Kiora Pharmaceuticals, Inc.

The company is a clinical-stage specialty pharmaceutical company focused on developing and commercializing treatments for retinal diseases. Its primary development candidate is KIO-301, a potential vision-restoring small molecule designed to act as a "photoswitch" to restore vision in patients with inherited and age-related degenerative retinal diseases, including retinitis pigmentosa (RP). An initial focus for KIO-301 is on patients with later stages of vision loss due to RP. The company completed a Phase 1b clinical trial for KIO-301 in September 2023 and presented topline results in November 2023. In January 2024, the company partnered with Théa Open Innovation (TOI). In October 2024, regulatory approval was received to initiate a Phase 2 clinical trial, named ABACUS-2, for KIO-301 in patients with RP. The ABACUS-2 trial is a 36-patient, multicenter, double-masked, randomized, controlled, multiple-dose study enrolling patients with ultra-low vision or no light perception, regardless of their underlying gene mutation associated with RP. Enrollment in the ABACUS-2 trial is currently ongoing. KIO-301, formerly known as B-203, was acquired through the Bayon Therapeutics, Inc. transaction, which closed on October 21, 2021. The company is also developing KIO-104 for retinal inflammatory diseases, including Diabetic Macular Edema (DME) and Posterior Non-Infectious Uveitis. KIO-104 is a non-steroidal small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH) formulated for intravitreal delivery.