Kiora Pharmaceuticals, Inc. · NASDAQ:KPRX
Kiora Pharmaceuticals — Eyegate Pharmaceuticals — Investor Presentation (May 2021)
36 slides from Kiora Pharmaceuticals's investor presentation, May 27, 2021
Slide 1: Treating inflammatory and immune diseases
Treating inflammatory and immune diseases May 2021
Slide 2: Forward Looking Statements
2 Forward Looking Statements Some of the statements 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 commercialization effo
Slide 3: Investment Highlights
3 Investment Highlights • PP - 001 completed safety studies for eye drop and intravitreal injection formulations • OBG completed multiple studies for wound healing and dry eye Clinical Stage • PP - 001 eye drops for dry eye and allergic conjunctivitis • OBG re - classified from a device to a drug fo
Slide 4: Company Overview
4 Company Overview PP - 001: fourth generation small - molecule inhibitor of Dihydroorotate Dehydrogenase (DHODH) – Validated class, first approved for RA (Sanofi - Arava) and then approved for MS (Sanofi - Aubagio) – Best - in - class with picomolar potency to overcome off - target side effects of
Slide 5: Transformed from a Device Company to a Drug Pharma Company
5 Transformed from a Device Company to a Drug Pharma Company – OBG ocular surface franchise : • U.S. IND submission and initiation of Phase 2 dry eye study planned in Q4:21 • Proof - of - concept study for persistent epithelial defects expected to initiate in Q4:21 – PP - 001 ocular surface franchis
Slide 6: Creating a World-Class Ocular Surface Franchise with a Deep Pipeline
6 Creating a World - Class Ocular Surface Franchise with a Deep Pipeline 1. Amendment to Healthy Volunteer Study in Austria, allowing Cohort 4 treating dry eye patients 2. Reclassified from a device to a drug in Q2 2021 Two unique platforms addressing inflammation and healing of the ocular surface S
Slide 7: DHODH Inhibitor
DHODH Inhibitor PP - 001
Slide 8: DHODH is an essential mitochondrial enzyme in the pyrimidine pathway
8 DHODH is an essential mitochondrial enzyme in the pyrimidine pathway for activated / abnormally proliferating cells – targeting DHODH ideal for disease specific therapeutic intervention PP - 001, an Inhibitor of Dihydroorotate Dehydrogenase (DHODH) • Inhibition of activated lymphocyte proliferatio
Slide 9: DHODH Inhibitors – Validated Drug Class for Autoimmune Diseases
9 DHODH Inhibitors – Validated Drug Class for Autoimmune Diseases 1. Arava (leflunomide) approved in 1998 for rheumatoid arthritis 2. Aubagio (teriflunomide) approved in 2012 for multiple sclerosis • Teriflunomide is the active metabolite of leflunomide Sanofi: 2 versions of once - daily oral tablet
Slide 10: Designed for Increased Target Specificity and Picomolar Potency
10 415.6 160.2 0.2 0 50 100 150 200 250 300 350 400 450 PP - 001, Designed for Increased Target Specificity and Picomolar Potency • 4 th - generation, non - steroidal, immuno - modulatory, small - molecule inhibitor of Dihydroorotate Dehydrogenase (“DHODH”) • Acquired from 4SC in 2013 2 – To overcom
Slide 11: First-in-Class Drug for Ophthalmology Indications
11 PP - 001 is a First - in - Class Drug for Ophthalmology Indications • First clinical development efforts focused in ophthalmology – High medical need for novel new immunomodulators/anti - inflammatories – Multiple diseases in the anterior and posterior regions of the eye offering substantial mark
Slide 12: Nano-Carrier Eye Drop with Immune-Modulating Potency
12 PP - 001: Nano - Carrier Eye Drop with Immune - Modulating Potency Unique and proprietary formulation optimizes solubility and retention on the ocular surface • These characteristics are provided by loading PP - 001 onto human albumin • Human albumin is a natural tear component • PP - 001 has bee
