Slide 1: Kiora Pharmaceuticals Q4 2024 Corporate Overview
Kiora Pharmaceuticals, Inc. NASDAQ: KPRX Q4 2024 | Corporate Overview
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: Developing Treatments for Retinal Diseases
3 Developing Treatments for Retinal Diseases Improve Sight in Patients with Severe Vision Loss and High Unmet Needs Patient Perspective Individual’s burden of disease Physicians Perspective Efficient, cost-effective treatments Societal Perspective 70% of blind patients are unemployed
Slide 4: Why Retinal Diseases?
4 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 5: Investment Highlights
5 Investment Highlights Innovative Modalities KIO-301 Molecular photoswitch KIO-104 Anti-inflammatory DHODH inhibitor Significant Market Need KIO-301 100K patients in US with RP and other IRDs KIO-104 >50M patients in US with pseudophakia 17M patients in US with retinal vein occlusion 800k pat
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-104 Intravitreal Small Molecule DHODH Inhibitor
7 KIO-104 Intravitreal Small Molecule DHODH Inhibitor Steroid-Sparing Approach to Retinal Inflammation
Slide 8: Retinal Inflammation Underlies Macular Edema
8 Retinal Inflammation Underlies Macular Edema Fluid leakage into the retina can cause separation of cell layers leading to blurring and distortion of vision and potentially permanent vision loss https://www.waterlooeye.ca Common Causes of Macular Edema: Posterior Non-Infectious Uveitis Diabetic
Slide 9: KIO-104 Mechanism of Action
9 T-cells are known to play a fundamental role in inducing ocular inflammation Dihydroorotate Dehydrogenase (DHODH) inhibitors are well established, disease-modifying agents in autoimmune conditions KIO-104 Mechanism of Action DHODH inhibition reduces the cellular pyrimidine pool resulting in:
Slide 10: Need for steroid-sparing anti-inflammatory delivered locally to the eye
10 Need for steroid-sparing anti-inflammatory delivered locally to the eye Posterior Non-Infectious Uveitis: a T-Cell Driven Disease Retinal Inflammation in the Back of the Eye Can Lead to Vision Loss Major Causes Autoimmune disorders (i.e., lupus, multiple sclerosis, psoriasis, rheumatoid arthritis
Slide 11: KIO-104 in Uveitis − Preclinical
11 KIO-104 in Uveitis − Preclinical KIO-104 reduced the number of relapses Placebo Days Post Immunization Cl in ic al U ve iti s Sc or e KIO-104 J Neuroinflammation. 2018 Feb 21;15(1):54
Slide 12: Phase 1/2a Study Design
12 Phase 1/2a Study Design Safety and tolerability Improvement of inflammation Blood PK of KIO-100 Single intravitreal injection of 300ng, 600ng, and 1200ng (any systemic therapy at enrollment was continued) Patients with chronic, posterior non-infectious uveitis Prospective, open label,
Slide 13: KIO-104 Phase 1/2a Results
13 KIO-104 Phase 1/2a Results Safety & Tolerability ✓ No SAE ✓ Excellent tolerability at all doses Pharmacokinetics ✓ KIO-100 was not detected in the peripheral blood at any time
Slide 14: KIO-104 Improved Visual Acuity and Reduced Cystoid Macular Edema
14 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 15: KIO-104 Phase 2 Study Design (KLARITY)
15 KIO-104: Phase 2 Study Design (KLARITY) 2-Step, Randomized, Open-Label, Steroid-Sparing, Dose Expansion Trial Step 1: Dose informing short-term study in macular edema (multiple clinical indications) Step 2: Assuming positive data from Step 1, dose expansion study with highest tolerated dose
Slide 16: KIO-301 Small Molecule Targeting Vision Restoration
16 KIO-301 Small Molecule Targeting Vision Restoration Inherited Retinal Diseases
