Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage
biopharmaceutical company focused on developing novel gene
therapies for life-threatening rare genetic diseases, today
announced that updated Phase 1/2 clinical trial data on the EB-101
gene therapy program and supportive natural history data for
patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)
will be highlighted at the upcoming Society for Investigative
Dermatology (SID) conference to be held April 26-29, 2017 in
Portland, OR.
“Recessive Dystrophic Epidermolysis Bullosa,
also known as butterfly skin syndrome, is a painful and ultimately
disfiguring disease that results in early deaths. The EB-101
clinical data update highlights the wound closure and collagen
biomarker expression continues for over two years in multiple
patients, which are critically important parameters of efficacy in
patients with RDEB,” said Timothy J. Miller, Ph.D., President and
CEO.
EB-101 was administered to non-healing chronic
wounds [mean length of time wounds were unhealed (unclosed) was 8.5
years] on each subject and assessed for wound healing at predefined
time points over years. The primary endpoint of the clinical trial
is to assess safety and evaluate wound closure compared to
untreated wounds. Secondary endpoints include expression of
full-length C7 and restoration of anchoring fibrils at three and
six months post-administration.
Significant wound healing, defined as >75%
closure from baseline, was observed in 94% (27/36 grafts) at 3
months, 67% (16/24 grafts) at 6 months and 50% (12/24 grafts) at 12
months post-administration. In addition, C7 collagen expression and
morphologically normal NC2 reactive anchoring fibrils – the
“zipper” that holds skin onto the underlying tissue and the primary
deficit in RDEB patients - have been observed in tissue biopsies in
the 4 subjects that are through two years of follow-up. By
comparison, all untreated control wounds remained unhealed (0%
wound closure) over the same time frame. Importantly, all subjects
(n=35) in the supportive natural history study failed to close
wounds (0% wound healing) using a comparison product of non-gene
corrected keratinocyte graft (Apligraf®) treatment by 7 weeks
post-application. This is a significant 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.
Details for the oral presentations and poster
sessions are listed below:
Presentation Title: “Phase I/IIa Clinical Trial for
Recessive Dystrophic Epidermolysis Bullosa
Using Genetically Corrected Autologous
Keratinocytes”Presenter: Zurab Siprashvili, Ph.D.Abstract Final ID:
519Oral Presentation, Friday, April 28th, 2:00 p.m. – 4:30 p.m. PT
Room: Oregon Ballroom 204
Abstract Title: “Quality of Life in Recessive Dystrophic
Epidermolysis Bullosa: The AltaVoice Patient Registry,
2012-2015”Presenter: Sara ChoiAbstract Final ID: 224Poster
Session I: Thursday, April 27th, 2017, 10:15 a.m. – 12:15 p.m.
PTRoom: Exhibit Hall A
Abstract Title: “Natural History of Chronic Wounds in
Patients with Recessive Dystrophic Epidermolysis Bullosa”Presenter:
Daniel C. Solis, BAAbstract Final ID: 219Poster Session II: Friday,
April 28th, 2017, 11:30 a.m. – 1:30 p.m. PT Room: Exhibit Hall
A
Abstract Title: Phase I/IIa Clinical Trial for
Recessive Dystrophic Epidermolysis Bullosa
Using Genetically Corrected Autologous
Keratinocytes”Presenter: Zurab Siprashvili, Ph.D.Abstract Final ID:
519Poster Session III: Saturday, April 29th, 10:15 a.m. – 12:15
p.m. PTRoom: Exhibit Hall A
About EB-101: Recessive
Dystrophic Epidermolysis Bullosa (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. 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 Phase 2 portion of
the ongoing Phase 1/2 clinical trial in RDEB patients
(NCT01263379).
About Society of Investigative
Dermatology (SID): The Society for Investigative
Dermatology was founded in 1937 to highlight and promote discovery
in investigative dermatology and to better represent skin-related
scientific investigation amongst medical societies. Soon after, it
launched the Journal of Investigative Dermatology (JID), a source
for all advancements in cutaneous biology. The first Annual Meeting
was held in New York City in 1938. The 76th Annual SID
Meeting, like those before it, will bring together researchers,
lecturers, and industry leaders in dermatology. Throughout the
four-day conference, cutting-edge research, findings and trends in
skin health and disease will be shared. All abstracts will be
published in upcoming editions of the JID. For more information,
please visit the conference website
at http://www.sidnet.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) 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.
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)-786-6208alucca@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 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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