Ocugen (NASDAQ: OCGN) reports strong Phase 2 OCU410 results in geographic atrophy
Rhea-AI Filing Summary
Ocugen, Inc. reported positive 12‑month topline Phase 2 ArMaDa data for OCU410, its modifier gene therapy for geographic atrophy secondary to dry age‑related macular degeneration. The optimal medium dose showed a 31% reduction in GA lesion growth versus control at 12 months and a 27% slower loss of the ellipsoid zone, a structural marker linked to visual function. Across treated patients there were no OCU410‑related serious adverse events or adverse events of special interest, supporting a favorable safety and tolerability profile. Based on these results, Ocugen plans to initiate a global Phase 3 registrational trial of OCU410 with up to 300 subjects in the third quarter of 2026, as part of its broader goal of three biologics license applications in three years.
Positive
- Phase 2 OCU410 efficacy looks clinically meaningful: the optimal medium dose achieved a 31% reduction in geographic atrophy lesion growth versus control at 12 months, with an 18% overall reduction across treated eyes and 27% slower ellipsoid zone loss, strengthening the case to proceed to Phase 3.
- Favorable safety profile supports late‑stage development: no OCU410‑related serious adverse events or adverse events of special interest were reported after single subretinal dosing in a 51‑patient Phase 2 trial, de‑risking the planned global Phase 3 program.
Negative
- None.
Insights
Strong Phase 2 efficacy and safety de‑risk OCU410 ahead of Phase 3.
Ocugen released 12‑month Phase 2 data for OCU410 in geographic atrophy showing a 31% reduction in lesion growth at the optimal medium dose versus control, and an 18% overall reduction across treated eyes. These effects are measured on an FDA‑accepted structural endpoint.
Safety looks clean: no OCU410‑related serious adverse events or adverse events of special interest were reported, despite a single subretinal administration in patients aged 50 and older. This supports moving directly into a pivotal program rather than repeating mid‑stage work.
The company plans a global Phase 3 trial of up to 300 subjects with an adaptive design powered at over 95%, targeting initiation in Q3 2026. For investors, this meaningfully advances OCU410 along the value chain and strengthens Ocugen’s broader strategy of three biologics license applications in three years, though ultimate outcomes still depend on Phase 3 performance and regulatory review.
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Item 7.01 Regulation FD Disclosure.
On March 24, 2026, Ocugen, Inc. (the “Company”) issued a press release announcing topline 12-month data from Phase 2 ArMaDa clinical trial evaluating OCU410 modifier gene therapy for geographic atrophy secondary to dry age-related macular degeneration. A copy of the press release is filed as Exhibit 99.1 to this Current Report on Form 8-K (“Form 8-K”) and is incorporated herein by reference.
The Company has scheduled a conference call and webcast for 8:00 a.m., EDT, on March 24, 2026, to discuss the full data set from the Phase 2 ArMaDa clinical trial evaluating OCU410 for geographic atrophy, late-stage dry age-related macular degeneration. Attached as Exhibits 99.2 and 99.3 hereto and incorporated herein by reference are presentation materials that the Company will (i) use in connection with its conference call and webcast (see Exhibit 99.2) and (ii) post on its website at www.ocugen.com on March 24, 2026 and may use from time to time in presentations or discussions with investors, analysts, or other parties (see Exhibit 99.3).
The information contained in Item 7.01 of this Form 8-K, including Exhibits 99.1, 99.2 and 99.3 attached hereto, is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”), nor shall it be deemed incorporated by reference in any filing with the U.S. Securities and Exchange Commission, whether made by the Company before or after the date hereof, regardless of any general incorporation language in such filing, under the Securities Act or the Exchange Act, except as expressly set forth by specific reference in such a filing.
