Kiora Pharmaceuticals presented preliminary results from an ongoing clinical trial showing its investigational drug, KIO-301, has the potential to restore light perception in patients who are blind or living with ultra-low vision due to retinitis pigmentosa. KIO-301 is a light-sensing small molecule designed to reactivate visual function of the eye in response to light. The results were presented at the Association for Research in Vision and Ophthalmology (ARVO) annual meeting in New Orleans.

Quantitative and qualitative measures from early patients in the study were reported. The highlighted case study presented is a patient with no light perception and received a low dose injection of KIO-301 in one eye. The following observations were reported: Patient-reported improvement in the ability to perceive a contrast between light and dark at days seven 14 and 29.

Improvement in object identification. Positive impact in overall functional vision as it relates to the use of vision in everyday activities. Substantial improvement in quality of life, as measured by the National Eye Institute's visual functional questionnaire.

There was an increase of six points (an increase of two to four points is considered clinically meaningful). Imaging data, using functional MRI, showed significantly increased activity in areas of the brain (striate V1 region of the visual cortex) at days three and 15 post-injection compared to baseline. The initial dose was safe and well-tolerated at day 29, with no adverse events reported throughout the study.

The duration of effect is consistent with preclinical pharmacokinetic data. Retinitis pigmentosa is a group of rare, inherited diseases that involve a breakdown and loss of the photoreceptor cells in the retina, the light-sensing tissue that lines the back of the eye. This progressive disease usually initially presents with difficulty seeing at night and a loss of side (peripheral) vision, extending to tunnel vision and eventually, full blindness.

The trial, called the ABACUS study, is a Phase 1b open-label, single ascending dose clinical trial for people living with retinitis pigmentosa. The study comprises the enrollment of six patients and the evaluation of 12 eyes, following monitoring for 29 days. The first cohort of three patients includes individuals with no or bare light perception due to the progression of RP.

The second cohort includes three 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, fMRI and other ophthalmic and quality-of-life assessments. This study is being conducted at multiple sites in Adelaide, South Australia, including The Royal Adelaide Hospital.

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.