Slide 1: Rethinking Ophthalmology: OCS-01 DIAMOND Trial DME Phase 3 Stage 1 Results
Rethinking Ophthalmology OCS-01 | DIAMOND Trial—DME Phase 3 Stage 1 Results May 22, 2023
Slide 2: Disclaimers
Disclaimers This presentation is made pursuant to Section 5(d) and/or Rule 163B of the Securities Act of 1933, as amended, and is intended solely for investors that are qualified institutional buyers or certain institutional accredited investors solely for the purposes of familiarizing such investor
Slide 3: Agenda
Agenda Opening Remarks Sylvia Cheung 01 Chief Financial Officer OCS-01 Phase 3 DIAMOND Riad Sherif, M.D. 02 Stage 1 Chief Executive Officer Q&A Session David Boyer, M.D., Keck School of Medicine (USC); Co-PI for DIAMOND Study Moderated by: Arshad Khanani, M.D., M.A., Sierra Eye Associates, Universit
Slide 4: OCS-01 DIAMOND Trial DME Phase 3 Stage 1 Results
Rethinking Ophthalmology OCS-01 | DIAMOND Trial—DME Phase 3 Stage 1 Results May 22, 2023
Slide 5: Phase 3 Stage 1: OCS-01 Eye Drops for DME Meets Primary Endpoint
Ph 3 Stage 1: OCS-01 Eye Drops for DME Meets Primary Endpoint Rapid and sustained improvements in vision and anatomic structure with robust statistical significance Primary Objective • Results validated loading and maintenance regimen to optimize OCS-01 efficacy Achieved potential in DME Primary End
Slide 6: DME is a Large and Growing Market with Critical Unmet Needs
DME is a Large and Growing Market with Critical Unmet Needs OCS-01 Eye Drops: potential to expand pool of treated DME patients & improve outcomes for those currently treated Growing DME patient Only invasive Late start population size(1) treatments approved of treatment Global DME Patients DME –Trea
Slide 7: Current DME Treatment Paradigm Leaves Two Patient Segments Undertreated
OCS-01 | Current DME Treatment Paradigm Leaves Two Patient Segments Undertreated and Losing Vision Patient presents with DME with DME DME with moderate to severe DME symptoms 24% 43% mild visual 33% recent onset visual impairment Diagnosed by OCT(1) impairment (2) (2) (2) DME Disease Progression and
Slide 8: First Eye Drop for DME – Results Consistent with Previous Trials
OCS-01 | First Eye Drop for DME – Results Consistent with Previous Trials OCS-01 shown to be superior to vehicle on BCVA and CST endpoints in Phase 2 trial Unique product candidate with clinically Positive results in exploratory and Phase Positive Phase 2 results5 validated MOA 2 studies in DME OCS-
Slide 9: DIAMOND Stage 1 Trial Results
Diamor DlAbetic Macular edema patients ON a Drop Stage 1 Trial Results
Slide 10: OCS-01 Phase 3 Program in DME Patients
OCS-01 | Phase 3 Program in DME Patients Loading dose regimen & enriched population increase probability of success Key Enrollment Criteria Stage 1 (N = 148) Stage 2 (N = 350 – 450 per trial; 1:1 Randomization) Age 18-85 years 52 weeks Diabetes mellitus 1 and 2 6 weeks 6 weeks ETDRS BCVA letter scor
Slide 11: OCS-01 Phase 3 in DME Patients – Stage 1