The press release and the presentations attached hereto as Exhibits 99.1, 99.2 and 99.3 include “safe harbor” language pursuant to the Private Securities Litigation Reform Act of 1995, as amended, indicating that certain statements contained therein are “forward-looking” rather than historical.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
| Exhibit No. |
Document | |
| 99.1 | Press Release, dated March 24, 2026. | |
| 99.2 | Webcast Presentation, dated March 24, 2026. | |
| 99.3 | Corporate Presentation, dated March 24, 2026. | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document). |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
Date: March 24, 2026
| OCUGEN, INC. | ||
| By: | /s/ Shankar Musunuri | |
| Name: Shankar Musunuri | ||
| Title: Chairman, Chief Executive Officer, & Co-Founder | ||
Exhibit 99.1
Ocugen Announces Topline 12-month Data from Phase 2 ArMaDa Clinical Trial Evaluating OCU410 Modifier Gene Therapy for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
March 24, 2026
| · | Optimal dose intended for Phase 3 demonstrates statistically significant reduction in lesion growth (31%) versus control at 12 months (p< 0.05) | |
| · | Potential 2X treatment benefit compared to 15% and 22% reductions reported for currently approved therapies at 12 and 24 months, respectively | |
| · | No serious adverse events and no adverse events of special interest related to OCU410 reported to date |
MALVERN, Pa., March 24, 2026 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced positive 12-month data from the Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The global prevalence of dAMD is 266 million worldwide, and GA affects approximately 2-3 million people in the United States (U.S.) and Europe. Importantly, this number is expected to increase significantly as populations age.
There are limited options for patients with dAMD in the U.S. and current therapies require 6–12 injections per year indefinitely, leading to substantial burden and significant dropout rates in real-world practice. Outside of the U.S., there are no approved products available.
Key findings from Phase 2 include:
| · | 31% reduction in lesion growth in the optimal dose (medium) group compared to control (p< 0.05) | |
| · | 27% slower rate of ellipsoid zone (EZ) loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function | |
| · | 55% of treated patients demonstrated ≥30% lesion size reduction vs. control | |
| · | Subgroup analysis (subjects with baseline GA lesions ≥5 mm2 and ≤17.5 mm2) showed 33% reduction in lesion growth compared to control in medium dose OCU410 with similar reductions in the high dose group |
The Phase 2 clinical trial builds directly on the clean safety profile observed in Phase 1 with no OCU410-related serious adverse events observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy reported to date.
GA is a multifactorial disease with a complex etiology that involves genetic and environmental factors. The current treatment options for GA in the U.S. are limited to those targeting a single mechanism—the complement pathway. By contrast, OCU410 is a first-in-class RORA-based gene therapy designed to support central retina and photoreceptor integrity through a multi-pathway mechanism—targeting drusen, inflammation, oxidative stress, and complement activation.
“We have confirmed robust treatment effect from a well-controlled Phase 2 trial of a genetic medicine for GA. Now we can move on to Phase 3 with a high degree of confidence,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This moves us one step closer to bringing a transformative one-time treatment to GA patients globally who are desperately seeking rescue from vision loss.”
“Our Phase 2 data consistently demonstrates statistically significant reduction of GA lesion growth after treatment with OCU410 optimal dose, and we continue to benchmark these results against natural history data to contextualize the magnitude of effect,” said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. “We are incorporating these learnings into an anticipated Phase 3 pivotal confirmatory trial with up to 300 subjects and an adaptive design powered at over 95%.”
“There remains a considerable unmet need in treating patients with GA and I am encouraged by the various analyses of the Phase 2 OCU410 data,” said Lejla Vajzovic, MD, FASRS, Director, Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure, Adult and Pediatric Vitreoretinal Surgery and Disease, Duke University Eye Center, and Chair, Ocugen Retina Scientific Advisory Board. “In addition to the strong efficacy and safety data, OCU410 has the potential to eliminate the chronic treatment burden associated with monthly or every-other-month intravitreal injections and to reduce treatment attrition driven by patient fatigue.”
