Abeona Therapeutics Receives FDA Orphan Drug Designation for ABO-201 Juvenile Batten Disease Gene Therapy Program
June 29 2017 - 8:15AM
Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare diseases, announced today that
the FDA has granted Orphan Drug Designation (ODD) for Abeona’s
ABO-201 program (AAV-CLN3), the AAV-based single intravenous gene
therapy program for juvenile Batten disease, a fatal lysosomal
storage disease of the nervous system caused by autosomal-recessive
mutations in the CLN3 gene.
“Receiving FDA orphan drug designation for
ABO-201 augments Abeona’s suite of regulatory achievements,
providing certain benefits and incentives, including marketing
exclusivity, that are strategically important from a regulatory and
commercial perspective,” stated Timothy J. Miller, Ph.D., President
& CEO of Abeona Therapeutics Inc. “The published ABO-201
preclinical data from Dr. Tammy Kielian’s lab support the clinical
translation for patients with juvenile Batten disease, and
demonstrated the importance of selecting the right vector and
delivery route for potential CNS benefit and to remove the
underlying pathology associated with the disease. This
designation helps advance the ABO-201 program and we look forward
to initiating human clinical trials later this year.”
Preclinical data supporting clinical trials for
ABO-201 (AAV-CLN3), the AAV-based single intravenous gene therapy
program for juvenile Batten disease, (juvenile neuronal ceroid
lipofuscinosis, JNCL), were published in the September 2016 issue
of the Journal of Neuroscience (doi:
10.1523/JNEUROSCI.1635-16.2016). The publication article can be
accessed by clicking on the following link:
http://www.jneurosci.org/content/36/37/9669.short.
About Orphan Drug Designation:
Orphan drug designation is granted by the FDA to novel drugs or
biologics that treat rare diseases or conditions affecting fewer
than 200,000 patients in the U.S. The designation allows the drug
developer to be eligible for a seven-year period of U.S. marketing
exclusivity upon approval of the drug, as well as tax credits for
clinical research costs, the ability to apply for annual grant
funding, clinical trial design assistance, and the waiver of
Prescription Drug User Fee Act (PDUFA) filing fees.
About ABO-201: ABO-201 (AAV
CLN3) is an AAV-based gene therapy which has shown promising
preclinical efficacy in delivery of a normal copy of the defective
CLN3 gene to cells of the central nervous system with the aim of
reversing the effects of the genetic errors that cause juvenile
neuronal ceroid lipofuscinosis, also known as juvenile Batten
disease (JBD). JBD is a rare, fatal, autosomal recessive
(inherited) disorder of the nervous system that typically begins in
children between 4 and 8 years of age. Often the first noticeable
sign of JBD is vision impairment, which tends to progress rapidly
and eventually results in blindness. As the disease progresses,
children experience the loss of previously acquired skills
(developmental regression). This progression usually begins with
the loss of the ability to speak in complete sentences. Children
then lose motor skills, such as the ability to walk or sit. They
also develop movement abnormalities that include rigidity or
stiffness, slow or diminished movements (hypokinesia), and stooped
posture. Beginning in mid- to late-childhood, affected children may
have recurrent seizures (epilepsy), heart problems, behavioral
problems, and difficulty sleeping. Life expectancy is greatly
reduced, and there are no approved treatments for JNCL.
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), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts)
for recessive dystrophic epidermolysis bullosa (RDEB). Abeona
is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type
B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JBD), ABO-202 (AAV-CLN1) for treatment of infantile Batten
disease (INCL), EB-201 for epidermolysis bullosa (EB), 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.
Investor Contact:Christine
SilversteinVice President, Investor RelationsAbeona Therapeutics
Inc. +1 (212)-786-6212csilverstein@abeonatherapeutics.com
Media Contact: Andre’a Lucca Vice President,
Communications & Operations Abeona Therapeutics Inc. +1
(212)-786-6208alucca@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, FDA orphan drug designation for ABO-201
provides Abeona certain benefits and incentives, including
marketing exclusivity, that are strategically important from a
regulatory and commercial perspective, our preclinical work for
ABO-201 and the recently published data supporting its clinical
translation for patients with juvenile Batten disease demonstrated
the importance of selecting the right vector and delivery route to
target tissues in the CNS and treat the symptoms associated with
the underlying disease pathology, we look forward to advancing the
ABO-201 program and initiating human clinical trials later this
year, 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 ability to secure licenses for any technology that
may be necessary to commercialize our products; 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.
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Aug 2024 to Sep 2024
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Sep 2023 to Sep 2024