Kiora Pharmaceuticals Doses First Patient in ABACUS Phase 1b Study Evaluating KIO-301 for Retinitis Pigmentosa

Kiora Pharmaceuticals announced the dosing of the first patient in a first-in-human open-label clinical trial for KIO-301, intended to restore vision loss in patients with retinitis pigmentosa (RP). 

"We are thrilled the first patient has been treated and I'm very encouraged with our observations to date," said Robert Casson, MB,BS (Hons), of the Royal Adelaide Hospital, principal investigator on the study. "Notably, the patient is clinically doing well and the drug appears to be safe and well tolerated. While early in the study, patient feedback supports improvement in vision. We look forward to assessing additional data with this first and additional patients as we continue enrolling."

ABACUS is a phase 1b open-label, single ascending dose clinical trial for people living with retinitis pigmentosa. The study will enroll six patients and evaluate 12 eyes. The first cohort of three patients will include individuals with no or bare light perception due to the progression of RP. The second cohort will include patients able to detect hand motion and count fingers. The primary endpoints are safety and tolerability, with secondary efficacy endpoints including object identification and contrast assessment, navigation, functional MRI and other ophthalmic and quality-of-life assessments. This single-site study is being conducted at The Royal Adelaide Hospital (RAH) in Adelaide, South Australia.

“We believe KIO-301 is capable of restoring meaningful vision in patients living with retinitis pigmentosa,” said Brian M. Strem, PhD, President and Chief Executive Officer of Kiora. “This is a pivotal development for Kiora, as KIO-301 could become the first available vision restoring treatment option for patients suffering substantial vision loss due to the deleterious effects of RP. An ability to demonstrate safety in this first-in-man study will be a big step forward in establishing proof-of-concept for KIO-301 and photoswitches for retinal reanimation.”

KIO-301 is a visible light-sensitive small molecule that acts as a reversible 'photoswitch,' specifically designed to restore the eyes' ability to perceive and interpret light in visually impaired patients. KIO-301 selectively enters retinal ganglion cells (those downstream of degenerated rods and cones) and 'switches' them into light sensing cells, capable of signaling the brain as to the presence or absence of visible light.