Abeona Therapeutics Enrolls First High Dose Subject in Ongoing Phase 1/2 Gene Therapy Clinical Trial in Sanfilippo Syndrome Type A
(Thomson Reuters ONE) -
* ABO-102, the leading clinical gene therapy program for Sanfilippo syndrome
type A patients, has demonstrated central nervous system (CNS) and
peripheral organ disease biopotency
* First high dose cohort patient is enrolled, and all patients (n = 4) have
cumulative 644 days post-injection with no Serious Adverse Events (SAEs)
reported to date
* Global ABO-102 enrollments in Europe and Australia commencing in the second
quarter
NEW YORK and CLEVELAND, Feb. 01, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics
Inc. (Nasdaq:ABEO), a clinical-stage biopharmaceutical company focused on
developing therapies for life-threatening rare genetic diseases, announced today
that the first high-dose subject was enrolled in the ongoing Phase 1/2 trial for
ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes a single
intravenous injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo
syndrome type A), a devastating lysosomal storage disease that affects every
cell and organ resulting in neurocognitive decline, speech loss, loss of
mobility, and premature death.
"The encouraging clinical data from the low-dose patients continue to support a
whole-body treatment approach using an intravenously delivered AAV to deliver
and drive expression of the SGSH enzyme in all organs of the body, particularly
the brain," stated Kevin M. Flanigan, MD, principal investigator of the clinical
trial, Director of the Center for Gene Therapy and Neuromuscular Disorders
Program at Nationwide Children's Hospital and Professor of Pediatrics and
Neurology at The Ohio State University College of Medicine. "Additionally, we
are encouraged that the 6-month biopotency in two initial subjects are
suggestive of sustained disease modification, and we look forward to presenting
additional data at the WORLDSymposium(TM) lysosomal storage conference later
this month."
The ongoing Phase 1/2 clinical trial, which has received FastTrack designation
by the FDA, is designed to evaluate safety and preliminary indications of
efficacy of ABO-102 in patients suffering from MPS IIIA. Per the design of the
trial, subjects in the low-dose and high-dose cohorts 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, particularly the CNS. Subjects are evaluated
at multiple time points over the initial 6-months post-injection for safety
assessments and initial signals of biopotency.
Previously announced 30-day post-injection data for the low dose cohort
indicated:
* ABO-102 reduced GAG (heparan sulfate) in urine 57.6% +/- 8.2%
* ABO-102 reduced GAG (heparan sulfate) in the CSF 25.6% +/- 0.8%
* Reduction in liver volume of 17.7% +/- 1.9%
* Reduction in spleen volume of 17.6% +/- 7.1%
"The combination of CSF and urinary heparan sulfate GAG reduction, liver and
spleen volume reduction, and neurological effects support our world leading gene
therapy treatment paradigms for patients with MPS III," stated Timothy J.
Miller, Ph.D., President and CEO of Abeona Therapeutics. "We look forward to Dr.
Flanigan's presentation of the biopotency and neurological data at the upcoming
WORLDSymposium."
Abeona's MPS IIIA program, ABO-102, has been granted Orphan Product Designation
in the USA and Europe, and has also received the Rare Pediatric Disease
Designation in the United States.
About ABO-102 (AAV-SGSH): ABO-102 is an adeno-associated viral (AAV)-based gene
therapy for MPS IIIA (Sanfilippo syndrome), which involves a one-time delivery
of a normal copy of the defective gene to cells of the central nervous system
(CNS) with the aim of reversing the effects of the genetic errors that cause the
disease. ABO-102 has been well tolerated in initial subjects of the low-dose
cohort with no safety or tolerability concerns identified through 30 day post-
injection in patients suffering from MPS IIIA, or Sanfilippo syndrome Type A, a
rare autosomal recessive disease caused by genetic mutations that result in a
deficiency of SGSH enzyme activity, leading to abnormal accumulation of GAG
(specifically, heparan sulfate) in the CNS and systemic tissues and organs. This
accumulation of heparan sulfate results in neurocognitive decline, speech loss,
loss of mobility, and premature death. Encouraging signs of early biopotency
have been observed in urinary and CSF GAG (glycosaminoglycan, specifically,
heparan sulfate) measurements, as well as potential disease-modifying effects in
the liver and spleen of the initial subjects enrolled and treated in the trial.
The clinical study is supported by neurocognitive evaluations, biochemical
assessments and MRI data generated in a 25-subject MPS III Natural History
Study, also conducted at Nationwide Children's Hospital, where patients
continued through one-year of follow up assessments.
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; 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 Berni-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: 01.02.2017 - 13:43 Uhr
Sprache: Deutsch
News-ID 521325
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contact information:
Town:
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