The Choroideremia Research Foundation (CRF) has granted funding to Kiora Pharmaceuticals, Inc. funding in support of its inherited retinal disease (IRD) clinical trial endpoints and testing validation for KIO-301, an investigational therapy.
Let’s start with KIO-301.
As a visible light-sensitive small molecule, KIO-301 is designed as a (molecular) reversible photoswitch with the potential to restore the eye’s ability to perceive and interpret light in visually-impaired retinitis pigmentosa (RP) patients.
Its purpose: to specifically enter photoreceptors’ key downstream retinal cells—bipolar and retinal ganglion cells (RGCs)—and turn them into light-sensing cells capable of notifying the brain of the presence / absence of light.
Didn’t we just hear news about this therapeutic?
Indeed! In February 2023, we reported that Théa Open Innovation, a sister company of Laboratoires Théa, acquired the exclusive rights to KIO-301 via a strategic development and commercialization agreement.
Among the details of the deal is the stipulation that Kiora will be primarily responsible for clinical development design and implementation through phase 2; Théa, on the other hand, will be largely responsible for phase 3 clinical trials + securing regional marketing authorizations for KIO-301.
See here for our full coverage.
So … what’s the clinical data on it?
Most recently (and by “recently” we mean May 2023), promising preliminary data was reported from the ongoing, open-label, dose-escalating phase 1b ABACUS study (NCT05282953) that assessed KIO-301 for RP in two cohorts and 4 dose levels.
The findings suggested that the initial low dose of KIO-301 has a favorable safety profile, considered well-tolerated, and capable of potentially improving light perception in patients with ultra-low vision (ULV) or blindness.
I’m up-to-date! Now talk about this grant.
Gifted by the CRF—the largest 501(c)(3) organization in the world dedicated to supporting research, education, and advocacy of choroideremia (CHM)—the funding will be put toward further validation of the Multilumiance Orientation & Mobility (MLOM) suite of tests that were designed by Kiora (in partnership with Ora, Inc.)
More about MLOM: These tests relate to mobility and functional visionunder controlled lighting.
The data on them so far: Reported during the American Academy of Ophthalmology (AAO) annual meeting, topline data from the ABACUS study found that KIO-301 trended toward improvement from 24.7 ± 15.2% at baseline to 60.0 ± 20.7% at day 28 (p=NS).
And how will MLOM be used?
Per the company, this suite of tests will be utilized in the upcoming ABACUS-2 phase 2 trial on KIO-301, which was recently granted clinical validation.
Study details: The controlled, double–masked, randomized, open-label, ascending-dose study is dosing up to 20 participants (aged 18+) diagnosed with late-stage RP and choroideremia.
Dosings: Randomized into four cohorts (cohorts 1-3 for RP; cohort 3 for only choroideremia), participants will receive ascending doses of KIO-310 via intravitreal injection over a 90-day period.
What’s being measured?
The phase 2 study will evaluate the number of participants with treatment-related adverse events (TRAEs) over 84 days, as determined based on:
- Overall changes in retinal thickness and appearance
- Clinical chemistry and hematology
- Other eye assessments
Gotcha. So who will be performing the validation of this study?
Kiora reported a collaboration between the company and principal investigator Robert Casson, MBBS (Hons), M.Biostat, DPhil, FRANZCO, a professor and consultant ophthalmologist at the Royal Adelaide Hospital and hHad of Ophthalmology and Visual Science at Adelaide University in Australia.
And the goal of this?
The company intends to work with IRD patients in order to “refine and validate specific endpoints to objectively and reliably measure functional vision changes, providing a standard and acceptable assessment for global regulatory bodies.”.
Note: This validation process will be conducted in Australia as an extension to the ABACUS-1 trial.