Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at the 13th Annual WORLDSymposium(TM) 2017
February 17 2017 - 10:04AM
NEW YORK and CLEVELAND, Feb. 17, 2017 (GLOBE
NEWSWIRE) -- 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(TM) 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(TM) 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(TM)
(alpha-1 protease inhibitor) for inherited COPD, using its
proprietary SDF(TM) (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
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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