Cash-strapped Kiora sharpens focus on rare retinal diseases, looks to partner off other assets

Kiora Pharmaceuticals is narrowing its line of sight to only two assets that tackle rare retinal diseases, while two other programs will be handed off to prospective partners.

The biotech is prioritizing small-molecule therapy KIO-301 in several indications, according to second-quarter earnings documents shared Aug. 8. The asset is designed to restore vision for individuals with genetic or age-related retinal degeneration and is currently being studied in a phase 1b clinical trial for retinitis pigmentosa called ABACUS.

Based on early data that found the lowest dose of KIO-301 safe and tolerable, Kiora hopes to expand the candidate into other orphan retinal disease programs, such as the genetic disorders choroideremia and Stargardt disease. There currently aren’t treatments available for any of the three diseases, according to Kiora.  

The biotech believes KIO-301’s mechanism of action should be applicable in choroideremia and Stargardt disease because there’s significant overlap with the pathology of retinitis pigmentosa, according to an Aug. 8 letter to investors. The biotech also recently partnered with the Choroideremia Research Foundation—a nonprofit patient advocacy group—to help advance KIO-301 in the indication.

Kiora only has the one KIO-301 trial at the moment and expects to complete all patient dosing for the phase 1b in the third quarter of this year, with top-line results forecast for the fourth quarter.

The biotech cited the initial trial findings as a major reason for sharpening its clinical focus to rare retinal diseases, a shift that means only one other asset—known as KIO-104—will remain on board. The therapy is designed to treat posterior noninfectious uveitis, a rare intraocular inflammatory disease.

Kiora intends to funnel the resources previously allocated to KIO-101, a midstage small molecule designed to treat the ocular presentation of rheumatoid arthritis, to KIO-104. That therapy uses the same active ingredient as KIO-101 but is formulated for intravitreal delivery. The California biotech said any further development of KIO-101 and KIO-201—a form of hyaluronic acid made to speed up corneal wound healing—is dependent on potential partners who have the resources to pick up the therapies.  

At the end of the second quarter, Kiora had $8 million in cash and cash equivalents on hand.