Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare diseases, announced today
guidance from a recent Type-C meeting with the FDA which has
recommended accelerating the EB-101 program into a pivotal Phase 3
trial. The Company continues to engage the FDA on the final
Phase 3 clinical trial design, planned to commence early 2018, and
will provide an update on the program in the coming months.
“The FDA guidance is an important milestone in
our clinical development plan for EB-101, and we are pleased to be
moving forward into a registrational Phase 3 clinical study in
2018. 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 addresses the underlying disease pathology to offer
significant therapeutic benefit for RDEB patients, and we look
forward to the collective work ahead in advancing this therapy,”
stated Timothy J. Miller, Ph.D., President and CEO of Abeona
Therapeutics Inc.
Abeona’s EB-101 product is an autologous,
ex-vivo gene therapy in which the COL7A1 gene is inserted into a
patient’s own skin cells (keratinocytes) for the treatment of the
underlying disease in Recessive Dystrophic Epidermolysis Bullosa.
The EB-101 program has been granted Orphan Drug and Rare Pediatric
Disease Designations from the US Food and Drug Administration (FDA)
and Orphan Drug Designation from the European Medicines Agency
(EMA).
“EB Research Partnership (EBRP), along with EB
Medical Research Foundation, are honored to have helped support
EB-101 development by the dedicated researchers at Stanford
University. Their tireless efforts in combination with Abeona’s
leadership is driving real progress in the RDEB patient
community. EBRP is encouraged by clinical results to date and
looks forward to realizing the promise of EB-101 in addressing the
devastating effects on RDEB patients’ quality of life and disease
burden,” stated Alexander Silver, co-founder and chairman EB
Research Partnership.
About EB-101 Phase 1/2 Clinical
Trial:In the Phase 1/2 clinical trial, EB-101 was
administered to non-healing chronic wounds on each subject which
were assessed for wound healing at predefined time points up to
several years to date. The primary endpoints of the clinical trial
assessed safety and evaluated wound healing after EB-101
administration compared to control untreated wounds. Secondary
endpoints included expression of collagen C7 and restoration of
anchoring fibrils at three and six months post-administration.
Clinical data were 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 two years
post-administration. The data included:
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 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 and to the FDA. Notably, 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 Epidermolysis Bullosa (EB) and
Recessive Dystrophic Epidermolysis Bullosa (RDEB): EB is a
group of devastating, life-threatening genetic skin disorders
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, pseudosyndactyly and a
shortened life span. 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.
Patients with RDEB lack functional type VII collagen owing to
mutations in the gene COL7A1 that produces C7 collagen and is the
main component of anchoring fibrils, the “velcro” that helps
stabilize the skin on the basement membrane.
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 EB Research Partnership
(EBRP): EBRP is the largest 501(c)(3) nonprofit dedicated
to funding research aimed at treating and ultimately curing
Epidermolysis Bullosa, a group of devastating and life-threatening
skin disorders that affect children from birth. EBRP uses a
sustainable philanthropic model via venture philanthropy for all of
its research commitments. To learn more, please visit
www.ebresearch.org.
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 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
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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