Kiora Pharmaceuticals (KPRX) LD Micro Conference summary

Unmet need and disease focus

• Retinal diseases cause severe vision loss and blindness, with high patient despair and no current treatments.

• Blindness is ranked as one of the most feared conditions, above Alzheimer's and cancer.

• Two main assets target inherited and inflammatory retinal diseases with significant market opportunity.

KIO-104: Inflammation and clinical progress

• KIO-104 is a non-steroidal small molecule for retinal inflammation, targeting conditions like posterior non-infectious uveitis.

• Current steroid treatments have adverse effects; KIO-104 aims to reduce inflammation and relapses.

• Phase I/II study showed no serious adverse events, excellent tolerability, and improved visual acuity.

• Phase II trial is planned for the first half of 2025, expanding to multiple disease states with macular edema.

KIO-301: Photoswitch technology and clinical data

• KIO-301 is a small molecule photoswitch that confers light sensitivity to retinal ganglion cells, bypassing dead photoreceptors.

• Preclinical and mouse models showed restored light response and behavioral changes.

• Phase I-B ABACUS study in retinitis pigmentosa showed improved visual field, acuity, and light perception, especially in patients with no prior light perception.

• Functional MRI and patient testimonials confirmed positive impact and safety.