Kiora Pharmaceuticals (NASDAQ: KPRX) today announced preclinical data demonstrating that KIO-300, a novel ion channel modulator, significantly suppressed seizure-associated electrophysiological activity in an ex vivo temporal lobe epilepsy (TLE) model. The findings were presented in a poster at the Epilepsy Foundation Pipeline Conference taking place June 18-19, 2026, in Leesburg, VA.
KIO-300 is the active pharmaceutical ingredient of KIO-301, which is currently in a Phase 2 clinical program for vision restoration in patients with retinitis pigmentosa. The new data highlight the potential of Kiora's ion channel modulator platform beyond ophthalmology, addressing a significant unmet need in epilepsy. Temporal lobe epilepsy is one of the most common forms of epilepsy, and many patients do not achieve adequate seizure control with existing treatments.
The preclinical study utilized an ex vivo model of TLE, where brain slices from epileptic animals were treated with KIO-300. Results showed a marked reduction in epileptiform activity, indicating that KIO-300 can modulate neuronal excitability and suppress seizure-like events. This mechanism of action is distinct from many current antiseizure medications, which often target synaptic transmission or ion channels in a less selective manner.
"These promising preclinical results suggest that KIO-300 may have therapeutic potential in epilepsy, a condition affecting millions worldwide," said a company spokesperson. "We are encouraged by the data and look forward to exploring further applications of our ion channel modulator platform."
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing advanced therapies for retinal disease, but this announcement signals a potential pipeline expansion into neurology. The company's lead candidate, KIO-301, is a molecular photoswitch designed to restore vision in patients with inherited retinal degenerations. KIO-104, another asset, is a small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH) for macular edema. The success of KIO-300 in the epilepsy model could open new avenues for the company and provide a novel treatment option for drug-resistant epilepsy.
The Epilepsy Foundation Pipeline Conference brings together researchers, clinicians, and industry leaders to discuss emerging therapies. Kiora's presentation underscores the growing interest in ion channel modulation as a therapeutic strategy for neurological disorders. While these results are preclinical, they lay the groundwork for future studies, including potential in vivo models and ultimately clinical trials.
Forward-looking statements in this release involve risks and uncertainties, including the ability to conduct clinical trials on a timely basis and obtain regulatory approvals. Kiora's results may be affected by factors described in its SEC filings, including its Annual Report on Form 10-K and Quarterly Report on Form 10-Q. The company disclaims any obligation to update forward-looking statements.
