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 fo r t he preclinical and clinical develop
Slide 3: Financing: Private Offering of $115 Million of Convertible Senior Notes
3 • Announced offering of $115 million aggregate principal amount ( $130 million if overallotment is exercised) of 6.75% Convertible Senior Notes due 2034 in a private offering to qualified institutional buyers • $32.7 million of net proceeds from the recently announced Convertible Senior Notes to r
Slide 4: Targeting Three BLAs in Three Years
4 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 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: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene - agnostic, targets 100+ genes
Slide 7: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
7 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 8: Long-Term Durability, Safety and Tolerability Data at 3 Years
8 Long - Term Durability, Safety and Tolerability Data at 3 Years Year 1 Year 2 Year 3 0 5 10 15 Year 1 Year 2 Year 3 0 5 10 15 OCU400 demonstrated a durable improvement in visual function (LLVA) in all evaluable treated subjects at 3 Yr when compared to untreated eyes Mean Change in LLVA (ETDRS Let
Slide 9: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 - associated retinopathies >1,200 mutations
Slide 10: 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 (Completed/
Slide 11: GARDian3 Phase 2/3 Pivotal Confirmatory Trial
11 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 12: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age - related macular degeneration ( dAMD )
Slide 13: OCU410 First-in-Class Gene Therapy for GA Patients
13 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 14: OCU410 Aims to Disrupt GA Treatment Driven by a Novel MOA
14 1 Akula 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 Treatmen
Slide 15: OCU410 Demonstrates 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), Iveric GATHER 2 (Liao 2023, Khanani 2024, Lancet/Ophthalmology, Product Insert), Natur
Slide 16: 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 lo
Slide 17: Anticipated Milestones
3Q 17 Milestones Anticipated Milestones Retinitis Pigmentosa Stargardt Disease Geographic Atrophy OCU400 OCU410ST OCU410 100% Enrollment Completion Interim Outcome Analysis 1 Initiate Rolling BLA Submission Phase 3 topline data Initiate Phase 3 Phase 2 Study Results 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q Phase
Slide 18: Financial Update
Financial Update Three months ended March 31, Statement of Operations 2025 2026 $9.5 $11.3 Research and development expense 6.5 $8.1 General and administrative expense (0.9) (1.3) Other (expense) income, net $(15.3) $(19.2) Net loss $(0.05) $(0.06) Net loss per share of common stock — basic and dilu
Slide 19: Advancing cures for blindness
Advancing cures for blindness IR @ocugen.com
Slide 20: Q&A
Q&A