Kiora Pharmaceuticals (KPRX) investor relations material

Pipeline and product overview

• Two lead drugs: KIO-301 for inherited retinal diseases and KIO-104 for retinal inflammation, both addressing significant unmet needs in large patient populations.

• KIO-301 targets over 100,000 US patients with retinitis pigmentosa and related disorders; KIO-104 targets 1.2 million US patients with retinal inflammatory diseases.

• KIO-301 acts as a photoswitch modulating ion channels to restore vision, while KIO-104 is a steroid-sparing DHODH inhibitor for inflammation.

• Partnerships with Théa (global rights outside Asia) and Senju (option for Asia) provide milestone and royalty opportunities.

• Cash runway extends into late 2028, supporting ongoing development.

Clinical development and results

• KIO-301 demonstrated reactivation of retinal ganglion cells and improved visual function in preclinical and Phase 1b studies.

• Phase 1b ABACUS trial showed improvements in visual field, acuity, quality of life, and increased cortical activity on fMRI in patients with severe vision loss.

• Phase 2 ABACUS-2 trial is ongoing, double-masked, and randomized, with 50% enrollment as of May 2026.

• KIO-104 Phase 1/2a study in posterior non-infectious uveitis showed improved visual acuity and reduced cystoid macular edema.

• KIO-104 is advancing to a Phase 2 MAD study (KLARITY) for macular edema and other indications.

Market opportunity and medical need

• Retinitis pigmentosa affects ~100,000 in the US, with no available treatments and significant impact on quality of life and healthcare costs.

• Posterior non-infectious uveitis and other retinal inflammatory diseases affect over 1.2 million in the US, with current treatments limited by steroid-related side effects.

• Proliferative vitreoretinopathy is a rare but severe complication leading to blindness and surgical failure.