Encinitas, California--(Newsfile Corp. - December 1, 2025) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) today announced that the U.S. Patent and Trademark Office issued a new patent (US-12,472,263) on additional and novel formulations of the KIO-100 family of compounds, This patent allows for additional delivery options, expanding the potential therapeutic utility of the active pharmaceutical ingredient (API) in KIO-104.
This newly issued patent specifically covers a novel formulation of the API in KIO-104. The API in KIO-104 is a non-steroidal anti-inflammatory, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH). A Phase 2 clinical trial (KLARITY) of KIO-104 for the treatment of retinal inflammation is actively screening and dosing patients.
"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," said Brian M. Strem, PhD, President & Chief Executive Officer of Kiora Pharmaceuticals. "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 multicenter KLARITY study will investigate KIO-104 in up to 28 patients with macular edema, a condition where inflammation causes a build-up of fluid behind part of the retina and can be associated with adverse vision changes and permanent scarring. KIO-104, as a potent, locally delivered small molecule, could provide a needed alternative to steroids or systemic anti-inflammatory drugs.
About Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing advanced therapies for retinal disease. We target critical pathways underlying retinal diseases using innovative small molecules to slow, stop, or restore vision loss. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of retinal inflammation. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH).
In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on X and LinkedIn as well as to visit our website and/or subscribe to email alerts.
Forward-Looking Statements
Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, Kiora's ability to execute on development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104 and KIO-301, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, the sufficiency of existing cash and short-term investments on hand to fund operations for specific periods, the timeline of anticipated readouts, the potential for cash runway extension through partnership milestones, the potential to add trial centers, expand the geographic footprint of trials and/or accelerate enrollment, the potential for KIO-301 and KIO-104 to address multiple indications, and the possibility of future registration studies and global commercialization. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to conduct clinical trials on a timely basis, market and other conditions and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2025 or described in Kiora's other public filings, including on Form 10-Q filed with the SEC on November 7, 2025. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law.
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Contact:
investors@kiorapharma.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/276367
This newly issued patent specifically covers a novel formulation of the API in KIO-104. The API in KIO-104 is a non-steroidal anti-inflammatory, small molecule inhibitor of dihydroorotate dehydrogenase (DHODH). A Phase 2 clinical trial (KLARITY) of KIO-104 for the treatment of retinal inflammation is actively screening and dosing patients.
"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," said Brian M. Strem, PhD, President & Chief Executive Officer of Kiora Pharmaceuticals. "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 multicenter KLARITY study will investigate KIO-104 in up to 28 patients with macular edema, a condition where inflammation causes a build-up of fluid behind part of the retina and can be associated with adverse vision changes and permanent scarring. KIO-104, as a potent, locally delivered small molecule, could provide a needed alternative to steroids or systemic anti-inflammatory drugs.
About Kiora Pharmaceuticals
Kiora Pharmaceuticals is a clinical-stage biotechnology company developing advanced therapies for retinal disease. We target critical pathways underlying retinal diseases using innovative small molecules to slow, stop, or restore vision loss. KIO-301 is being developed for the treatment of retinitis pigmentosa, choroideremia, and Stargardt disease. It is a molecular photoswitch that has the potential to restore vision in patients with inherited and/or age-related retinal degeneration. KIO-104 is being developed for the treatment of retinal inflammation. It is a next-generation, non-steroidal, immuno-modulatory, and small-molecule inhibitor of dihydroorotate dehydrogenase (DHODH).
In addition to news releases and SEC filings, we expect to post information on our website, www.kiorapharma.com, and social media accounts that could be relevant to investors. We encourage investors to follow us on X and LinkedIn as well as to visit our website and/or subscribe to email alerts.
Forward-Looking Statements
Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, Kiora's ability to execute on development and commercialization efforts and other regulatory or marketing approval efforts pertaining to Kiora's development-stage products, including KIO-104 and KIO-301, as well as the success thereof, with such approvals or success may not be obtained or achieved on a timely basis or at all, the sufficiency of existing cash and short-term investments on hand to fund operations for specific periods, the timeline of anticipated readouts, the potential for cash runway extension through partnership milestones, the potential to add trial centers, expand the geographic footprint of trials and/or accelerate enrollment, the potential for KIO-301 and KIO-104 to address multiple indications, and the possibility of future registration studies and global commercialization. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the ability to conduct clinical trials on a timely basis, market and other conditions and certain risk factors described under the heading "Risk Factors" contained in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2025 or described in Kiora's other public filings, including on Form 10-Q filed with the SEC on November 7, 2025. Kiora's results may also be affected by factors of which Kiora is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. Kiora expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions, or circumstances on which any such statement is based, except as required by law.
###
Contact:
investors@kiorapharma.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/276367