Kiora Pharmaceuticals Gains Approval to Launch Phase 2 Trial of KIO-301 for Retinitis Pigmentosa Treatment
Wednesday, October 30, 2024
Kiora Pharmaceuticals has received regulatory clearance to commence a Phase 2 clinical trial to assess KIO-301 for vision restoration in individuals with retinitis pigmentosa.
The ABACUS-2 study will involve 36 patients in a multi-centre, double-masked, randomised, controlled, multiple-dose format, specifically enrolling those with ultra-low or no light perception, regardless of the genetic mutation associated with their retinitis pigmentosa.
Initial dosing is anticipated for next year, pending validation of novel functional vision endpoints that could serve as primary endpoints for future registration studies across the US, Europe, and other regions.
Currently, no approved treatments exist for retinitis pigmentosa, and Kiora's approach seeks to meet regulatory guidance by focusing on functional vision outcomes that reflect everyday life impacts.
Extensive preliminary work on validating these functional endpoints is underway, aiming to optimise success in a potential single Phase 3 trial for US and European market approval. This validation, supported by a grant from the Choroideremia Research Foundation, is being performed in collaboration with Théa Open Innovation.
Kiora has selected trial sites and clinical investigators from leading inherited retinal disease centres in Australia. Participants will be randomised 2:1 to receive intravitreal doses of KIO-301 or a control treatment in both eyes.
The active arm will administer either a high (100 micrograms) or low (50 micrograms) dose across three doses six weeks apart, with participants monitored for three months following the final dose. Those initially in the control arm will have the option to cross over to the active treatment following this phase.
Primary study outcomes will focus on safety and tolerability, while key efficacy assessments will include functional vision, visual acuity (assessed by the Berkeley Rudimentary Vision Test), visual fields (assessed by perimetry), and quality-of-life evaluations specific to ultra-low vision.
Since establishing a partnership with Théa Open Innovation, Kiora has made rapid advancements towards launching ABACUS-2. Preliminary data from Phase 1b trials suggests KIO-301 has meaningful potential for vision restoration and, due to its unique mechanism, may benefit individuals regardless of the specific genetic mutation underlying their retinitis pigmentosa.
This characteristic is critical given the range of gene mutations associated with inherited retinal diseases.
KIO-301, a small molecule photoswitch, is designed to restore light sensitivity in retinal ganglion cells (RGCs), bypassing damaged rods and cones and activating in response to light. This process is reversible, enabling the cells to turn on in the presence of light and off in its absence.
By conferring light-sensing capabilities to RGCs, KIO-301 aims to support visual processing, offering promise as a broad-reaching therapeutic for patients with inherited retinal diseases.
Source: kiorapharma.com
