Kiora Pharmaceuticals, Inc. Q4 2026 | Corporate Overview
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, statements concerning Kiora's planned development and potential commercialization of KIO-401; the anticipated timing, design, initiation, conduct, enrollment, endpoints and results of clinical trials in CRAO and NAION; the potential efficacy, safety, therapeutic benefit and commercial opportunity of KIO-401; the interpretation and applicability of clinical results generated by Senju Pharmaceutical in Japan; the potential for calpain inhibition in additional ophthalmic indications; anticipated regulatory interactions and approvals; the ability to leverage existing clinical development infrastructure; the expected progression of the KIO-301 program and related clinical operations; the Company's plans for KIO-104; the expected capital requirements associated with its development programs, including the clinical trials of KIO-401 in CRAO and NAION; and Kiora's ability to secure additional financing to support its operations. These forward-looking statements are based on management's current expectations and assumptions and involve risks and uncertainties that could cause actual results to differ materially from those expressed or implied. Such risks include, among others, uncertainties associated with clinical development and regulatory review; the possibility that results observed in previous clinical studies will not be reproduced in future trials; the cost, timing, enrollment, conduct and outcomes of clinical trials; the ability to obtain sufficient capital to fund planned development activities; the availability and terms of future financing; the potential dilution to existing stockholders; the Company's ability to execute its development strategy and licensing obligations; and other risks described under "Risk Factors" in Kiora's Annual Report on Form 10-K filed with the SEC on March 25, 2026, and in its subsequent filings with the SEC. Kiora's results may also be affected by factors of which the Company is not currently aware. Forward-looking statements speak only as of the date of this release. Except as required by law, Kiora undertakes no obligation to publicly update or revise any forward-looking statement to reflect subsequent events, circumstances or changes in expectations. Legal Disclaimers
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 inhibitor § Positive Ph2 and Ph3 studies in Japan § Rare acute conditions with no currently approved treatments § Expansion opportunity beyond retina KIO-301 Existing Phase 2 asset for late-stage Retinitis Pigmentosa § Global partnerships with Laboratoires Théa, Senju Pharmaceutical & CKD Pharmaceuticals ⨠ $118M in potential development and regulatory milestones ⨠ $431M in potential commercial milestones ⨠ Royalties into the low 20% § Orphan Drug Designation § Phase 2 trial actively enrolling
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 Global Rights (outside Asia) NAION Inherited Retinal Disease KIO-301 Molecular Photoswitch Retinitis Pigmentosa (advanced) Partnered GloballyOther Inherited Retinal Diseases
5 KIO-401 Small Molecule Calpain Inhibitor
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 § ↑Intracellular Ca2+ § Release from intracellular stores (ER, mitochondria) § Calpains are a family of calcium- activated cysteine proteases § Ubiquitously expressed in the cytoplasm of neurons and other cells § Calpains are endogenously inhibited by calpastatin Calcium Overload Proteolysis of Substrates and Cell Death § Cleavage of cytoskeletal proteins § Cleavage of ion channels, pumps and synaptic proteins § Mitochondrial dysfunction § Membrane destabilization § Neuronal cell death How Ischemia Activates Calpains Reduced perfusion Loss of ion homeostasis Increased Intracellular Ca2+ Calpain (active) Neuronal injury and cell death Ca2+ Calpastatin Calpastatin (inhibitor) Calpain (inactive) Calpastatin Calpain ↓ ATP X Ca2+ KIO-401 is a neuroprotective, oral small molecule calpain inhibitor, designed to protect retinal cells during ischemia Calpains: Calcium-Activated Proteases in Ischemia Injury
