Kiora to Launch Clinical Trial in Australia for Vision-Restoring Small Molecule for RP Patients

Kiora Pharmaceuticals has received authorization to launch a clinical trial for KIO-301, its emerging small-molecule therapy to restore vision in people with advanced retinitis pigmentosa (RP) and potentially other retinal conditions. Known as the ABACUS study, the Phase 1B clinical trial will take place at The Royal Adelaide Hospital (RAH) in Adelaide, South Australia, and will begin enrolling patients in the third quarter of 2022.

KIO-301 is known as a “photoswitch,” a light-sensitive small molecule designed to bestow light sensitivity to ganglion cells that are downstream from degenerated rods and cones. KIO-301 will be delivered through monthly intravitreal injections.

“We are excited to see this promising approach for restoring vision move toward human studies. It has the potential to restore vision for people with little or no vision remaining, regardless of the mutated gene causing their RP,” says Claire Gelfman, PhD, chief scientific officer, Foundation Fighting Blindness. “While this approach is similar in some respects to optogenetics, it is different in that it doesn’t use a gene to deliver light sensitivity to the retina. Rather, it uses a small molecule, which is administered to the eye on a regular basis. Both the dosing levels and formulation can be modified to meet the needs of the patient, whereas optogenetics is a one-time, irreversible approach.”

The Foundation Fighting Blindness provided $1.3 million in funding through its Translational Research Acceleration Program and a Gund Harrington Scholar Award to Richard Kramer, PhD, University of California, Berkeley, for the development of related photoswitches for restoring vision.