A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)

Phase 1/2 Completed 32 enrolled Kiora Pharmaceuticals, Inc.

NaSingle-groupOpen-labelTreatment

Summary

A phase I/II dose-escalating study of the safety, tolerability and efficacy of KIO-301 administered intravitreally to patients with retinitis pigmentosa and choroideremia (ABACUS).

Timeline

Start: 2022-11-10
Primary completion: 2023-08-10
Completion: 2026-03-24

Outcome

Met primary endpoint

paper The primary safety outcome was met, with no serious adverse events or dose-limiting toxicities observed at any point. reads as met primary endpoint PMID 41981307 ↗

release “A presentation will report interim results on safety, tolerability and efficacy from the ABACUS study, the ongoing, first-in-human clinical trial of KIO-301” reads as unknown kiorapharma.com ↗

release “The key findings from ABACUS-1 demonstrated that KIO-301 is safe & tolerable in this population” reads as met primary endpoint kiorapharma.com ↗

Publications

Casson RJ, Daniels E, Barras CD, Dwyer A, Strem BM, Wykoff CC, Gregorio-King C, Schuh C, Kramer RH, Van Gelder RN. Intravitreal photoswitch therapy in advanced retinitis pigmentosa: a phase 1 open-label trial. Nat Med. 2026 May;32(5):1865-1870. doi: 10.1038/s41591-026-04317-6. Epub 2026 Apr 14.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	KIO-301	Small molecule	—	Intravitreal