Slide 1: Modifier Gene Therapy—Clinical Programs Update
a — ocugen. > ee Courageous Innovation Dedicated to Bringing Game-Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally Modifier Gene Therapy—Clinical Programs Update David Almeida, MD, MBA, PhD Erie Retina Research, Erie, PA COPHy April
Slide 2: Potential for Three Biologics License Applications (BLAs) in the Next Three Years
Potential for Three Biologics License Applications (BLAs) in the Next Three Years p U.S./EU T: t BLA/MAA Product Candidate Status Bice oe ocu400 Phase 3 in Progress Ret IGS er eutocs (iD) (Largest Orphan Gene Therapy 300,000 2026 (Gene-agnostic targeting Clinical Trial) >100 genes, broad indication)
Slide 3: OCU410ST for Stargardt Disease (GARDian)
OCU410ST to Assess the Safety And Efficacy Of Stargardt Disease (GARDian) NCTO5956626
Slide 4: OCU410ST First-in-Class Novel Modifier Gene Therapy Product for Stargardt Disease
OCU410ST First-in-Class Novel Modifier Gene Therapy Product for Stargardt Disease Unmet Need ee as ~100,000 Patients in U.S./EU and 1 million globally who suffer from ABCA4- - associated retinopathies' (>1,200 mutations) Lipid deposits/ Drusen Inflammation Currently there are no approved products in
Slide 5: • • 6M 0.0 0.2 0.4 0.6 0.8 Timeline Treated Eye Untreated Eye 54%
• • 6M 0.0 0.2 0.4 0.6 0.8 Timeline Treated Eye Untreated Eye 54%
Slide 6: Improvement of Visual Function (BCVA) in OCU410ST Treated vs Untreated Eyes
Improvement of Visual Function (BCVA) in OCU410ST Treated vs Untreated Eyes 10 < g -@ TreatedEye -® Untreated Eye (2) 3 Improvement a i Baseline £a i 5 < = ae ge Baseline —~_M3 Me | Stabilization on nN ef os eee oO ea Decrease 3 ii from Baseline 6/6 (100%) evaluable treated eyes demonstrated improve
Slide 7: Overall Safety - OCU410ST Phase GARDian Trial
Overall Safety - OCU410ST Phase 1 GARDian Trial * OCU410ST demonstrated a favorable safety and tolerability profile * Nostudy drug or study procedure related SAEs (deemed related to study drug or study procedure) were reported including: * Nodevelopment of exudation * Noinfectious endophthalmitis *
Slide 8: Clinical Study Design for Phase 2/3 Pivotal Confirmatory BLA Trial
Clinical Study Design for Phase 2/3 Pivotal Confirmatory BLA Trial Phase 2/3 Pivotal Confirmatory BLA Trial Study Endpoints MTD established from Phase 1 trial Primary Efficacy Endpoint: * Change from baseline to Month 12 in the atrophic lesion size [measured in mm? by fundus autofluorescence (FAF)]
Slide 9: OCU400 for Retinitis Pigmentosa
OCU400 for Retinitis Pigmentosa
Slide 10: OCU400 First-in-Class Novel Modifier Gene Therapy for RP
OCU400 First-in-Class Novel Modifier Gene Therapy for RP Unmet Need ~300,000 people in the U.S./EU and 1.6 million globally suffer from RP, which affects >100 genes OCU400 Market Potential Luxturna treatment for —— > withapatient population of ~2,000 (2023 report) One approved product (Luxturna®), a
Slide 11: Safety and Durability of Visual Function (LLVA) - Long-Term Follow-up
Safety and Durability of Visual Function (LLVA) - Long-Term Follow-up Multiple Mutations* (N=10) RHO(N=8) * No Severe Adverse Events (SAE) related to OCU400 p=0.021 p=0.055 have been reported ° o u lo iS 3 a ° Mean ALLVA (+SEM) from Baseline a Mean ALLVA (+SEM) from Baseline ° a 100% (10/10) treated
Slide 12: OCU400 Phase 3 liMeliGhT Trial: Design and Key Criteria
OCU400 Phase 3 liMeliGhT Trial: Design and Key Criteria OCU400 Phase 3 MTD established from Phase 1/2 trial Randomization 2:1 (RHO, Gene-agn Arms Control Group Gene-agnostic, n=25) Dose No treatment Treatment Group 2.5x10"° vg/eye in 250 pL — ocugen. anes Study Endpoints Primary Efficacy Endpoint *
Slide 13: OCU410 (RORA) ArMaDa Trial for the Treatment for Geographic Atrophy
OCU410 (RORA) ArMaDa Trial for the Treatment for Geographic Atrophy Secondary to (AMD — NCTO6018558 ocugen. —
Slide 14: OCU410 First-in-Class Novel Modifier Gene Therapy for GA
OCU410 First-in-Class Novel Modifier Gene Therapy for GA Unmet Need ~19 million patients in U.S. and EU and 260 million globally suffer from dAMD!' ~2-3 million patients in U.S. & EU and ~8 million globally suffer from GA-advanced dAMD? Currently, approved products target only one disease pathway as
Slide 15: OCU410 is Relatively Safe Compared to Approved Drugs for GA
OCU410 is Relatively Safe Compared to Approved Drugs for GA Endophthalmitis and Retinal Detachments / s Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization Intraocular Inflammation Optic ischemic neuropathy Treatment Emergent Serious Adverse Events so ~ bo «bas 1 Treatm
Slide 16: Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes
Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes é 10 SE 5 = & é Stabilization Treated eyes demonstrate £a 4-line (23-letter*) gainin the ) € 2 Hecreass visual acuity when Ss = -10 from Baseline compared to untreated ° 2 15 fellow eyes sa -e Treated Eye ® 3 {i -20 -=- Untre
Slide 17: Substantial slowing of GA Lesion Growth after single sub-retinal injection
Substantial slowing of GA Lesion Growth after single sub-retinal injection 25: 4 Natural History Studies + Untreated Eye nal o a * 41% slower GA lesion growth in treated eye versus fellow eyes after single injection > Mean change from Baseline in GA lesion (mm?) ° & 2 = BL 3M 6M 9M 12M Timeline * Th
Slide 18: OCU410 Treatment Preserves Retinal Tissue in GA Lesions
OCU410 Treatment Preserves Retinal Tissue in GA Lesions o8 07 2 @ ° a Pe a Single sub-retinal injection of OCU410 at 12M is more effective when compared to PM and PEOM treatment 2 rs 01 | 0.4 Os 0.76 Difference in Mean GA lesion Size between Treated and Untreated Eyes (mm?) ° @ PEOM (12M) PM (12M) o
Slide 19: OCU410 Phase 2 Study: Design and Key Criteria
OCU410 Phase 2 Study : Design and Key Criteria MTD established Randomization 1:1:1 Medium-dose High-dose Group cup Control Group n=17 n=i7 n=17 Arms Dose Control Group No treatment Low-dose Group 2.5x 10" vg/mL in 200 pL Medium-doseGroup 5x10" vg/mL in 200 pL High-dose Group 1.5x 10" vg/mL in 200 pL
Slide 20: Near-Term Targeted Milestones—2025
Near-Term Targeted Milestones—2025 * Complete OCU400 Phase 3 recruitment and continue to monitor patients to ensure BLA/MAA filings are complete mid-2026 * Provide safety and efficacy updates from OCU410 Phase | and Phase 2 (interim) * — Initiate OCU410ST Phase 2/3 pivotal confirmatory clinical tria
Slide 21: Ocugen Vision
Ocugen” Vision Fully integrated, patient-centric biotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation — ocugen. —