EX-99.1 2 corporatedeck_march26x20.htm EX-99.1 corporatedeck_march26x20
Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market and Working Even Harder to Provide Access to Patients Globally


 
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 of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events, or otherwise, after the date of this presentation. Forward Looking Statement Ocugen - March 2026


 
Pioneering biotechnology company leading the way to address major blindness diseases with novel modifier gene therapy Leader in Ophthalmology Gene Therapy


 
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 Ocugen - March 2026


 
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 macular degeneration (dAMD) Phase 3 in Progress (Largest Orphan Gene Therapy Clinical Trial) Phase 2/3 Pivotal Confirmatory Clinical Trial in Progress Plan to Initiate Phase 3 in 2026 2026 2027 2028 100,000 (U.S. + EU) 2-3 million (U.S. + EU) Retinitis Pigmentosa Stargardt Disease Geographic Atrophy OCU400 OCU410ST OCU410 Designations: RMAT1, ODD2, OMPD3 & ATMP4 Designation: ODD, RPDD5 & OMPD Designation: ATMP 1 Regenerative Medicine Advanced Therapy (RMAT); 2 Orphan Drug Designation (ODD); 3 Orphan Medicinal Product Designation (OMPD); 4 Advance Therapy Medicinal Products (ATMP); 5 Rare Paediatric Disease Designation (RPDD); Rolling Submission * Market Authorization Application will follow BLA submission Ocugen - March 2026 Phase 3 Enrollment Completed


 
Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions Modifier Gene Therapy Platform


 
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 diseases such as RP, Stargardt and others Diseased retina Healthy retina ONL ONL INL OS OPL INL GCL 1 Jones BW, Marc RE, Pfeiffer RL. Retinal Degeneration, Remodeling and Plasticity. 2016 Oct 28. In: Kolb H, Fernandez E, Nelson R, editors. Webvision: The Organization of the Retina and Visual System [Internet]. Salt Lake City (UT): University of Utah Health Sciences Center; 1995-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK482309/ 2 https://www.nature.com/articles/s41434-024-00440-6 Ocugen - March 2026


 
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 affecting the retina have already been identified 65% 785 250+ Photoreceptor development Inflammation and cell survival Phototransduction Cone cell development Metabolism Ocugen - March 2026


 
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 multifactorial diseases (e.g dAMD). Gene-Agnostic Multifactorial Durable Effect Broad Impact Ocugen - March 2026


 
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 & Tolerability • Proven Clinical Efficacy Modifier Gene Therapy: A Platform Technology Based on AAV5 Vector and Nuclear Hormone Receptor Genes 10Ocugen - March 2026


 
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene-agnostic, targets 100+ genes


 
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.S. + EU approved treatment availableglobally suffer from RP $52M peak annual sales Luxturna® only addresses one gene (RPE65) Regulatory Milestones (Anticipated) One product for all 100 genes delivered via a single, subretinal injection Designations OCU400 Phase 3 trial underway — largest orphan gene therapy trial for RP • Enrollment Completed • Manufacturing process validation Rolling Submission U.S. (BLA) Followed by EU (MAA) FDA (RMAT + ODD) EMA (ATMP+ OMPD) Patients going untreated 298,000 Regenerative Medicine Advanced Therapy (RMAT); Orphan Drug Designation (ODD); Orphan Medicinal Product Designation (OMPD); EAP= Expanded Access Program 2026 Ocugen - March 2026


 
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 Letters) from Baseline Results from Phase 1/2 Study Improvement in visual function in treated eyes when compared to untreated eyes, demonstrates gene -agnostic Mechanism of Action 0 Severe Adverse Events Reported related to OCU400 88 % treated evaluable subjects demonstrated improvement or preservation in visual function compared to untreated eyes at 3 Years M e a n ∆ L L V A ( ± S E M ) fr o m B L (T re a te d -U n tr e a te d ) M e a n ∆ L L V A ( ± S E M ) f ro m B L (T re a te d -U n tr e a te d ) Multiple Mutations RHO Evaluable, consented subjects for multiple mutations at Year 1 (N=11), Year 2 (N=11), Year 3 (N=8) LogMar equivalent of ETDRS letters are represented for Year 3 Improvement or Preservation in evaluable Treated Eyes Preservation = -/+4 letters from Baseline, Improvement: ≥5 Letters from Baseline Clinically meaningful Clinically meaningful


 
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 Acuity (LLVA) Target Population Early- to late-stage disease in broad RP population including pediatrics (3+ years) 12-month change in function vision assessed by LDNA* Improvement in Lux Level in LDNA from baseline to 12 months Primary Secondary 2 1 *LDNA= Luminance Dependent Navigation Assessment is a mobility test administered on a maze under different lux levels Exploratory: Patient Global Impression of Change (PGIC) Top Genes Associated with RP Ocugen - March 2026


