Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — H.C. Wainwright 5th Annual Ophthalmology Virtual Conference (August 2025)
25 slides from Kiora Pharmaceuticals's conference presentation, Aug 13, 2025
Slide 1: Corporate Overview
Kiora Pharmaceuticals, Inc. NASDAQ: KPRX 13 Aug 2025 | Corporate Overview H.C. Wainwright 5th Annual Ophthalmology Virtual Conference
Slide 2: Forward Looking Statements
2 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 develo
Slide 3: Why Retinal Diseases?
3 Why Retinal Diseases? “…the last light sensations faded and the dark discs had finally overwhelmed me. I had fought them bravely, as it seemed to me, for thirty-six years, but to no avail. It was then I began to sink into the deep ocean, and finally learn how to touch the rock on the far side of d
Slide 4: Corporate Highlights
4 Corporate Highlights Developing Two Innovative Drugs KIO-301 Molecular photoswitch has potential to restore vision lost to an IRD KIO-104 Anti-inflammatory, disease modifying drug for retinal inflammation Targeting Significant Patient Need KIO-301 100K patients in US with RP and other IRDs KIO-104
Slide 5: Financial Metrics & Capital Structure
5 Financial Metrics & Capital Structure Cash & short-term investments $20.7 MM Receivables & tax credits $3.1 MM Runway Late 2027 Phase 2 readouts expected Early 2027 Market cap (inc. PFWs) ~$11 MM Common TSO 3.4 MM shares PFWs 0.9 MM shares Effective TSO 4.3 MM shares Owner Shares 1 AIGH Capital Ma
Slide 6: Targeting the Retina to Slow, Stop, or Restore Vision Loss
6 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 KIO-301 Intravitreal Retinitis Pigmentosa (Mutation Agnostic)* Théa Open Innovation (global less Asia)
Slide 7: KIO-301 Small Molecule Targeting Vision Restoration Inherited Retinal Diseases
7 KIO-301 Small Molecule Targeting Vision Restoration Inherited Retinal Diseases
Slide 8: Downstream Neurons Remain Viable
8 Downstream Neurons Remain Viable Many Inherited Retinal Diseases, including Retinitis Pigmentosa (RP), result in death of photoreceptors Bipolar Cells and Retinal Ganglion Cells (RGCs) remain intact and retain ability to send signals to the brain Photoreceptors (rods and cones) sense light and
Slide 9: KIO-301 (MOA): Turns RGCs ON in the Presence of Light
9Neuron. 92, 100-113 (2016) trans cis 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* ‡ P2X7 is solely expressed on RGCs and amacrine cells in the retina * Visual light c
