ABEONA THERAPEUTICS INC. Q1 FY2025 earnings call

• Abeona is proud to have ZEVASKYN, approved by the U.S. FDA for RDEB, with launch underway. First Qualified Treatment Center (QTC) at Lurie Children's Hospital activated, with first patient expected in Q3 2025.
• Sold priority review voucher (PRV) for $155M to strengthen balance sheet.
• Abeona Assist received 30+ patient and family inquiries in two weeks post-ZEVASKYN approval.
• Successful physician engagements at Society for Investigative Dermatology meeting, including late-breaking abstracts and Advisory Board discussion.
• Executed outcomes-based agreements with two payer contracting organizations to ensure patient access.

Abeona is a commercial stage cell and gene therapy company. Its main product is ZEVASKYN, the first and only autologous cell-based gene therapy for RDEB. No other product segments are detailed in terms of financial performance and revenue contribution as the focus is primarily on ZEVASKYN's launch and commercial progress.

• Aim to treat 10 to 14 patients in 2025 as previously guided.
• Anticipate profitability in the second half of 2026.
• Ophthalmology pipeline's RS1 program on track for clinical trials in the second half of 2026, with other pre-clinical programs to follow.

Q: What does 'activated' mean for the first site and are staff and surgeons trained?

A: Activation means sites are trained with processes in place to identify patients for ZEVASKYN, secure payer authorizations, and begin manufacturing scheduling.

Q: How many eligible patients are at Lurie Children's and other centers?

A: Exact number at Lurie isn't known, but there are dozens, with other centers expected to have eligible patients that will build over time.

Q: Factors for first site being Lurie Children's and patient triaging?

A: Lurie's collaboration and speed in engaging with Abeona, with patient triaging considering insurance type and patient preference.

Q: Scaling commercial supply and PRV net proceeds?

A: Current facilities can support 10 patients per month, net proceeds from PRV sale ~$152M, pro forma cash for Q2 to be determined.

Q: Prior treatment history of RDEB patients for ZEVASKYN?

A: Majority of patients have been previously treated with VYJUVEK or FILSUVEZ, and physicians are comfortable with discussing this.

Q: Billing and reimbursement timeline?

A: Revenue recognized after patient treatment, payment depends on discussions with QTCs and insurers, tracking similar to other cell and gene therapies.

Q: Inbound interest from patients and enrollment?

A: ~30 patients inquired, with next step being enrolling in Abeona Assist program to continue updates and outreach.