Okyo Pharma Limited · NASDAQ:OKYO
Okyo Pharma — Investor Presentation (May 2024)
37 slides from Okyo Pharma's investor presentation, May 2024
Slide 1: Targeting Dry Eye Disease and Neuropathic Corneal Pain
Targeting Dry Eye Disease Overcoming& Limitations of Current Dry Eye Therapies Neuropathic Corporate Presentation Corneal Pain MAY 2024 Nasdaq: OKYO Non-Confidential
Slide 2: Disclaimer
Disclaimer By accepting and using this presentation, you will be deemed to agree not to disclose any information contained herein except as may be required by law. Additionally, certain information contained in this presentation has been obtained from published sources prepared by other parties, whi
Slide 3: Dry Eye Disease: Overview
Dry Eye Disease: Overview Ocular Surface Damage Inadequate or unstable tears resulting in lack of moisture and progressive damage to the ocular surface Inflammation & Pain: Key Symptoms of Dry Eye Disease Tear film instability triggers chronic inflammation which leads to symptoms of pain, itchiness,
Slide 4: Limits of Current Standard of Care
Limits of Current Standard of Care 6 FDA-Approved Drugs in the Market with Significant Limitations, Slow Onset of Action, and Numerous Side Effects API Limitations* Restasis 0.05% Delayed response, up to 6 months to improve symptoms, burning sensation Allergan cyclosporine 70% patients do not refill
Slide 5: Global DED Market Expected to Reach ~$11 Billion by 2030
Global DED Market Expected to Reach ~$11 Billion by 2030* ~$3.8 Billion Annual Healthcare Costs** ~$50 Billion Annual Costs of Managing DED to US Economy ** Current Treatment Options Inadequate; More Effective Treatment May Increase Market Size Bausch & Lomb Recent Acquisition of Xiidra for $2.5 Bil
Slide 6: OK-101: A Lipid-Conjugated Chemerin Peptide
OK-101: A Lipid-Conjugated Chemerin Peptide Chemerin Peptide Linker Drug candidate with anti-inflammatory and Chemerin Receptor ocular pain reducing properties Modulates Anchored Lipid Lipid conjugated peptide chemistry minimizes Inflammation OK-101 drug washout and enhances the potency Receptor loc
Slide 7: Pipeline Focus: OK-101 to Treat Ocular Diseases
Pipeline Focus: OK-101 to Treat Ocular Diseases Indication Pre-clinical Phase 1 Phase 2 Phase 3 Dry Eye Disease (“DED”) Phase 2 Trial Completed Neuropathic Corneal Pain Plan to open trial in Q2 2024 (“NCP”) OK-101 Allergic Conjunctivitis Uveitis 7
Slide 8: Chemerin Derived Peptide: A Potential Regulator of Inflammation & Pain
Chemerin Derived Peptide: A Potential Regulator of Inflammation & Pain 1 OK-101 Stable, Protease Resistant 1. Pro-inflammatory chemerin Chemerin Peptide Fragment activates immune cells at the Chemerin 3 Linker inflammation site CHEMR23 Anchored 2 Lipid 2. Peptides derived from chemerin 4 physiologic
Slide 9: OK-101: Targeting Chemerin Receptor
OK-101: Targeting Chemerin Receptor Chemerin Peptide Chemerin Receptor Linker Anchored Lipid Immune Cells Anti-inflammatory Response 9
Slide 10: Membrane Anchoring Improves Potency, Durability
Membrane Anchoring Improves Potency, Durability *In-vitro studies Enhanced Potency Increased Durability Human Chemerin Receptor Human Chemerin Receptor (Wash Resistant) 125 125 (Max soluble unwashed =100%) 100 (Max soluble unwashed =100%) Luciferase activity 100 75 Luciferase activity OK-101 Anchore
Slide 11: Validation: OK-101 Efficacy in Dry Eye Mouse Model
Validation: OK-101 Efficacy in Dry Eye Mouse Model 1600 1400 OK-101 and cyclosporine were 1200 administered topically twice a day Corneal Permeability 1000 Corneal permeability significantly reduced with OK-101 vs phosphate 800 buffer alone 600 Potency of OK-101 was 400 comparable to cyclosporine, a
Slide 12: OK-101 Normalized Goblet Cells & Reduced Inflammatory CD4 T Cells
OK-101 Normalized Goblet Cells & Reduced Inflammatory CD4 T Cells* Enucleated Mouse Eyes from Scopolamine-Treated Dry-Eye Mouse Study Increased Mucin-Secreting Goblet Cells* Reduced Inflammatory Biomarkers* OK-101 normalized goblet cell density OK-101 reduced count of CD4+ T cells Goblet cell densit
Slide 13: OK-101 Decreased Activated Immune MHCII+ Cells in DED Mice