Slide 17: Inherited Retinal Diseases (IRDs) Lead to Loss of Vision
17 Inherited Retinal Diseases (IRDs) Lead to Loss of Vision Healthy Vision Rods and cones, the photoreceptors of the retina, process light and relay an electrical signal to downstream cells One of these cell types, retinal ganglion cells (RGCs), transmit the signal to the visual cortex The vis
Slide 18: KIO-301 is a Molecular Photoswitch Designed to Restore Vision
18 KIO-301 is a Molecular Photoswitch Designed to Restore Vision KIO-301 without Light When photoreceptors die, RGCs undergo some remodeling, including expressing specific proteins that allow KIO-301 to selectively enter the cell with ion channels Without light, KIO-301 remains in its linear “of
Slide 19: KIO-301 Reanimates the Retina & Changes Behavior
19 KIO-301 Reanimates the Retina & Changes Behavior Extensive Validation in Preclinical Models Neuron 2014: 81, 800-813. Behavioral study used a homologue molecule to KIO-301 API
Slide 20: Inherited Retinal Disease Landscape
20https://taiwanird.com.tw/ Inherited Retinal Disease Landscape Leber Congenital Amaurosis (LCA) Cone-Rod Dystrophy (CRD) Color Vision Defects (CVD) Stargardt Disease (SD) Vitreo-retinopathy Bietti Crystalline Dystrophy (BCD) Retinoschisis Choroideremia (CHM) Congenital Night Blindness (CNB) Retinit
Slide 21: Market Opportunity
21 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 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: ABACUS-1 – Primary Endpoint Achieved
23 ABACUS-1 – Primary Endpoint Achieved Single Dose IVT KIO-301 is Safe & Well Tolerated @ 7.5μg, 25μg, 50μg MedDRA Term KIO-301 7.5µg (N=3); n (%) KIO-301 25µg (N=6); n (%) KIO-301 50µg (N=3); n (%) Severity Drug Related Total N=12; n (%) Ocular Hypertension 1 (33%) 0 (0%) 0 (0%) Mild Possible 1 (8
Slide 24: Kinetic Visual Field
24 Kinetic Visual Field KIO-301 May Improve Visual Field ‡ Cohort 2 includes 3 patients (6 eyes) CL = Confidence Limit Goldmann perimetry Performed at baseline (BL), and each study visit Performed by same group of orthoptists to reduce variability Greater improvement observed in Cohort 2 Kin
Slide 25: Visual Acuity — Berkeley Rudimentary Vision Test
25 Visual Acuity — Berkeley Rudimentary Vision Test (BRVT) Optometry and Vision Science 2012:89(9), 1257-1264 Cohort 2 (3 patients, 3 eyes) 0 10 20 30 1.50 1.75 2.00 2.25 2.50 Visual Acuity Mean ± SEM Day lo gM AR Off-Chart Counting Finger Hand Motion
Slide 26: Light Perception (Intensity & Contrast Assessment)
26 Light Perception (Intensity & Contrast Assessment) Assessment: Series of visual stimuli (a series of letters are presented on a screen to the patient via a rear projector) Binary outcome (yes/no) The subject is asked to acknowledge (verbally and/or physically) when a change in light is perc
Slide 27: Light Perception – KIO-301 May Improve Light Perception
27 Light Perception – Cohort 1 KIO-301 May Improve Light Perception in the NLP/BLP Population Insights: Cohort 1 subjects demonstrate improved odds ratio on drug Odds Ratio – strength of association, OR=2 → 100% increase in the odds of an outcome > e.g., duration of diabetes mellitus (> 15 years
Slide 28: Functional Vision - Multiluminance Orientation & Mobility (MLOM)
28 2’x2’ tile Door Location Test Setup Functional Vision - Multiluminance Orientation & Mobility (MLOM) 2’x2’ tile Window Location Test Setup 2’x2’ tile Walking Direction Test Setup Start End 2’x2’ tile HCRE Course Setup Screen Screen Technician 1 Technician 2Door Window Left Center Right Left Cente
