Abeona Acquires Experimental Swallowing-Disorder Drug

Rare Daily Staff

Abeona Therapeutics has acquired an experimental once-daily treatment for eosinophilic esophagitis, a chronic inflammatory disease that can make swallowing difficult and cause food to become stuck in the esophagus.

The company said it plans a new late-stage trial after two earlier studies failed to meet a key measure of patient-reported symptom improvement.

The drug, previously known as APT-1011 and now renamed ABO-801, was acquired from Ellodi Pharmaceuticals for $20 million in Abeona stock, with no upfront cash payment. Beyond the initial stock payment, Ellodi could receive $30 million if the FDA approves ABO-801, up to another $30 million tied to sales milestones, and royalties ranging from the low teens to the mid-teens as a percentage of net sales.

Abeona also announced an agreement to raise approximately $30 million through a private placement to support its pipeline and commercialization efforts.

Eosinophilic esophagitis, or EoE, affects the esophagus, the tube that carries food from the mouth to the stomach.

ABO-801 is a rapidly dissolving tablet containing the corticosteroid fluticasone propionate. It is designed to deliver the medicine directly to the esophagus while limiting the amount reaching the rest of the body. Abeona hopes the once-daily formulation will offer a convenient option for managing the chronic disease. The U.S. Food and Drug Administration has granted the treatment orphan drug and Fast Track designations.

The treatment has been evaluated in several clinical studies, including two completed phase 3 trials that together enrolled 361 patients. Neither phase 3 study met its prespecified co-primary endpoint measuring symptoms reported by patients. Both, however, showed statistically significant improvements in several measures assessed through endoscopy, a procedure that allows doctors to examine the esophagus.

A key question for the new trial is whether the treatment can demonstrate improvement in the symptoms patients experience, in addition to changes observed in the esophagus.

Abeona said findings from the earlier studies will guide the design of another phase 3 trial, which it expects to begin in mid-2027. Initial results could be available by the end of 2028, depending on when the study starts and how quickly it enrolls participants. The company described the safety findings across the clinical program as favorable.

“ABO-801 brings an established clinical foundation, with learnings from two completed phase 3 studies that inform the design of the planned phase 3 study,” said Vish Seshadri, CEO of Abeona.

Photo: Vish Seshadri, CEO of Abeona.