Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epiderm...
March 08 2017 - 8:15AM
Abeona’s Fourth Gene Therapy Program to
Receive EMA Orphan Designation
Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading 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 for Abeona’s EB-101 gene therapy
program for patients with recessive dystrophic epidermolysis
bullosa (RDEB), a devastating, life-threatening genetic skin
disorder that is characterized by skin blisters and erosions all
over the body.
“EB-101 is Abeona’s fourth gene therapy program
to be granted EMA Orphan Designation and it further builds on our
commercial portfolio of clinical-stage gene therapies that have
received FDA and EMA orphan drug designations, which is an
important validation of the clinical translation of these
treatments for severely underserved patient populations,” stated
Timothy J. Miller, Ph.D., President & CEO of Abeona
Therapeutics Inc. “The orphan designation also provides 10 years of
market exclusivity in the European Union from similar medicines for
similar indications, an important value driver for Abeona as we
expand our clinical programs in Europe.”
The ongoing phase 1/2 clinical trial with
gene-corrected skin grafts has shown promising wound healing and
safety in patients with RDEB. Investigators at Stanford University
are enrolling adolescent and adult patients for the phase 2 portion
of the EB-101 clinical trial to determine the efficacy of COL7A1
gene-corrected grafts on wound healing (NCT01263379).
Typically, wounds in patients with RDEB, also
known as "butterfly skin" syndrome, can remain unhealed for months
to years due to the inability of the skin to stay attached to the
underlying dermis and can cover a large percentage of the body.
Results from the initial four patients of the clinical study
demonstrated that treatment with EB-101 restored type VII collagen
(C7) expression at the dermal-epidermal junction at the graft sites
in 90% of the biopsy samples at 3 months post-treatment, in 66% at
6 months post-treatment, and in 42% samples at 12 months
post-treatment. Importantly, correct type VII collagen localization
was observed at anchoring fibrils. Wounds that demonstrated type
VII collagen at graft sites displayed 87% healing at 3 months, 67%
at 6 months, and 50% at 12 months compared with baseline wound
sites.
“The encouraging EB-101 clinical results
advances our support to address the significant unmet medical needs
that RDEB patients experience and underscores our commitment to
collaborating with outstanding research groups such as Stanford
University,” noted Steven H. Rouhandeh, Executive Chairman of
Abeona Therapeutics. “We are also very proud to collaborate with
dedicated patient advocacy groups such as EB Research Partnership
(EBRP) and EB Medical Research Foundation (EBMRF) to advance EB-101
for the RDEB community.”
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.
Authorised orphan medicines, such as EB-101, benefit from ten years
of protection from market competition with similar medicines for
similar indications once they are approved, and provides Abeona
with incentives and benefits in the EU that include protocol
assistance and reduced fees once EB-101 is approved for EB
patients.
About EB-101: EB-101 is an
autologous, ex-vivo gene therapy in which COL7A1 is transduced into
autologous keratinocytes for the treatment of recessive dystrophic
epidermolysis bullosa (RDEB). EB-101 has been well tolerated to
date and demonstrated promising efficacy in the ongoing phase 1/2
clinical trial in RDEB patients (NCT01263379).
About Epidermolysis Bullosa
(EB): EB is a group of devastating, life-threatening
genetic skin disorders that is characterized by skin blisters and
erosions all over the body. The most severe form, recessive
dystrophic epidermolysis bullosa (RDEB), is characterized by
chronic skin blistering, open and painful wounds, joint
contractures, esophageal strictures, pseudosyndactyly, corneal
abrasions and a shortened life span. Patients with RDEB lack
functional type VII collagen (C7) owing to mutations in the gene
COL7A1 that encodes for C7 and is the main component of anchoring
fibrils that attach the dermis to the epidermis. EB patients suffer
through intense pain throughout their lives, with no effective
treatments available to reduce the severity of their symptoms.
Along with the life-threatening infectious complications associated
with this disorder, many individuals often develop an aggressive
form of squamous cell carcinoma (SCC).
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 SilversteinVice President, Investor
RelationsAbeona Therapeutics Inc.+1
(212)-786-6212csilverstein@abeonatherapeutics.com
Media Contact: Andre’a LuccaVice President, Communications &
OperationsAbeona Therapeutics Inc.+1
(212)-786-6208alucca@abeonatherapeutics.com
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