Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — Investor Presentation (2026)
24 slides from Kiora Pharmaceuticals's investor presentation, 2026
Slide 1: Kiora Pharmaceuticals, Inc. Q2 2026 Corporate Overview
Kiora Pharmaceuticals, Inc. NASDAQ: KPRX Q2 2026 | Corporate Overview
Slide 2: Forward Looking Statements
Forward Looking Statements Some of the statements in this presentation are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements include statements relating to, among other things, the developm
Slide 3: Corporate Highlights
Corporate Highlights KIO-301 Ion channel modulator, acting as photoswitch, to restore inherited vision loss Two Innovative Drugs KIO-104 Anti-inflammatory, disease modifying drug for retinal inflammation KIO-301 100K+ patients in US with RP and other IRDs Significant Patient Need KIO-104 1.2M patien
Slide 4: Targeting the Retina to Slow, Stop, or Restore Vision Loss
Targeting the Retina to Slow, Stop, or Restore Vision Loss Development Pipeline of Proprietary Small Molecule Therapeutics Product Indication Preclinical Phase 1 Phase 2 Phase 3 Commercial Rights Retinitis Pigmentosa Théa Open (Mutation Agnostic)* Innovation (global less Asia) KIO-301 Choroideremia
Slide 5: KIO-301
KIO-301 Small Molecule "Photoswitch" Modulates Ion Channels Targeting Vision Restoration in Inherited Retinal Diseases 5
Slide 6: In Patients with RP, Downstream Neurons Remain Viable
In Patients with RP, Downstream Neurons Remain Viable RGC Bipolar Cell Rod Cone To the brain (connection intact) Bipolar cells and RGCs relay signal from rods and cones to Photoreceptors (rods and cones) sense light and pass the brain, where the image is “processed” the signal to special transmittin
Slide 7: KIO-301 (MOA): Turns RGCs "ON" in the Presence of Light
KIO-301 (MOA): Turns RGCs “ON” in the Presence of Light ▪ When photoreceptors die ⟶ downstream neurons (RGCs) are not capable of being activated ▪ KIO-301 preferentially enters these RGCs and turns them “ON” in the presence of light* KIO-301 Extracellular P2X7‡ KV/HCN Channels trans • KIO-301 lodges
Slide 8: KIO-301 Reanimates the Retina & Changes Behavior
KIO-301 Reanimates the Retina & Changes Behavior Extensive Validation in Preclinical Models Before KIO-301 After KIO-301 Light Dark Neuron 2014: 81, 800-813. 8
Slide 9: Retinitis Pigmentosa
Retinitis Pigmentosa A Disease with No Available Treatments Normal Vision Market Opportunity ▪ ~100k patients in US (Provider: Retina Specialists [~3k]) ▪ Estimated total cost to US healthcare system in 2019: $3.7B Clinical Presentation ▪ Night blindness, reduced visual field range and eventual loss
Slide 10: KIO-301: Phase 1b Study Design (ABACUS)
KIO-301: Phase 1b Study Design (ABACUS) Open Label, Single Ascending Dose Trial – 2 Sites (Australia) Cohort 1: KIO-301 Cohort 1: KIO-301 (7.5 µg) Contralateral (25 µg) N=6 3/cohort Binocular Cohort 2: KIO-301 Cohort 2: KIO-301 (25 µg) Contralateral (50 µg) Day 7-8 Day 29-30 Visit 3 Visit 5 Day -120
Slide 11: Changes in Vision & Patient QoL
Changes in Vision & Patient QoL Kinetic Visual Field Visual Acuity Mean Change from BL ± 80% CL Mean ± SEM 2.50 D29-BL 2.25 HM logMAR D14-BL 2.00 * D7-BL CF 1.75 D2-BL Off-Chart 1.50 -20 -10 0 10 20 30 40 0 10 20 30 Degrees of Change from Baseline Day *p<0.05 High Contrast Room Exit Test Quality of
Slide 12: Functional MRI
Functional MRI Significantly Increased of Cortical Activity Occipital Cortex (Mean ± SEM) Voxels (Change from Baseline) 2500 * * p<0.05 2000 * * 1500 1000 500 0 2 14 28 Day All Eyes (n=12) NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers 12