In the Phase 2 study, the safety and efficacy of OCU410 in patients with GA secondary to dAMD are being assessed. Fifty-one (51) patients aged 50 years and older with GA lesions within the foveal or non-foveal region were randomized 1:1:1 to receive a single subretinal administration of OCU410 at a medium dose of 1 × 1010 vector genomes per eye, a high dose of 3 × 1010 vector genomes per eye, or no treatment in the control group; each injection volume was 200 microliters. Of note, choroidal neovascularization in the fellow eye was not exclusionary, and patients with prior exposure to pegcetacoplan or avacincaptad pegol were eligible following a three-month washout.
The primary endpoint was change in GA lesion size at 12 months, measured in square millimeters by fundus autofluorescence, an FDA-accepted structural endpoint used in recent GA registration trials. Exploratory endpoints included EZ preservation on OCT a key biomarker for photoreceptor integrity, which correlates with visual function.
Ocugen plans to initiate the OCU410 Phase 3 registrational trial in the third quarter of 2026 in line with the Company’s goal of three BLA filings in three years.
About dAMD and Geographic Atrophy
Geographic atrophy is an advanced form of dAMD characterized by progressive degeneration of the macula, leading to irreversible central vision loss. Millions of patients worldwide are affected by GA, with a particularly high burden in aging populations in the United States and Europe. Despite recent approvals, treatment options remain limited and require chronic intravitreal injections, underscoring the need for innovative, durable therapies that address multiple disease mechanisms. dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function, and central vision impairment. dAMD accounts for 85-90% of all AMD cases.
About OCU410
OCU410 is an investigational, subretinal injection, AAV5-based gene therapy that delivers RORA (retinoid-related orphan receptor alpha), a nuclear receptor that regulates key pathways involved in retinal homeostasis, including oxidative stress response, complement regulation, inflammation, and lipid metabolism. OCU410 is being developed as a one-time gene therapy for patients with GA secondary to dry AMD. OCU410 has received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies to address major blindness diseases and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, 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; the ability of OCU410 to perform in humans in a manner consistent with nonclinical, preclinical or previous clinical study 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 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 press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact:
Tiffany Hamilton
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com
Exhibit 99.2
Courageous Innovation Topline Data for Phase 2 ArMaDa Trial A Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy (late - stage dry AMD)
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 development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and potential sa fety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, th e a bility to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectati ons for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and un certainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipat es, ” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future ev ent s 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 lim ited to, the risks that preliminary, interim and top - line clinical trial results may not be indicative of, and may differ from, final clinica l data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical tr ial 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 E xch ange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual rep ort s that we file with the SEC. Any forward - looking statements that we make in this presentation speak only as of the date of this presentati on. Except as required by law, we assume no obligation to update forward - looking statements contained in this presentation whether a s a result of new information, future events, or otherwise, after the date of this presentation. Forward Looking Statement Ocugen – OCU410 Phase 2 Topline Results
