Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — IPO Roadshow (May 2023)
47 slides from Kiora Pharmaceuticals's IPO roadshow presentation, May 15, 2023
Slide 1: Kiora Pharmaceuticals Q2 2023 Corporate Overview
Kiora Pharmaceuticals, Inc. NASDAQ: KPRX Q2 2023 | Corporate Overview Filed pursuant to Rule 433 under the Securities Act of 1933, as amended Dated 5/15/2023 Registration Statement No. 333-271699
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: Corporate Highlights: Developing Therapeutics for Rare & Underserved Ophthalmic Diseases
3 Developing Therapeutics for Rare & Underserved Ophthalmic Diseases Priority Asset – KIO-301: Vision Restoration in Retinitis Pigmentosa (RP) ▪ Small molecule “photoswitch” is gene mutation agnostic, easy to deliver ▪ Study fully enrolled, dosing ongoing ▪ Case study presented in Q2 2023, anticipat
Slide 4: Diverse Pipeline Offers Near Term Milestones
4 Indication Product Formulation Development Stage Pre-clinical Phase 1 Phase 2 Phase 3 P os te ri or Se gm en t Retinitis Pigmentosa (Mutation Agnostic) KIO-301 IVT* A nt er io r Se gm en t Ocular Presentation of Rheumatoid Arthritis + KIO-101 Eye Drop Persistent Corneal Epithelial Defects KIO-201
Slide 5: Upcoming Milestones
5 Upcoming Milestones Q3 ‘23 Q4 ‘23 Q1 ‘24 Q2 ‘24 Q3 ‘24 Q4 ‘24 KIO-301 ABACUS Interim Data KIO-301 ABACUS Full Data KIO-301 ABACUS MAD Initiation KIO-301 ABACUS MAD Interim Data KIO-301 ABACUS MAD Full Data KIO-101 OPRA+ Ph2 Full Data KIO-201 Seek FDA Guidance
Slide 6: KIO-301 Vision Restoration in Retinitis Pigmentosa
6 KIO-301 Vision Restoration in Retinitis Pigmentosa
Slide 7: Initially Targeting RP: A Disease with No Available Treatments
7 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 Marke
Slide 8: Downstream Neurons Remain Viable in RP
8 Downstream Neurons Remain Viable in RP ▪ RP results 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
Slide 9: KIO-301 (MOA): Turns RGCs ON in the Presence of Light
9Neuron. 92, 100-113 (2016) trans cis ▪ In RP, 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 10: KIO-301-1101: Phase 1b Study Design (ABACUS)
10 KIO-301-1101: Phase 1b Study Design (ABACUS) Open Label, Single Ascending Dose Trial – 2 Sites (Australia) 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 ▪ Two cohorts, non-randomized, open-label, single IVT injection per eye ▪ Co
Slide 11: KIO-301: ABACUS Aims
11 KIO-301: ABACUS Aims Explore objective assessments in these cohorts • No predefined approvable endpoints in ultra-low vision patients Do all of the above in an environment of safety and tolerability Obtain patient feedback • What patients report is important at this stage Demonstrate Proof-of-Mec
Slide 12: KIO-301: ABACUS – What is Measured?