Slide 13: Patient Testimonials
Patient Testimonials www.youtube.com/@kiorapharma 13 Additional videos available at https://kiorapharma.com/inherited-retinal-diseases/
Slide 14: KIO-301-2101: Phase ABACUS-2
KIO-301-2101: Phase 2 ABACUS-2 Multiple Dose (6 wk interval), Bilateral Injection, Double-Masked, 36-Patient, Randomized, Controlled Trial Cohort 1 (Start Dose): Cohort 2 (Dose Escalated): ▪ Same as Cohort 1 but dosed at 100 µg ▪ RP patients who are ⨠ NLP (logMAR ≥ 3.0 OU) (n=6, Cohort 1A) ⨠ ULV (lo
Slide 15: Ion Channel Modulators: Value in Ophthalmic and Beyond
Ion Channel Modulators: Value in Ophthalmic and Beyond Vision (Partnered) Neuro Epilepsy Chronic Cardiology KIO-300 Family pain Pulmonary 15
Slide 16: KIO-104
KIO-104 Intravitreal Small Molecule DHODH Inhibitor Steroid-Sparing Approach to Retinal Inflammation 16
Slide 17: DHODH Inhibition Causes Nucleotide Starvation in Activated T-Cells
DHODH Inhibition Causes Nucleotide Starvation in Activated T-Cells Active Inflammation DNA (RNA) De Novo Pathway of Pyrimidine Synthesis A T(U) KIO-104 DHODH G C Mitochondrion Purines Pyrimidines Nucleotides Protein & Lipid Phospholipid (New RNA/DNA) Glycosylation Metabolism Homeostasis Cell Adhesio
Slide 18: Retinal Inflammation: Posterior Non-Infectious Uveitis
Retinal Inflammation: Posterior Non-Infectious Uveitis T Cell Driven Inflammation in the Back of the Eye Can Lead to Vision Loss Need for steroid-sparing anti-inflammatory delivered locally to the eye Major Causes Autoimmune disorders (i.e., lupus, multiple sclerosis, psoriasis, rheumatoid arthritis
Slide 19: Phase 1/2a SAD Study Design
Phase 1/2a SAD Study Design Posterior Non-Infectious Uveitis Duration [Study Days] -14 -7 0 2 7 14 21 28 KIO-104 Tasks Screening / Baseline Exam Exam Exam Exam Exam Injection ▪ Patients with chronic, posterior non-infectious uveitis Study Design ▪ Prospective, open label, multi-center, dose escalati
Slide 20: Phase 1/2a Results: KIO-104 Improved Visual Acuity and Reduced Cystoid Macular Edema
Phase 1/2a Results: KIO-104 Improved Visual Acuity and Reduced Cystoid Macular Edema Visual Acuity Cystoid Macular Edema‡ 16 14 Mean Change in Letters 12 from Baseline at D28 10 Steroids* Baseline (558µm) 8 6 4 Humira* 2 0 0.3 μg 0.6 μg 1.2 μg N=4 N=4 N=4 Day 28 (421µm) ‡ 40% of eyes with vision thr
Slide 21: KIO-104: Phase 2 MAD Study Design (KLARITY) – Macular Edema
KIO-104: Phase 2 MAD Study Design (KLARITY) – Macular Edema 2-Step, Randomized, Open-Label, Steroid-Sparing, Dose Expansion Trial Low Dose (3.5 μg) HTD, E2W, Single Dx N=8 N=20 Macular Edema* High Dose (10 μg) HTD, E4W, Single Dx // Day -30 to -1 Day 1 Day 90 Day 1 Day 90-120 Screening 3 doses E2W 3
Slide 22: PVR Overview
PVR Overview Proliferative vitreoretinopathy (PVR) is a rare inflammatory disorder of the retina that leads to severe retinal scarring and blindness. It is the leading cause of failure of retinal reattachment surgery. • Stage 1: • BRB breakdown • INFLAMMATORY reaction • Influx of blood-derived cells
Slide 23: KIO-104: Proliferative Vitreoretinopathy
Percentage of Eyes with Scars (%) K 100 0 20 40 60 80 IO -1 Ve 04 h K IO Lo icle -1 w 04 D H os e ig h D os e Rabbit Model of Retinal Detachment Number of Scars K 0 5 10 15 20 25 IO -1 Ve 04 h K IO Lo icle -1 w 04 D H os e ig h D os e KIO-104: Proliferative Vitreoretinopathy Mean Scar Length (μm) K
Slide 24: Thank You
Thank You NASDAQ: KPRX