3 • Introduction – Shankar Musunuri , PhD, MBA, Chairman, Chief Executive Officer, and Co - founder of Ocugen • Phase 2 GA Data Updates • Lejla Vajzovic , MD, FASRS, Professor of Ophthalmology, CME Program Director, Duke Ophthalmology, Duke University School of Medicine & Chairman, Ocugen Scientific Advisory Board • Jay Chhablani, MD, Professor, University of Pittsburgh and UPMC Vision Institute, and President of NetraMind • Topline Data on Clinical Safety and Tolerability – Huma Qamar, MD, MPH, CMI, Chief Medical Officer • Panel Discussion • Lejla Vajzovic , MD, FASRS, Professor of Ophthalmology, CME Program Director, Duke Ophthalmology, Duke University School of Medicine & Chairman, Ocugen Scientific Advisory Board • Victor H. Gonzalez, MD, Retinal Surgeon, Valley Retina Institute, McAllen, Texas, Faculty at University of Texas Rio Grande Valley • Jay Chhablani, MD, Professor , University of Pittsburgh and UPMC Vision Institute, and President of NetraMind • Syed M Shah, MD, Vice Chair for Research and Digital Medicine, Director of Retina Service, Department of Ophthalmology at Emplfiy Health, La Crosse, WI, Ibn al - Haytham Professor, Department of Ophthalmology, Agha Khan University • Dr. Arun Upadhyay, PhD, Chief Scientific Officer, Head of Research & Development, Ocugen • Closing Remarks – Shankar Musunuri , PhD, MBA, Chairman, Chief Executive Officer, and Co - founder of Ocugen OCU410 GA Webcast Meeting Agenda Ocugen – OCU410 Phase 2 Topline Results
Ocugen’s Bold Mission Leader in ophthalmic gene therapies Address major blindness diseases with novel modifier gene therapy Bring transformative one - time treatments to market Work even harder to provide access to patients globally 1 2 3 4
5 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 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 MOA 1 Addresses all disease pathways – marketed therapies only address the complement system Ocugen – OCU410 Phase 2 Topline Results
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); O CU410 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% Mean Change of GA Area (mm²) from BL Ocugen – OCU410 Phase 2 Topline Results 6
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: Geographic atrophy secondary to dry AMD 7 • 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 mm 2 by FAF at Month 12 Phase 2 ( ArMaDa Trial): NCT06018558 Ocugen – OCU410 Phase 2 Topline Results
Phase 2 Baseline Demographics and Characteristics 8 All participants >60 yrs, all White except 1 (Control Group) which was not reported, * Criteria for approved trials (for Syfovre TM ) include baseline lesion of ≥2.5mm 2 and ≤18.5m m 2 Nonfoveal lesions becoming foveal by M12: 1 in Control Group, 1 in Medium Dose Group, 3 in High Dose Group ; LTF/U= Lost to Follow Up ; # - Upper Respiratory Tract Infection ; ITT= Intent to Treat population; mITT = modified Intent - to Treat population Control Group (N=13) High Dose (N=16) Medium Dose (N=16) Factor 77.3 ( 5.4) 78.4 ( 6.5 ) 74.9 ( 5.7) Age, mean (SD) 8 (17.8%) 11 (24.4%) 9 (20.0%) Female, N=28 5 (11.1%) 5 (11.1%) 7 (15.6%) Male, N=17 92.3 88.0 94.0 Bilateral GA (%) 9.1 (5.7) 9.1 (5.4) 8.4 (3.9) GA lesion size (mm 2 ), mean (SD) 2.9 (0.9) 2.8(0.7) 2.9 (0.9) Square root GA size (mm 2 ) mean (SD) 10 (77%) 3 (23%) 8 (50%) 8 (50%) 9 (56%) 7 (44%) Foveal, N= 27 Non foveal lesion, N= 18 3 (23%) 5 (31%) 7 (44%) Lens status - pseudophakic, N=15 47.2 (19.2) 57.8 (18.0) 57.4 (20.3) BCVA (ETDRS) mean (SD) 3 (21.4%) 5 (35.7%) 6 (42.8%) Unifocal, N=14 Ocugen – OCU410 Phase 2 Topline Results Subjects Dosed (N=51) Control (N=17) Med Dose (N=17) High Dose (N=17) 1 - SAE unrelated to OCU410 # 1 - not meeting lesion criteria 1 - surgical procedure issue 1 - inevaluable GA lesion 4 LTF/U 1 LTF/U N=45 Subjects - Overall Safety N=42 Subjects – Overall Efficacy (ITT) N=39 Subjects – Overall Efficacy ( mITT ) Lesion Size*: ≥2.0 mm 2 and ≤20.5mm 2 Lesion Size: ≥2.5 mm 2 and ≤17.5mm 2 (Criterion for approved therapies) N=31 Subjects – Overall Efficacy (Potential Phase - 3 ITT) Lesion Size: ≥5 mm 2 and ≤17.5mm 2 Clinical Trial Flowchart
9 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 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* ϭ ͓ 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 – OCU410 Phase 2 Topline Results
Lesion Size 31%, 16% reduction in medium, high dose group, respectively compared to control 10 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 criteria (≥5 mm 2 and ≤17.5mm 2, 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 mm 2 and ≤17.5mm 2 (N=31) Lesion Size 33%, 31% reduction in medium, high dose group, respectively compared to control Lesion ≥2.5 mm 2 and ≤17.5mm 2 (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 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 Medium Dose High Dose 12 Months Natural History - 16% - 31% 12 Months - 31% - 33% Ocugen – OCU410 Phase 2 Topline Results Lesion Size 18% reduction in treated eyes compared to controls Overall Analysis (N=42) ≥2.0 mm 2 and ≤20.5mm 2 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 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 -18% 12 Months
