Pivotal Phase 3 clinical trial is planned to commence in early 2018
EB-101 gene therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) has demonstrated
promising efficacy and safety in the Phase 1/2 clinical trial
EB-101 has received Orphan Drug and Rare Pediatric Disease Designations in the USA and Orphan Drug
Designation in the EU
NEW YORK and CLEVELAND, July 18, 2017 (GLOBE NEWSWIRE) -- 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.