OCS-01 | Phase 3 in DME Patients – Stage 1 Loading dose regimen & enriched population increase probability of success Key Enrollment Criteria Age 18-85 years 6-week Loading Phase 6-week Maintenance Phase Diabetes mellitus 1 and 2 n = 100 OCS-01 6x/day OCS-01 3x/day ETDRS BCVA letter score: 24—65 Ret
Slide 12: Patient Disposition
Patient Disposition ITT population Screened n = 288 Randomized n = 148 OCS-01 Vehicle n = 100 n = 48 Discontinued trial Discontinued trial Withdrawn: n = 1 Withdrawn: n = 3 AE: n = 9 AE: n = 5 Other: n = 5 Other: n = 1 Completed 12 weeks Completed 12 weeks n = 85 n = 39 AE, adverse event; ITT, inten
Slide 13: Demographics: Well-balanced Between Arms
Demographics: Well-balanced Between Arms Parameter OCS-01 (n = 100) Vehicle (n = 48) Age, mean (SD), years 61.9 (9.0) 63.9 (7.3) Male, n (%) 53 (53.0) 26 (54.2) Duration of DME, mean (SD), years 2.0 (2.6) 1.9 (2.7) BCVA, mean (SD), ETDRS letter score 57.5 (9.3) 58.3 (7.5) CST, mean (SD), µm 453.0 (1
Slide 14: Primary Endpoint Achieved with Robust Statistical Significance
Primary Endpoint Achieved with Robust Statistical Significance Rapid improvement in vision with OCS-01 treatment, as assessed by BCVA Strong Improvement in Vision with OCS-01 at Week 6 9.0 OCS-01 (N = 100) 8.0 7.2 Baseline letters 7.0 Vehicle (N = 48) P = 0.007 From 6.0 ETDRS (95% CI, 1.1-7.0) 6, 5.
Slide 15: Improvement in Vision with OCS-01 Sustained to Week 12
Improvement in Vision with OCS-01 Sustained to Week 12 Rapid improvement in BCVA with loading dose regimen sustained with maintenance regimen Loading Phase (6x/day) Maintenance Phase (3x/day) in P = 0.007 9 OCS-01 (N = 100) (95% CI, 1.1-7.0) 8 7.6 7.2 7.1 P = 0.016 Vehicle (N = 48) Baseline 7 (95% C
Slide 16: 25% of OCS-01 Patients Achieve ≥ 3-Line Improvement in BCVA at Week 6
25% of OCS-01 Patients Achieve ≥ 3 Line Improvement in BCVA at Week 6 3-line (15 letter) improvement in BCVA deemed highly clinically relevant Vision Gainers Analysis at Week 6 ≥ 15-Letter Gain 30 6 25.3% 25 Week to P = 0.015 Gaining 20 Baseline OCS-01 (N=100) Vehicle (N=48) Patients 15 From of ters
Slide 17: 27% of OCS-01 Patients with ≥ 3-Line Improvement in BCVA at Week 12
27% of OCS-01 Patients with ≥ 3-Line Improvement in BCVA at Week 12 3-line (15 letter) improvement in BCVA deemed highly clinically relevant Vision Gainers Analysis at Week 12 ≥ 15-Letter Gain 30 27.4% to P = 0.009 25 Gaining Baseline OCS-01 (N=100) 20 Vehicle (N=48) From12 Patients 15 of ters Week
Slide 18: 63.6 μm Reduction in CST Achieved with OCS-01 at Week 6
63.6 μm Reduction in CST Achieved with OCS-01 at Week 6 Central subfield thickness (CST) is a key metric used by physicians to manage DME patients CST as Assessed by Optical Coherence Tomography (OCT) 20 10 Vehicle 5.5 µ m OCS-01 CST 0 in 6, OCS-01 (N = 100) -10 Vehicle (N = 48) Week -20 Changeto P
Slide 19: Reduction in CST Achieved with OCS-01 Sustained to Week 12
Reduction in CST Achieved with OCS-01 Sustained to Week 12 Rapid improvements in CST with loading dose regimen sustained with maintenance regimen CST as Assessed by Optical Coherence Tomography (OCT) Loading Phase (6x/day) Maintenance Phase (3x/day) 30 20 10.2 10 5.50 From 1.10 CST 0 -1.40 in -10 -1
