NEW YORK and CLEVELAND, Jan. 19, 2017 (GLOBE
NEWSWIRE) --
- ABO-101, Abeona's
third AAV gene therapy program to receive EMA Orphan
Designation
- Clinical trials
anticipated to begin enrolling in second quarter 2017
- Natural History
Study in 25 patients has established efficacy outcome
measures
- FDA previously
granted Orphan Drug and Rare Pediatric Disease
Designations
Abeona Therapeutics Inc.
(Nasdaq:ABEO), a clinical-stage biopharmaceutical company focused
on developing gene therapies for life-threatening rare diseases,
announced today that the European Medicines Agency (EMA) Committee
for Orphan Medicinal Products has granted Orphan Drug Designation
(EMA/OD/226/16) for Abeona's gene therapy program ABO-101 for
children impacted by Sanfilippo syndrome type B (MPS IIIB), a rare
autosomal recessive disease that causes neurocognitive decline,
speech loss, loss of mobility, and premature death in children.
"This designation builds on our
commercial portfolio of AAV gene therapies that have received FDA
and EMA orphan drug designations, which is an important validation
of the scientific and clinical translation of these products for
severely underserved patient populations," stated Timothy J.
Miller, Ph.D., President & CEO of Abeona Therapeutics Inc.
"Accomplishing the designation would not have been possible without
the contributions of Nationwide Children's Hospital researchers
Drs. Doug McCarty and Haiyan Fu, the Stop Sanfilippo Fundacion,
Fundacion Sanfilippo B, Red Sanfilippo Fundacion, the Sanfilippo
Children's Research Foundation, Ben's Dream, the Sanfilippo Medical
Research Foundation, Team Sanfilippo and the National MPS Society
USA."
ABO-101 has previously been
granted the U.S. Food and Drug Administration (FDA) Orphan Product
Designation in the United States and received the Rare Pediatric
Disease Designation as a pre-requisite part of the FDA's Priority
Review Voucher (PRV) process. The FDA has allowed the
Investigational New Drug (IND) for a Phase 1/2 clinical trial, and
enrollments are anticipated to begin in the second quarter of
2017.
About
European Union (EU) Orphan Drug Designation: The European
Commission grants orphan drug designation status to provide
incentives to develop medicinal products to treat, prevent or
diagnose diseases or conditions that affect no more than five in
10,000 persons in the European Union. The orphan drug designation
provides Abeona with incentives and benefits in the EU, including
protocol assistance, reduced fees and protection from market
competition once ABO-101 is approved for the treatment of MPS IIIB
patients.
About
ABO-101 (AAV-NAGLU): ABO-101, the company's
first-in-human, adeno-associated viral (AAV)-based gene therapy for
MPS III (Sanfilippo syndrome), which involves a one-time
intravenous delivery of a normal copy of the Naglu gene to cells of the central nervous system
and peripheral organs with the aim of reversing the effects of the
genetic errors that cause the disease. After a single dose of
ABO-101 in Sanfilippo preclinical models, ABO-101 induced cells in
the CNS and peripheral organs to produce the missing enzyme and
remove the underlying sugar (GAG) storage pathology in cells.
Preclinical in vivo efficacy
studies in Sanfilippo syndrome have demonstrated functional
benefits that remain for months after treatment. A single dose of
ABO-101 significantly restored normal cell and organ function,
corrected cognitive defects that remained months after drug
administration, increased neuromuscular function and normalized the
lifespan of animals with MPS IIIB after treatment compared to
untreated control animals. These results are consistent with
studies from several laboratories suggesting AAV treatment could
potentially benefit patients with Sanfilippo syndrome. Safety and
efficacy studies of AAV gene therapy treatments for Sanfilippo
syndrome have recently been published in several peer-reviewed
scientific journals.
About
Sanfilippo syndromes (or
mucopolysaccharidosis): Mucopolysaccharidosis (MPS)
type III (Sanfilippo syndrome) is 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. Mucopolysaccharides (GAGs) are long
chains of sugar molecules used in building 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 of GAGs within the central nervous system
(CNS), including the brain and spinal cord, and other tissues,
which result 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
include, without limitation, our belief that the designation by the
EMA is an important validation of the scientific and clinical
translation of our products for severely underserved patient
populations. 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 ability to successfully continue our 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.