Kiora Pharmaceuticals Secures New U.S. Patent for Novel KIO-100 Formulations

Kiora Pharmaceuticals has received a new U.S. patent (Patent No. 12,472,263) covering additional and novel formulations of the KIO-100 family of compounds, further strengthening the intellectual property portfolio for its pipeline of non-steroidal anti-inflammatory therapies. This announcement follows the July 2025 issuance of a previous patent (Patent No. 12,364,680) related to KIO-104, the company’s lead ocular therapy.

Novel Formulation Targets Retinal Inflammation

The newly granted patent protects a novel formulation of the active pharmaceutical ingredient (API) in KIO-104, a non-steroidal small molecule inhibitor of dihydroorotate dehydrogenase (DHODH). Unlike steroids or systemic anti-inflammatories, KIO-104 offers a targeted approach by inhibiting DHODH, a mitochondrial enzyme essential in the synthesis of DNA and RNA. This mechanism positions KIO-104 as a promising alternative in the treatment of retinal inflammatory diseases.

According to Brian M. Strem, PhD, President & CEO of Kiora Pharmaceuticals, “We believe DHODH inhibitors have significant potential to slow, stop, or reverse inflammatory-related vision loss, where steroids or biologics are not providing appropriate benefit today.”

Advancing Clinical Development: KIO-104 in Phase 2 KLARITY Trial

KIO-104 has already demonstrated clinical proof-of-concept in a Phase 1 trial for non-infectious uveitis. The company is now enrolling patients in Phase 2 of the KLARITY trial, a multicenter study evaluating higher doses of KIO-104 in posterior non-infectious uveitis and diabetic macular edema, as well as other forms of retinal inflammation.

The ongoing KLARITY study will involve up to 28 patients across two dosing levels, with results from the initial stage guiding future dose expansion strategies.