Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Ty...
January 19 2017 - 7:43AM
- 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™
(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 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.
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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