Kiora in-licenses late-stage KIO-401 for retinal artery occlusion

Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX) announced on October 29, 2026 that it has obtained exclusive development and commercialization rights outside Asia to KIO-401 (mitucapistat), a late-stage, oral, neuroprotective small-molecule calpain inhibitor, from Senju Pharmaceutical Co., Ltd. The announcement is Exhibit 99.1 to a Form 8-K. KIO-401 is being developed for central retinal artery occlusion (CRAO), a rare, acute retinal disease that can cause severe, permanent vision loss. No approved therapies exist for it today.

The license terms

Kiora will make an upfront payment of $3.5 million. It will also make a one-time milestone payment in the low teen millions upon technology transfer, which the company expects in 2027. Senju is eligible for further regulatory and commercial milestones and single-digit royalties on net sales outside Asia. Senju keeps its rights within Asia.

The agreement builds on an existing relationship between the companies involving KIO-301, Kiora's photoswitch therapy for advanced retinitis pigmentosa. After reviewing Senju's previous work, Kiora identified KIO-401 as a compelling late-stage asset. Chief executive Brian M. Strem, Ph.D., said the company plans to build on those findings. "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."

The clinical data

Senju ran a randomized Phase 2a study in Japan in which KIO-401 showed a statistically significant improvement from baseline in ETDRS visual acuity compared with a prospective, observational registry (p=0.0398). A later randomized, double-masked Phase 3 study in Japan showed improvement in best-corrected visual acuity, including a statistically significant improvement among patients who were legally blind at baseline according to World Health Organization criteria (p=0.0195).

Brian Mac Grory, MB BCh BAO, MRCP, an associate professor at Duke University School of Medicine, chairs the writing group for an American Heart Association scientific statement on CRAO. According to the press release, he said the reported outcomes are the first encouraging results for a neuroprotective drug for CRAO.

Planned development and financial position

Kiora plans a US-focused, multinational Phase 3 registration trial in CRAO, targeted to start in 2027, subject to funding and regulatory alignment. The trial will evaluate efficacy and safety in patients with acute CRAO, with visual function outcomes expected to play a central role. The sample size is estimated to be more than 100 patients, and the final design will be set after discussions with regulatory authorities. A Phase 2b trial in non-arteritic anterior ischemic optic neuropathy (NAION) is targeted for 2028, subject to funding and regulatory considerations.

Kiora's annual report describes it as a clinical-stage specialty pharmaceutical company developing therapies for retinal diseases. Its KIO-301 program is partnered with Théa Open Innovation (TOI), which reimburses R&D expenses and holds commercialization rights outside Asia. Kiora is eligible for up to $285 million in additional milestones and tiered royalties reaching the low 20% range. Kiora has discontinued development of KIO-104 and will pursue out-licensing for it.

Reported figures show revenue of $0 for 2025, compared with $16.0 million for 2024. Research and development expense was $10.8 million in 2025 and $7.8 million in 2024. Cash used in operations was $10.0 million in 2025. Year-end cash and cash equivalents were $8.7 million for 2025, and year-end short-term investments were $8.4 million. At June 30, 2026, cash and cash equivalents were $7.1 million and short-term investments were $9.8 million. Melissa Tosca, CPA, the chief financial officer, said the priority is to secure capital to advance KIO-401 through late-stage development. Kiora is evaluating financing alternatives.

The risks ahead

Kiora has to raise money before it can run the KIO-401 Phase 3 trial, which the company says is subject to funding and regulatory alignment. It reported $0 revenue for 2025 and says it is evaluating financing alternatives. The release gives no patient numbers for either Japanese study. The Phase 2a comparison was against an observational registry, not a placebo group, and the Phase 3 p-value cited covers only the subgroup that was legally blind at baseline, with no statistic given for the overall group. The company is targeting 2027 for the Phase 3 start and 2028 for the NAION trial.

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