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

Commercial progress and product launch

• Transitioned to commercial stage with ZEVASKYN approval and launch for recessive dystrophic epidermolysis bullosa, showing 31% quarter-over-quarter revenue growth and $11.4 million in revenue.

• Seven qualified treatment centers now cover about 40% of the addressable patient market, including major institutions like Cincinnati Children's.

• Over 95% of commercial lives and all Medicaid plans in 50 states provide coverage for ZEVASKYN, with no insurance denials reported.

• Twelve patients have been commercially treated as of August 13, with robust patient support and travel programs in place.

• Administrative bottlenecks are being addressed, with onboarding times for new centers dropping to as low as two months.

Product differentiation and clinical impact

• ZEVASKYN is the only autologous cellular sheet gene therapy for both adults and pediatric patients, offering durable wound healing and pain relief from a single surgical application.

• Clinical trials demonstrated years of wound healing, with some wounds remaining closed for up to 12 years after treatment.

• Flat pricing model per treatment cycle, regardless of the number of sheets used, provides revenue certainty.

• Commercially treated patients include those previously on other therapies, with the youngest treated at two years old.

• Market research shows 40% of patients live outside states with qualified centers, highlighting the importance of patient support for travel.

Pipeline and strategic partnerships

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

• Ophthalmology capsid technology licensed to Beacon Therapeutics for preclinical retinal disease programs.

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

• Cash reserves of $146 million expected to fund commercial and pipeline activities.