Slide 29: KIO-301 May Improve Functional Vision
29 Functional Vision − Multiluminance Orientation & Mobility (MLOM) KIO-301 May Improve Functional Vision 0.1 1 10 100 Day 2 Day 7 Day 14 Day 29 Door Location Test* Change From Baseline ± 80% CL Odds Ratio 0.1 1 10 100 Day 2 Day 7 Day 14 Day 29 Walking Direction Test‡ Change From Baseline ± 80% CL O
Slide 30: Visual Function Questionnaire (NEI VFQ-25)
30 Visual Function Questionnaire (NEI VFQ-25) KIO-301 May Improve Patients’ Overall Quality of Life 0 30 35 40 45 50 Quality of Life Survey Day VF Q -2 5 C om po si te S co re n=12 *HMSA Medical Policy – Luxturna – 2016 National Eye Institute generated survey assess daily functions related to genera
Slide 31: Functional MRI
31 Functional MRI Qualitative Supportive of Cortical Activation NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers
Slide 32: Functional MRI Quantification of Checkerboard Stimulus
32 1-02 Checkerboard with arrow Statistical Activation Map (Subject 2-05 [CF] - 50µg) 2d 4 weeks2 weeks Functional MRI Quantification of Checkerboard Stimulus 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 33: Patient Testimonials
33 “…in my last few months and years, my vision has been deteriorating fairly rapidly. It’s like a dimmer switch has been turned down and the rooms were getting darker. Going into areas would be like going into a cave. In my living room, there are two lights in the ceiling, cluster lights. I would h
Slide 34: ABACUS-1 Takeaways
34 ABACUS-1 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 Approvable outcome assessments discussed with regulators Positive US FDA pIND meeting
Slide 35: Théa Open Innovation Partnership
35 Théa Open Innovation Partnership: Co-Development & Commercialization Transaction Highlights Théa Open Innovation (TOI) granted exclusive development & commercialization rights to KIO-301 for IRDs (≠ Asia) $16M cash upfront payment to Kiora Kiora eligible for up to an additional $285M in cli
Slide 36: ABACUS-2 Study Design: Received Approval to Initiate
36 KIO-301-2101: Phase 2 ABACUS-2 | Received Approval to Initiate Double-masked, Randomized, Controlled, Multiple Dose Study 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 1
Slide 37: ABACUS-2 Double-masked, Randomized, Controlled Study
37 ABACUS-2 (IVT Q6W for 3 Months) Double-masked, Randomized, Controlled, Multiple Dose Study – 5 Sites (Australia), 36 Patients St ud y A ss es sm en ts St ud y V is its Treatment Period Study visits occur every 3 weeks for study assessments with KIO-301 or control administered every 6 weeks (OU)
Slide 38: Choroideremia Research Foundation Grant & Ora Partnership
38 Choroideremia Research Foundation Grant & Ora Partnership Run-in Study Ongoing to Validate Novel Functional Vision Endpoints 2’x2’ tile 360-Degree Door Location Test Door Door D oorD oo r Start End 2’x2’ tile High Contrast Room Exit Test 2’x2’ tile 360-Degree Room Light Direction Test Light Array
Slide 39: Leadership Team
39 Brian M. Strem, PhD President & CEO Eric J. Daniels, MD, MBA Chief Development Officer Melissa Tosca, CPA Chief Financial Officer Stefan Sperl, PhD EVP – CMC & Operations Leadership Team
Slide 40: Upcoming Milestones
40 Upcoming Milestones Initiate ABACUS-2 Phase 2 clinical trial 2H 2024 Complete run-in study to validate novel functional vision endpoints Q1 2025 Complete enrollment of ABACUS-2 2H 2025 KIO-301 KIO-104 Round out non-clinical package Q4 2024 Initiate KLARITY Phase 2 clinical trial 1H 2025
Slide 41: Thank You
Thank You NASDAQ: KPRX