12 KIO-301: ABACUS – What is Measured? Assessment Description Objective Intensity & Contrast Light Perception Goldmann Perimetry Visual Field MLOM Suite of ‘functional’ tests fMRI Cortical Imaging Subjective Interviews Subject Feedback VFQ-25 Quality of Life (QoL) Survey
Slide 13: KIO-301: ABACUS - Subject Status Update
13 KIO-301: ABACUS - Subject Status Update Subject ID 1st Eye Completed* Dose Responder‡ 2nd Eye Completed* Dose Responder‡ Cohort 1 NLP/BLP 1-01 Completed 7.5 μg Completed 25 μg 1-02 Completed 7.5 μg In-process 25 μg 1-04 Completed 7.5 μg To Be Scheduled 25 μg Cohort 2 CF/HM 1-03 In-process 25 μg T
Slide 14: ABACUS Completed Subjects – Responder Matrix
14 KIO-301: ABACUS Completed Subjects – Responder Matrix* Subject ID Intensity & Contrast Visual Field MLOM fMRI VFQ-25 (QoL) Subject Feedback Dose Response 1-01 1-02 N/A In-process 1-04 N/A In-process In-process 1-05 N/A In-process In-process In-process *+ve objective response above baseline at any
Slide 15: KIO-301: ABACUS Safety & Tolerability
15 KIO-301: ABACUS Safety & Tolerability IOP – all patients normal and no change to baseline, except 1 patient had bilateral increase in IOP (21→27 mmHg). Rapidly responded to pharmacological intervention (timolol). OCT - no macula edema, no change in thickness. Dilated Fundus w/ photography* - abno
Slide 16: PT 1-02: Intensity & Contrast Assessment
16 PT 1-02: Intensity & Contrast Assessment* Visit 1 (Baseline) Visit 3 (Day 7) * - A series of six (6) visual stimuli (“X” at logMAR 2.0) presented to the patient at 3 light intensities
Slide 17: Pt 1-02: Intensity & Contrast Assessment Key Takeaways
17 Key Takeaways: • Light perception increases over first 2 weeks following injection • Return to baseline expected Pt 1-02: Intensity & Contrast Assessment V is it 1 (B as el in e) V is it 2 (D ay 2 ) V is it 3 (D ay 7 ) V is it 4 (D ay 1 4) V is it 5 (D ay 2 9) V is it 1 (B as el in e) V is it 2 (
Slide 18: Kinetic Visual Field
18 Kinetic Visual Field Goldmann Haag-Streit Total Horizontal Degrees-OD Total Vertical Degrees-OD Baseline 8 9 1-month 28 14 • Assessment by orthoptists • Measure total horizontal and vertical degrees baseline 1 mo Pt1-01
Slide 19: Functional MRI – Setup & Analysis
19 Cohort 1 Paradigms (BLP/NLP) Processing and Analysis • BOLD signal acquired • Preprocessing (e.g., spatial normalization) • Quantitative analysis using FSL (GLM) - ongoing PROJECTOR MIRROR SCREEN Functional MRI – Setup & Analysis
Slide 20: Pt 1-02: fMRI – Qualitative Overlap of 3 Paradigms
20 Key Takeaways • Clear striate (V1) increase in activity at Visits 2 & 4 compared to Visit 1 • Visit 5 returns to similar activity as baseline Pt 1-02: fMRI – Qualitative Overlap of 3 Paradigms Visit 1 (Baseline) Visit 2 (Day 3) Visit 4 (Day 15) Visit 5 (Day 30)
Slide 21: MLOM Example (High Contrast Room Exit)
21 MLOM Example (High Contrast Room Exit - HCRE) A test of functional vision Suited best for CF/HM Patients (Cohort 2) Start End 2' x 2' tile HCRE Course Setup #1-05 Visit # Pass/Fail V1 (Baseline) fail V2 (Day 2) fail V3 (Day 7) pass V4 (Day 14) pass V5 (Day 28) pass Baseline Visit #5 (Day 28)
Slide 22: ABACUS Quality of Life Survey (VFQ-25)
22 NEI-VFQ-25 administered at Visits 1 & 5 KIO-301: ABACUS Quality of Life Survey (VFQ-25) 2 - 4 point increase is accepted by payers as clinically meaningful* * - HMSA Medical Policy – Luxturna - 2022 Baseline V5 (Day 29) 20 30 40 50 60 C o m p o s it e S c o re 1-01 1-02 +6.3 +6.6 1-04 +0.5
