Kiora Pharmaceuticals (KPRX) Lytham Partners Fall 2024 Investor Conference summary

Strategic focus and pipeline overview

• Developing treatments for retinal diseases with significant unmet need, aiming to improve sight in patients with severe vision loss due to inherited or age-related diseases.

• Pipeline includes KIO-301, a small molecule photoswitch for retinitis pigmentosa and other inherited retinal diseases, and KIO-104, a non-steroidal small molecule for retinal inflammation.

• KIO-301 is mutation agnostic, potentially benefiting all patients with inherited retinal diseases regardless of genetic mutation.

• KIO-104 leverages knowledge from rheumatology, targeting conditions like posterior non-infectious uveitis, diabetic macular edema, and retinal vein occlusion.

• Kiora’s pipeline leverages innovative modalities, including molecular photoswitches and DHODH inhibitors, to address gaps in current treatments.

Clinical development and results

• KIO-301 showed promising Phase 1b results in retinitis pigmentosa, including improved visual function and no safety concerns, with a Phase 2 trial (ABACUS-2) set to begin in 2H 2024.

• KIO-104 demonstrated improved visual acuity and reduced macular edema in Phase 1/2a for posterior non-infectious uveitis; a Phase 2 trial (CLARITY) is planned for Q4 2024.

• Functional MRI data post-KIO-301 treatment demonstrated increased neural activity in the brain's visual processing center, supporting the drug's mechanism of action.

• KIO-104 phase I/II study in posterior non-infectious uveitis showed no serious adverse effects, excellent tolerability, and meaningful improvements in visual acuity and retinal swelling.

• Phase II trial for KIO-104 planned for H1 2025, targeting multiple disease states with macular edema.

Regulatory and partnership updates

• Ongoing dialogue with FDA and European regulators; phase II trial design for KIO-301 aligned with regulatory expectations for functional endpoints.

• Strategic partnership with Théa Open Innovation for global (excluding Asia) rights to KIO-301, with $16 million upfront, full phase II/III funding, and up to $285 million in milestones plus royalties.

• Partnership de-risks KIO-301 and provides non-dilutive capital, enabling continued development of KIO-104, for which worldwide commercial rights are retained.

• Kiora is pursuing additional partnerships for KIO-301 rights in Asia.

• Strategic relationships, including the Théa partnership, are expected to support late-stage development and commercialization.