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 phase of disease Central retinal artery becomes occluded Blood flow stops Time is critical Early treatment may save vision Irreversible vision loss Limited oxygen is not enough Oxygen and nutrients are cut off Inner retina becomes ischemic Over 80% of patients have permanent, severe vision loss (worse than 20/200)
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 (ETDRS), Visual Fields § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Within 48 hrs of onset of symptoms Day 85 Visit 7 Cohort 1: KIO-401 (n=9) (100mg/day) Day 2 Visit 2 Day 57 Visit 6 Post Treatment Observation Cohort 2: KIO-401 (n=10) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Prospective Observational Registry (n=9) * Study was conducted by Senju
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
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 Baseline in BCVA at Weeks 1-8, BCVA Responder Rates of 3 Lines at Week 12, Change from Baseline in Visual Field at Week 12, Treatment Emergent Adverse Events, ECG & labs § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Within 48 hrs of onset of symptoms Day 85 Visit 7 Day 2 Visit 2 Day 57 Visit 6 Post Treatment Observation KIO-401 (n=33) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Placebo Control (n=30) Post Treatment Observation * Study was conducted by Senju
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 analysis performed with Fisher’s Exact Test
12 KIO-401: Phase 3 Study Results* Categorical Visual Function Improvement * Study was conducted by Senju
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 Significant TEAEs 0 0 0 0 Any study drug-related TEAEs 1 (3.0) 2 1 (3.0) 1 Mild 1 (3.0) 2 1 (3.0) 1 Moderate 0 0 0 0 Severe 0 0 0 0 Serious study drug-related TEAEs 0 0 0 0 Study drug-related TEAEs leading to death 0 0 0 0 Serious study drug-related TEAEs except death 0 0 0 0 Significant study drug-related TEAEs 0 0 0 0 * Study was conducted by Senju TEAE: Treatment Emergent Adverse Event KIO-401: Phase 3 Study Results* Safety – TEAE Summary (Treatment and Post-treatment Phases)
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 20/400) § Primary – Best Corrected Visual Acuity (ETDRS) § Secondary – Visual Fields, AEs & labs § Imaging – Fundus, OCT, Fluorescein Angiography, Slit Lamp Study Design Endpoints Power Analysis Parameters: 80% power, p=0.05, 2-sided based on Senju P3 trial BCVA data (responder analysis will increase total patients by ~2x) Within 48 hrs of onset of symptoms Day 85 Visit 7 Day 2 Visit 2 Day 57 Visit 6 KIO-401 (n~72) (200 mg/day) Day 8 Visit 3 Day 15 Visit 4 Day 29 Visit 5 Day 1 Visit 1 Placebo Control (n~72) Post Treatment Observation Post Treatment Observation
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 Today No medical or surgical treatment has been shown to improve the prognosis3 – steroids used off-label to help w edema 91% of specialists do not recommend routine treatment4 Limited Near-Term Options Many agents have been assessed but none has clearly shown benefit2,5 GLP-1 Link May Expand The Unmet Need. EU labels were updated in Sept ‘25 to add NAION for Ozempic, Rybelsus and Wegovy, and the UK followed in Feb ‘26.6,7 At roughly one extra case per 10,000 person-years against about 15 million US users, that is on the order of 1,000 additional US cases a year.8,9 1. WHO. Semaglutide medicines and risk of NAION. 27 Jun 2025. 2. Gibbons A, Henderson AD. Front Ophthalmol. 2022;2:848710. 3. Wu KY, Evoy F. NAION: Diagnosis and Management. AAO EyeNet. Aug 2022:29-31. 4. Bialer OY, Stiebel-Kalish H. Graefes Arch Clin Exp Ophthalmol. 2024;262:1399-1410. 5. Ischemic Optic Neuropathy: A Review of Current and Potential Future Pharmacotherapies. Pharmaceuticals. 2024;17(10):1281. 6. EMA PRAC, 6 Jun 2025; EU product information updated 30 Sep 2025. 7. MHRA Drug Safety Update, 5 Feb 2026. 8. KFF Health Tracking Poll, May 2024; 6% of US adults currently using a GLP-1. 9. Kiora calculation from references 6 and 8. NAION: Traditional Therapy Has No Current Options Sudden, Irreversible Vision Loss with No Approved Therapy
