Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (March 2025)
32 slides from Ocugen's investor presentation, Mar 3, 2025
Slide 1: Courageous Innovation
—_ ~ ocugen. > ee Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies, Cell Therapies, and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally OCGN March 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical
Slide 3: We’re Here to Make an Impact Through Courageous Innovation
We’re Here to Make an Impact Through Courageous Innovation Company Overview Founded 2013 HQ &Manufacturing Malvern, PA Employees ~100 Ticker Symbol OCGN India Business Center Hyderabad Our Values Respect = - San Integrity Teamwork Accountability ocugen. 8 —
Slide 4: Modifier Gene Therapy Platform
Modifier Gene Therapy Platform — ocugen. — Novel approach to treating inherited retinal diseases as well as blindness diseases affecting millions First-in-Class * Differentiator: Master gene regulator; gene-agnostic approach * Pipeline: OCU400 Phase 3 liMeliGhT clinical trial (retinitis pigmentosa)
Slide 5: 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 6: Modifier Gene Therapy Platform
Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions — ocugen. 6 —
Slide 7: Traditional Gene Therapy is Highly Inefficient for Retinal Diseases
Traditional Gene Therapy is Highly Inefficient for Retinal Diseases Red-green colour _First Retinitis Pigmentosa Human Whole exome and whole vision deficit gene (RHO) discovered - Genome _genome sequencing gene Southern Blotting Linkage and Sanger sequence discovery structural sequencing of candidat
Slide 8: The Photoreceptor is Dependent on Cellular Homeostasis Regulated by Key Transcription Factors
The Photoreceptor is Dependent on Cellular Homeostasis Regulated by Key Transcription Factors (TFs) such as NR2E3(OCU400) Untreated — RP Treated -OCU400 (Rhomutation) os ONL ONL INL INL RP Impact on the Photoreceptor Recovery after treatment with OCU400 Normal Photoreceptor * Immunolabeling of rod p
Slide 9: OCU400 for Retinitis Pigmentosa
OCU400 for Retinitis Pigmentosa — ocugen. —
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 One approved product (Luxturna®), a gene therapy, which can address only one | gene (RPE65) Luxturna peak annua
Slide 11: Durability of Visual Function (LLVA) - Long-Term Follow-up
Durability of Visual Function (LLVA) - Long-Term Follow-up Multiple Mutations* (N=10) RHO(N=8) ‘ puael s p=0.055 * 100% (10/10) treated evaluable subjects showed 3 3 10 p=0.008 improvement or preservation in visual function a a ® compared to untreated eyes = = * Treated eyes with multiple mutations
Slide 12: Phase 1/2 Summary: Safety and Efficacy of OCU400
Phase 1/2 Summary: Safety and Efficacy of OCU400 at 2 Years * OCU400 is observed to have a favorable safety and tolerability profile in all subjects across multiple mutations * 100% (10/10) treated evaluable subjects showed improvement or preservation in visual function compared to untreated eyes *
Slide 13: 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. <e Study Endpoints Primary Efficacy Endpoint * Ch
Slide 14: OCU410ST for Stargardt Disease
OCU4IOST for Stargardt Disease — ocugen. —
Slide 15: 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 ~100,000 Patients in U.S./EU and 1 million globally who suffer from ABCA4- OCU410ST Market Potential associated retinopathies' (>1,200 mutations) Currently there are no approved products in the market and it
Slide 16: OCU410ST Stabilizes or Improves Retinal Structure and Visual Function in Phase 1 Clinical Trial
OCU410ST Stabilizes or Improves Retinal Structure and Visual Function in Phase 1 Clinical Trial Parameters for Assessment Low | Low | Low | Med | Med | Med | High | High Overall i e 3 1 2 3 1 3 Measures Primary Endpoint: Atrophic lesion growth (mm?) * 5/7 (71.5%) compared to untreated eyes @ ) ) 6 S