 
OCU410ST Stargardt Disease ABCA4 -associated retinopathies >1,200 mutations


 
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/anticipated) for upregulation of networks of key genes improving the cell environment and survival with a single, subretinal injection Designations OCU410ST 2025 Initiated pivotal Phase 2/3 2027 Topline Data, BLA submission FDA (RPDD + ODD) EMA (ATMP+ OMPD) 100% of Patients going untreated 100,000 Potential Patients Advanced Therapeutic Medicinal Product (ATMP); Orphan Drug Designation (ODD); Orphan Medicinal Product Designation (OMPD) 2026 Complete enrollment Interim analysis 16Ocugen - March 2026


 
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 pediatrics (3+ years) 12-month change in atrophic lesion size from baseline vs. control Measured in mm2 by fundus autofluorescence (FAF) 34 Treatment Group 3×1010 vg per eye in 200 µL 17 Control Group No Treatment First Second 17 17 All Subjects MTD Established in Phase 1 Primary Secondary Adaptive design with sample size re-estimation [Mid-2026] Interim Analysis: 24 subjects complete 8 months post-OCU410ST (16 treated and 8 controls) DMC Interim-Data Analysis Ocugen - March 2026


 
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 eyes Improvement1 from Baseline Decrease from Baseline Stabilization Improvement or Preservation in evaluable Treated Eyes Preservation = -/+4 letters from Baseline, Improvement: ≥5 Letters from Baseline No Serious Adverse Events Reported N=6 N=6 *Khanani et al., Nature Eye, January 10, 2026 (https://doi.org/10.1038/s41433-025-04202-5 ) Ocugen - March 2026


 
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 RPE loss and visible atrophy in GA and Stargardt disease What is Ellipsoid Zone (EZ) • EZ loss predicts future visual acuity decline and impacts functional outcomes Structure-Function Correlation • EZ changes occur before visible RPE atrophy expansion in GA progression • Enables earlier intervention and more sensitive treatment effect detection in GA and Stargardt • Correlates to earlier functional therapeutic benefit (effect as early as 1 year) compared to other measures such as visual acuity (effect ≥ 2 years) Why it Matters: Early and Sensitive Detection • EZ metrics predicted treatment response in high-EZ/RPE ratio eyes as well as it is clinically meaningful • EZ is established as clinically relevant AMD/GA endpoint Proven Success in Recent Trials CS (≤1mm diameter) Normal/Intact Ellipsoid Zone Parafoveal Area (PAA) (>1mm to ≤3mm) Ocugen - March 2026


 
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 included in the analysis M12 -2 -1 0 1 2 E Z a re a l o s s ( m m 2 ) M e a n ( ± S E M ) c h a n g e f ro m B L Treated Eye Untreated Fellow Eye • 116% relative decline in EZ loss rate with treatment • Untreated eyes show expected disease decline/atrophy 116% relative reduction in EZ in treated eyes vs. untreated eyes OCU410ST Treatment Provides Retinal Structural Preservation by Reducing EZ Loss & Photoreceptor Degeneration – Phase 1 GARDian Trial OCU410ST 20Ocugen - March 2026


 
OCU410 Geographic Atrophy Advanced dry age-related macular degeneration (dAMD)


 
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 address only 1 of the 4 pathways involved in disease progression globally suffer from advanced dAMD Regulatory Milestones (Anticipated) Designed to address all four pathways associated with GA without 6-12 injections per year and related side effects Designations OCU410 2026 Initiate Phase 3 2028 Topline data, BLA submission EMA (ATMP) SYFOVRE® and IZERVAY® >$1B combined annual sales2-3M Patients Recent Milestone Positive Preliminary 12- month Phase 2 data Approved Products in US 2026 Phase 2 Topline data released 2027 Complete enrollment 22Ocugen - March 2026


 
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 Driven by a Novel MOA Driving global change at the patient level (2-3M patients in U.S. and EU ) GA Patient Experience RORA 4-way MOA1 Addresses all disease pathways – marketed therapies only address the complement system Ocugen - March 2026 OCU410


 
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: Geographic atrophy secondary to dry AMD 24 • Subjects 50 years and older • BCVA of ≥21 ETDRS Letters • Total GA area ≥2.0 and ≤ 20.5 mm2 (1 to 8 disk areas) • GA within foveal and nonfoveal region • CNV in fellow eye is not exclusionary • Subjects who had a history of pegcetacoplan or avacincaptad pegol use were enrolled with 3M washout period Key Protocol Inclusion Criteria: Change in GA lesion size measured in mm2 by FAF at Month 12 Phase 2 (ArMaDa Trial): NCT06018558 Ocugen - March 2026 OCU410