Slide 13: Eye Drop Safety Study in Healthy Volunteers
13 Phase 1 Dose - Ascending Study Completed • Double - masked, placebo - controlled, randomized, dose escalating study in 24 healthy volunteers (Austria) • Purpose: safety, tolerability, and systemic PK assessment • Results: ✓ Established safety and no SAEs ✓ Excellent tolerability with no burning o
Slide 14: IVT Injection for Phase 1b/2a in Posterior Uveitis PoC Study
14 PP - 001: IVT Injection for Phase 1b/2a in Posterior Uveitis PoC Study • A dose - escalating trial with 3 different drug concentrations (n=12, 1 intravitreal injection/patient) • Treating patients with chronic, non - infectious uveitis – An autoimmune disease with T - cell involvement (IFN - γ an
Slide 15: DHODHi Mechanism of Action Applicable Across Several Categories
15 DHODHi Mechanism of Action Applicable Across Several Categories • Oral formulation underway • Targeting IND H1 2022 Oral PP - 001 • IV formulation completed – Preclinical tox ongoing – HVS targeted for Q4 2021 (Austria) – Pre - IND meeting by year - end IV PP - 001 • Will assess post the HV study
Slide 16: Preparing for the Treatment of Systemic Disease
16 PP - 001: Preparing for the Treatment of Systemic Disease Intravenous Formulation • Grant received from Austrian government in 2020 for development of IV formulation – IV formulation of anti - inflammatory required to treat late - stage (intubated) COVID patients – Formulation work completed and
Slide 17: Oncology Development Strategy
17 PP - 001: Oncology Development Strategy PP - 001 as the most potent DHODHi showed in vitro activity in solid and hematological tumors 1. Completed in vitro efficacy – ~200 tumor cell lines screened Results • Inhibits proliferation of 10 - 15% of solid and hematological tumor cell lines at sub - m
Slide 18: OBG Crosslinked Hyaluronic Acid Eyedrop for Dry Eye
18 OBG Crosslinked Hyaluronic Acid Eyedrop for Dry Eye
Slide 19: HA, a Natural Substance Promoting Healing and Lubrication
19 HA, a Natural Substance Promoting Healing and Lubrication • Ocular Bandage Gel (“OBG”), is based on a modified form of the natural polymer hyaluronic acid (HA) • HA is a material with a high viscosity that promotes wound healing and provides lubrication – Lubricant : can bind up to 1,000 times it
Slide 20: Covalent Crosslinking Creates Unique Attributes Ideal for Ocular Surface
20 Covalent Crosslinking Creates Unique Attributes Ideal for Ocular Surface • Prevents degradation, predominately excreted in crosslinked form • Much longer retention on the ocular surface over non - crosslinked HA (2 hours vs minutes) • Higher shear - thinning properties: – Able to achieve concentr
Slide 21: OBG Program – Recent Conversion from Device to Drug Regulation
21 OBG Program – Recent Conversion from Device to Drug Regulation • OBG is designed to protect the ocular surface; permit re - epithelialization of the cornea and improve ocular surface integrity • Being developed to address ophthalmic conditions where epithelial cells are either missing (wounds) or
Slide 22: Treating Ocular Surface Indications
22 PP - 001 AND OBG TREATING OCULAR SURFACE INDICATIONS • Dry eye: PP - 001 and OBG • Allergic conjunctivitis: PP - 001 • Ocular surface wounds: OBG
Slide 23: Dry Eye: Opportunity
23 Dry Eye: Opportunity • A multifactorial disease of the ocular surface characterized by a loss of homeostasis of the tear film – Inflammation is the common denominator of pathogenesis • Significantly affects the quality of life – Chronicity, pain and irritation, blindness in severe form • Tens of
Slide 24: Dry Eye: T-Cell Mediated Disease