Slide 17: Improvement of Visual Function in OCU410ST Treated vs Untreated Eyes
Improvement of Visual Function in OCU410ST Treated vs Untreated Eyes 10 < g -@ TreatedEye -® Untreated Eye (3) 3 Improvement te i Baseline £0 i Se ge Baseline ~~__M3 Me |] Stabilization OG _ pxsssuocuueuus bo } oX 5 gO oe Decrease 3 r from Baseline 6/6 (100%) evaluable treated eyes demonstrated impr
Slide 18: • • • • • 6M 0.0 0.2 0.4 0.6 Timeline Treated Eye Untreated
• • • • • 6M 0.0 0.2 0.4 0.6 Timeline Treated Eye Untreated Eye 52%
Slide 19: Overall Safety - OCU410ST Phase 1 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 20: 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 21: OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
on lary to Dry Age-Related cular Degeneration
Slide 22: 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 dAMD2 * 2approved products for GA: ‘ Currently, approved products tar
Slide 23: 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 Group Control Group n=16 n=17 n=14 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
Slide 24: Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes in Phase 1/2 Study
Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes in Phase 1/2 Study Mean change in LLVA (ETDRS) from Baseline — ocugen. aes Stabilization Treated eyes demonstrate 2-line (10-letter*) gain in the — visual acuity when from Baseline compared to untreated fellow eyes -e Treated
Slide 25: Substantial Slowing of GA Lesion Growth after Single Sub-Retinal Injection of OCU410
Substantial Slowing of GA Lesion Growth after Single Sub-Retinal Injection of OCU410 ~~ Natural History Studies —* Untreated Eye -@ Treated Eye nN 1694039 ° 44% slower GA lesion growth in 4 Faa% | treated eye versus fellow eyes a after single injection 0.95£0.45 ¢ The slope of the untreated eye over
Slide 26: OCU410 Treatment Preserves Retinal Tissue in GA Lesions
OCU410 Treatment Preserves Retinal Tissue in GA Lesions ° a ° B Pere eer Single sub-retinal injection of OCU410 at 9M is more effective when compared to PM and PEOM treatment ° ® Ser Seema Beers 9° ib 2 ee PEOM (9M) PM (9M) 0CU410(9M) Difference in Mean GA lesion Size between Treated and Untreated E
Slide 27: OCU410 is Relatively Safe Compared to Approved Drugs for GA
OCU410 is Relatively Safe Compared to Approved Drugs for GA Endophthalmitis and Retinal Detachments wf = Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization Intraocular Inflammation Optic ischemic neuropathy Treatment Emergent Serious Adverse Events — ~ co < eos Treatme
Slide 28: OCU200
OCU200 Novel biologic for treating Diabetic Macular Edema (DME), Diabetic Retinopathy (DR) and Wet Age-Related Macular Degeneration (Wet AMD) — ocugen. 28 —
Slide 29: OCU200: Phase 1 Clinical Trial Targeting Diabetic Macular Edema (DME)
OCU200: Phase 1 Clinical Trial Targeting Diabetic Macular Edema (DME) Novelbiologics candidate for sight-threateningconditions * Arecombinantfusion protein of transferrinandtumstatin ———— * Potentialtoaddress diabetic macularedema (DME), diabetic retinopathy (DR), and wet AMD: total prevalence=213M
Slide 30: Ocugen’s Cell Therapy and Mucosal Vaccine Platforms
Ocugen’s Cell Therapy and Mucosal Vaccine Platforms * Therapeutic Focus: Articular cartilage lesions Regene rative Cel * Differentiator: 3D scaffold Therapy Platform * Received RMAT designation First-in-Class + Pipeline: * NeoCart® (Ph3): articular cartilage defects in the knee Therapeutic Focus: Fl
Slide 31: 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 in 1H 2026 * Provide safety and efficacy updates from OCU410 Phase | and Phase 2 (interim) * — Initiate OCU410ST Phase 2/3 pivotal confirmatory clinical tr
Slide 32: 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. 32 —