Abeona Therapeutics (ABEO) Corporate presentation summary

Commercial launch and market opportunity

• ZEVASKYN launched with strong interest from the RDEB community and is available at multiple qualified treatment centers, with more activations planned for 2026 and beyond.

• Identified eligible patient numbers are growing, and demand is expected to increase as more centers are activated.

• The US market includes approximately 750 ZEVASKYN-eligible RDEB patients, with an estimated $4B cumulative revenue opportunity and $3.1M WAC per treatment.

• Profitability is anticipated upon treating more than three patients per month, expected to be reached in Q2 2026.

• Strong balance sheet with $191.4M in cash, cash equivalents, and short-term investments as of December 31, 2025.

Clinical efficacy and patient impact

• ZEVASKYN is the first gene therapy using a patient's own skin cells to treat wounds in children and adults with RDEB, offering single-application treatment and years of healing.

• In the Phase 3 VIITAL study, 81% of treated wounds achieved at least 50% healing at week 24, compared to 16% in controls, with significant pain reduction.

• Long-term follow-up showed multi-year wound healing and sustained collagen VII expression at treated sites.

• Patient testimonials highlight life-changing improvements in wound healing, pain relief, and quality of life.

Disease background and unmet need

• RDEB is caused by COL7A1 gene mutations leading to lack of functional C7 protein, resulting in chronic, painful wounds and high risk of infection and squamous cell carcinoma.

• The disease significantly reduces life expectancy and quality of life, with high economic burden and healthcare resource utilization.