Kiora Pharmaceuticals In-Licenses Late-Stage, Oral Neuroprotective Calpain Inhibitor for Central Retinal Artery Occlusion (CRAO) with Planned Expansion into NAION; Asset Completed Phase 3 Clinical Trial in CRAO in Japan
Key Highlights
- ➤Kiora (KPRX) licenses ex-Asia KIO-401 rights from Senju for $3.5 million upfront
- ➤KIO-401 Phase 3 CRAO study showed significant BCVA improvement in legally blind patients
- ➤Kiora targets multinational Phase 3 CRAO trial initiation in 2027, subject to funding
- ➤Kiora plans Phase 2b NAION trial initiation in 2028, subject to funding
- ➤Senju eligible for low-single-digit royalties on applicable ex-Asia net sales
Expert Statements
Brian M. Strem, President and Chief Executive Officer of Kiora Pharmaceuticals
“KIO-401 addresses a significant unmet need for patients with CRAO and NAION, two rare, acute ophthalmic diseases that result in permanent vision loss and have no approved therapies today.”
Brian M. Strem, President and Chief Executive Officer of Kiora Pharmaceuticals
“We have an established relationship with Senju through our KIO-301 partnership, which facilitated our partnership on KIO-401.”
Brian M. Strem, President and Chief Executive Officer of Kiora Pharmaceuticals
“After reviewing results from Senju's previous work, we identified KIO-401 as a compelling late-stage asset.”
Brian M. Strem, President and Chief Executive Officer of Kiora Pharmaceuticals
“We now plan to build on those findings with a larger, adequately powered multinational Phase 3 trial designed to more definitively evaluate its clinical benefit.”
Brian M. Strem, President and Chief Executive Officer of Kiora Pharmaceuticals
“We believe KIO-401 has the potential to meaningfully improve patient outcomes and achieve significant commercial value in the U.S., Europe and other regions.”
Brian Mac Grory, Associate Professor of Neurology and Ophthalmology at Duke University School of Medicine and Chair of the writing group for the American Heart Association scientific statement on CRAO
“CRAO remains an under-recognized medical emergency, and even when diagnosed, there is no approved therapy specifically shown to preserve vision.”
Brian Mac Grory, Associate Professor of Neurology and Ophthalmology at Duke University School of Medicine and Chair of the writing group for the American Heart Association scientific statement on CRAO
“An effective oral treatment could galvanize systems of care for acute CRAO recognition and management and expand treatment access to patients in regional and community hospitals.”
Brian Mac Grory, Associate Professor of Neurology and Ophthalmology at Duke University School of Medicine and Chair of the writing group for the American Heart Association scientific statement on CRAO
“The reported clinical trial outcomes are the first encouraging results for a neuroprotective drug for CRAO and strongly support further evaluation of KIO-401.”
Shuhei Yoshida, President & CEO of Senju Pharmaceutical
“The clinical results generated in Japan support continued global development of this compound as a potential treatment for acute retinal ischemia.”
Shuhei Yoshida, President & CEO of Senju Pharmaceutical
“Kiora brings retinal drug development expertise and a focused strategy for advancing the program outside Asia.”
Shuhei Yoshida, President & CEO of Senju Pharmaceutical
“Combining Senju's expertise in Japan and broader Asia with Kiora's leadership in global development, we view Kiora as a strong partner to lead development in the United States, Europe and other ex-Asia markets, with a shared goal of bringing a new treatment option to patients suffering from these ischemic insults.”
Melissa Tosca, Chief Financial Officer of Kiora Pharmaceuticals
“Our priority is to secure capital to advance KIO-401 through late-stage development while leveraging our existing clinical infrastructure.”
Melissa Tosca, Chief Financial Officer of Kiora Pharmaceuticals
“We will prioritize KIO-401 and partnered KIO-301 while pursuing out-licensing opportunities for KIO-104 following discontinuation of its development.”
* The oral small-molecule calpain inhibitor demonstrated meaningful visual acuity improvement in Phase 2 and Phase 3 CRAO studies conducted in Japan compared to controls. * No therapeutics exist today to treat patients that experience CRAO vision loss. * Kiora plans to advance KIO-401 (mitucapistat) into a multinational Phase 3 registration trial in CRAO, with initiation targeted for 2027. * Company also plans to evaluate KIO-401 in a Phase 2b clinical trial in non-arteritic anterior ischemic optic neuropathy ("NAION"). San Diego, California--(Newsfile Corp. - October 29, 2026) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) ("Kiora" or the "Company") today announced that it has obtained exclusive development and commercialization rights, outside Asia, to KIO-401, a late-stage neuroprotective, oral small-molecule calpain inhibitor, from Senju Pharmaceutical Co., Ltd. ("Senju"). KIO-401 is being developed as a potential treatment for central retinal artery occlusion ("CRAO"), a rare, acute ischemic retinal disease described as "stroke of the eye" that can result in severe, permanent vision loss and for which there are currently no approved therapies.
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