Kiora Pharmaceuticals has secured grant funding from the Choroideremia Research Foundation (CRF) in support of validating functional vision assessments for patients with profound blindness.

Functional vision assessments are task-oriented challenges designed to mimic real-world environments. Importantly, functional vision assessments have served as approvable endpoints in registration/marketing authorization studies by worldwide regulators. This grant support will aid in funding further validation of the Multiluminence Orientation and Mobility (MLOMTM) suite of tests, designed and developed in partnership with Ora, Inc., enabling their use in Kiora's upcoming ABACUS-2 Phase 2 clinical trial assessing KIO-301.

ABACUS-2 is a randomized, controlled trial investigating KIO-301 for vision restoration in patients with late-stage retinitis pigmentosa; and if successful, could serve to restore vision in patients with choroideremia and other inherited retinal diseases (IRDs).

Approval for the clinical validation study was recently granted. The validation work can now begin shortly and will be performed in collaboration with Professor Robert Casson, MBBS (Hons), M.Biostat, DPhil, FRANZCO, consultant ophthalmologist at the Royal Adelaide Hospital and hHad of Ophthalmology and Visual Science at Adelaide University. The goal is to work with IRD patients to refine and validate specific endpoints to objectively and reliably measure functional vision changes, providing a standard and acceptable assessment for global regulatory bodies.

"Our patient community is encouraged by the new technologies and treatments under development for Choroideremia. Importantly, we also recognize that rigorous, standardized assessments of vision correlating to improvements in everyday life are required to bring these treatments to the marketplace. The CRF's contribution under this grant will help ensure appropriate tests are available for all drug developers. This grant is ultimately about providing greater clarity on the pathway to approvability," said Kathi Wagner, Executive Director of the CRF.

This validation work will build upon learnings from Kiora's ABACUS-1 trial, a feasibility study assessing the safety and efficacy of its investigational drug, KIO-301. With feedback from Kiora's pre-IND meeting with the US FDA, Kiora and Ora will validate the latest generation of functional endpoints.

According to Keith Lane, Vice President, Posterior Segment of Ora, Inc., "We are thrilled to play this important role in developing vision restoring therapies to those most in need and look forward to having the MLOM tests front and center for next-generation therapies."

The validation work will be performed in Australia as part of an extension to ABACUS-1.

"We want to thank the CRF for their financial support and entrusting us with taking on this important work. As we look to advance KIO-301 through clinical development across multiple IRDs, including choroideremia, input from regulators, physicians, and ultimately patients are essential in helping shape the pathway to market for not only our drug but for therapies potentially benefiting the entire community," added Eric Daniels, MD, MBA, Chief Development Officer of Kiora.