Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX

Kiora Pharmaceuticals — Investor Presentation (February 2024)

43 slides from Kiora Pharmaceuticals's investor presentation, Feb 12, 2024

  1. Slide 1: Kiora Pharmaceuticals H1 2024 Corporate Overview

    Kiora Pharmaceuticals, Inc. NASDAQ: KPRX H1 2024 | Corporate Overview

  2. 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

  3. Slide 3: Sharpened Focus on Orphan Retinal Diseases

    3 Kiora is developing retinal therapeutics to improve sight in patients with severe vision loss due to inherited or age-related diseases Sharpened Focus on Orphan Retinal Diseases Patient Perspective Individual’s burden of disease Physicians Perspective Efficient, cost-effective treatments Societal

  4. 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

  5. Slide 5: Pipeline

    5 Pipeline Product Route of Delivery Indication Development Stage Prevalence* Pre-clinical Phase 1 Phase 2 Phase 3 (US, EU5, JP) KIO-301 Intravitreal Retinitis Pigmentosa (Mutation Agnostic) 250,000 Choroideremia 16,000 Stargardt Disease 99,000 KIO-104 Intravitreal Posterior Non-Infectious Uveitis 1

  6. Slide 6: Upcoming Clinical/Regulatory Milestones

    6 Q4 ‘23 Q1 ‘24 Q2 ‘24 Q3 ‘24 Q4 ‘24 KIO-301 ABACUS-1 ✓ Top Line Data KIO-301 ABACUS-2 Ph2 Initiation KIO-301 ABACUS-2 Ph2 Top Line Data* KIO-301 Initiated Choroideremia ‘Extension’ KIO-301 Stargardt Ph2 Initiation KIO-301 ✓ FDA pIND RP KIO-301 EMA Meeting KIO-301 ODD Choroideremia KIO-301 ODD Starg

  7. Slide 7: KIO-301 Partnership Non-Binding Term Sheet Major Terms

    7 Final Major Terms Territory Global less Asia Field of Use Ophthalmology (all indications) with development milestones for each indication (RP, CHM, SD) or others Development Responsibilities Kiora responsible until Ph3 (JSC) Development Costs for Ph2 Thea to reimburse Kiora for KIO-301 Research &

  8. Slide 8: Who is Théa?

    8  Private, family owned, ophthalmic focused company  Chibret family founded the French Society of Ophthalmology in 1883  >100 commercial ophthalmic products globally including: Azasite® Cosopt® Ivizia® Virgan® Zioptan® Other partnerships include: Théa products available in over 75 countries dire

  9. Slide 9: KIO-301 Small Molecule Targeting Vision Restoration

    9 KIO-301 Small Molecule Targeting Vision Restoration Retinitis Pigmentosa, Choroideremia, Stargardt Disease

  10. Slide 10: Inherited Retinal Diseases Lead to Loss of Vision

    10 Inherited Retinal Diseases 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 visual c

  11. Slide 11: KIO-301 is a Molecular Photoswitch Designed to Restore Vision

    11 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 “o

  12. Slide 12: KIO-301 Reanimates the Retina & Changes Behaviour

    12 KIO-301 Reanimates the Retina & Changes Behaviour Extensive Validation in Preclinical Models Neuron. 81, 800-813 (2014). Behavioural study used a homologue molecule to KIO-301 API

  13. Slide 13: Retinitis Pigmentosa: A Disease with No Available Treatments

    13 Clinical Presentation  Night blindness, reduced visual field range and eventual loss of central vision  Visual acuity declines  50% of patients are not qualified to drive by age 37 and legally blind by 55 Etiology  50+ genetically distinct subtypes from 150+ mutations  Inherited disease Mark

  14. Slide 14: KIO-301-1101: Phase 1b Study Design (ABACUS)

    14 KIO-301-1101: 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 (sho

  15. Slide 15: Patient Testimonials

    15 Patient Testimonials Patient 1-02 Baseline VA: NLP Cohort 1 Patient 2-05 Baseline VA: CF Cohort 2 VA – Visual Acuity, NLP – No Light Perception, CF – Counting Fingers, HM – Hand Motion Patient 1-03 Baseline VA: HM Cohort 2

