Abeona Therapeutics Receives Rare Pediatric Disease Designation for EB-101 Gene Therapy Product for Patients with Epidermolys...
May 30 2017 - 8:05AM
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 FDA has granted Rare Pediatric Disease Designation 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.
“These designations are granted to drugs with
high promise that may address areas of unmet medical need for
children with rare diseases. RDEB is a debilitating and life
threatening inherited disorder with no approved treatment options
available for patients today," stated Timothy J. Miller, Ph.D.,
President & CEO of Abeona Therapeutics Inc. “Building upon the
already granted FDA and EMA Orphan Drug Disease Designations for
the EB-101 gene therapy program, receiving the Rare Pediatric
Disease Designation is another important validation of the science
and clinical approach to developing a novel gene therapy for RDEB
patients.”
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 ongoing 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.
About Rare Pediatric Disease
Designation: The rare pediatric disease designation
indicates that the FDA may give the company a pediatric priority
review voucher if the drug is approved for the pediatric
indication. That voucher could then be used by the company for
another drug—any drug—to be given a priority review. A priority
review mandates that the FDA will review a BLA drug submission
within six months instead of the standard 10 months. Normally, a
priority review designation would only be given to a drug that is
for a serious condition and has demonstrated the potential to be a
significant improvement in safety and effectiveness. The priority
review voucher may be used by the sponsor, sold or transferred.
EB-101 Gene Therapy Program
Highlights:
- Clinical data were recently presented at the Society of
Investigative Dermatology (SID) conference by the Company’s
Stanford University collaborators, and demonstrated that EB-101
treated wounds were significantly healed >50% for more than 2
years post-administration.
- EB-101 demonstrated significant wound healing (defined as
greater than 50% healed) in 100% of treated wounds (36/36) at 3
months; 89% (32/36) at 6 months, 83% (20/24) at 12 months, 88%
(21/24) at 24 months and 100% (6/6) 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.
- Supportive Natural History Study: A 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.
- The EB-101 program has also been granted orphan drug
designation from FDA and the European Medicines Agency
(EMA).
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. Patients are being enrolled 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 by the FDA and 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, the expected receipt of a
Priority Review Voucher 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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