Track designation by the US FDA.
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) and
ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene
therapies for Sanfilippo syndrome (MPS IIIA and IIIB,
respectively). Abeona is also developing EB-101 (gene-corrected
skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB),
EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene
therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene
therapy for treatment of infantile Batten disease (INCL), and
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.
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 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 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; our belief that the
data demonstrate an early and robust systemic delivery of ABO-102,
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 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.
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)-786-6208
alucca@abeonatherapeutics.com
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