  16. Slide 16: ABACUS-1 Primary Endpoint Achieved

    16 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

  17. Slide 17: Kinetic Visual Field (Goldmann Perimetry)

    17 Kinetic Visual Field (Goldmann Perimetry) Assessment & Insights:  Applicability in this population  Performed by experienced orthoptists  Limited to 2-axis  The patient is asked to acknowledge (using a buzzer) when light stimulus is visualized within the dome  Method facilitates limitation o

  18. Slide 18: KIO-301 May Improve Visual Field

    18 Kinetic Visual Field KIO-301 May Improve Visual Field 0 10 20 30 30 40 50 60 70 80 Kinetic Visual Field‡ LS Mean ± 80% CL Day D eg re es -20 -10 0 10 20 30 40 D2-BL D7-BL D14-BL D29-BL Kinetic Visual Field‡ Mean Change ± 80% CL Degrees of Change from Baseline * *p<0.05 ‡ Cohort 2 includes 3 patie

  19. Slide 19: Visual Acuity — Berkeley Rudimentary Vision Test

    19 Visual Acuity — Berkeley Rudimentary Vision Test (BRVT) 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 Optometry and Vision Science 2012:89(9), 1257-1264 Cohort 2 (3 patients, 3 eyes)

  20. Slide 20: Light Perception (Intensity & Contrast Assessment)

    20 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

  21. Slide 21: KIO-301 may improve light perception

    21 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

  22. Slide 22: Functional Vision - Multiluminance Orientation & Mobility

    22 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

  23. Slide 23: KIO-301 May Improve Functional Vision

    23 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

  24. Slide 24: Visual Function Questionnaire (NEI VFQ-25)

    24 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

  25. Slide 25: Functional MRI Supportive of Cortical Activation

    25 Functional MRI Supportive of Cortical Activation NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers

  26. Slide 26: ABACUS-1 Takeaways

    26 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

  27. Slide 27: KIO-301-2101: Study Design (ABACUS-2)

    27 KIO-301-2101: Phase 2 Study Design (ABACUS-2) Randomized (3:1), Controlled*, Double Masked, Multiple Dose Study – 4 Sites (Australia) Cohort A: 16 patients with RP who have NLP (logMAR >3.0) Cohort B: 16 patients with RP who have ULV (logMAR > 1.6 and < 3.0 OU) Cohort 1A and Cohort 1B • 50µg KIO-

  28. Slide 28: Choroideremia: Inherited Disease that Leads to Blindness

    28  Orphan Disease: prevalence of 1:50,000, ~12,000 patients in US/EU  X-linked recessive disease primarily affecting males  Cause: Inherited mutation in the Choroideremia (CHM) gene encoding Rab escort protein-1 (REP1)  REP1 is involved in the regulation of intracellular trafficking of Rab prot

  29. Slide 29: Partnership with the Choroideremia Research Foundation

    29 The Choroideremia Research Foundation (CRF) is the largest global not-for-profit organization focused on the search for a cure for Choroideremia (CHM). Partnership with the Choroideremia Research Foundation Education and Awareness of CHM and KIO-301 CHM KOL Network to Assist in Clinical Protocol

  30. Slide 30: Stargardt Disease: No Approved Therapeutics

    30 Stargardt Disease: No Approved Therapeutics Foundation Fighting Blindness, Makari Wellness  Orphan Disease: prevalence of 1:10,000 ~30,000 patients in US  Autosomal recessive disease inherited from parent carriers, typical onset in 2nd decade of life, vision loss in 4th-5th decade.  Cause: Mut

  31. Slide 31: KIO-301-3101: Study Designs (CHM & Stargardt)

    31 KIO-301-3101: Phase 2 Study Designs (CHM & Stargardt) Controlled, Randomized Clinical Trial – Australia  Three cohorts, randomized, controlled, multiple bilateral IVT injections (days 1, 30, 60)Study Design Endpoints Review  Safety review conducted by Investigators after sentinel subject  Prim

