Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (October 2026)
32 slides from Ocugen's investor presentation, Oct 15, 2026
Slide 1: Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market
Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market and Working Even Harder to Provide Access to Patients Globally
Slide 2: Forward-looking statements
2 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 and clinical development
Slide 3: Leader in Ophthalmology Gene Therapy
Pioneering biotechnology company leading the way to address major blindness diseases with novel modifier gene therapy Leader in Ophthalmology Gene Therapy
Slide 4: Targeting Three BLAs by 2028 Pipeline
4 Targeting Three Biologics License Applications (BLAs) by 2028 Pipeline Phase I Phase II Phase III Target BLA/ MAA* Submission Program ABCA4-associated retinopathies caused by 1,200+ mutations Phase 2/3 Pivotal ConfirmatoryClinical Trial in Progress Mid 2027 100,000 (U.S. + EU) Stargardt Disease OC
Slide 5: Modifier Gene Therapy Platform
Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions Modifier Gene Therapy Platform
Slide 6: Traditional therapy has limited therapeutic potential
Traditional therapy has limited therapeutic potential Traditional therapy can only target one single gene at a time, limiting therapeutic potential. Problem 6 of all human proteins are expressed in the retina genes are highly specialized to it, interacting through complex pathways mutations affectin
Slide 7: The Solution is a Gene-Agnostic Approach
7 The Solution is a Gene-Agnostic Approach Solution Our breakthrough technology is designed to address rare diseases and complex diseases Targeting master regulators Master regulators control entire gene networks. By targeting them, Ocugen’s gene therapy platform addresses the root cause of IRDs and
Slide 8: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene-agnostic, targets 100+ genes
Slide 9: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
9 OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa Retinitis Pigmentosa Retinitis Pigmentosa (RP) is a group of rare, inherited retinal diseases caused by mutations in over 100 genes, leading to progressive vision loss and, in many cases, blindness. 1.6 million 2,000 1 Market Potential U.
Slide 10: The largest orphan gene therapy trial ever run
10 The largest orphan gene therapy trial ever run, targeting approval across all RP mutations 140 patients enrolled in Phase 3 liMeliGhT trial (largest orphan gene therapy Ph3) 100+ RP-causing genes addressed by a single subretinal injection 1H 2027 topline data expected: 1Q BLA filing target: 2Q PO
Slide 11: Phase 1/2 Subject Disposition: Safety and Post-hoc Efficacy Analysis
Phase 1/2 Subject Disposition: Safety and Post-hoc Efficacy Analysis 18 Safety analysis All Phase 1/2 subjects −2 → 16 Post-hoc efficacy 9 RHO 3 AD-NR2E3 4 AR-NR2E3 −3 → 13 Post-hoc efficacy 9 RHO 4 AR-NR2E3 −6 → 7 Phase 3 ITT* Post hoc efficacy 5 RHO 2 AR-NR2E3 18 → 16 2 with surgery-related AEs Su
Slide 12: Phase 1/2 Population Meeting Intent to Treat Criteria for Phase 3
Phase 1/2 Population Meeting “Intent to Treat Population” Criteria for Phase 3* at 12M *RHO +AR-NR2E3 Subjects (- Adverse Events, Sentinel); and Ceiling Effect (RHO+AR-NR2E3) Subjects, Sub#= Subject numbers # AR-NR2E3 Subjects: Baseline MLMT at 5 Lux level; 1 Lux level improvement resulted in ceilin
Slide 13: OCU400 Durable LLVA Treatment Effect Through 3 Years
OCU400 Durable LLVA Treatment Effect Through 3 Years 13 7.0 6.2 8.9 0 5 10 15 Year 1 Year 2 Year 3 Δ LLVA, treated − untreated (letters) Multiple mutations Treated − untreated (mean ±SEM) Clinically meaningful (5 letters) 6.5 7.3 7.6 0 5 10 15 Year 1 Year 2 Year 3 Δ LLVA, treated − untreated (letter
Slide 14: OCU400 Phase 3 Study Statistical Power
OCU400 Phase 3 Study: >99% power as per current enrollment and effect size assumption Statistical Method • Power calculated using SAS PROC POWER. • One-sided Fisher's Exact Test. • Significance level (α): 0.025. • Binary responder endpoint. Study Design • Total sample size: N = 140 subjects.(TRT-92,
Slide 15: OCU400 Conclusions
OCU400 Conclusions 15 OCU400 was generally safe and well tolerated across doses and mutations through 3 years, with no SAEs related to OCU400; visual acuity SAEs were attributed to surgery (foveal detachment). Month 12 (N = 18) 89% (16/18) of subjects showed preservation or improvement in the treate
Slide 16: EAP Program in the Bahamas marks first OCU400 approval
16 Highlights: • Bahamas Regulatory Agency, (LARTA1 ), provided provisional approval and priority designation of OCU400 • Full LARTA approval, focused on logistics – site readiness and activation, and patient dosing expected within 90 days under a regulated EAP2 • The EAP is open to patients around
Slide 17: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 -associated retinopathies >1,200 mutations
