Abeona’s Third Gene Therapy Program to
Receive FDA Orphan Designation
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
EB-101 gene therapy program for patients with dystrophic
epidermolysis bullosa (DEB), including recessive dystrophic
epidermolysis bullosa (RDEB), which are life-threatening
genetic skin disorders characterized by skin blisters and erosions
that cover the body.
“Abeona is committed to advancing innovative
gene therapies that address the unmet needs of patients suffering
with dystrophic epidermolysis bullosa, a devastating rare skin
disease. We are grateful that the FDA has recognized EB-101
as a rare disease product that may offer a significant therapeutic
benefit for patients with dystrophic forms of epidermolysis
bullosa, including RDEB. EB-101 is Abeona’s third gene
therapy program to be granted Orphan Drug Designation by the FDA,
an important value driver for the program as it provides seven
years of market exclusivity from similar medicines for similar
indications,” stated Timothy J. Miller, Ph.D., President & CEO
of Abeona Therapeutics Inc.
Typically, wounds on 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.
In the Phase 1/2 clinical trial, EB-101 was administered to
non-healing chronic wounds on each subject and assessed for wound
healing at predefined time points over years. The primary endpoints
of the clinical trial assess safety and evaluate wound healing
after EB-101 administration compared to control untreated wounds.
Secondary endpoints include expression of collagen C7 and
restoration of anchoring fibrils at three and six months
post-administration.
Clinical data were recently presented at the
Society of Investigative Dermatology (SID) conference by Stanford
collaborators, and demonstrated that EB-101 treated wounds were
significantly healed >50% for more than 2 years
post-administration. The data showed:
Wound healing, defined as >50% closure after
EB-101 administration, was observed in:--100% (36/36 treated
wounds, n=6 subjects) at 3 months, --89% (32/36 treated wounds, n=6
subjects) at 6 months,--83% (20/24 treated wounds, n=4 subjects) at
12 months, --88% (21/24 treated wounds, n=4 subjects) at 24
months,--100% (6/6 treated wounds, n=1 subject) at 36 months
post-administration.
Collagen VII (C7) expression: C7 and
morphologically normal NC2 reactive anchoring fibrils – the
“zipper” that holds skin onto the underlying tissue and the primary
deficit in RDEB patients – were observed in EB-101 treated wounds
up to two years post administration.
Importantly, data from a supportive natural
history study of 1,436 wounds from 128 patients with RDEB,
established by Stanford and EBCare Registry, were also presented at
the conference. Notably, in the natural history study, 13 RDEB
patients with a total of 15 chronic wounds were treated with an
allograft product, including Apligraf® and Dermagraft®. Of these
wounds treated with allografts, only 7% (1/15 treated wounds)
remained healed after 12 weeks, and 0% (0/15 treated wounds)
remained healed after 24 weeks. This is a meaningful finding of the
natural history study, as there are no approved therapies for RDEB
patients that demonstrate significant wound closure after two
months post-application.
About Orphan Drug Designation:
Under the FDA’s Orphan Drug Designation program, 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 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). RDEB is a subtype of an inherited
genetic skin disorder 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 owing to
mutations in the gene COL7A1 that encodes for C7 and is the main
component of anchoring fibrils, which stabilize the
dermal-epidermal basement membrane. Investigators at Stanford
University are enrolling patients in the ongoing Phase 2 portion of
the Phase 1/2 clinical trial (NCT01263379). The EB-101 program has
also been granted orphan drug designation from the European
Medicines Agency (EMA).
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), 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 (JNCL), 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 include, without limitation, our
plans for continued development and internationalization of our
clinical programs, that patients will continue to be identified,
enrolled, treated and monitored in the EB-101 clinical trial, and
that studies will continue to indicate that EB-101 is
well-tolerated and may offer significant improvements in wound
healing. 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 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.
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