Kiora Pharmaceuticals receives patent for additional and novel formulations of KIO-100 compounds

The US Patent and Trademark Office has issued a new patent (US Patent No. 12,472,263) on additional and novel formulations of the KIO-100 family of compounds from Kiora Pharmaceuticals. In July 2025, the company announced it had secured a patent for the company’s ocular therapy KIO-104 (US Patent No. 12,364,680).

According to the company, the newly received patent covers a novel formulation of the API, a non-steroidal anti-inflammatory small molecule inhibitor of dihydroorotate dehydrogenase (DHODH), in KIO-104. KIO-104 offers an alternative treatment method to steroids or systemic anti-inflammatory drugs by inhibiting the mitochondrial enzyme DHODH, which plays a key role in the synthesis of key building blocks of both DNA and RNA.

Brian M. Strem, PhD, president & CEO of Kiora Pharmaceuticals, commented on the patent, saying, "We believe DHODH inhibitors have significant potential to slow, stop or reverse inflammatory-related vision loss, where steroids or other biologics are not providing appropriate benefit today. KIO-104, our DHODH inhibitor formulated for local delivery into the retina, previously demonstrated clinical proof-of-concept in a Phase 1 trial in non-infectious uveitis. We're now enrolling in a Phase 2 study to evaluate higher doses of KIO-104 in multiple retinal inflammatory conditions, including posterior non-infectious uveitis and diabetic macular edema."

The phase 2 clinical trial KLARITY is evaluating KIO-104 for the treatment of retinal inflammation and is actively screening and dosing patients. The KLARITY study is a multicenter trial of KIO-104 in up to 28 patients at 2 dose levels across various forms of inflammatory retinal diseases.^1,2 The results from the initial stage study will inform dose expansion.

The collection of patents is expected to further strengthen the protection of KIO-104 and extend market exclusivity into 2043, the company said in a press release. In addition to KIO-104, Kiora Pharmaceuticals is developing KIO-301 for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. Most recently, the company granted Senju Pharmaceutical an exclusive option to exercise rights to an exclusive development and commercialization agreement for KIO-301 for the treatment of ophthalmic diseases. Areas covered by the agreement would cover key Asian marketplaces, including Japan and China.³

References:

1. Kiora Pharmaceuticals Granted U.S. Patent Covering Additional Formulations for the KIO-100 Family of Anti-Inflammatory Compounds. Published December 1, 2025. Accessed December 1, 2025. https://ir.kiorapharma.com/news-events/news-releases/detail/242/kiora-pharmaceuticals-granted-u-s-patent-covering-additional-formulations-for-the-kio-100-family-of-anti-inflammatory-compounds

2. Filkins K. Kiora Pharmaceuticals Secures US Patent for KIO-104. Published July 24, 2025. Accessed December 1, 2025. https://www.ophthalmologytimes.com/view/kiora-pharmaceuticals-secures-u-s-patent-for-kio-104

3. Harp MD. Kiora Pharmaceuticals and Senju Pharmaceutical partner on KIO-301 for retinal disease treatment in Asia. Published June 5, 2025. Accessed December 1, 2025. https://www.ophthalmologytimes.com/view/kiora-pharmaceuticals-and-senju-pharmaceutical-partner-on-kio-301-for-retinal-disease-treatment-in-asia