24 Dry Eye: T - Cell Mediated Disease • Desiccating stress (tear film instability and hyperosmolarity) leads to compromised corneal epithelial cells and increased expression of pro - inflammatory cytokines on the ocular surface • Inflammatory cytokines lead to APC activation – Migrate to lymph nodes
Slide 25: Dry Eye: Immunopathogenesis
25 Dry Eye: Immunopathogenesis PP - 001 blocks T - cell proliferation preventing Th1 and Th17 infiltration Dry Eye A T - cell mediated disease • PP - 001 acts upstream to inhibit proliferation of T helper cells (Th1 and Th17) in lymph node • OBG, a natural lubricant, facilitates healing of the ocula
Slide 26: Preclinical In Vivo Proof-of-Concept
26 PP - 001: Preclinical In Vivo Proof - of - Concept • Scopolamine patches applied for 20 days and placed in a controlled environment chamber – 16 eyes per group were treated with 10 µl eyedrop QID for 20 days • EU CTA approved: POC study in patients (n=21) in Austria planned for Q3 • Targeting IND
Slide 27: OBG: Completed Dry Eye POC Study – Trial Design
27 OBG: Completed Dry Eye POC Study – Trial Design ClinicalTrials.gov Identifier: NCT04055324 ( https://clinicaltrials.gov/ct2/show/NCT04055324 ) Primary Objective: to evaluate the effect of Ocular Bandage Gel (OBG) vs. Refresh ® Plus Preservative - Free Lubricant (RPF) in patients with punctate epi
Slide 28: OBG: Demonstrates Healing of the Central Region of the Cornea
28 OBG: Demonstrates Healing of the Central Region of the Cornea • There are 5 regions of the cornea, but the central region is the most important as it covers pupil area, which is responsible for vision quality and corneal sensitivity • Central corneal staining demonstrated a positive treatment eff
Slide 29: Path to Phase 2 Dry Eye Study and IND
29 • Quickest route to proof - of - concept data is study in Austria expected to commence in Q3 – Adding additional cohort with patients to previous safety study in healthy volunteers – 21 patients (14 PP - 001 and 7 vehicle) treated b.i.d. over 12 days – Endpoints contain safety signs and symptoms
Slide 30: Allergic Conjunctivitis
30 ALLERGIC CONJUNCTIVITIS • Overview and PP - 001
Slide 31: Allergic Conjunctivitis Overview and Development
31 • Conjunctiva is a thin, translucent membrane lining the anterior part of the sclera and inside of the eyelids • Conjunctivitis is characterized by inflammation and swelling of the conjunctival tissue, accompanied by engorgement of the blood vessels, ocular discharge, pain, and itchiness (i.e., a
Slide 32: Ocular Wound Healing
32 OCULAR WOUND HEALING • Overview and OBG
Slide 33: Ocular Surface Wound Healing Market
33 Ocular Surface Wound Healing Market • Corneal epithelial and stromal defects are common ocular conditions (2 million annually in the U.S.) – Eye injuries represent up to 18% of emergency room traumas 1 – Corneal abrasions account for up to 4% of all U.S. occupational injuries 1 – Approximately 20
Slide 34: OBG Demonstrates Accelerated Healing post Surgical Procedure
34 OBG Demonstrates Accelerated Healing post Surgical Procedure • Two - arm, positive - controlled, masked via reading center with 234 subjects randomized • Primary endpoint was complete corneal re - epithelialization on Day 3 – 79% receiving OBG were completely healed compared with 63% in the BCL g
Slide 35: Anticipated Milestones
35 Anticipated Milestones • PP - 001 Ocular – Initiate POC dry eye study in Austria – Pre - IND meeting in U.S. for dry eye • PP - 001 Systemic – Topline P1 safety data (Austria) – IND filing in the U.S. and Initiate P1b/2a oncology study • PP - 001 Oral – File IND • PP - 001 Ocular – Topline P2 dat
Slide 36: pharma
pharma May 2021
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