Slide 23: PROs: Direct Feedback Interviews
23 PROs: Direct Feedback Interviews Subject 1-02 Subject 1-05
Slide 24: Dose Response Observed
24 Dose Response Observed 0 10 20 30 0 20 40 60 80 100 Pt 1-01 - Intensity/Contrast Visit Day % C o rr e c t 7.5 g 25 g B as el in e 1- m onth B as el in e 1- m onth B as el in e 1- m onth B as el in e 1- m onth 0 10 20 30 40 Pt 1-01 - KVF D e g re e s Total Horizontal Total Vertical 7.5 μg 25 μg
Slide 25: KIO-301: ABACUS Key Takeaways
25 KIO-301: ABACUS Key Takeaways ✓ Intravitreal KIO-301 is safe and tolerable, to date ✓ All patients treated demonstrate objective and subjective responses ✓ Dose response ✓ Appears to have more robust response & longer duration of effect ✓ Enrollment complete, dosing ongoing with full data expecte
Slide 26: Beyond RP for Photoswitch Platform
26 Beyond RP for Photoswitch Platform ▪ Other inherited retinal diseases (rod-cone dystrophies, choroideremia, …) ▪ Age-related macular degenerative diseases ＞ Geographic atrophy ＞ Late-stage wAMD ▪ In combination with any and all gene-replacement therapies ▪ Screening for optogenetics Indications E
Slide 27: Retinal Disease Therapies Experiencing Strong Adoption
27 Retinal Disease Therapies Experiencing Strong Adoption Indication Therapies & Pricing Patients Wet AMD Vabysmo (Roche): $13,140 year 1, $6570 after Eylea (Regeneron): Wet AMD & DME, $16,000 8+ million (US) Geographic atrophy Syfovre (Apellis): $26,500 per year ($18.4 MM 1st Qtr of sales) Zimura (
Slide 28: KIO-101 Ocular Presentation of Rheumatoid Arthritis & Other Autoimmune Diseases (OPRA+)
28 KIO-101 Ocular Presentation of Rheumatoid Arthritis & Other Autoimmune Diseases (OPRA+)
Slide 29: KIO-101: Selectively Targets T-Cell Mediated Inflammation in the Eye
29 KIO-101: Selectively Targets T-Cell Mediated Inflammation in the Eye *Dihydroorotate Dehydrogenase ‡ Sanofi 10-K 2022 KIO-101 has Demonstrated Greater Specificity & Potency IMU-838 (Vidofludimus) Aubagio KIO-101 415.6 160.2 0.2 IC50 (ȠM) Systemic approaches do not deliver sufficient drug to ocula
Slide 30: Novel Approach to Address Major Need Among RA Patients & Beyond
30 Novel Approach to Address Major Need Among RA Patients & Beyond Ocular Surface Discomfort is the Most Common Non-Articular Complaint Rheumatol. Int. 2017 Sep;37(9):1551-1557. Eyenet Magazine. 2016 Nov:37-9. IVOS. 2015 Jun;56(7):4437. Access Patients through Specialists DHODH Inhibition Ideally Su
Slide 31: KIO-101: Exploratory Phase 1b Ocular Surface Inflammation Trial
31 KIO-101: Exploratory Phase 1b Ocular Surface Inflammation Trial Enrolled Patients with Ocular Surface Inflammation* (N=21, 14/7) Randomization Days 1-12 BID Dosing Day 20 Final Follow-up Cohort 4: KIO-101 (0.15%) Cohort 4: Placebo Dosing Days Follow-Up Days ▪ Two cohorts, randomized, double maske
Slide 32: KIO-101-1101 Key Data Summary
32 KIO-101-1101 Key Data Summary Slide* Safety & Tolerability ▪ 0.15% was well tolerated in patients with ocular surface inflammation * Presented April 26, 2022 @ American Society of Cataract & Refractive Surgery (ASCRS) Annual Conference
Slide 33: KIO-101: Phase 2 OPRA+ Trial Design
33 KIO-101: Phase 2 OPRA+ Randomized, Multicenter, Double Masked, Multiple Ascending Dose Trial ▪ Three-arm, randomized (1:1:1), controlled, double masked, 90-day BID topical dosing ▪ Dx of RA (or other AI disease), conjunctival hyperemia, ODS-VAS ▪ Up to 120 patients ▪ ODS-VAS, Schirmer’s, conjunct
Slide 34: KIO-201 Proprietary, Modified Form of Hyaluronic Acid to Heal Ocular Surface Wounds
