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: 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 Headquarters, Malvern, PennsylvaniaGMP Facility Values Ocuge
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* SubmissionProgram 300,000 (U.S. + EU) Gene-agnostic targeting >100 genes, broad indication ABCA4-associated retinopathies caused by 1,200+ mutations Advanced dry age-related ma
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 3million+ 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 disease
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 and
Slide 10: 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 & Tole
Slide 11: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene-agnostic, targets 100+ genes
Slide 12: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
12 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 13: Long-Term Durability, Safety and Tolerability Data at 3 Years
13 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 Lett
Slide 14: Phase 3 liMeliGhT Trial: Largest RP Data Set
14 Phase 3 liMeliGhT Trial—Largest RP Data Set OCU400 Phase 3 Study Design Endpoints MTD Determined in Phase 1/2 Control Group No Treatment Treatment Group 2.5×1010 vg per eye 250 µL140 RP patients 2:1 ratio Visual function improvement in treated eye vs. control eye Assessed by Low Luminance Visual
Slide 15: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 -associated retinopathies >1,200 mutations
Slide 16: 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 availableglobally suffer from ABCA4- associated retinopathies Regulatory Milestones (Completed/anticip
Slide 17: GARDian3 Phase 2/3 Pivotal Confirmatory Trial
17 GARDian3- Phase 2/3 Pivotal Confirmatory Trial OCU410ST EndpointsTrial 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 Including ped
Slide 18: Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit
18 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 untreated ey
Slide 19: Why Ellipsoid Zone (EZ)?
19 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 20: OCU410ST Reduces EZ Loss and Photoreceptor Degeneration
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 foveal detachment during surgery were not
Slide 21: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age-related macular degeneration (dAMD)
Slide 22: OCU410 First-in-Class Gene Therapy for GA Patients
22 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 23: OCU410 Aims to Disrupt GA Treatment Driven by a Novel MOA
23 1Akula 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 Treatment D
Slide 24: 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 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 Target Population: G
Slide 25: Phase 2 Safety and Tolerability Data
25 Retinal Vasculitis and/or Retinal Vascular Occlusion Choroidal Neovascularization (CNV) Intraocular Inflammation Ischemic Optic Neuropathy Treatment Emergent Serious Adverse Events Treatment Emergent Adverse Events C onsidered Severe OCU410 Med Dose (N=16) Endophthalmitis and Retinal Detachments
Slide 26: 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 ag
Slide 27: OCU410 Provides Treatment Benefit at 12 Months
0.0 0.5 1.0 1.5 2.0 2.5 3.0 G A L e s io n A re a ( m m 2 ) M e a n ( ± S E M ) c h a n g e f ro m B L Control Medium Dose High Dose 12 Months Natural History Lesion Size 31%, 16% reduction in medium, high dose group, respectively compared to control 27 OCU410 Provides Treatment Benefit at 12 Months
Slide 28: Treatment Benefit with Optimal Dose 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 inc
Slide 29: Consistent Treatment Benefit at 12 Months (Preliminary vs Final)
29 OCU410 Optimal Dose Demonstrates Consistent Treatment Benefit at 12 Months (Preliminary vs Final Analysis) References for Natural History (dotted line above) : Mones and Biarnes, 2018, TVST, N=117 0 1 2 3 4 G A L es io n A re a (m m 2 ) M ea n (± S E M ) c ha ng e fro m B L Medium Dose-Topline Co
Slide 30: Clinical Effects Observed with OCU410 Treatment in GA Participants
Clinical Effects Observed with OCU410 Treatment in GA Participants 30 Efficacy Highlights (N=42) • Robust response – 55% achieved ≥30% reduction • Central therapeutic benefit – median 33% reduction • Superior to natural history (p<0.05 vs natural history/control) Fisher's exact p<0.05 vs. Natural Hi
Slide 31: Ocugen Hopes to Transform the Treatment Landscape for GA Patients
Ocugen Hopes to Deliver on Its Promise to Transform the Treatment Landscape for Patients with GA 31 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 32: Anticipated Near-Term Targeted Milestones
32 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 3Phase 2 Study Results 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q Phas
Slide 33: Advancing cures for blindness
Advancing cures for blindness Advancing cures for blindness IR@ocugen.com