Slide 20: No Unexpected Safety Findings
No Unexpected Safety Findings Treatment Emergent Adverse Events Treatment Emergent Serious Adverse Events (SAE) OCS-01 (N = 100) Vehicle (N = 48) OCS-01 (N = 100) Vehicle (N = 48) n (%) n (%) n (%) n (%) Any TEAE 70 (70.0) 30 (62.5) Any ocular SAE 1 (1.0) 0 Diabetic retinal edema 10 (10.0) 9 (18.8)
Slide 21: IOP Increase Consistent with Literature
IOP Increase Consistent with Literature OCS-01 Vehicle n=100 n=48 n (%) n (%) Any IOP related AE 22/100 (22.0) 1/48 (2.1) 10 mmHg IOP change from baseline at any visit 16/97 (16.5) 0/47 (0) Higher than 25 mmHg IOP at any visit 19/97 (19.6) 1/47 (2.1) Higher than 35 mmHg IOP at any visit 1/97 (1.0) 0
Slide 22: Minimal Mean IOP Increase is Similar Across Loading and Maintenance
Minimal Mean IOP Increase is Similar Across Loading and Maintenance 35 Loading Phase (6x/day) Maintenance Phase (3x/day) 30 From OCS-01 (N = 100) 25 IOP Vehicle (N = 48) Hg in 20 mm Change 15 10 2.79 Baseline, 2.39 2.1 (SD) 1.61 5 0.64 0.0 0.44 0.08 Mean 0 -0.05 -5 -0.17 2 4 6 8 12 Weeks IOP, intrao
Slide 23: OCS-01 Met Primary and Secondary Endpoints in Phase 3 Stage 1
OCS-01 Met Primary and Secondary Endpoints in Ph 3 Stage 1 OCS-01 Vehicle Vehicle Adjusted P Value (n = 100) (n = 48) Change Mean Change in BCVA at Week 6 +7.2 letters +3.1 letters +4.1 letters 0.007 Mean Change in BCVA at Week 12 +7.6 letters +3.7 letters +3.9 letters 0.016 % with 3-line gain in BC
Slide 24: Next Step: Phase 3 DME Trial Stage 2
OCS-01 | Next Step: Phase 3 DME Trial Stage 2 Two global Ph 3 trials (N = 350-450 each) to support NDA filing for treatment of DME Key Enrollment Criteria Age 18-85 years N = 350 – 450 per trial 52 weeks No participation in Stage 1 Confirmed diagnosis of DME OCS-01: 6x/day weeks 1—6; 3x/day weeks 7—
Slide 25: Summary
Summary
Slide 26: OCS-01 Phase 3 Stage 1 Recap
OCS-01 Ph 3 Stage 1 Recap • Trial objectives met: Results validated loading and maintenance regimen to optimize OCS-01 efficacy potential in DME with robust statistical significance • OCS-01 met all Functional and Clinical benefit endpoints in a robust, statistically superior manner (in 3-month tria
Slide 27: Innovative, Diversified and Late-stage Pipeline
Innovative, Diversified and Late-stage Pipeline Next Catalysts Product Investigational Pre-clinical Phase 1 Phase 2 Phase 3 2023 2024 Candidate(s) Indication(s) DIABETIC MACULAR EDEMA 1 endpt. met Stage 1 OCS-01 Optireach® INFLAMMATION AND PAIN FOLLOWING OCULAR SURGERY Ph3 readout NDA technology CYS
Slide 28: Uniquely Positioned to Build Significant Value
Uniquely Positioned to Build Significant Value OCS-01: 1st Eye drop for Diabetic Macular Edema (DME) in Ph3 Targeting critical unmet needs in 3 major st OCS-02: 1 Biologic eye drop for Dry Eye Disease (DED) in Ph2b ophthalmology segments (upside potential from biomarker-driven precision medicine app
Slide 29: Our Purpose
Our Purpose To drive innovation to save sight and improve eye care
Slide 30: Q&A Session
Q&A Session Moderated by: Dr. Pravin Dugel Director, Oculis Holding AG 30