Slide 18: OCU410ST First-in-Class Gene Therapy for Stargardt Disease
OCU410ST First-in-Class Gene Therapy for Stargardt Disease Stargardt Disease A rare IRD associated with 1,200+ mutations of the ABCA4 gene 1 million 0 Market Potential U.S. + EU globally suffer from ABCA4- approved treatments available associated retinopathies Regulatory Milestones (Completed/antici
Slide 19: Phase 1/2 GARDian1 Trial Demonstrated Clinically Meaningful Benefit
19 Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit OCU410ST Lesion Size Reduction 54% treated vs. Control ImprovementorPreservationinevaluable TreatedEyes Preservation = -/+4 letters from Baseline, Improvement: ≥5 Letters from Baseline NoSeriousAdverseEventsReported N=6 *Khanani et
Slide 20: GARDian1 Visual Function Results
20 Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit Ocugen – October 2026 OCU410ST Treated Eyes UntreatedEyes Visual Function* 100% StabilizedorImproved compared to untreated eyes Nearly 1-line gain In visual acuity compared to untreated eyes Improvement1 from Baseline Decreasefrom
Slide 21: GARDian3 Phase 2/3 Pivotal Confirmatory Trial
21 GARDian3- Phase 2/3 Pivotal Confirmatory Trial OCU410ST Trial Design Endpoints Randomized 2:1(N=51) DSMB 4-week Data Reviews Functional improvementinvision vs. control eye Assessed by LLVAand BCVA EZ analysis (exploratory) TargetPopulation Early- to late-stage disease population Including pediatr
Slide 22: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age-related macular degeneration (dAMD)
Slide 23: OCU410 First-in-Class Gene Therapy for GA Patients
23 OCU410 First-in-Class Gene Therapy for GA Patients Geographic Atrophy Geographic Atrophy (GA) is an advanced form of dry AMD. GA causes irreversible degeneration of retina cells in the macula, leading to loss of central vision. ~8 million 2 Market Size U.S. + EU approved treatments available that
Slide 24: OCU410 Aims to Disrupt GA Treatment Driven by a Novel MOA
24 1Akula et al. Gene Ther 2024; MOA= Mechanism of Action: anti-drusen activity (improves retinal function), anti-inflammatory (suppresses inflammation in HMC3 cells), anti-oxidative (improves ARPE19 cell survival), anti-complement (increases Cd59 protein) OCU410 Aims to Disrupt GA Treatment Driven
Slide 25: Phase 2 ArMaDa Trial Design
Endpoints Randomization 1:1:1 EZ preservation (correlates to visual function) 17 ControlGroup No Treatment Primary: Exploratory: 17 Medium Dose 1x 1010 vg per eye, 200 µL 17 High Dose 3x 1010 vg per eye, 200 µL Phase 2 ArMaDa Trial: To Assess Safety and Efficacy of OCU410 in GA TargetPopulation: Geo
Slide 26: OCU410 Demonstrates Favorable Safety and Tolerability Profile
26 Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization (CNV) Intraocular Inflammation Ischemic Optic Neuropathy Treatment Emergent Serious Adverse Events Treatment EmergentAdverse Events Considered Severe OCU410 Med Dose (N=16) Endophthalmitis and Retinal Detachments Ad
Slide 27: Phase 2 Data Driving Optimal Dose for Phase 3
0 10 20 30 Control Medium Dose Lesion Size Reduction 31% treated vs control Phase 2 Data Driving Optimal Dose for Phase 3 References for Natural History: Mones and Biarnes, 2018, TVST, N=117; For Primary Endpoint analysis evaluable subjects include controls (N=12) and medium dose (N=16); for EZ loss
Slide 28: OCU410 Statistically Significant Reduction in Lesion Size at 12 Months
OCU410 Demonstrates Statistically Significant Reduction in Lesion Size at 12 Months OCU410 shows ~2X effect size compared to approved therapies References: Apellis OAKS/DERBY (Heier et al, Lancet, Product Insert), IvericGATHER 2 (Liao 2023, Khanani 2024, Lancet/Ophthalmology, Product Insert), Natura
Slide 29: Phase 3 Trial Design of OCU410 in GA
29 Phase 3 – Assess Efficacy, Safety and Tolerability of OCU410 in GA Trial Design Endpoints Randomized 2:1(N=237) Proportion of subjects with LLVA ≥15 Letter Loss from Baseline to M12 as assessed by ETDRS visual charts at two consecutive visits 12-month change in EZ Loss Area in study eyes vs. cont
Slide 30: Ocugen Hopes to Transform the Treatment Landscape for GA
Ocugen Hopes to Deliver on Its Promise to Transform the Treatment Landscape for Patients with GA 30 OCU410 potentially creates a new standard of care • First-in-class RORA MOA designed to support central retina and photoreceptor integrity • Promising Phase 2 results indicate 31% reduction in lesion
Slide 31: Anticipated Milestones – Three BLAs by 2028
31 Milestones Anticipated Milestones – Three BLAs by 2028 Retinitis Pigmentosa Stargardt Disease Geographic Atrophy OCU400 OCU410ST OCU410 100% Enrollment Completion Phase 3 Topline Data Phase 2 Study Results Initiate Phase 3 2026 2027 1Q 2Q 3Q 4Q Phase 3 Topline Data BLA Submission Approval/ Launch
Slide 32: Advancing cures for blindness
Advancing cures for blindness Advancing cures for blindness IR@ocugen.com