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 placebo (p=0.0195) Simple Route of Administration Oral administration – no surgery, no device, no IV procedure, no novel delivery system Rapid Path to Value Creation Target label expansion timing expected in 2033 The signal is strongest where the need is greatest. A known molecule with human proof of concept, given as a pill, in a disease with no approved therapy. The largest treatment effect was seen in patients who were already legally blind when they presented. KIO-401 Phase 3 study results, data on file. Figures shown are for the subgroup with baseline BCVA worse than 20/400 (WHO definition of legal blindness); the overall trended positive on its primary endpoint (p=0.0949). KIO-401 Is Poised to Offer a New Solution for NAION Pts An Oral Neuroprotectant with a Phase 3 Signal in Acute Ischemic Optic Injury
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 fundus photography § Primary – Best Corrected Visual Acuity (ETDRS) § Secondary – Visual Fields, AEs & labs § Imaging – Fundus, OCT (RNFL), Slit Lamp Study Design Endpoints Power Analysis Parameters: 90% power, p=0.05, 2-sided based on 20-point difference in responder analysis Within 14 days of onset of symptoms Day 14 Visit 2 Day 168 Visit 7 KIO-401 (n~136) (200 mg/day) Day 28 Visit 3 Day 56 Visit 4 Day 112 Visit 6 Day 1 Visit 1 Placebo Control (n~136) Day 84 Visit 5
18 KIO-301
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 causes reversible isomeric shift, altering ion flux through Kv/HCN channels Intracellular KIO-301 P2X7‡ Extracellular KV/HCN Channels • KIO-301 lodges inside ion channels KIO-301 (MOA): Turns RGCs “ON” in the Presence of Light • In presence of light, alters ion flow, causing cell activation
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 cones) sense light and pass the signal to special transmitting cells Bipolar cells and RGCs relay signal from rods and cones to the brain, where the image is “processed” RG C Bipolar Cell To the brain (connection intact) Cone Rod
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
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 only for Cohort 1 above) is repeated for each cohort per eye; intensity & contrast assessment, kinetic perimetry, functional MRI, etc. § Safety review conducted by Investigators between after sentinel subject Study Design Endpoints Review N=6 3/cohort Binocular Day 90 Cohort 1: KIO-301 (7.5 µg) Day -30 to -2 Visit 1-Screening Day 29-30 Visit 5 Day 60 Day 120 Cohort 1: KIO-301 Contralateral (25 µg) Cohort 2: KIO-301 (25 µg) Cohort 2: KIO-301 Contralateral (50 µg) Day 1-3 Visit 2 Day 7-8 Visit 3 Day 14-15 Visit 4 Day -120 to -30 fMRI feasibility NLP – No Light Perception, BLP – Bare Light Perception, HM – Hand Motion, CF – Counting Fingers Nature Medicine v32, 1865–1870 (2026)
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)
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)
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 Cohort begins with 6 patients for Safety Review Committee review § Primary (safety): AEs, PK, labs § Secondary (efficacy): functional vision (multi-luminance object identification), BCVA, peripheral field, FST, PROs Study Design * Also includes further test/retest validation to functional vision assessment OLE – Open Label Extension, BCVA – Best Corrected Visual Acuity, FST – Full-Field Stimulus Testing, PROs – Patient Reported Outcomes Day 42 Day 63 Ultra Low Vision (ULV, n=8) No Light Perception (NLP, n=4) Day 1 Day -30 to -1 Screening* Vehicle Controls (n=6) 50 µg KIO-301 Day 21 ULV (n=8) NLP (n=4) Vehicle Controls (n=6) 100 µg KIO-301 Day 84 Day 105 Day 165 Day 42 Day 63 Day 21 Day 84 Day 105 Day 165 // // Day 1 OLE for Controls Endpoints
26 KIO-301: Global Partnership Network Significant Upside Opportunity via Milestones and Royalties
27 Leadership and KOLs
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
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