Abeona Therapeutics (ABEO) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Key clinical insights

• Pz-cel demonstrated durable wound healing and significant pain reduction in patients with recessive dystrophic EB, with effects lasting multiple years after a single treatment application.

• Clinical trials included patients with severe, chronic wounds, some open for up to 21 years, and showed over 50% and 75% wound healing rates, as well as reduced itch and favorable safety profile.

• No serious treatment-emergent adverse effects or squamous cell carcinoma were reported at treated sites, with safety data extending up to 11 years post-treatment.

• The therapy uses retroviral gene integration into patient-derived skin cells, producing functional skin grafts that can cover large or multiple wound areas.

• Pz-cel is the only investigational therapy with a single-application approach for large, chronic wounds in this patient population.

Regulatory and commercialization progress

• BLA resubmission for Pz-cel is on track for this year, with anticipated approval in the first quarter of next year.

• FDA's previous complete response letter was limited to CMC-related issues, with no new clinical studies required; alignment has been reached with the FDA on the resubmission path.

• Commercial readiness efforts include engaging with high-volume EB centers and building a nimble infrastructure for both manufacturing and launch.

• An ICD-10 procedure code specific to the genetic nature of Pz-cel has been secured, supporting reimbursement discussions with payers.

• Market research indicates strong payer willingness to cover Pz-cel, with projected peak annual revenue exceeding $550 million.

Market opportunity and strategy

• Approximately 750 patients are estimated to be eligible for Pz-cel, with each patient potentially requiring two treatment cycles.

• The strategy focuses on onboarding five to seven geographically dispersed centers of excellence, aiming for one to two patients per center per month post-launch.

• Over 50% of the target patient population is commercially insured, with additional coverage from Medicaid and Medicare.

• Payers value the therapy's durability and clinical impact, supporting positive reimbursement outlook.

• Ongoing lifecycle management and future program development are being assessed alongside launch preparations.