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 clini cal developm
Slide 3: Targeting Three BLAs in Three Years
3 Targeting Three Biologics License Applications (BLAs) in Three Years Pipeline Phase I Phase II Phase III Target BLA/ MAA * Submission Program 300, 000 (U.S. + EU) Gene - agnostic targeting >100 genes, broad indication ABCA4 - associated retinopathies caused by 1,200+ mutations Advanced dry age - r
Slide 4: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene - agnostic, targets 100+ genes
Slide 5: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
5 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 Potenti
Slide 6: Phase 3 liMeliGhT Trial — Largest RP Data Set
6 Phase 3 liMeliGhT Trial — Largest RP Data Set OCU400 Phase 3 Study Design Endpoints MT D Determined in Phase 1/2 Control Group No Treatment Treatment Group 2.5 п ͳͲ ଵ vg per eye 250 µL 140 RP patients 2:1 ratio Visual function improvement in treated eye vs. control eye Assessed by Low Luminance V
Slide 7: Exclusive licensing agreement with Kwangdong
Executed exclusive licensing agreement with Kwangdong Pharmaceutical Co., Ltd. for South Korean rights to OCU400 Potential sales milestones of $180 million or more in first 10 years of commercialization and royalties equaling 25% of net sales — Kwangdong ocugen. » —
Slide 8: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 - associated retinopathies >1,200 mutations
Slide 9: 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 approved treatments available globally suffer from ABCA4 - associated retinopathies Regulatory Milestone s (Anticipate
Slide 10: GARDian3 - Phase 2/3 Pivotal Confirmatory Trial
10 GARDian3 - Phase 2/3 Pivotal Confirmatory Trial OCU410ST Endpoints Trial Design Randomized 2:1 (N=51) DSMB 4 - week Data Reviews Functional improvement in vision vs. control eye Assessed by LLVA and BCVA EZ analysis (exploratory) Target Population Early - to late - stage disease population Includ
Slide 11: Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit
11 Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit OCU410ST Treated Eyes Untreated Eyes Atrophic Lesion Growth * 54 % slower compared to untreated eyes Visual Function * 100% Stabilized or Improved compared to untreated eyes Nearly 1 - line gain In visual acuity compared to untreat
Slide 12: Why EZ?
12 Source : Velaga et al., BMJ Open Ophthalmology 2021 Ocugen - Fourth Quarter & Full Year 2025 Business Update Why EZ? OCU410ST • EZ is a hyperreflective band representing photoreceptor inner/outer segment junction • Indicates photoreceptor health line OCT • Biomarker for photoreceptor structural i
Slide 13: OCU410ST Provides Retinal Structural Preservation by Reducing EZ Loss
Data points at 12M (N=6, 3 Low Dose, 2 Med Dose, 1 High Dose) CFB= Change from Baseline, EZ preservation/restoration = ≤ +0.5 mm²/year For N=6 Subjects; 1 High dose subject with loss - to - follow up, 1 High dose subject with cataract and 1 Med dose subject with fove al detachment during surgery wer
Slide 14: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age - related macular degeneration ( dAMD )
Slide 15: OCU410 First-in-Class Gene Therapy for GA Patients
15 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
Slide 16: Phase 2 — Reduction in GA Lesion Growth at 12 Months
12 Months 0 1 2 3 4 G A L e s i o n A r e a ( m m 2 ) M e a n ( ± S E M ) c h a n g e f r o m B L Control Treatment (OCU410) -46% BL M6 M12 0 1 2 3 4 G A L e s i o n A r e a ( m m 2 ) M e a n ( ± S E M ) c h a n g e f r o m B L Control Treatment (OCU410) 46% Includes both foveal and subfoveal GA: ge
Slide 17: Phase 2 — Superior Clinical Effects in GA Patients
Phase 2 — Demonstrates Superior Clinical Effects in GA Patients Significant Lesion Size Reduction Compared to Control Responder Rate to OCU410 Treatment 46% 22% OCU410 Approved Therapies with monthly injections 1 Percentage Reduction in GA Lesion Growth (Treatment vs Control) Approved therapies benc
Slide 18: Similar GA Lesion Growth for Medium and High Dose
Similar GA Lesion Growth at 12 Months for Medium and High Dose for Lesion Size ≥7.5mm 2 at Baseline Includes both foveal and sub foveal GA: geographic atrophy, Data points for Control, N=3; Treated Medium Dose, N=5; Treated H igh - Dose, N=6 OCU410 Lesion Size 57% vs 56% lesion reduction in medium d
Slide 19: Phase 2 — Retinal Structural Preservation, Reducing EZ Loss
Phase 1 — Treatment Provides Retinal Structural Preservation by Reducing EZ Loss & Photoreceptor Degeneration 19 M=Months Data points at 12M (N=7, 3 Low Dose, 2 Med Dose, 2 High Dose) 1 High dose subject with loss - to - follow up, and 1 Med dose subject with foveal detachment during surgery were no
Slide 20: Additional Programs Pipeline Progress & Strategic Updates
20 Additional Programs Pipeline Progress & Strategic Updates OCU200 • Targeting diabetic macular edema (DME) • No serious adverse events or OCU200 - related AEs reported across dose - escalation cohorts to date • Enrollment on track for 1Q 2026 completion OCU500 • Inhaled vaccine (mucosal immunity)
Slide 21: Anticipated Near-Term Targeted Milestones
21 Executive Summary Anticipated Near - Term Targeted Milestones RP Stargardt Disease GA — Advanced dry AMD OCU400 OCU410ST OCU410 100% Enrollment Completion Interim data Initiate Rolling BLA Submission Phase 3 T opline D ata Initiate Phase 3 Phase 2 Study Results 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q Phase 3
Slide 22: Appointments to Executive Leadership Team
22 Executive Summary Appointments to Executive Leadership Team Ocugen - Fourth Quarter & Full Year 2025 Business Update Abhi Gupta Executive Vice President, Commercial and Business Development Rita Johnson - Greene Chief Financial Officer Paul Staid Executive Vice President, Operations
Slide 23: Financial Update
Financial Update Three months ended December 31, 2025 Statement of Operations 2024 2025 $8.3 $10.7 Research and development expense 6.3 6.1 General and administrative expense (0.2) (1.2) Interest (expense) income, net $(13.9) $(17.7) Net loss $(0.05) $(0.06) Net loss per share of common stock — basi
Slide 24: Questions & Answers
Questions & Answers na
Slide 25: Thank You
Thank You