Kiora Receives Authorization for Phase 2 Clinical Trial of its Photoswitch for Advanced RP

Kiora Pharmaceuticals, a company developing treatments for orphan retinal diseases, has received authorization in Australia to launch the Phase 2 ABACUS-2 clinical trial for its photoswitch, a small molecule that is designed to restore some vision to people with advanced retinal diseases. The controlled Phase 2 trial will enroll 36 patients with ultra-low vision or no light perception from advanced retinitis pigmentosa (RP).  The trial is expected to begin at multiple sites in Australia in 2025. If the Phase 2 trial is successful, and outcome measures used to evaluate vision changes are validated, the company hopes to launch a Phase 3 clinical trial in the US, Europe, and other regions. Kiora believes the photoswitch may also restore vision in people with advanced choroideremia, Stargardt disease, and other retinal diseases. Known as KIO-301, the photoswitch is designed to work regardless of the mutated gene causing the retinal disease.

KIO-301 enables retinal ganglion cells to respond to light, thereby working like a backup system for lost photoreceptors. Retinal ganglion cells, which are downstream from photoreceptors, often survive in advanced retinal disease but don’t naturally respond to light. KIO-301 is delivered by a standard injection into the vitreous, the soft gel in the middle of the eye. One administration of the therapy appears to be effective for about a month.

Six patients were treated in ABACUS-1, Kiora’s Phase 1/2 clinical for KIO-301. Patients were split into two groups of three. Cohort 1 patients had no light perception or bare light perception. Cohort 2 patients had enough vision to see hand motion or count fingers. Three doses of KIO-301 were evaluated.

Cohort 1 patients, after treatment, appeared to have improvements in their ability to perceive the direction of movement and/or location of a window or lighted exit. Cohort 2 patients had improvements in visual fields (peripheral vision) as measured by a Goldmann perimeter. Cohort 2 patients receiving the high dose had improvements in visual acuity, as measured by a Berkeley Rudimentary Vision Test, a test designed to evaluate vision in people unable to read any letters on a typical eye chart.

The clinical development of KIO-301 was made possible through $1.3 million in funding from the Foundation through its Translational Research Acceleration Program and a Gund Harrington Scholar Award to Richard Kramer, PhD, University of California, Berkeley, for his research of photoswitches for restoring vision.

Kiora has a strategic partnership with Théa Open Innovation for the co-development and commercialization of KIO-301.