Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — Investor Presentation (November 2023)
38 slides from Kiora Pharmaceuticals's investor presentation, Nov 9, 2023
Slide 1: Kiora Pharmaceuticals, Inc. Q4 2023 Corporate Overview
Kiora Pharmaceu-cals, Inc. NASDAQ: KPRX Q4 2023 | 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 retinal therapeutics for severe vision loss; focus on orphan retinal diseases
3 Kiora is developing re9nal therapeu9cs to improve sight in pa9ents 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 Perspec/ve Efficient, cost-effec8ve treatments Societal Persp
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: 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
Slide 6: Upcoming Clinical/Regulatory Milestones
6 Q4 ‘23 Q1 ‘24 Q2 ‘24 Q3 ‘24 Q4 ‘24 KIO-301 ABACUS Topline Data KIO-301 ABACUS-2 Ph2 Initiation KIO-301 ABACUS-2 Ph2 Topline Data* KIO-301 Initiate Choroideremia Ph2 Trial KIO-301 Choroideremia Ph2 Topline Data* KIO-301 IniOate Stargardt Ph2 Trial KIO-301 FDA pIND RP KIO-301 Stargardt Ph2 Topline D
Slide 7: KIO-301 Small Molecule Targeting Vision Restoration
7 KIO-301 Small Molecule Targeting Vision Restoration Retinitis Pigmentosa, Choroideremia, Stargardt Disease
Slide 8: Inherited Retinal Diseases Lead to Loss of Vision
8 Inherited Retinal Diseases Lead to Loss of Vision Healthy Vision § Rods and cones, the photoreceptors of the re7na, process light and relay an electrical signal to downstream cells. § One of these cell types, re7nal ganglion cells (RGCs), transmit the signal to the visual cortex. § The visual cort
Slide 9: KIO-301 is a Molecular Photoswitch Designed to Restore Vision
9 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 10: Retinitis Pigmentosa: A Disease with No Available Treatments
10 Clinical Presenta-on § Night blindness, reduced visual field range and eventual loss of central vision § Visual acuity declines § 50% of pa\ents are not qualified to drive by age 37 and legally blind by 55 E-ology § 50+ gene\cally dis\nct subtypes from 150+ muta\ons § Inherited disease Market Opp
Slide 11: KIO-301 Reanimates the Retina & Changes Behaviour
11 KIO-301 Reanimates the Retina & Changes Behaviour Extensive Validation in Preclinical Models Neuron 2014: 81, 800-813. Behavioural study used a homologue molecule to KIO-301 API
Slide 12: KIO-301-1101: Phase 1b Study Design (ABACUS)
12 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
Slide 13: Patient Testimonials
13 Patient Testimonials Pa\ent 1-02 Baseline VA: NLP Cohort 1 Patient 2-05 Baseline VA: CF Cohort 2 VA – Visual Acuity, NLP – No Light PercepVon, CF – CounVng Fingers, HM – Hand MoVon Patient 1-03 Baseline VA: HM Cohort 2 Videos also available at: https://kiorapharma.com/technology/kio-301/
Slide 14: Kinetic Visual Field (Goldmann Perimetry)
14 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
Slide 15: KIO-301 May Improve Visual Field
15 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
Slide 16: Visual Acuity — Berkeley Rudimentary Vision Test (BRVT)
16 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)
Slide 17: Light Perception (Intensity & Contrast Assessment)
17 Light Perception (Intensity & Contrast Assessment) Assessment: § Series of visual s8muli (a series of lelers are presented on a screen to the pa8ent via a rear projector) § Binary outcome (yes/no) § The subject is asked to acknowledge (verbally and/or physically) when a change in light is perceiv
Slide 18: KIO-301 May Improve Light Perception in the NLP/BLP Population
18 Light PercepSon – Cohort 1 KIO-301 May Improve Light PercepNon in the NLP/BLP PopulaNon 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) w
Slide 19: Functional Vision - Multiluminance Orientation & Mobility (MLOM)
19 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 20: KIO-301 May Improve Functional Vision
20 FuncEonal Vision - MulEluminance OrientaEon & Mobility (MLOM) KIO-301 May Improve FuncNonal 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 Odds
Slide 21: Visual Function Questionnaire (NEI VFQ-25)
21 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 assesses daily functions related to gen
Slide 22: Functional MRI Supportive of Cortical Activation
22 Functional MRI Supportive of Cortical Activation NLP – No Light Perception, BLP – Bare Light Perception, CF – Counting Fingers
Slide 23: ABACUS-1 Takeaways
23 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 need to be defined § US FDA pIND scheduled for Q4 2023 K
Slide 24: KIO-301-2101: Phase 2 Study Design (ABACUS-2)
24 KIO-301-2101: Phase 2 Study Design (ABACUS-2) Sham Controlled, Masked, Randomized, Multiple Ascending Dose Trial – 4 Sites (Australia) 5 RP pts with NLP randomized to receive 50μg KIO-301 or sham (3:2) OU monthly for 3 months 5 RP pts with LP randomized to receive 50μg KIO-301 or sham (3:2) OU mo
Slide 25: Choroideremia: Inherited Disease that Leads to Blindness
25 § 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
Slide 26: Partnership with the Choroideremia Research Foundation
26 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
Slide 27: Stargardt Disease: Inherited Disease that Leads to Blindness
27 § 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: Mutation in the ABCA4 or ELOVL4 gene § Accumulation of lipofusion plaques in the retinal pigmen
Slide 28: KIO-301-3101: Phase 2 Study Designs (CHM & Stargardt)
28 KIO-301-3101: Phase 2 Study Designs (CHM & Stargardt) Sham 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 §
Slide 29: KIO-104 Intravitreal Small Molecule DHODH Inhibitor
29 KIO-104 Intravitreal Small Molecule DHODH Inhibitor Steroid Sparing Approach to Retinal Inflammation
Slide 30: KIO-104 Overview (DHODH Inhibitor)
30 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-𝛾 KIO-104 Overview (DHODH Inhibitor) O F F F F F F F NH O S OH O Existing immunosuppressive agents have a fundamentally different…
Slide 31: Non-Infectious Uveitis
31 Non-InfecSous UveiSs 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-2032
Slide 32: KIO-104-1101: Phase 1 Study Design
32 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
Slide 33: KIO-104 Improves VA and CME After Single IVT Dose
33 KIO-104 Improves VA and CME After Single IVT Dose * Historical Controls (Yeh et al, Retina 00 2018: 1-9. Suhler et al, Visual III Ophthalmology 2018:125, 7) 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 N=4
Slide 34: Corporate Overview
CORPORATE OVERVIEW 34 &KIORA
Slide 35: Leadership Team
35 Brian M. Strem, PhD President & CEO Eric J. Daniels, MD, MBA Chief Development Officer Melissa Tosca, CPA EVP – Finance Stefan Sperl, PhD EVP – CMC & Opera7ons Leadership Team
Slide 36: Board of Directors
36 Board of Directors Brian M. Strem, PhD President & CEO Aron ShapiroKen Gayron Praveen Tyle, PhD Chairman David Hollander, MD, MBA Erin Parsons Carmine Stengone
Slide 37: Scientific Advisory Board
37 Scientific Advisory Board Russel Van Gelder, MD, PhD Charlie Wykoff, MD, PhD Allen Ho, MD, PhD Chris/ne Kay, MD, PhD Vitreo Retinal ASSOCIATES Mark Pennesi, MD, PhD
Slide 38: Contact: info@kiorapharma.com
Contact: info@kiorapharma.com
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