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: 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: Introduction: We're Here to Make an Impact Through Courageous Innovation
4 Introduction We're Here to Make an Impact Through Courageous Innovation Dedicated to Bringing Game - Changing Gene Therapies to Market and Working Even Harder to Provide Access to Patients Globally Respect Integrity Teamwork Accountability Head quarters , Malvern, Pennsylvania GMP Facility Values
Slide 5: Targeting Three BLAs in Three Years
5 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 6: 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 7: Problem: Millions living with retinal disease
7 Problem 3 million+ people living with retinal disease globally Most retinal therapies treat symptoms or target single genes, leaving millions with progressive vision loss and no effective, lasting treatment options. No therapy available for many retinal diseases, specially inherited retinal diseas
Slide 8: 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 8 of all human proteins are expressed in the retina genes are highly specialized to it, interacting through complex pathways mutations affectin
Slide 9: The Solution is a Gene-Agnostic Approach
9 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 a
Slide 10: The Platform: Modifier Gene Therapy Platform
10 The Platform Modifier Gene Therapy Platform AAV5 C arries Modifier Gene ( M )* 1. Modifier Gene (M) Regulation of target gene Expression 2. Molecular Reset of the Gene Network Restoration of Molecular and cellular homeostasis 3. Retina Increased survival of retinal cells 4. * Modifier Genes: NR2E
Slide 11: Modifier Gene Therapy: A Platform Technology Based on AAV5 Vector
Key Attributes of AAV5 vector: • Non - integrating Vector • Enhanced Tissue Specificity • High Transduction Efficiency • Long Term Gene Expression • Low Immunogenicity The Platform Product Highlights: • Novel Gene Agnostic Therapy • Broad Patient Eligibility • Durable Benefit • Favorable Safety & To
Slide 12: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene - agnostic, targets 100+ genes
Slide 13: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
13 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 Potent
Slide 14: A Novel Modifier Gene Therapy for RP Patients
14 A Novel Modifier Gene Therapy for RP Patients OCU400 A gene - agnostic, broad - spectrum approach targeting retinal health at the root Potential curative therapy with a single subretinal injection using NR2E3 rd1 Rho - / - Rho P23H Preclinical data demonstrates activity across multiple retinal de
Slide 15: Preclinical studies demonstrating MOA
Preclinical studies demonstrating MOA OCU400 (AAV encoding NR2E3) Rho -/- Rho P23H+/- Rd7 (NR2E3) Rd1 (PDE6B) Rd16 (CEP290) Adapted from: § Li et al., Nature Gene Therapy, 2021 § McNamee et al., Nature Gene Therapy, 2024 Fundus Imaging Improvement in Retinal Structure OCU400 Untreated Treated Histol
Slide 16: Improved Visual Function and Field in Phase 1/2 Clinical Trials
Patient 1 Patient 2 Patient 3 Patient 4 Patient 5 Patient 6 Patient 7 Patient 8 16 Improved Patient Visual Function and Field in Phase 1/2 Clinical Trials OCU400 *RHO +AR - NR2E3 Subjects ( - Adverse Events, Sentinel); and Ceiling Effect (RHO) Subjects; ^ ceiling effect (AR - NR2E3) # AR - NR2E3 Sub
Slide 17: Long-Term Safety & Durability Data
17 Long - Term Safety & Durability Data OCU400 Improvement in visual function in treated eyes when compared to untreated eyes, demonstrates gene - agnostic Mechanism of Action 0 Se rious Adverse Events Reported 100% treated evalu a ble subjects showed improvement or preservation in visual function c
Slide 18: Phase 3 liMeliGhT Trial: Largest RP Data Set
18 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
Slide 19: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 - associated retinopathies >1,200 mutations
Slide 20: 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 21: GARDian3 Phase 2/3 Pivotal Confirmatory Trial
21 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 22: Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit
22 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 23: Why EZ? Ellipsoid Zone
23 Source : Velaga et al., BMJ Open Ophthalmology 2021 Why EZ? OCU410ST • EZ is a hyperreflective band representing photoreceptor inner/outer segment junction • Indicates photoreceptor health line OCT • Biomarker for photoreceptor structural integrity and metabolic health • EZ disruption precedes RP
Slide 24: 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 25: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age - related macular degeneration ( dAMD )
Slide 26: OCU410 First-in-Class Gene Therapy for GA Patients
26 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 27: OCU410 Aims to Disrupt GA Treatment Driven by a Novel MOA
27 1 Akula et al. Gene Ther 2024; MOA= Mechanism of Action: a nti - drusen activity (improves retinal function), anti - inflammatory (suppresses inflammation in HMC3 cells), anti - oxidative (impr oves ARPE19 cell survival), anti - complement (increases Cd59 protein) OCU410 Aims to Disrupt GA Treatm
Slide 28: Phase 2 ArMaDa Trial design
Endpoints Randomization 1:1:1 EZ preservation (correlates to visual function) 17 Control Group No Treatment Primary: Exploratory: 17 Medium Dose 1x 10 10 vg per eye, 200 µL 17 High Dose 3x 10 10 vg per eye, 200 µL Phase 2 ArMaDa Trial: To Assess Safety and Efficacy of OCU410 in GA Target Population:
Slide 29: Phase 2 Safety and Tolerability Data
29 Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization (CNV) Intraocular Inflammation I schemic Optic Neuropathy Treatment Emergent Serious Adverse Events Treatment Emergent Adverse Events C onsidered S evere OCU410 Med Dose (N=16) Endophthalmitis and Retinal Detachment
Slide 30: OCU410 Phase 2 Data Show Significant Reduction in Lesion Size at 12 Months
OCU410 Phase 2 Data Show Significant Reduction in Lesion Size at 12 Months References: Apellis OAKS/DERBY (Heier et al, Lancet), Iveric GATHER 2 (Liao 2023, Khanani 2024, Lancet/Ophthalmology), Natural History Meta - analysis (Fleckenstein 2018, Ophthalmology). Cha nge from Baseline for OCU410 was a
Slide 31: OCU410 Provides Treatment Benefit at 12 Months
Lesion Size 31%, 16% reduction in medium, high dose group, respectively compared to control 31 OCU410 Provides Treatment Benefit at 12 Months For lesion criteria (≥2.5 mm 2 and ≤17.5mm 2 ) evaluable subjects (N=39)include controls (N=12), medium dose (N=16), and high dose (N=11) and for lesion crite
Slide 32: Treatment Benefit with Optimal Dose (Medium) in Phase 2 ArMaDa Trial
0 10 20 30 12 Months Control Medium Dose Lesion Size 31% reduction in medium dose group compared to control Treatment Benefit with Optimal Dose (Medium) in Phase 2 ArMaDa Trial References for Natural History: Mones and Biarnes , 2018, TVST, N =117; For Primary Endpoint analysis evaluable subjects in
Slide 33: 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 33 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 leg
Slide 34: Anticipated Near-Term Targeted Milestones
34 Milestones Anticipated Near - Term Targeted Milestones Retinitis Pigmentosa Stargardt Disease Geographic Atrophy OCU400 OCU410ST OCU410 100% Enrollment Completion Interim data Initiate Rolling BLA Submission Phase 3 topline data Initiate Phase 3 Phase 2 Study Results 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q P
Slide 35: Advancing cures for blindness
Advancing cures for blindness IR @ocugen.com