Abeona Therapeutics (NASDAQ:ABEO) plans to advance its newly acquired eosinophilic esophagitis drug candidate into another Phase 3 study while expanding the commercial launch of ZEVASKYN, executives said at a Stifel investor event.
Chief Executive Officer Vish Seshadri said Abeona acquired ABO-801, previously known as APT-1011, after reviewing dozens of potential assets. The drug has been evaluated in two Phase 3 studies that each missed a co-primary endpoint measuring patient-reported symptoms.
Despite those results, Seshadri said the company believes changes to the study design could improve the chances of a successful trial. He also said the acquisition would not disrupt the teams responsible for commercializing and supplying ZEVASKYN.
New Study Planned for ABO-801
ABO-801 is an orally dissolving formulation of fluticasone propionate designed to treat eosinophilic esophagitis, or EoE, an inflammatory disease of the esophagus. Seshadri said the drug has orphan drug designation and formulation and method-of-use patents extending to 2042.
The CEO pointed to histological and endoscopic results from the earlier studies as evidence of the drug’s potential. He said a pooled analysis also showed a statistically significant difference in complete symptom response: 18% of treated patients reported no difficulty swallowing during a specified 14-day period, compared with 6% of the control group.
Seshadri attributed the missed symptom endpoints in part to the patient-reported outcome tool used in the earlier trials. He said Abeona plans to use the Dysphagia Symptom Questionnaire, or DSQ, in the next study. Unlike the earlier tool, the DSQ asks whether a patient ate solid food before proceeding with symptom questions.
Abeona also plans to size the study to detect an approximately 13-percentage-point difference in complete response, assess a 12-week endpoint with a 24-week durability confirmation, and adjust the patient mix to include more people with moderate or severe disease, Seshadri said.
He cautioned that the company’s retrospective analysis mapping the earlier trial results to the DSQ relied on assumptions and could not itself be submitted to regulators as trial results.
Deal Structure and Financing
Chief Financial Officer Joe Vazzano said Abeona issued $20 million in company stock to Ellodi as part of the acquisition, with no cash paid upfront. The first milestone payment to Ellodi would not be due until ABO-801 is approved, he said.
Vazzano said Abeona had more than $135 million in cash at Sept. 30, excluding $30 million in PIPE proceeds. He said that amount, together with ZEVASKYN revenue and FAYUVI royalties, would provide sufficient capital for the estimated $50 million to $60 million development program over roughly 2.5 years.
Seshadri said Abeona expects to add a handful of specialists for ABO-801 while using existing clinical operations personnel. The company’s ZEVASKYN commercial and manufacturing teams would remain focused on that product, he said.
ZEVASKYN Treatment Centers Expand
Abeona treated six patients with ZEVASKYN in the third quarter, bringing the total to 15 since its December launch, Seshadri said. The product was approved by the FDA in 2025.
Eight qualified treatment centers are active across the U.S., four of which have treated patients. Seshadri said Abeona would like all eight centers to have treated their first patient by around the middle of next year.
Delays at some centers have reflected factors including out-of-state Medicaid enrollment and clinical issues affecting patients initially selected for treatment, he said. Chief Commercial Officer Madhav Vasanthavada said Abeona is speaking with additional centers and expects a few more to come online in 2027.
Vasanthavada added that physicians are sharing wound-healing observations in confidential advisory settings, while some have expressed interest in a more formal scientific forum. He said the follow-up period physicians want to see before treating subsequent patients varies, with some looking for three months and others seeking about six months.
About Abeona Therapeutics (NASDAQ:ABEO)
Abeona Therapeutics, Inc. is a biotechnology company focused on developing cell and gene therapies for serious and rare diseases. Its work includes treatments designed to address the underlying genetic causes of disease. The company’s lead product, ZEVASKYN (prademagene zamikeracel), is an autologous gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, a rare inherited skin disorder. Made from a patient’s skin cells, the therapy uses genetic modification to help cells produce type VII collagen and is applied to wounds.