OK-101 Decreased Activated Immune MHCII+ Cells in Draining Lymph Nodes of DED Mice Flow cytometry of draining lymph nodes gated on CD45 Isotype Vehicle OK-101 2.72 67.8 45.1 CD 45 (MHC) class II cell surface receptors involved in antigen presentation are increased in DED
Slide 14: OK-101 Increased Tregs in Draining Lymph Nodes of DED Mice
OK-101 Increased Tregs in the Draining Lymph Nodes of DED Mice Flow cytometry of draining lymph nodes gated on CD45+ CD3+ and CD4+ T cells Isotype Vehicle OK-101 2.36 58.6 80.9 CD 25 FoxP3 Inflammation in DED is mainly mediated by Th17 resulting in decreased Tregs cells
Slide 15: Corneal Neuropathic Pain in Dry Eye Disease (DED)
Corneal Neuropathic Pain in Dry Eye Disease (DED) DED patients suffer from corneal neuropathic pain, making their condition more resistant to anti-inflammatory drugs No Current FDA approved topical treatment for ocular pain ChemR23 receptor on leukocytes targeted by OK-101 is also expressed on neuro
Slide 16: OK-101 Reduced Neuropathic Corneal Pain in Mouse Model
OK-101 Reduced Neuropathic Corneal Pain (“NCP”) in Mouse Model OK-101** Reduced Corneal Pain Response Ciliary Ligation Model* Illustrates OK-101 Comparable to Gabapentin*** (GBP) Potential to Reduce Ocular Pain Ciliary nerve ligation surgery to create the 70 Vehicle corneal neuropathic pain (CNP) mo
Slide 17: OK-101 Addresses Inflammation and Pain Components of Dry Eye
OK-101 Addresses Inflammation and Pain Components of Dry Eye in Mouse Models Dry Eye Cycle Extrinsic Factors: OK-101 Reduces Contact lens Inflammatory Inflammation Loss of Digital screen time Markers of Ocular Goblet Cells Surface Allergies Surgery Inflammation OK-101 Normalizes Blepharitis C Goblet
Slide 18: OK-101 DED Development Timeline
OK-101 DED Development Timeline Phase 2 Phase 3 • 240 Subjects; 80 per arm • 12 Week Dosing • 6-8 Months Duration • Potential Registration Trial 2022 2023 2024 2025 Trial Start Announced Top-Line Data CRO Partner on May 2, 2023 Announced Jan 2024 Completed Full Enrollment of 240 Patients 18
Slide 19: OK-101 DED Phase 2 Trial Design
ENDPOINTS OK-101 DED Phase 2 Trial Design Primary Endpoints Note: First Human Trial of OK-101 (through Day 85) Inferior Corneal Staining (Sign) Day -14 Day 1 Day 15 Day 29 Day 57 Day 85 Ocular Discomfort Score Placebo run- (Symptom) in period Screening Secondary Endpoints Visit 1 Visit 2 Visit 3 Vis
Slide 20: Chemistry of OK-101
CHEMISTRY OF OK-101 OK-101 -- A LIPIDATED PEPTIDE Hydrophilic region • AMPHIPATHIC MOLECULE (both hydrophilic and hydrophobic) Peptide • POTENTIAL TO FORM MICELLES • MOUSE DRY EYE STUDY ONLY USED 0.04% OK-101 Lipid HUMAN TRIAL PERFORMED AT 0.05% AND 0.10% OK-101 Hydrophobic region ONLY SHOWING 0.05%
Slide 21: US Regulatory Requirements for Dry Eye Disease
US Regulatory Requirements for Dry Eye Disease Dry Eye: Developing Drugs for Treatment Guidance for Industry Requirements Acceptable Signs and Symptoms of Dry Eye Sign and Symptoms (co-primaries, p<0.05, Signs: corneal staining, conjunctival no unit difference required) – can be staining, TFBUT, Sch
Slide 22: Approved DED Drugs
Approved DED Drugs Company Labelled Onset of Secondary Endpoint Drug Name Primary Endpoint (Sign) Most common Adverse Effects (Year approved) indication Action (Symptom) Restasis; Allergan Sign; BID up to 6 Tear production N/A Ocular burning (17%) Cyclosporine 0.05% (2003) months Xiidra Bausch & Lom
Slide 23: Improvement in Conjunctival Sum Staining (Sign)
Improvement in Conjunctival Sum Staining (Sign) Data shown is change from baseline in Intent-to-Treat Population P values are vs placebo based on Wilcoxon rank sum test Lissamine Green Staining Fluorescein Staining Day 15 Day 29 Day 57 Day 85 Day 15 Day 29 Day 57 Day 85 -0.6 0.2 LSM (SE) change from
Slide 24: Significant Improvement in Tear Film Break Up Time (Sign)
Day 15 Day 29 Day 57 Day 85 0.7 Significant Improvement 0.6 in Tear Film Break Up LSM (SE) change from baseline (s) 0.5 Time (Sign) 0.4 P = 0.02 Data shown is change from 0.3 baseline in Intent-To-Treat P = 0.01 P = 0.05 P = 0.03 0.2 Population P values are vs placebo based on 0.1 Wilcoxon rank sum
