Abeona Therapeutics (ABEO) investor relations material

ZEVASKYN launch and treatment-center expansion

• FDA-approved in 2025; launch began in December, with the first patient treated then. By Q3 2026, 15 patients had been treated since launch, including 6 in Q3.

• Eight QTCs were activated across the U.S.; four had treated patients. Goal: all eight centers to treat their first patient by around mid-2027; a few additional centers are expected in 2027.

• First treatments can be delayed by payer enrollment or patient clinical issues; centers with experience are seeing repeat treatments, while throughput is expected to vary across sites.

• ZEVASKYN commercial and CMC teams will remain undisturbed; a separate ABO-801 development team will use existing trial infrastructure, with commercial capabilities added in stages nearer potential approval.

• Wound-healing outcomes are being shared in confidential physician advisory settings; some physicians seek three months of follow-up, while others prefer six months, and a more formal scientific forum is being explored.

ABO-801 opportunity and clinical evidence

• ABO-801 is an oral fluticasone formulation for eosinophilic esophagitis (EoE), designed to dissolve on the tongue in 30 seconds and deliver drug locally along the esophagus; systemic absorption is less than 1%.

• Management cited an estimated EoE population of 360,000 and limited innovation in oral corticosteroids, with EOHILIA the only approved drug in that category.

• Two Phase III studies missed a co-primary patient-reported endpoint; management highlighted endoscopic improvement by week 12 sustained at week 52 and complete symptom response of 18% vs. 6%.

• Post hoc mapping of PROSE results to DSQ at an assumed cutoff of NRS ≤2 yielded ~34% response vs. 20% for placebo; the analysis was exploratory and not suitable for regulatory submission.

• Next Phase III plan: use DSQ, power the study to detect the observed 13% response difference, assess a 12-week co-primary endpoint with 24-week durability confirmation, and include more moderate-to-severe patients.

Acquisition economics and execution

• Search criteria prioritized a late-stage, data-backed asset with limited upfront cash; ABO-801 was selected after review of roughly 50–60 assets and extensive clinical, KOL and payer diligence.

• Consideration included $20 million in stock, with no upfront cash; the first milestone payment to Ellodi is due only upon ABO-801 approval.

• Planned Phase III spend is $50–$60 million over 2.5 years; management cited $135 million cash at September 30, an additional $30 million in PIPE proceeds, ZEVASKYN revenue and FAYUVI royalties as sufficient to fund development through approval.

• Formulation and method-of-use patents extend to 2042; FDA orphan-drug approval provides seven years of U.S. exclusivity.

• FAYUVI, licensed to Ultragenyx, was approved in September; a separate Rett syndrome asset was licensed to Taysha, reflecting prior development and licensing experience.