34 KIO-201 Proprietary, Modified Form of Hyaluronic Acid (HA) to Heal Challenging Ocular Surface Wounds
Slide 35: Persistent Corneal Epithelial Defects (PCED)
35 Persistent Corneal Epithelial Defects (PCED) * American Academy of Ophthalmology, Ocular Surgery News: April 10, 2019, Med. Hypothesis Discov. Innov. Ophthalmol. 8 (2019): 163-176. Unmet Medical Need (Orphan Indication) ▪ Failure of normal closure of a corneal injury in 10–14 days, despite standa
Slide 36: KIO-201: Proprietary, Cross-Linked Form of HA
36 KIO-201: Proprietary, Cross-Linked Form of HA * Regulated as a medical device until 2020 American Academy of Ophthalmology, Ocular Surgery News: April 10, 2019, Med. Hypothesis Discov. Innov. Ophthalmol. 8 (2019): 163-176. Ideally Suited to Promote Healing ▪ HAs known to promote epithelium healin
Slide 37: KIO-201: Phase 2 PCED Study
37 KIO-201: Phase 2 PCED Study Single-Arm, Open Label Trial ▪ Single-arm, open label, 28 days 6x daily topical dosing ▪ Dx of Stage 1 or 2 PCED ▪ Up to 10 patients ▪ Safety & tolerability ▪ % of patients healed (<0.5 mm lesion size) at week 4, time to complete healing Day 21 Visit 4 KIO-201 (0.75%)
Slide 38: KIO-201: PCED Study Results
38 KIO-201: PCED Study Results Baseline Day 7 Day 14 Day 21 Day 28 Fluorescein staining of a representative patient in the study
Slide 39: KIO-201: PCED Study Results
39 KIO-201: PCED Study Results * p<0.003 versus baseline 0 10 20 30 0 5 10 15 Day A re a ( m m 2 ) Mean Defect Size * * * * 0 7 14 21 28 0 25 50 75 100 PCED Patients Healed Day P e rc e n ta g e o f P a ti e n ts Healed Not Healed
Slide 40: Substantial Clinical History
40 Substantial Clinical History 6 clinical studies to date • PRK Recovery 1 • N=39 subjects; 78 eyes • PRK Recovery 2 • N=45 subjects; 90 eyes • PRK Recovery 3 • N=234 subjects; 468 eyes • Punctate Epitheliopathies 1 • N=30 subjects; 60 eyes • Punctate Epitheliopathies 2 • N=20 subjects; 40 eyes • P
Slide 41: Corporate Overview: Financials, Management & Milestones
41 CORPORATE OVERVIEW Financials, Management & Milestones
Slide 42: Financials & Capitalization
42 Financials & Capitalization Capitalization as of May 11, 2023 Common Stock Equivalents Common Stock 2,024,270 Series D Convertible Preferred (convertible @ $141.28/ share) 52 Warrants (WAEP $16.33) 1,613,483 Options (WAEP $17.01) 211,578 RSAs 70,550 ESPP 191 Available Option Pool 11,175 Total Ful
Slide 43: Capital Raise and Milestones
43 Capital Raise and Milestones S-1 Follow-On Offering Raising $15M in common stock offering + 50% warrant coverage (5 yr) ~$3M existing + $15M raise $18M Burn Rate ~$850k/mo ~21 mo Operating Capital Capital until March 2025 Q3 ‘23 Q4 ‘23 Q1 ‘24 Q2 ‘24 Q3 ‘24 Q4 ‘24 KIO-301 ABACUS Interim Data KIO-3
Slide 44: Leadership Team
44 Leadership Team Brian M Strem, PhD President & CEO Stefan Sperl, PhD EVP – CMC & Operations Eric J Daniels, MD, MBA Chief Development Officer Melissa Tosca, CPA EVP – Finance
Slide 45: Board of Directors
45 Board of Directors Brian M Strem, PhD President & CEO Paul Chaney Chairman Aron Shapiro Ken Gayron Praveen Tyle David Hollander, MD, MBA Erin Parsons
Slide 46: Scientific Advisory Board
46 Scientific Advisory Board Daniel Durrie, MD Russel Van Gelder, MD, PhD Charlie Wykoff, MD, PhD Victor Perez, MDFrancis Mah, MDPaul Karpecki, OD, FAAO Allen Ho, MD, PhD Christine Kay, MD, PhD Vitreo Retinal ASSOCIATES Mark Pennesi, MD, PhD
Slide 47: Contact
Contact: info@kiorapharma.com