Abeona Therapeutics Provides Update from ABO-102 Phase 1/2 MPS IIIA Clinical Trial at the 13th Annual WORLDSymposium(TM) 2017
(Thomson Reuters ONE) -
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 13(th) 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(at)abeonatherapeutics.com
Media Contact:
Andre'a Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)-786-6208
alucca(at)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
Unternehmensinformation / Kurzprofil:
Datum: 17.02.2017 - 16:04 Uhr
Sprache: Deutsch
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