Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — Investor Presentation (January 2023)
28 slides from Kiora Pharmaceuticals's investor presentation, Jan 9, 2023
Slide 1: Kiora Pharmaceuticals Corporate Outlook
Kiora Pharmaceuticals, Inc. NASDAQ: KPRX January 2023 | Corporate Outlook
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 d
Slide 3: Corporate Highlights
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 ▪ Dosed 1 st patient Q4 2022 in Phase 1b study ▪ Anticipate preliminary results 1H
Slide 4: Diverse Pipeline Offers Near Term Milestones
4 S egme n t Diverse Pipeline Offers Near Term Milestones Indication Product F o rm u lati o n Development Stage Pre - clinical Phase 1 Phase 2 Phase 3 P o st e ri o r Segment Retinitis Pigmentosa (Mutation Agnostic) K I O - 301 IVT Granted Orphan Drug De signation – March 2022 Anterior Ocular Prese
Slide 5: 2022 Recap: Milestones Achieved
Achieved Several Clinical and Regulatory Development Milestones ▪ Initiated ABACUS, a first - in - human Phase 1b trial for KIO - 301 in RP ▪ Received Orphan Drug Designation (ODD) for KIO - 301 in RP ▪ Initiated and completed enrollment of Phase 2 trial of KIO - 201 for PCED ▪ Reported full results
Slide 6: KIO-301 Vision Restoration in Retinitis Pigmentosa
6 KIO - 301 Vision Restoration in Retinitis Pigmentosa
Slide 7: Targeting RP: A Condition with No Available Treatments
7 Prevalence ▪ 1 : 3,500 worldwide ▪ Approximate 100,000 in US Etiology ▪ 50+ genetically distinct subtypes from 150+ mutations ▪ Inherited disease Clinical Presentation ▪ Night blindness, reduced visual field range and eventual loss of central vision ▪ Visual acuity declines Diagnosis ▪ Retinal exa
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 R GC Bipolar
Slide 9: KIO-301: Turns RGCs ON in the Presence of Light
9 Neuron. 92, 100 - 113 (2016) tr an s ci s ▪ In RP, photoreceptors are no longer viable => companion “signal” cells (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 ce
Slide 10: KIO-301: Acts Selectively in Diseased Retinas
10 KIO - 301: Acts Selectivity in Diseased Retinas Sci. Rep. 7, 45487 (2017) KIO - 301 is selective for RGCs in Degenerating Retinas Normal Retina Diseased Retina Light Dark Light Dark U n t r e a t e d KIO - 301
Slide 11: KIO-301: Behavioural Changes in Diseased Mice
11 KIO - 301: Behavioural Changes in Diseased Mice rd1 mouse has similar retinal degeneration as observed in RP patients Homologue molecule to KIO - 301 API Distance traveled is a relative measure N=10 mice/group Mice evaluated 1d after IVT inj Neuron. 81, 800 - 813 (2014)
Slide 12: KIO-301: Phase 1b Study Design (ABACUS)
12 KIO - 301: Phase 1b Study Design (ABACUS) Ongoing, Open Label, Single Ascending Dose Trial ▪ Two cohorts, non - randomized, open - label, single IVT injection per eye ▪ Cohort 1 – NLP/LP patients; Cohort 2 – HM/CF patients ▪ 6 patients, 12 eyes ▪ Primary – safety (AEs), PK & labs ▪ Key secondary
Slide 13: Functional Assessments in Ultra-Low Vision Patients
13 Functional Assessments How Do You Measure Success in Ultra - Low Vision Patients? Objective ▪ Functional MRI ▪ Mobility/maze testing ▪ Object identification ▪ Perimetry Subjective ▪ Patient reported feedback ▪ VFQ - 25 H eal thy RP
Slide 14: Beyond RP for Photoswitch Platform
14 Beyond RP for Photoswitch Platform ▪ Other inherited retinal diseases (rod - cone dystrophies) ▪ Age - related macular degenerative diseases > Geographic atrophy > Late - stage wAMD ▪ In combination with any and all gene - replacement therapies ▪ Screening for optogenetics Indications Expanding E
Slide 15: KIO-101 Ocular Presentation of Rheumatoid Arthritis & Other Autoimmune Diseases
15 KIO - 101 Ocular Presentation of Rheumatoid Arthritis & Other Autoimmune Diseases (OPRA+)
Slide 16: KIO-101: Selectively Targets T-Cell Mediated Inflammation in the Eye
16 KIO - 101: Selectively Targets T - Cell Mediated Inflammation in the Eye *Dihydroorotate Dehydrogenase KIO - 101 has Demonstrated Greater Specificity & Potency IMU - 838 (Vidofludimus) A u b a g i o 415 . 6 160 . 2 0.2 KIO - 101 IC 50 ( Ƞ M) KIO - 101 Locally Inhibits DHODH in Eye ▪ Decreases T H
Slide 17: Novel Approach to Address Major Need Among RA Patients
17 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 Idea
Slide 18: KIO-101: Initiating Phase 2 Study in 2023
18 KIO - 101: Initiating Phase 2 Study in 2023 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 ▪ OD
Slide 19: KIO-201 Modified Hyaluronic Acid for Ocular Surface Wounds
19 KIO - 201 Proprietary, Modified Form of Hyaluronic Acid (HA) to Heal Challenging Ocular Surface Wounds
Slide 20: Ocular Surface Wounds: Opportunity Set
20 Ocular Surface Wounds: Opportunity Set Persistent Corneal Epithelial Defects (PCED) Ocular Surface Surgical Recovery ▪ Failure of normal closure of a corneal injury in 10 – 14 days, despite standard of care ▪ Orphan Drug Designation (ODD) opportunity ▪ PRK – Surgical correction of refractive erro
Slide 21: KIO-201: Proprietary, Cross-Linked Form of HA
21 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
Slide 22: KIO-201: Phase 2 PCED Study
22 KIO - 201: Phase 2 PCED Study Single - Arm, Open Label Trial ( Enrollment Completed ) ▪ 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
Slide 23: Corporate Overview: Management & Milestones
23 CORPORATE OVERVIEW Management & Milestones
Slide 24: 2023 Anticipated Milestones
24 2023 Anticipated Milestones Milestone Timing KIO - 301 | Retinitis Pigmentosa Complete enrollment in Phase 1b RP study (ABACUS) Q2 2023 Report initial results from ABACUS 1H 2023 Report complete results from ABACUS 2H 2023 KIO - 201 | Ocular Wound Repair Receive Orphan Drug Designation PCED Q1 20
Slide 25: Leadership Team
25 Leadership Team Brian M Strem, PhD President & CEO Stefan Sperl, PhD EVP – CMC & Operations Angela Dentiste, MBA VP – Clinical Operations Eric J Daniels, MD, MBA Chief Development Officer Melissa Tosca, CPA EVP – Finance
Slide 26: Board of Directors
26 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 27: Scientific Advisory Board
27 Scientific Advisory Board Daniel Durrie, MD Russel Van Gelder, MD, PhD Charlie Wykoff, MD Victor Perez, MD Francis Mah, MD Paul Karpecki, OD, FAAO Allen Ho, MD, PhD Christine Kay, MD, PhD Vitreo Retinal ASSOCIATES
Slide 28: Contact
Contact: info@kiorapharma.com
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