Kiora Pharmaceuticals (NASDAQ: KPRX) announced that the results of its Phase 1 ABACUS‑1 study of the retinal photoswitch KIO‑301 have been published in the journal Nature Medicine.
The open‑label, first‑in‑human, dose‑escalation trial enrolled six participants with late‑stage retinitis pigmentosa and delivered KIO‑301 intravitreally to 12 eyes. The primary safety endpoint was met, with no serious adverse events, dose‑limiting toxicities, or structural retinal changes observed over 30 days.
Secondary and exploratory endpoints included functional vision testing, visual acuity, kinetic visual fields, functional MRI of the visual cortex, and participant‑reported quality‑of‑life scores. Light‑induced neural activity changes were observed in the visual cortex, supporting the photoswitch mechanism of action.
“Publication of the KIO‑301 Phase 1 findings in Nature Medicine is a meaningful milestone that brings early clinical support for a molecular photoswitch into the peer‑reviewed forum.” – Dr. Robert Casson, Principal Investigator of the study from the Royal Adelaide Hospital.
“The results of the trial provide evidence of short‑term ocular safety and feasibility, while underscoring that larger, controlled studies are still needed to determine whether any functional changes translate into reliable, everyday vision benefit.” – Dr. Robert Casson, Principal Investigator of the study from the Royal Adelaide Hospital.
“Based on Phase 1 results, we initiated a randomized, controlled Phase 2 clinical trial, dubbed ABACUS‑2,” said Brian M. Strem, Ph.D., Chief Executive Officer of Kiora. “The trial is designed to evaluate higher doses of KIO‑301 with the goal of measuring, among other endpoints, functional visual improvements and comparing them to a control group, who will be eligible for KIO‑301 treatment as part of an open‑label extension.”
“Our success in 2025 was defined by transitioning from trial preparation to trial execution for our two lead programs,” said Brian M. Strem, Ph.D., President and Chief Executive Officer of Kiora.
“The fMRI results reinforce our understanding of KIO‑301's mechanism of action, as well as the assertion that conferring light sensitivity to cells other than the native rods and cones, can positively impact vision,” added Eric Daniels, MD, Chief Development Officer of Kiora. “Importantly, this data is consistent with improvements in functional endpoints assessing change in patients' everyday activities that we and regulatory bodies believe are essential for drug approval.”
Investors responded positively to the publication, reflecting confidence in the clinical trajectory of KIO‑301 and the broader photoswitch platform.
Kiora reported a net loss of $10.8 million for the year ended December 31 2025, compared with a net income of $3.6 million in 2024, as the company continues to invest heavily in clinical development.
As of March 25 2026, Kiora held $17.1 million in cash, cash equivalents, and short‑term investments, providing a projected runway into late 2027 and supporting the upcoming Phase 2 readout.
The company has secured an option agreement with Senju Pharmaceutical Co., Ltd. for development and commercialization of KIO‑301 in key Asian markets, with a potential deal value of up to $110 million plus royalties, and a co‑development and commercialization agreement with Théa Open Innovation.
Kiora also continues to develop KIO‑104, a candidate for retinal inflammation, expanding its pipeline beyond the photoswitch platform.
The Nature Medicine publication validates the photoswitch approach, strengthens investor confidence, and positions Kiora to compete with gene therapies and optogenetic strategies for inherited retinal diseases, while the company’s financial position and strategic partnerships provide a foundation for advancing its clinical program.
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