Kiora Pharmaceuticals (NASDAQ: KPRX) announced today that its novel ion channel modulator, KIO-300, significantly suppressed seizure-associated electrophysiological activity in an ex vivo temporal lobe epilepsy (TLE) model. The preclinical 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, a genetic form of blindness. The new data indicate that KIO-300 may have therapeutic potential beyond ophthalmology, specifically in neurological disorders such as epilepsy. Temporal lobe epilepsy is the most common form of focal epilepsy, and current treatments often fail to control seizures, highlighting the need for novel therapies.
The study demonstrated that KIO-300 significantly reduced the frequency and amplitude of epileptiform discharges in brain slices from animal models of TLE. These results suggest that the compound's mechanism of action—modulating ion channels involved in neuronal excitability—may be effective in dampening hyperexcitability associated with seizure activity. The company believes that KIO-300's ability to cross the blood-brain barrier and its favorable safety profile in ongoing clinical trials position it as a promising candidate for epilepsy.
Kiora Pharmaceuticals is a clinical-stage biotechnology company focused on developing advanced therapies for retinal disease, targeting critical pathways to slow, stop, or restore vision loss. However, the new epilepsy data suggest a potential pipeline expansion. KIO-301, the lead candidate, is a molecular photoswitch that aims to restore vision in patients with inherited retinal degenerations. Additionally, KIO-104 is a next-generation, non-steroidal immuno-modulatory small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH) being developed for macular edema.
"These preclinical results are encouraging and support the potential of our ion channel modulator platform to address neurological conditions beyond our core retinal focus," said a company representative at the conference. The company plans to further evaluate KIO-300 in additional preclinical models and explore potential clinical development in epilepsy.
Forward-looking statements in the press release note that Kiora's ability to execute on development and commercialization efforts for its products, including KIO-301 and KIO-104, is subject to risks and uncertainties. These include the timing of clinical trials, regulatory approvals, and market conditions. The company's filings with the SEC, including its Annual Report on Form 10-K filed on March 25, 2026, and its Quarterly Report on Form 10-Q filed on May 8, 2026, provide further details on risk factors.
The Epilepsy Foundation Pipeline Conference brings together researchers, clinicians, and industry to discuss emerging therapies for epilepsy. Kiora's presentation adds to the growing body of evidence supporting ion channel modulation as a viable strategy for seizure control.
