Abeona Therapeutics (ABEO) H.C. Wainwright 28th Annual Global Investment Conference summary

Commercial progress and market access

• Achieved $11.4 million in revenue with 31% quarter-over-quarter growth, supported by a growing network of seven qualified treatment centers covering 40% of the addressable market.

• Over 95% of commercial lives and all 50 states' Medicaid now cover ZEVASKYN, with no insurance denials reported.

• Twelve patients have been commercially treated as of August 13th, with administrative bottlenecks improving as centers gain experience.

• Patient identification to treatment time is decreasing, aided by cross-center best practice sharing and robust patient support programs.

• $146 million in cash reserves expected to fund commercial launch and pipeline development.

Product innovation and clinical impact

• ZEVASKYN is the only autologous cellular sheet gene therapy for recessive dystrophic epidermolysis bullosa, offering durable wound healing and pain relief after a single surgical application.

• Clinical trials demonstrated wound closure and pain reduction lasting up to 12 years, with commercial use averaging nine sheets per patient at a fixed price per treatment cycle.

• Patients previously treated with other therapies are now receiving ZEVASKYN, including children as young as two years old.

• Market research shows 40% of patients travel out of state for treatment, highlighting the importance of expanding the network of leading centers.

• Revenue is recognized upon patient treatment, with a predictable 25-day manufacturing process and rapid patient discharge.

Pipeline and partnership developments

• Partnered programs with Ultragenyx (Sanfilippo syndrome type A, PDUFA date upcoming) and Taysha (Rett syndrome) offer milestone and royalty opportunities.

• Beacon Therapeutics partnership leverages licensed AAV204 capsids for preclinical ophthalmology programs targeting rare and prevalent retinal diseases.

• Recently acquired PSMA-SIR-T, a preclinical synthetic immune receptor T-cell therapy for prostate cancer, with plans to enter human trials next year.

• CAR-T cell therapy program aims to address unmet needs in solid tumors, showing differentiation in animal models.

• Multiple value-driving events and inflection points anticipated across the pipeline in the coming year.