Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — Investor Presentation (October 2026)
29 slides from Kiora Pharmaceuticals's investor presentation, Oct 29, 2026
Slide 1: Kiora Pharmaceuticals, Inc. Q4 2026 Corporate Overview
Kiora Pharmaceuticals, Inc. Q4 2026 | Corporate Overview
Slide 2: Forward-looking statements disclaimer
2 This presentation is provided for informational purposes only. Some of the material and statements in this presentation are "forward-looking" statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include, without limitation
Slide 3: Expanding Kiora's Late-Stage Retinal Therapeutics Pipeline
3 Expanding Kiora’s Late-Stage Retinal Therapeutics Pipeline Added neuroprotective oral calpain inhibitor for CRAO with planned development in NAION KIO-401 New Phase 3-ready asset for Central Retinal Artery Occlusion (from Senju Pharmaceutical) § Global rights outside Asia § Small molecule calpain
Slide 4: Targeting the Retina to Slow or Stop Vision Loss or Restore Sight
4 Targeting the Retina to Slow or Stop Vision Loss or Restore Sight Development Pipeline of Proprietary Small Molecule Therapeutics Program Therapeutic Approach Indication Discovery Preclinical Phase 1 Phase 2 Phase 3 Commercial Rights Diseases of Retinal Ischemia KIO-401 Calpain Inhibitor CRAO Glob
Slide 5: KIO-401 Small Molecule Calpain Inhibitor
5 KIO-401 Small Molecule Calpain Inhibitor
Slide 6: What are Calpains?
6 What are Calpains? Ischemia § Reduced blood flow § Decreased oxygen and glucose § Cellular stress Energy Failure § ↓ATP production § Failure of ion pumps § Loss of ionic homeostasis Calpain Activation § Excess Ca2+ causes dissociation of calpain from calpastatin, resulting in activated calpain § ↑
Slide 7: Central Retinal Artery Occlusion – An Acute Stroke of the Eye
7 Central Retinal Artery Occlusion – An Acute Stroke of the Eye A Disease with No Available Treatments Unfortunately, ocular massage, thrombolytics (tPA) and other methods have limited-to-no efficacy Retinal ischemia activates calpains within 48 hours Patients typically recover ~2 lines after acute
Slide 8: KIO-401: Phase 2a Study Design
8 KIO-401: Phase 2a Study Design* Randomized, Dose Escalating, Multi-Center (Japan Only), Clinical Trial § Multicenter, two cohort, randomized, double-masked versus prospective registry § Baseline logMAR > 1.0 § Primary – Adverse Events (AEs), PK & labs § Secondary – Best Corrected Visual Acuity (ET
Slide 9: KIO-401: Phase 2a Study Results
9 KIO-401: Phase 2a Study Results* Mean Change from Baseline in ETDRS Letters p = 0.0398} p = 0.070 * Study was conducted by Senju
Slide 10: KIO-401: Phase 3 Study Design
10 KIO-401: Phase 3 Study Design* Randomized, Controlled, 14 Sites (Japan Only) Clinical Trial § Multicenter, randomized, controlled, double-masked clinical trial § Baseline logMAR > 1.0 § Primary – Change from Baseline in Best Corrected Visual Acuity (BCVA) at 12 Weeks § Secondary – Change from Bas
Slide 11: KIO-401: Phase 3 Study Results
11 KIO-401: Phase 3 Study Results* Mean Change from Baseline in ETDRS Letters (Observed Data Only) p = 0.0949} p = 0.0195} B as el in e B C V A W o rs e T h an 2 0 /4 0 0 ** * Study was conducted by Senju ** 20/400 (logMAR = 1.3) is World Health Organization definition of legal blindness Responder a
Slide 12: KIO-401: Phase 3 Study Results Categorical Visual Function Improvement
12 KIO-401: Phase 3 Study Results* Categorical Visual Function Improvement * Study was conducted by Senju
Slide 13: KIO-401: Phase 3 Study Results Safety – TEAE Summary
13 Placebo KIO-401 (200 mg) (N=33) (N=33) n (%) events n (%) events Any TEAEs 10 (30.3) 17 13 (39.4) 21 Mild 7 (21.2) 13 9 (27.3) 17 Moderate 2 (6.1) 3 4 (12.1) 4 Severe 1 (3.0) 1 0 0 Serious TEAEs 3 (9.1) 3 1 (3.0) 1 TEAEs leading to death 0 0 0 0 Serious TEAEs except death 3 (9.1) 3 1 (3.0) 1 Sign