 
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 Adverse Events (AE), Serious AEs, Adverse Event of Special Interest (AESI) 0 0 1* 0 0 0 0 OCU410 High Dose (N=16) Control (N=13) 0 0 2* 0 0 0 0 0 0 1* 1# 0 0 0 # Intraocular Inflammation deemed related to study procedure – resolved *CNV reported as AEs were not related to OCU410 based on DSMB review No OCU410 -related SAEs and AESIs reported to date Phase 2 Safety and Tolerability Data Ocugen - March 2026 OCU410


 
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 against ArMaDa control subjects; Dropout rates for approved therapies reported after 10 injections (PIPER|SANDLER Industry Note, June 2025); OCU410 Optimal Dose = Medium Dose Potentially addresses current unmet need in treating GA: • Potential one-and-done treatment for life versus 6-12 injections per year • May overcome up to 40% drop-out reported in the current standard of care Topline, 12-month efficacy results in Phase 1 and Phase 2: • Promising efficacy in Phase 1 and Phase 2 • Apparent structural preservation on GA lesion • EZ preservation may support visual function Topline data suggests favorable safety and tolerability profile: • No SAEs and AESIs deemed related to OCU410 % Reduction in lesion size compared to control in reported studies 1.0 1.5 2.0 2.5 Pegacetacoplan Monthly @ 24M Pegacetacoplan EOM @ 24M Avacincaptad pegol @ 12M OCU410 - Optimal dose @ 12M Control (Natural History) -22% -19% -15% -31% M ea n C ha ng e of G A Ar ea (m m ²) fr om B L 26Ocugen - March 2026 OCU410


 
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 For lesion criteria (≥2.5 mm 2 and ≤17.5mm2 ) evaluable subjects (N=39)include controls (N=12), medium dose (N=16), and high dose (N=11) and for lesion criteria (≥5 mm 2 and ≤17.5mm2, N=31 ) Controls (N=9), medium dose (N=13) and high dose (N=9). References for Natural History: Mones and Biarnes, 2018, TVST, N=117 Lesion ≥5 mm2 and ≤17.5mm2 (N=31) Lesion Size 33%, 31% reduction in medium, high dose group, respectively compared to control Lesion ≥2.5 mm2 and ≤17.5mm2 (N=39) (Lesion criteria in pivotal trials supporting approval) 0.0 0.5 1.0 1.5 2.0 2.5 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 -16% -31% 12 Months -31%-33% Lesion Size 18% reduction in treated eyes compared to controls Overall Analysis (N=42) ≥2.0 mm2 and ≤20.5mm2 Approved products: • Avacincaptad pegol at 12M (15%) • Pegcetacoplan EM at 24M (22%) 0.0 0.5 1.0 1.5 2.0 2.5 12 Months Control OCU410 - Overall Treated (Medium+High Dose) 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 -18% 12 Months Ocugen - March 2026 OCU410


 
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 include controls (N=12) and medium dose (N=16); for EZ loss analysis, Contro ls (N=12) and medium dose (N=13) GA Lesion ≥2.5 mm 2 and ≤17.5mm2 (Lesion criteria in prior pivotal trials supporting approval); FAF= Fundus Autofluorescence; SD -OCT= Spectral Domain Optical Co herence Tomography; Primary analysis conducted by MMRM and p -value <0.05 EZ loss 27% slower in treated eyes compared to control 0.0 0.5 1.0 1.5 2.0 2.5 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 12 Months Natural History -31% Exploratory Endpoint - Ellipsoid Zone Loss (SD-OCT; N=25) (Correlates to Visual Function) -27% Primary Endpoint – Change in GA Lesion Area (FAF; N=28) EZ A re a lo ss (% ) C ha ng e fr om B Lp < 0.05 Ocugen - March 2026 28 OCU410


 
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 Controls-Preliminary Analysis Medium Dose-Preliminary Analysis Controls-Topline Natural History -31% -35% Ocugen – OCU410 Phase 2 Topline Results -54% -32% against NH -36% against NH N=5 N=10 N=10N=12 N=12 N=16 • Study demographic representative of GA Natural History progression • Study control consistent with Natural History Lesion growth rate thus validating treatment benefit OCU410


 
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 History Responder Rates by Threshold 69% 62% 55% 38% 31% R e d u ct io n T h re sh o ld ≥10% ≥20% ≥30% ≥40% ≥50% Ocugen – OCU410 Phase 2 Topline Results


 
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 size and 27% slower EZ loss • Potential to eliminate treatment burden and patient fatigue to reduce treatment attrition • Optimized Phase 3 trial design and targeted GA lesion size for vision preservation • Upcoming Global Phase 3 , n=~300, adaptive design, >95% power, 3Q 2026 (Target) Ocugen - March 2026 OCU410


 
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 Phase 3 top line data BLA Submission Ocugen - March 2026


 
Advancing cures for blindness Advancing cures for blindness IR@ocugen.com