Kiora Pharmaceuticals To Begin Phase 2 Trial Of KIO-301 For Retinitis Pigmentosa
Kiora Pharmaceuticals gets Phase 2 trial clearance for KIO-301, aiming to restore vision in retinitis pigmentosa patients.
Breaking News
Oct 30, 2024
Simantini Singh Deo

Kiora Pharmaceuticals, Inc. announced that it has received regulatory clearance to begin a Phase 2 clinical trial for KIO-301, a treatment designed to restore vision in patients with retinitis pigmentosa. The ABACUS-2 trial will enroll 36 patients with ultra-low vision or no light perception, regardless of the genetic mutation causing their retinitis pigmentosa.
This multi-center, double-masked study will include randomised and controlled doses of KIO-301, with patient dosing set to begin next year after validating new functional vision endpoints. These assessments may serve as primary endpoints for future regulatory studies in the U.S., Europe, and other regions.
Eric Daniels, M.D., Chief Development Officer at Kiora, said in a statement, "There are, unfortunately, no approved therapies for patients with retinitis pigmentosa. This study represents a significant step toward addressing this challenge. Before submission for approval, we engaged with European and US regulators to incorporate their expectations and guidance for approvable endpoints. Consistent with historical approval in other inherited retinal diseases, both regulatory bodies emphasized the need to measure a therapy's effect on everyday functional vision.”
He further commented, “For this reason, we are investing time upfront to validate the functional endpoints for ABACUS-2, increasing our likelihood of success in a potential single Phase 3 trial for market approval in the US and Europe. This validation work is being performed in collaboration with our partner Théa Open Innovation, with the support of the Choroideremia Research Foundation as part of a grant to design a standard endpoint for investigational therapies of inherited retinal diseases."
Kiora is currently securing partnerships with clinical sites and investigators at key retinal disease centers across Australia, where the trial will be conducted at five locations. Patients will be randomized 2:1 to receive KIO-301 or control treatment, with both eyes treated. This trial will evaluate two doses, a high and a low dose (100 & 50 micrograms, respectively). After three doses administered six weeks apart, patients will be monitored for three months. Those in the control group will have the option to switch to the active treatment afterwards. The primary trial goals include safety and tolerability, while key efficacy metrics will assess functional vision, visual acuity, visual fields, and quality of life.
"In a short window since entering our partnership with Kiora, tremendous progress has been made on advancing KIO-301 toward ABACUS-2. What makes KIO-301 compelling is that it appears, based on the Phase 1b data, to have the potential for meaningful vision restoration. Further, because of its unique mechanism of action, it has the potential to work across all 150-plus underlying gene mutations associated with retinitis pigmentosa and other inherited retinal diseases,” stated Dr. Céline Olmiere, Head of Théa Open Innovation.
KIO-301 is a small-molecule photoswitch designed to restore light sensitivity in retinal ganglion cells (RGCs), which are located downstream of the damaged rods and cones that normally convert light into vision. Activated in light and deactivated in darkness, KIO-301 enables RGCs to perform visual processing by adjusting the flow of ions in response to light, effectively allowing the cells to "see." Since it is activated independently of specific gene mutations, KIO-301 offers hope as a treatment for a wide range of inherited retinal diseases.