ABEO Abeona Therapeutics Inc.

Abeona Therapeutics Announces Upcoming Data Presentations at the Society for Pediatric Dermatology 45th Annual Meeting

Abeona Therapeutics Announces Upcoming Data Presentations at the Society for Pediatric Dermatology 45th Annual Meeting

NEW YORK and CLEVELAND, June 18, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that two abstracts related to its clinical program for recessive dystrophic epidermolysis bullosa (RDEB) will be presented at the upcoming Society for Pediatric Dermatology (SPD) 45th Annual Meeting, to be held virtually during July 10-12, 2020. RDEB is a rare connective tissue disorder without an approved treatment, in which patients suffer from severe epidermal wounds that severely impact their lives.

The first abstract features a more detailed examination than previously disclosed of data on long-term pain relief following durable healing of wounds in RDEB patients treated with EB-101 in a Phase 1/2 study. Separately, a literature review on the clinical, humanistic and economic burden of RDEB will be presented.

The posters will be presented by Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University Medical Center and Principal Investigator of the EB-101 pivotal Phase 3 VIITALTM study, and M. Peter Marinkovich, M.D., Investigator on the VIITALTM study and Bullous Disease Clinic Director, Stanford University Medical Center. Details for the presentations are as follows:

Title: Durable Healing and Pain Reduction in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Following EB-101 Treatment of Large, Chronic Wounds

Presenter: Jean Tang, M.D. Ph.D.

Virtual Session Date: July 10-12, 2020

Title: The Full Burden of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Presenter: M. Peter Marinkovich, M.D.

Virtual Session Date: July 10-12, 2020

Following the conclusion of each virtual presentation, the posters will be available on the “Events” page under the “Investors & Media” section of Abeona’s website at .

More details about the programs for the SPD Annual Meeting are available at .

About EB-101

EB-101 is an autologous, gene-corrected cell therapy currently being investigated in the pivotal Phase 3 VIITALTM study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITALTM study is a multi-center, randomized clinical trial enrolling 10 to 15 RDEB patients with approximately 30 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver COL7A1 genes into a patient’s own skin cells (keratinocytes and its progenitors) and transplanting them back to the patient to enable normal Type VII collagen expression and facilitate wound healing. Abeona produces EB-101 for the VIITALTM study at its fully-functional gene and cell therapy manufacturing facility in Cleveland, OH. In a Phase 1/2a clinical trial, EB-101 provided durable wound healing for RDEB patients lasting 2+ to 5+ years, including for the largest, most challenging wounds that affect the majority of the RDEB population. More information on the clinical trials of EB-101 can be found at and .

About Recessive Dystrophic Epidermolysis Bullosa

Recessive 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.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeona’s clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Company’s portfolio of AAV-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Abeona’s library of novel, next-generation AIM™ capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeona’s fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITALTM study and is capable of clinical and commercial production of AAV gene therapies. For more information, visit .

Forward-Looking Statements

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 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 and its products and product candidates, future regulatory interactions with regulatory authorities, as well as 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 the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, 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 the forward-looking statements or to 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.

Investor Contact:

Greg Gin

VP, Investor Relations

Abeona Therapeutics

+1 (646) 813-4709

Media Contact:

Scott Santiamo

Director, Corporate Communications

Abeona Therapeutics

+1 (718) 344-5843

EN
18/06/2020

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