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 ≤17.5mm 2 (Lesion criteria in prior pivotal trials supporting approval); FAF= Fundus Autofluorescence; SD - OCT= Spectral Domain Optical Co herence Tomography; P rimary 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 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 Medium Dose 12 Months Natural History - 31% Exploratory Endpoint - Ellipsoid Zone Loss (SD - OCT; N=25) (Correlates to Visual F unction) - 27% Primary Endpoint – Change in GA Lesion Area (FAF; N=28) EZ Area loss (%) Change from BL p < 0.05 11 Ocugen – OCU410 Phase 2 Topline Results
Clinical Effects Observed with OCU410 Treatment in GA Participants 12 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% 69% Reduction Threshold ≥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 13 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 – OCU410 Phase 2 Topline Results
Advancing cures for blindness IR @ocugen.com
Exhibit 99.3
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 clini cal development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success an d data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs, statements regarding qualitat ive assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development time lin es, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipat es, ” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify th ese forward - looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results t o d iffer 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 ana lyses 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 tri als; 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 E xch ange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file wit h 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 a s 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 Head quarters , Malvern, Pennsylvania GMP 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 * 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 - related macular degeneration ( d AMD ) 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: RMAT 1 , ODD 2 , OMPD 3 & ATMP 4 Designation: ODD, RPDD 5 & 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
Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions Modifier Gene Therapy Platform
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 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
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: NR2E3 or RORA Single subretinal injection 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 Profile • Proven Clinical Efficacy Modifier Gene Therapy: A Platform Technology Based on AAV5 Vector and Nuclear Hormone Receptor Genes 11 Ocugen - March 2026
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene - agnostic, targets 100+ genes
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 Potential U.S. + EU approved treatment ava ila ble globally suffer from RP $52 M peak annual sales Luxturna® only addresses one gene (RPE65) Regulatory Milestone s (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 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
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 degeneration mouse models NR2E3 Retina - specific gene expression "Master Regulator" of many genetic pathways Resets transcriptional networks and restores retinal health rd16 rd7 Li S. Nature Gene Ther . 2021;28(5):223 - 241. Ocugen - March 2026
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 Histology Slow/Stop Retina Degeneration OCU400 Untreated Treated Biomarkers: Molecular reset Restoration of Expression of Key Transcription Factors Immunofluorescence (IF) Restoration of Phototransduction Gene Expression OCU400 Untreated Treated RP LCA Preclinical Animal Models: Electroretinography (ERG) Improvement in Photoreceptor Function Immunofluorescence (IF) Electroretinography Biomarkers: Molecular Reset Fundus Imaging Histology 15 Ocugen - March 2026
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 Subjects: Baseline MLMT at 5 Lux level; 1Lux level improvement resulted in ceiling effect on old scale on 0 - 6 Lux levels ¶ Subjects 001 - 003, 003 - 001, 001 - 005, 002 - 002 and 003 - 006 were responders based on the adapted LDNA Scale rounded to the nearest Lux level. Visual field is represented as improvements in VTOT or V30 compared to untreated eyes 63% of Treated Eyes Showed Improvement from baseline after 12 months 10s Improvement statistically significant improved in MT completion in Treated Eyes (p=0.031) 75% Improvement of VF in Treated Eyes in ITT subgroup demonstrated compared to untreated eyes (6/8) Eye Mobility Under Low Light Subject ID (with Mutation) Mobility Test Improvement Visual Field Improvement MLMT Scale LDNA Scale MLMT Scale (Phase 1/2) Lux Levels Lux Intensity 400 250 130 50 10 5 1 0.1 0 1 2 3 4 5 6 7 Lux Levels Lux Intensity LDNA Scale (Phase 3) 500 178 63 22 8 3 1 0.125 0 1 2 3 4 5 6 9 8 7 0.35 0.04 Ocugen - March 2026
