Full Press Release Details
Therapeutics Cleared to Initiate Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis
removes clinical hold; Company may proceed with VIITALTM study
expects to initiate study in the first quarter of 2020
endpoint confirmed as proportion of wounds with greater than 50% healing at 3 months vs control wounds
of potential subjects have been pre-screened for the study
YORK and CLEVELAND, Dec. 9, 2019 - Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy,
today announced that the U.S. Food and Drug Administration (FDA) has removed its clinical hold and provided clearance to proceed
with the VIITALTM study, the Company's pivotal Phase 3 clinical trial evaluating EB-101 for the treatment of
recessive dystrophic epidermolysis bullosa (RDEB). The FDA removed the clinical hold following the Company's submission
of additional data points on transport stability of EB-101 to clinical sites. Abeona expects to initiate the VIITALTM study
in first quarter of 2020.
Abeona team has worked diligently to provide a prompt and thorough response to the FDA, enabling us to proceed with our pivotal
Phase 3 trial for EB-101," said Jo o Siffert, M.D., Chief Executive Officer of Abeona. "Recently published
long-term follow up data from our Phase 1/2 trial leaves us increasingly confident that EB-101 can provide durable healing for
large, chronic wounds that afflict many RDEB patients. We are now focused on initiating the VIITALTM study in the first
quarter of 2020. The success in building and qualifying a state-of-the-art GMP manufacturing facility also represents a critical
step toward bringing this novel product to patients in dire need of effective treatment."
two to five years of follow-up, data from a Phase 1/2 clinical trial conducted by Stanford University evaluating EB-101 showed
that the gene-corrected cell therapy provided durable wound healing for RDEB patients, including for the largest, most challenging
wounds that constitute the majority of wounds in this population.
VIITALTM Phase 3 study will be a multi-center, randomized clinical trial assessing EB-101 in 10 to 15 RDEB patients,
with approximately 30 chronic wound sites treated in total. The primary study endpoint will be the proportion of wounds with greater
than 50% healing at three months, comparing treated with untreated wound sites on the same patient. Secondary endpoints include
the patient's global impression of change in pain from baseline as well as other patient reported outcomes assessing pain
during dressing changes, pain impact and physical function.
is an autologous, gene-corrected cell therapy in late-stage clinical development for the treatment of recessive dystrophic epidermolysis
bullosa (RDEB), a rare connective tissue disorder without an approved therapy. Treatment with EB-101 involves using gene transfer
to deliver COL7A1 genes into a patient's own skin cells (keratinocytes) and transplanting them back to the patient to enable
normal Type VII collagen expression and facilitate wound healing. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy,
Breakthrough Therapy, and Rare Pediatric designations for EB-101 and Orphan Drug designation in both the U.S. and EU.
Recessive Dystrophic Epidermolysis Bullosa
dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder characterized by severe skin wounds that cause pain
and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1
gene, leaving them unable to produce functioning Type VII collagen which is necessary to anchor the dermal and epidermal layers
of the skin. There is currently no approved treatment for RDEB.
Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company's
clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa,
as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB),
respectively. The Company's portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and
CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM AAV vector platform to address
all mutations of cystic fibrosis. Abeona has received twenty regulatory designations from the FDA and EMA for its pipeline candidates.
For more information, visit www.abeonatherapeutics.com.
press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of
1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties.
These statements include statements about the Company's clinical trials, including the timing and success thereof; the Company's
products and product candidates; EB-101 can provide durable healing in large, chronic wounds that afflict many RDEB patients;
future regulatory interactions with regulatory authorities; and the Company's goals and objectives. We have attempted to
identify forward looking statements by such terminology as "may," "will," "believe," "estimate,"
"expect," and similar expressions (as well as other words or expressions referencing future events, conditions or
circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially
from those indicated by such forward-looking statements as a result of various important factors, 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 outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition,
the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve
or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks
associated with data analysis and reporting, and other risks as may be detailed from time to time in the Company's Annual
Reports on Form 10-K and quarterly reports on Form 10-Q and other periodic reports filed by the Company with the Securities and
Exchange Commission. The Company undertakes no obligation to revise these forward-looking statements or update them to reflect
events or circumstances occurring after the date of this presentation, whether as a result of new information, future developments
or otherwise, except as required by the federal securities laws.
Director, Corporate Communications