Abeona Therapeutics Receives FDA Breakthrough Therapy Designation for EB-101 Autologous Cell Therapy in Epidermolysis Bullosa...
August 29 2017 - 9:05AM
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 U.S. Food and Drug Administration has granted
Breakthrough Therapy designation status to the Company’s EB-101
gene therapy program for patients with Recessive Dystrophic
Epidermolysis Bullosa (RDEB). The designation from the FDA
enables collaborative discussions with senior FDA personnel,
priority review and an expedited approval process to drug
candidates where preliminary clinical trials indicate that a
therapy may offer substantial treatment advantages over existing
options for patients with serious or life-threatening diseases.
“EB-101 is an autologous gene-corrected cell
therapeutic approach that utilizes a patient’s own cells and
genetically engineering them to produce the correct version of
collagen, which helps hold skin on to the body, thereby reducing
the number of painful blisters caused by injury and improving wound
healing,” stated Timothy J. Miller, Ph.D., Abeona’s President and
CEO. “We are grateful that the FDA has recognized the promising
clinical data from the EB-101 program with Breakthrough Therapy
designation and look forward to initiating our pivotal Phase 3
trial as we advance EB-101 for patients with this debilitating
disease.”
The Breakthrough Therapy designation is based on
data from the Phase 1/2 EB-101 clinical trial, which demonstrated
significant wound healing (greater than 50% healed) in treated
wounds for over two years. Breakthrough Therapy designation is
intended to expedite the development and review of drugs for
serious or life-threatening conditions. The criteria for this
particular designation require preliminary clinical evidence
that demonstrates the drug may have substantial improvement on at
least one clinically significant endpoint over available
therapy. A Breakthrough Therapy designation conveys all fast
track program features with more intensive FDA guidance on an
efficient drug development program, an organizational commitment
involving senior managers, and eligibility for rolling review and
priority review. This is the first Breakthrough Therapy designation
for Abeona since the FDA initiated the program in 2013,
highlighting the necessity to develop innovative therapies in
diseases where there is a significantly unmet clinical need like
RDEB.
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. Abeona’s EB-101 product is an
autologous, ex-vivo gene-corrected cell 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).
About EB-101 Phase 1/2 Clinical
Trial:In the recent 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. The trial met
the primary endpoints safety and efficacy, where wound healing
after EB-101 administration was compared to control untreated
wounds from a supporting natural history study that evaluated 128
patients. 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 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
proprietary vector platform, AIM™, for next generation product
candidates. 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: Lynn Granito Berry &
Company Public Relations +1 (212) 253-8881
lgranito@berrypr.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.
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Aug 2024 to Sep 2024
Abeona Therapeutics (NASDAQ:ABEO)
Historical Stock Chart
From Sep 2023 to Sep 2024