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 compared to untreated eyes OCU400 demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at 2 Yr when compared to untreated eyes Multiple Mutations (N=11 ) RHO (N=8 ) Mean Change in LLVA (ETDRS Letters) from Baseline Results from Phase 1/2 Study Ocugen - March 2026
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 Visual Acuity (LLVA) Target Population Early - to late - stage disease in broad RP population i ncluding pediatrics ( 3 + years) 12 - month change in f unction vision assessed by LDNA* Improvement in Lux Level i n 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 available globally suffer from ABCA4 - associated retinopathies Regulatory Milestone s (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 ( RPD D + 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 20 Ocugen - March 2026
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 Including pediatrics ( 3 + years) 12 - month change in atrophic lesion size from baseline vs. control Measured in mm 2 by fundus autofluorescence (FAF) 34 Treatment Group 3 п 10 10 vg per eye in 200 µL 17 Control Group No Treatment First Second 17 17 All Subjects M TD 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
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 untreated eyes Improvement 1 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
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 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 fove al detachment during surgery were not included in the analysis M12 -2 -1 0 1 2 E Z a r e a l o s s ( m m 2 ) M e a n ( ± S E M ) c h a n g e f r o 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 24 Ocugen - March 2026
OCU410 Geographic Atrophy Advanced dry age - related macular degeneration ( dAMD )
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 that address only 1 of the 4 pathways involved in disease progression globally suffer from advanced dAMD Regulatory Milestone s (Anticipated) Designed to address all four pathways associated with GA without 6 - 12 injections per year and related side effects Designation s OCU410 2026 Initiate Phase 3 2028 Topline data, BLA submission EMA (ATMP) SYFO V RE ® and IZERVAY ® > $ 1 B combined annual sales 2 - 3M Patients Recent Milestone Positive Preliminary 12 - month Phase 2 data Approved Products in US 2026 Phase 2 full data release 2027 Complete enrollment 26 Ocugen - March 2026
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 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 MOA 1 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 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: Geographic atrophy secondary to dry AMD 28 • 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 mm 2 by FAF at Month 12 Phase 2 ( ArMaDa Trial): NCT06018558 Ocugen - March 2026 OCU410
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 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* ϭ ͓ 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); O CU410 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% Mean Change of GA Area (mm²) from BL 30 Ocugen - March 2026 OCU410
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 criteria (≥5 mm 2 and ≤17.5mm 2, 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 mm 2 and ≤17.5mm 2 (N=31) Lesion Size 33%, 31% reduction in medium, high dose group, respectively compared to control Lesion ≥2.5 mm 2 and ≤17.5mm 2 (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 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 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 mm 2 and ≤20.5mm 2 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 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 -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 ≤17.5mm 2 (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 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 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 Area loss (%) Change from BL p < 0.05 Ocugen - March 2026 32 OCU410
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 legion 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
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 Phase 3 top line data BLA Submission Ocugen - March 2026
Advancing cures for blindness IR @ocugen.com
FAQ
What did Ocugen (OCGN) announce about the OCU410 Phase 2 ArMaDa trial?
How effective was OCU410 in reducing geographic atrophy lesion growth for Ocugen (OCGN)?
What does the safety profile of OCU410 look like in Ocugen’s Phase 2 trial?
What are Ocugen’s next steps for OCU410 after the Phase 2 results?
How large is the geographic atrophy and dry AMD market Ocugen (OCGN) targets with OCU410?
How does OCU410 differ from existing geographic atrophy treatments?
What broader pipeline goals did Ocugen (OCGN) highlight alongside OCU410 data?
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