Development of new neuroprotective treatment for retinal diseases—a phase 1/2 clinical trial on central retinal artery occlusion with KUS121

Abstract

Purpose : We have been developing the Kyoto University Substance (KUS) 121 which is an inhibitor of valosin-containing protein ATPase. It has a protective effect on retinal cells in vitro and in animal models of ocular diseases, including retinitis pigmentosa, retinal artery occlusion, and glaucoma. To examine the safety and efficacy of KUS121, we conducted an investigator-initiated clinical trial on patients with central retinal artery occlusion (CRAO).

Methods : Nine patients with CRAO with symptoms lasting between 3 and 48 hours were recruited for the clinical trial (phase 1/2, UMIN000023979). KUS121 was intravitreally injected daily for 3 days. We assessed the safety of the drug and the patients’ visual function (visual acuity and visual field) outcomes for 3 months.

Results : Between November 2016 and December 2017, 11 patients with non-arteritic CRAO were assessed, and nine patients were recruited for the study. All the nine patients completed the follow-up. No serious adverse events or side effects were observed. The final best corrected visual acuity (BCVA) was improved compared with that at baseline in all the patients. Seven out of nine (78%) patients showed BCVA equal to or better than 0.05. The average visual field area and scores were also improved.

Conclusions : We confirmed the efficacy and safety of KUS121 in preserving the visual function of patients with CRAO. We are currently preparing for the next phase of clinical trials. Additionally, KUS121 is being developed as a therapeutic agent for other retinal diseases such as age-related macular degeneration.

This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.