Slide 25: Significant Improvement in Pain (Symptom)
Day 15 Day 29 Day 57 Day 85 -6 -8 Significant Improvement LSM (SE) change from baseline (VAS) in Pain (Symptom) -10 P = 0.2 Data shown is change from baseline -12 in Intent-To-Treat Population -14 P values are vs placebo based on Wilcoxon rank sum test P = 0.03 P = 0.04 -16 P = 0.01 -18 -20 0.05% OK
Slide 26: Significant Improvement in Burning/Stinging & Blurred Vision
Significant Improvement in Burning/Stinging & Blurred Vision (Symptoms) Data shown is change from baseline in Intent-To-Treat Population P values are vs placebo based on Wilcoxon rank sum test Burning/Stinging Blurred vision Day 15 Day 29 Day 57 Day 85 Day 15 Day 29 Day 57 Day 85 8 0 6 LSM (SE) chan
Slide 27: Significant Improvement in Symptoms by Patient Reported Daily Diary
Significant Improvement in Symptoms by Patient Reported Daily Diary Within First Two Weeks Pain Burning/Stinging CHANGE FROM BASELINE CHANGE FROM BASELINE Day 1 to 15 Day 16 to 29 Day 30 to 57 Day 58 to 85 Day 1 to 15 Day 16 to 29 Day 30 to 57 Day 58 to 85 Average Average Average Average Average Ave
Slide 28: OK-101 Phase 2 Trial Highlights - Safety
OK-101 Phase 2 Trial Highlights - Safety 3.8% Treatment emergent adverse events (TEAEs) were observed to be similar to the placebo-treated group. 2.5% No severe drug related ocular TEAEs were seen. Possible drug-related TEAEs were observed in one patient in the OK-101 0.05% treatment group and 3 pat
Slide 29: OK-101 Phase 2 DED Study: Ocular Adverse Events
OK-101 Phase 2 DED Study: Ocular Adverse Events Category OK-101 (0.1%) OK-101 (0.05%) Placebo (N = 80) (N = 81) (N = 79) Number of Ocular AEs 7 19 7 Number of Ocular TEAEs 6 16 6 Number of Ocular SAEs 0 0 0 Number of Ocular TE-SAEs 0 0 0 Number of Subjects Withdrawn Study Drug due to Ocular TEAE: n
Slide 30: OK-101 Disrupts Dry Eye Cycle
OK-101 Disrupts Dry Eye Cycle by Targeting Inflammation, Improving Tear Film Stability and by Ameliorating Multiple Symptoms 38,000,000 US DED patients OK-101 Reduces Inflammatory Markers 18,000,000 Diagnosed OK-101 reduces pain, burning 1,200,000 /stinging, Treated for DED blurred vision, & eye dry
Slide 31: OK-101 to Treat Neuropathic Corneal Pain (NCP)
OK-101 to Treat Neuropathic Corneal Pain (NCP) 31 Confidential
Slide 32: Announced Plans for OK-101 to Treat NCP
July 28, 2023 – Announced Plans for OK-101 to Treat NCP* Announced Clinical Trial Agreement with Tufts Medical Center for Trial on NCP NCP Trial to be conducted by Pedram Hamrah, MD, Professor, at Tufts Medical Center IND filed with FDA for OK-101 to Treat NCP announced on Oct. 9, 2023 Open Label Tr
Slide 33: Plan to Start Phase 2 Trial for OK-101 in NCP
Plan to Start Phase 2 Trial for OK-101 to Treat Neuropathic Corneal Pain (NCP) Phase 2, Randomized, Double masked, Placebo-Controlled Study Assessing Safety and Efficacy of OK-101 in Subjects with NCP • 48 subjects to be enrolled • Three arms, 0.05% OK-101, 0.1% OK-101 and Placebo, (16 per arms) • F
Slide 34: Patent Protection through at least 2039
Patent Protection through at least 2039 OK-101 Technology: OK-201 Technology: Composition of Matter: US 10,233,219 Composition of Matter: US 10,899,796 Issued in US to 2034 with potential patent term extension up Issued in US to 2036 (+70 days of *PTA) with potential to 2039 patent term extension up
Slide 35: Experienced Team With Considerable Drug Development Expertise
Experienced Team With Considerable Drug Development Expertise Management Board Gary S. Jacob, PhD William Clementi, PhD Gabriele Cerrone Bernard Denoyer Chairman, Founder Non-Executive Director Chief Executive Officer and Director Chief Operating Officer Extensive experience founding, Extensive fina
Slide 36: Key Takeaways
Key Takeaways Significant drug effects were observed in multiple signs (conjunctival staining, TFBUT) and symptoms (ocular pain, burning/stinging and blurred vision) of DED as early as the 15-days after dosing. Drug effect was durable throughout the trial for a number of endpoints. Significant impro
Slide 37: Thank you
Overcoming Limitations of Current Dry Eye Therapies Thank you