Corporate Presentation June 2020 Abeona Therapeutics Corporate - PowerPoint PPT Presentation

Corporate Presentation June 2020 Abeona Therapeutics Corporate Presentation, June 2020

Safe Harbor Statement This presentation contains certain statements that may be forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, including statements relating to the product portfolio and pipeline and clinical programs of the company, the market opportunities for all of the company’s products and product candidates, and the company’s goals and objectives. These statements are subject to numerous risks and uncertainties, including but not limited to the risks detailed in the Company’s Annual Report on Form 10-K for the year ended December 31, 2019, including risks associated with the ongoing coronavirus/COVID-19 pandemic (as further explained in the Company’s “Stakeholder Letter in Response to the COVID-19 Pandemic” dated March 27, 2020) and other reports filed by the company with the Securities and Exchange Commission. This presentation does not constitute an offer or invitation for the sale or purchase of securities or to engage in any other transaction with Abeona Therapeutics or its affiliates. The information in this presentation is not targeted at the residents of any particular country or jurisdiction and is not intended for distribution to, or use by, any person in any jurisdiction or country where such distribution or use would be contrary to local laws or regulations. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this presentation 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. 2 Abeona Therapeutics Corporate Presentation, June 2020

Abeona: A Fully-Integrated Gene & Cell Therapy Company G ENE & C ELL T HERAPY C OMPREHENSIVE G ENE & L ATE -S TAGE B REAKTHROUGH T HERAPY AAV9 AND P ROPRIETARY R OBUST P IPELINE OF E XPERTISE AND AAV (AIM TM ) C ELL T HERAPY F IRST - TO -M ARKET WITH G ENE C ORRECTED C LINICAL S TAGE AND M ANUFACTURING C APABILITIES O PPORTUNITIES C ELL T HERAPY P ROGRAMS P RECLINICAL P ROGRAMS C APABILITIES • Pivotal Phase III clinical trial in RDEB initiated 1Q20 • Ongoing Phase I/II clinical trials in MPS IIIA and MPS IIIB • CLN program approaching the clinic • Next generation AIM™ AAV capsid platform: in vivo proof of concept data in various disorders • Fully-functional GMP facility validated and governed by comprehensive quality systems ⎼ Phase III manufacture for EB-101 ⎼ Capable of clinical and commercial production of AAV gene therapies • $116M in cash, cash equivalents and marketable securities (March 31, 2020) 3 Abeona Therapeutics Corporate Presentation, June 2020

Robust Pipeline 4 Abeona Therapeutics Corporate Presentation, June 2020

✓ O RPHAN D RUG D ESIGNATION (FDA) ✓ O RPHAN D RUG D ESIGNATION (EU) ✓ R ARE P EDIATRIC D ISEASE D ESIGNATION (FDA) ✓ B REAKTHROUGH T HERAPY D ESIGNATION (FDA) ✓ R EGENERATIVE M EDICINE A DVANCED T HERAPY D ESIGNATION (FDA) • RDEB Review • EB-101 Clinical Program Abeona Therapeutics Corporate Presentation, June 2020

EB: Devastating Inherited Connective Tissue Disorder Recessive Dystrophic Epidermolysis Bullosa (RDEB): • Primarily characterized by skin blisters and erosions • Caused by mutations in COL7A1 gene, which encodes type VII collagen • Estimated 2,500 patients in the U.S. Patients suffer from having up to 80% of their body covered in wounds, leading to: • Severe pain and widespread scarring • Numerous debilitating and life-threatening systemic complications • Inflammation, infections, loss of heat - high metabolic rate and malnutrition • 75-90% risk of developing squamous cell carcinoma (SCC) normal skin affected skin The lack of functioning anchoring fibrils in RDEB patients leads to 50% 75% of generalized severe skin blistering and tears with minor trauma die before 40 patients die before 35 6 Abeona Therapeutics Corporate Presentation, June 2020

Lifelong Burden with No Relief and No FDA Approved Treatments • Current wound care is wrapping and bandaging to prevent trauma, promote healing, prevent infection • These treatments are inadequate and: Reliance on opioids Do not address Are time intensive Are costly with due to pain underlying disease and burdensome spend over associated with cause with daily changes $150,000 per year cleaning wounds 7 Abeona Therapeutics Corporate Presentation, June 2020

