Abeona Therapeutics (ABEO) 2025 Conference Transcript

Summary of Abeona Therapeutics (ABEO) Conference Call Company Overview - Abeona Therapeutics is a commercial stage cell and gene therapy company that recently received approval for ZivaSkin, a therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB) [5][70] - The company has a market cap of approximately $300 million and anticipates becoming profitable in the early half of 2026 [6][71] Key Developments - ZivaSkin was approved just over a month ago, and the company has entered into a sale agreement for a Priority Review Voucher (PRV) for$155 million [5][70] - Abeona is funded for eight quarters of operations without accounting for revenue generation [6][71] Launch Metrics and Treatment Centers - Lurie Children's Hospital has been activated as the first qualified treatment center for ZivaSkin [7][72] - The company is tracking patient throughput and referral volume from other centers of excellence [7][72] - The timeline to fully activate all five treatment centers is by the end of 2025 [9][74] Patient Demand and Treatment Capacity - There are currently an upper double-digit number of patients eligible for treatment across the five centers [11][73] - The company expects to treat 10 to 14 patients this year, with the capacity to ramp up to 10 patients per month by the first half of 2026 [18][37] - The treatment window runs from August to November, with minimal disruption expected due to a planned December manufacturing shutdown [20][21] Pricing and Payer Engagement - ZivaSkin is priced at $3.1 million, which is considered reasonable compared to the standard of care costs [28][30] - The company has not faced pushback from payers and has entered agreements covering over 100 million commercially insured lives [31][36] Financial Outlook - Abeona expects to achieve cash flow positivity in 2026 with a target of treating 36 patients [37][39] - The company is confident in exceeding this target, potentially treating up to 50 patients [38][39] Future Plans and Market Expansion - Abeona is exploring regulatory submissions outside the U.S. and assessing reimbursement scenarios in major markets like Western Europe and Japan [46][47] - The company has identified approximately 750 patients in the U.S. with RDEB, with a significant opportunity for repeat treatments [49][51] Competitive Landscape - ZivaSkin differentiates itself by providing long-lasting wound coverage with a single application, addressing both wound healing and quality of life improvements [53][54] Partner Programs - Abeona is eligible for royalties and milestones from partnered programs with Ultragenyx and Atacea, which are not factored into current projections [55][58] Key Milestones Ahead - The company aims to achieve its treatment goals for ZivaSkin and activate additional qualified treatment centers, with several PDUFA action dates for partnered programs expected in 2025 [66][67]