Slide 14: KIO-401: CRAO Planned Global Phase 3 Study Design
14 KIO-401: CRAO Planned Global Phase 3 Study Design Randomized, Controlled, up to 50 Sites (US/EU/Asia) Registration, Clinical Trial § Multicenter, randomized (1:1), controlled, double-masked clinical trial § Baseline BCVA worse than 20/200 (predefined subgroup for patients with BCVA worse than 20/
Slide 15: Severe, Irreversible Vision Loss (NAION)
15 Severe, Irreversible Vision Loss Sudden, painless vision loss in one eye – generally irreversible, with no effective treatment available1 Tx Initiated Within 2 Weeks Most patients present days to weeks after onset2 Roughly 10% keep worsening over the following four weeks3 No Current Treatment Tod
Slide 16: Established Treatment: Oral neuroprotection for ischemic injury
16 Established Treatment Oral neuroprotection aimed at ischemic injury, with proof of concept established in CRAO Proven Mechanism of Action Signal In CRAO patients legally blind at presentation, KIO-401 produced roughly 21 ETDRS letters, over four lines of vision, at 12 weeks versus about 9 on plac
Slide 17: KIO-401: NAION Planned Phase 2b Study Design
17 KIO-401: NAION Planned Phase 2b Study Design Randomized, Controlled, Registration-able, Clinical Trial § Multicenter, randomized (1:1), controlled, double-masked clinical trial § Baseline BCVA 20/50-20/500 § Visual Field Mean Deviation worse than or equal to -3.0 dB § Documented disc edema via fu
Slide 18: KIO-301
KIO-301 ig €@KIORA
Slide 19: KIO-301 (MOA): Turns RGCs ON in the Presence of Light
19 Neuron. 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
Slide 20: In Patients with RP, Downstream Neurons Remain Viable
20 In Patients with RP, 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
Slide 21: KIO-301 Reanimates the Retina & Changes Behaviour
21 KIO-301 Reanimates the Retina & Changes Behaviour Extensive Validation in Preclinical Models Neuron 2014: 81, 800-813. Light Dark Before KIO-301 After KIO-301
Slide 22: KIO-301: Phase 1b Study Design (ABACUS)
22 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 23: Changes in Vision & Patient QoL
23 Changes in Vision & Patient QoL * *p<0.0 5 Kinetic Visual Field Mean Change from BL ± 80% CL Visual Acuity Mean ± SEM High Contrast Room Exit Test Mean ± SEM Quality of Life Survey Nature Medicine v32, 1865–1870 (2026)
Slide 24: Functional MRI: Significant Increase of Cortical Activity
24 Functional MRI Significant Increase of Cortical Activity NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers Nature Medicine v32, 1865–1870 (2026)
Slide 25: KIO-301: Phase 2 Randomized, Controlled Clinical Trial (ABACUS-2)
25 KIO-301: Phase 2 Randomized, Controlled Clinical Trial (ABACUS-2) Multiple Dose, Bilateral Injection, Double-Masked, 36-Patient, Randomized, Controlled Trial § Clinical diagnosis of retinitis pigmentosa with visual acuity of logMAR1.6-3.0 OU (ULV) or logMAR ≥ 3.0 (NLP) § Recruitment in each Cohor
Slide 26: KIO-301: Global Partnership Network
26 KIO-301: Global Partnership Network Significant Upside Opportunity via Milestones and Royalties
Slide 27: Leadership and KOLs
Leadership and KOLs 27 &@KIORA
Slide 28: Leadership Team
28 Melissa Tosca, CPA Chief Financial Officer LinkedIn Keith Lane, MBA EVP – Clin Dev & Regulatory LinkedIn Brian M. Strem, PhD President & CEO LinkedIn Stefan Sperl, PhD EVP – CMC, NonClin, IP LinkedIn Leadership Team
Slide 29: Key Opinion Leaders
29 Charlie Wykoff, MD, PhDTaiji Sakamoto, MD, PhD Allen Ho, MD, PhDRoger Goldberg, MD, PhD Brian Mac Grory, MD, PhD Christine Kay, MD, PhD Vitreo Retinal ASSOCIATES Mark Pennesi, MD, PhD Russell Van Gelder, MD, PhD KIO-301 KIO-401 Key Opinion Leaders
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