Recurrent and Chronic Wounds Have Distinct Time Courses Recurrent Recurrent Wounds Over Time (N=25) % Baseline Wound Area baseline 2 months 3 months Time to heal (6 weeks) Time to re-blister (3 weeks) Weeks Chronic % Baseline Wound Area Chronic Wounds Over Time (N=25) baseline 2 months 5 months Weeks Recurrent Wounds Over Time (N=25) Natural history of chronic wounds in patients with recessive dystrophic epidermolysis bullosa; Solis, D. et al.; Journal of Investigative Dermatology, Volume 137, Issue 5, S37 8 Abeona Therapeutics Corporate Presentation, June 2020

Size Distribution According to Wound Types Sequentially Photographed Wounds: N=25 patients, 62 wounds 70% Large wounds are the most urgent to 60% treat as they carry the greatest burden, including Pain, Pruritis, and Risk of 50% % of Wounds infection 40% 30% 20% 10% 0% ≤19 cm 2 20-39 cm 2 ≥40 cm 2 Recurrent 15% 26 cm 2 5 years 64% 21% 118 cm 2 7 years Chronic Open 20% 53% 27% Mean Size Mean Duration p = 0.001 p = NS Natural history of wounds in patients with recessive dystrophic epidermolysis bullosa; Teng et al., Abstract #251; Society of Investigational Dermatology Annual Meeting, 2019 9 Abeona Therapeutics Corporate Presentation, June 2020

Chronic Open Wounds Have Greater Pain and Itch Sequentially Photographed Wounds: N=25 patients, 62 wounds Chronic Open Recurrent 2.4 6 2.5 5.0 Wong Baker FACES™ 5 Pain Rating Scale 2 Itch Man Scale 1.5 4 1.5 2.3 3 1 2 0.5 1 0 0 Pain Itch Pain Itch Natural history of wounds in patients with recessive dystrophic epidermolysis bullosa; Teng et al., Abstract #251; Society of Investigational Dermatology Annual Meeting, 2019 10 Abeona Therapeutics Corporate Presentation, June 2020

Developing an Ideal RDEB Treatment The ideal RDEB treatment will: • Target the underlying genetic cause of disease • Correct the genetic defect in the appropriate cells – e.g. keratinocytes • Durably restore COL7 function by correcting keratinocyte progenitors • Address the most common and challenging patient wounds Chronic wounds, which average ~ 118 cm 2 ⎼ Recurrent wounds, average ~30 cm 2 ⎼ 11 Abeona Therapeutics Corporate Presentation, June 2020

EB-101: Ex-Vivo Autologous Gene Corrected Breakthrough Therapy G ENE - CORRECTED CELL THERAPY THAT RESTORES NORMAL FUNCTIONAL C OLLAGEN VII TO KERATINOCYTES AND THEIR PROGENITORS • • B IOPSY TO PATIENT - READY IN ~26 DAYS • 2 SKIN BIOPSIES = 6 SHEETS = 240 CM 2 PERSONALIZED TREATMENT EB-101 i EB 101 is transp nsplant nted o onto Two 8 8mm mm s skin b n biopsies s shi hipped t to patie ients’ w wounds i in as l little le a as Abeona, p a, produced ed i into 6 6-8 8 ~40 c 0 cm 2 2 26 days. 26 d sheet eets. . Keratin inocyte c cell lls expanded and p prepared t the f for n next t step. 10 10-12 d 12 days o s of continued c cell l maturatio ion a and growth. Functio ional T Type V VII c colla llagen transduce ced i into to k kerati tinocy cyte te cells ls; c cell m l maturatio ion l leads t to gene-correc ected ed t type V VII c collag agen en Kerati tinocy cyte te c cells e expanded a and expres ession. . harves ested ed as 5 5.5x7.5 .5cm s sheet ets. 12 Abeona Therapeutics Corporate Presentation, June 2020

The Phase III VIITAL TM Study: A Pivotal Clinical Evaluation of EB-101 for RDEB A Phase III Clinical Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Study Description Bullosa (RDEB) using EB-101 for autologous tissue transplantation • Multi-center, randomized trial led by Stanford University • 10-15 RDEB patients, with approximately 30 chronic wound sites treated in total Trial Design • Follow-up visits at 1 month-12 months, then yearly in a long-term follow-up protocol until year 5 The proportion of wounds with healing at three months, comparing treated with Primary Endpoint untreated wound sites on the same patient 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 Secondary Endpoints function. First patient treated in Q1 2020; additional 10 patients prescreened 13 Abeona Therapeutics Corporate Presentation, June 2020