Abeona Therapeutics Inc. Q2 FY2025 earnings call

• FDA approved ZEVASKYN in April 2025, the first autologous cell-based gene therapy for RDEB. • Actively executing ZEVASKYN launch; 2 qualified treatment centers identified over a dozen patients as candidates. • On track for first ZEVASKYN patient treatment in Q3 2025, anticipating first commercial revenue then. • Secured positive coverage with multiple payers, including UnitedHealthcare; entered NDRA with CMS for Medicaid coverage. • Engaged with RDEB community through webinars and regional meet-ups, with positive patient response. • R&D expenses reduced due to costs capitalized into inventory and reclassification of some costs to SG&A. • Pipeline updates: Beacon exercised option for AAV204 capsid; Ultragenyx faced CRL for UX111 BLA; Taysha commenced pivotal trial for TSHA-102.

No distinct product segments with revenue contribution detailed. Cash, cash equivalents, short-term investments and restricted cash were $225.9 million as of June 30, 2025. Research and development expenses were $5.9 million for Q2 2025 vs. $9.2 million in Q2 2024. SG&A expenses were $17.1 million for Q2 2025 vs. $8.6 million in Q2 2024. Net income was $108.8 million in Q2 2025 including gain from sale of PRV.

• Anticipate first commercial revenue from ZEVASKYN in Q3 2025. • Remain optimistic about treating 10 to 14 patients in 2025. • On track to increase manufacturing capacity to treat 10 patients per month by mid-2026. • Projected company-wide profitability in early 2026. • Plan to participate in investor conferences in September.

Q: How are identified patients defined?

A: Patients with severe RDEB, large wounds never healed, prioritized by physicians.

Q: How many cases does a center need to treat before adopting Pz-cel as routine therapy?

A: Sites like Chicago Lurie's are treating first commercial patients with no prior dry run, but have infrastructure for RDEB patients.

Q: Mechanics of payment and reimbursement for ZEVASKYN?

A: Revenue recognized after patient is treated; payment can come via direct from Abeona or specialty pharmacy. Prior auth process has 100% approval with no pushback on prior RDEB treatments.

Q: Prior authorizations submitted and biopsy/treatment timelines?

A: Prior auth numbers dynamic, process getting shorter; optimistic about 10-14 patient estimate for 2025.

Q: Production capacity ramp risk?

A: Current CGMP facility has capacity, ramp to 10 per month by mid-2026 on track with no anticipated catastrophic risks or FDA inspections beyond routine discussions.

Q: EU filing and launch?

A: Started looking at EU and Japan, but manufacturing footprint in other geographies is long lead time; focused on U.S. launch first with updates expected end of 2025.

Q: SG&A spend for next quarters?

A: Mix of expenses varies with production output; 3 patients per month is breakeven point, but mix may fluctuate quarter-to-quarter.