  32. Slide 32: KIO-104 Intravitreal Small Molecule DHODH Inhibitor

    32 KIO-104 Intravitreal Small Molecule DHODH Inhibitor Steroid Sparing Approach to Retinal Inflammation

  33. Slide 33: KIO-104 Overview (DHODH Inhibitor)

    33 KIO-104 is an intravitreal, non-steroidal, novel small molecule which mitigates:  Metabolic activity and proliferation of T-cells  Secretion of IL-17, VEGF and IFN-𝛾𝛾 KIO-104 Overview (DHODH Inhibitor) O F F F F F F F NH O S OH O Existing immunosuppressive agents have a fundamentally…

  34. Slide 34: Non-Infectious Uveitis

    34 Non-Infectious Uveitis Br J of Ophthalmol. 2004;88(9):1159-1162. Med Hypothesis Discov Innov Ophthalmol. 2013 Winter;2(4):113-120. Retina Today. 2016;47-51. Clin Ophthalmol. 2016;10:1983-2020. JAMA Ophthalmol. 2016 Nov 1;134(11):1237-1245. Uveitis Market Insights, Epidemiology & Market Forecast-2

  35. Slide 35: KIO-104-1101: Phase 1 Study Design

    35 Day 28 Cohort 1: 0.3 µg N=4 Day -14 to 0 Screening Cohort 3: 1.2 µg N=4 Day 1 Day 7 Chronic Posterior Uveitis (N=12) Day 14 Day 21 Cohort 2: 0.6 µg N=4 KIO-104-1101: Phase 1 Study Design Study Design Endpoints Key Results  Prospective, multi-center, open-label, dose ranging, single IVT injection

  36. Slide 36: KIO-104 Improves VA and CME After Single IVT Dose

    36 KIO-104 Improves VA and CME After Single IVT Dose * 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 et te rs fr om B as el in e at D 28

  37. Slide 37: KIO-104 Path Forward

    37 KIO-104 Path Forward Posterior Non-Infectious Uveitis ~$ 47 M • Ph2b Clinical Trial (EU): Q4 2024 – Q1 2026 • Non-Clinical, IND Enabling Studies: Q1 2025 – Q1 2026 • Ph3 Registration Study(s) in USA & EU: Q3 2026 – Q1 2028 • NDA: Late 2028 Proliferative Vitreoretinopathy and/or other retinal infl

  38. Slide 38: Corporate Overview

    CORPORATE OVERVIEW 38 €€KIORA

  39. Slide 39: Capitalization

    39 Capitalization Capitalization as of Feb 5, 2024 Common Stock Equivalents Common Stock 25,879,020 Series D Convertible Preferred (convertible @ $141.28/ share) 52 Series F Convertible Preferred (convertible @ $1.10/share) 381,780 Warrants (WAEP $0.84) 71,419,749 Options (WAEP $4.26) 812,945 ESPP 1

  40. Slide 40: Leadership Team

    40 Brian M. Strem, PhD President & CEO Eric J. Daniels, MD Chief Development Officer Melissa Tosca, CPA EVP – Finance Stefan Sperl, PhD EVP – CMC & Operations Leadership Team

  41. Slide 41: Board of Directors

    41 Board of Directors Brian M Strem, PhD President & CEO Aron Shapiro Ken Gayron Praveen Tyle, PhD Chairman David Hollander, MD, MBA Erin Parsons Carmine Stengone

  42. Slide 42: Scientific Advisory Board

    42 Scientific Advisory Board Russel Van Gelder, MD, PhD Charlie Wykoff, MD, PhD Allen Ho, MD, PhD Christine Kay, MD, PhD Vitreo Retinal ASSOCIATES Mark Pennesi, MD, PhD

  43. Slide 43: Contact

    Contact: info@kiorapharma.com

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