Kiora Pharmaceuticals, Inc. has announced it will be significantly shifting its clinical development pipeline to focus on rare retinal disease candidates —signaling a major opportunity for potential strategic partnerships in the near future.
Let’s take it from the top.
According to the company’s Q2 2023 Financial Results, announced on August 8, 2023, the company will transfer its clinical resources away from KIO-101—designed to treat the ocular presentation of rheumatoid arthritis (OPRA)—to KIO-104.
Hold up … refresh me on KIO-104.
Similar to KIO-101, KIO-104 is a third-generation small molecule dihydroorotate dehydrogenase (DHODH) inhibitor—part of a class of non-steroidal autoimmune disease drugs.
To note, both KIO-101 and KIO-104 contain the same active compound.
However, unlike KIO-101—which is formulated as a topical eye drop—KIO-104 is designed as an intravitreal injection for the potential to decrease intraocular inflammation in posterior non-infectious uveitis.
Any clinical data on it?
Yup! In October 2022, Kiora released data, published in Frontiers in Medicine, from a phase 1/2a study that demonstrated a single intravitreal injection of KIO-104 decreased inflammation in a dose-dependent fashion and significantly improved visual acuity (VA) for the duration of the study.
So what’s going to happen to KIO-101?
The company is looking for potential strategic partnerships to continue the development of its ancillary anterior segment program for both KIO-101 and KIO-201 (a topical eye drop intended to treat persistent corneal epithelial defects [PCED] and corneal surgical wounds).
Gotcha. Now what else are they going to focus on?
Also included is KIO-301, a visible light-sensitive small molecule (called a “molecular photoswitch”) designed as a reversible photoswitch to possibly restore the eye’s ability to perceive and interpret light in visually impaired retinitis pigmentosa (RP) patients.
How does KIO-301 work?
The therapeutic is designed 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.
To note, KIO-301 was granted Orphan Drug Designation (ODD) by the FDA in March 2022.
What’s the latest clinical data?
In May 2023, Kiora reported favorable preliminary data 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.
And future development plans?
The company expects to complete all dosings of KIO-301 in the ABACUS study in Q3 2023, with topline data for all patients and doses presented in Q4 2023.
Additionally, Kiora is planning to expand clinical development of KIO-301 to include choroideremia (in partnership with the Choroideremia Research Foundation) and Stargardt’s disease.