Slide 10: KIO-301 Reanimates the Retina & Changes Behavior
10 KIO-301 Reanimates the Retina & Changes Behavior Extensive Validation in Preclinical Models Neuron 2014: 81, 800-813.
Slide 11: Market Opportunity: Retinitis Pigmentosa
11 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 of central vision Visual acuity declines 50% of patients are not q
Slide 12: KIO-301: Phase 1b Study Design (ABACUS)
12 KIO-301: Phase 1b Study Design (ABACUS) Open Label, Single Ascending Dose Trial – 2 Sites (Australia) Two Cohorts, non-randomized, open-label, single IVT injection per eye Cohort 1 – NLP/BLP patients; Cohort 2 – HM/CF patients Primary – AEs, PK & labs Secondary – Assessment days (shown on
Slide 13: Changes in Vision & Patient QoL
13 Changes in Vision & Patient QoL -20 -10 0 10 20 30 40 D2-BL D7-BL D14-BL D29-BL Degrees of Change from Baseline * *p<0.05 0 10 20 30 1.50 1.75 2.00 2.25 2.50 Day lo gM AR Off-Chart CF HM 0 30 35 40 45 50 Day VF Q -2 5 C om po si te S co re 0 10 20 30 0 20 40 60 80 100 Day R es po ns e R at e (% )
Slide 14: Functional MRI Significantly Increased Cortical Activity
14 Functional MRI Significantly Increased of Cortical Activity NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers 2 14 28 0 500 1000 1500 2000 2500 Occipital Cortex (Mean ± SEM) Day Vo xe ls (C ha ng e fro m B as el in e) * * * * p<0.05 All Eyes (n=12)
Slide 15: Patient Testimonials
15 Patient Testimonials www.youtube.com/@kiorapharma Additional videos available at https://kiorapharma.com/inherited-retinal-diseases/
Slide 16: ABACUS-1 Phase 1b Clinical Trial Key Takeaways
16 ABACUS-1 Phase 1b Clinical Trial Key Takeaways Patients report improvements in vision Consistent with objective clinical assessments Follow-on study will include sham group Pilot study limitations Non-controlled Small sample size Clear pathway to approvability Functional vision improvem
Slide 17: KIO-301-2101: Phase 2 ABACUS-2
17 KIO-301-2101: Phase 2 ABACUS-2 Multiple Dose, Bilateral Injection, Double-Masked, 36-Patient, Randomized, Controlled Trial Recruitment in each Cohort begins with 6 patients (1:1) for SRC review and escalation/de-escalation recommendation Cohort 2 (Dose Escalated): Same as Cohort 1 but dosed at
Slide 18: KIO-104 Intravitreal Small Molecule DHODH Inhibitor
18 KIO-104 Intravitreal Small Molecule DHODH Inhibitor Steroid-Sparing Approach to Retinal Inflammation
Slide 19: DHODH Inhibition Causes Nucleotide Starvation in Activated T-Cells
19 DHODH Inhibition Causes Nucleotide Starvation in Activated T-Cells Pyrimidines are key co-factor for glycoprotein, glycolipid, and phospholipid synthesis Homeostasis Normal Cell Function Salvage Pathway (Recycling of Pyrimidines) Active Inflammation De Novo Pathway of Pyrimidine Synthesis Nucleot
Slide 20: KIO-104: Proliferative Vitreoretinopathy Rabbit Model
20 KIO-104: Proliferative Vitreoretinopathy Rabbit Model of Retinal Detachment CONFIDENTIAL Veh icl e KIO -10 4 L ow Dose KIO -10 4 H igh Dose 0 50 100 150 M ea n Sc ar L en gt h (μ m )
Slide 21: Need for steroid-sparing anti-inflammatory delivered locally to the eye
21 Need for steroid-sparing anti-inflammatory delivered locally to the eye Retinal Inflammation: Posterior Non-Infectious Uveitis T Cell Driven Inflammation in the Back of the Eye Can Lead to Vision Loss Major Causes Autoimmune disorders (i.e., lupus, multiple sclerosis, psoriasis, rheumatoid arthri
Slide 22: Phase 1/2a SAD Study Design
22 Phase 1/2a SAD Study Design Posterior Non-Infectious Uveitis Safety and tolerability Improvement of inflammation Blood PK of KIO-100 Single intravitreal injection of 300ng, 600ng, and 1200ng Patients with chronic, posterior non-infectious uveitis Prospective, open label, multi-center,
Slide 23: Phase 1/2a Results: KIO-104 Improved Visual Acuity
23 Phase 1/2a Results: KIO-104 Improved Visual Acuity and Reduced Cystoid Macular Edema * Historical Controls (Yeh et al, Retina 00, 1-9, 2018; Suhler et al. Visual III, Ophthalmology 125, 7, 2018.) IVT - Intravitreal 0 2 4 6 8 10 12 14 16 0.3 μg 0.6 μg 1.2 μg Steroids* Humira*M ea n C ha ng e in L
Slide 24: KIO-104: Phase 2 MAD Study Design (KLARITY)
24 KIO-104: Phase 2 MAD Study Design (KLARITY) 2-Step, Randomized, Open-Label, Steroid-Sparing, Dose Expansion Trial Part A: Dose informing short-term study in macular edema (multiple clinical indications) Part B: Dose expansion with highest tolerated dose Primary: AEs, PK, labs Secondary: B
Slide 25: Thank You
Thank You NASDAQ: KPRX