EX-99.2 3 tm267731d1_ex99-2.htm EXHIBIT 99.2

 

Exhibit 99.2

 

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, su ccess and data announcements of current ongoing preclinical and clinical trials, including the timing of enrollment and data readouts, the ability to init iat e new clinical programs, Ocugen’s financial condition and expected cash runway into the fourth quarter of 2026, statements regarding qualitative assessments of av ailable data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, statem ents regarding potential market size and commercial possibilities of Ocugen’s product candidates, and Ocugen’s projections under its license agreement with Kwa ngdong Pharmaceutical Co., Ltd., which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “po ten tial,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that con vey uncertainty of future events or outcomes to identify these forward - looking statements. Such statements are subject to numerous important factors, risks, and unc ertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that pr eli minary, 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 ma y e merge 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, includi ng by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Co mmi ssion (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 obligati on 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 - Fourth Quarter & Full Year 2025 Business Update

 

 

3 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 - Fourth Quarter & Full Year 2025 Business Update

 

 

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

 

 

5 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 enrollment complete — largest orphan gene therapy trial for RP Manufacturing process validation Rolling submission U.S. (BLA) 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 2027 Phase 3 trial topline data — largest orphan gene therapy trial for RP BLA completed with MAA to follow Recent Milestones Completed Phase 3 trial enrollment Positive long - term, 3 - year Phase 1/2 durability data Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

6 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 Ocugen - Fourth Quarter & Full Year 2025 Business Update Exploratory: Patient Global Impression of Change (PGIC) Top Genes Associated with RP

 

 

 

 

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 (Anticipated) F or 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 9 Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

10 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 - Fourth Quarter & Full Year 2025 Business Update

 

 

11 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 - Fourth Quarter & Full Year 2025 Business Update

 

 

12 Source : Velaga et al., BMJ Open Ophthalmology 2021 Ocugen - Fourth Quarter & Full Year 2025 Business Update 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)

 

 

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 Ocugen - Fourth Quarter & Full Year 2025 Business Update 13

 

 

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

 

 

15 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 15 Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

12 Months 0 1 2 3 4 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 Treatment (OCU410) -46% BL M6 M12 0 1 2 3 4 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 Treatment (OCU410) 46% Includes both foveal and subfoveal GA: geographic atrophy; Treated Eyes that received OCU410 Medium dose and OCU410 High Dose were combined for analysis Data points for Control, N=5; Treated Medium Dose, N=10; Treated High - Dose, N=8 Phase 2 — Treatment Demonstrates Reduction in GA Lesion Growth at 12 Months p=0.015 N=23 OCU410 Lesion Size 46% Reduction in treatment group compared to controls Disease Progression Slower Atrophy Growth compared to controls 16 Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

Phase 2 — Demonstrates Superior Clinical Effects in GA Patients Significant Lesion Size Reduction Compared to Control Responder Rate to OCU410 Treatment 46% 22% OCU410 Approved Therapies with monthly injections 1 Percentage Reduction in GA Lesion Growth (Treatment vs Control) Approved therapies benchmark 12 M 24 M OCU410 Oaks and Derby Study reports are obtained from Heier e tal ., 2023, PMID:37865470 1 Data presented are based on package inserts of approved therapies. OCU410 remains investigational, and no head - to - head trials have been conducted. 17 Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

Similar GA Lesion Growth at 12 Months for Medium and High Dose for Lesion Size ≥7.5mm 2 at Baseline Includes both foveal and sub foveal GA: geographic atrophy, Data points for Control, N=3; Treated Medium Dose, N=5; Treated H igh - Dose, N=6 OCU410 Lesion Size 57% vs 56% lesion reduction in medium dose and high dose compared to controls Disease Progression Slower Lesion Growth in both dose groups compared to controls 18 No difference observed between doses Baseline 6M 12M 0 1 2 3 4 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 OCU410 - Medium Dose OCU410 - High Dose Timeline (Months) -57% -56% p= 0.04 p= 0.04 Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

Phase 1 — Treatment Provides Retinal Structural Preservation by Reducing EZ Loss & Photoreceptor Degeneration 19 M=Months Data points at 12M (N=7, 3 Low Dose, 2 Med Dose, 2 High Dose) 1 High dose subject with loss - to - follow up, and 1 Med dose subject with foveal detachment during surgery were not included in th e analysis Preservation= reduction in the loss of EZ and EZ/RPE (photoreceptors) as a treatment outcome M12 0 1 2 3 4 5 6 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 Eyes Untreated Fellow Eyes OCU410 BLM12 BLM12 0.0 0.5 1.0 1.5 2.0 2.5 E Z / R P E a t r o p h y r a t i o M e a n ( ± S E M ) Treated Eye Untreated Fellow Eye EZ loss 60% slower compared to untreated fellow eyes EZ - RPE loss Reduced compared to untreated fellow eyes potential treatment effect Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

20 Additional Programs Pipeline Progress & Strategic Updates OCU200 • Targeting diabetic macular edema (DME) • No serious adverse events or OCU200 - related AEs reported across dose - escalation cohorts to date • Enrollment on track for 1Q 2026 completion OCU500 • Inhaled vaccine (mucosal immunity) • NIAID to initiate Phase 1 trial in 2Q 2026 OrthoCellix • New wholly owned subsidiary for regenerative assets (e.g., NeoCart ) • Independent financing to maximize value for patients and Ocugen shareholders Ocugen - Fourth Quarter & Full Year 2025 Business Update

 

 

21 Executive Summary Anticipated Near - Term Targeted Milestones RP Stargardt Disease GA — Advanced dry AMD OCU400 OCU410ST OCU410 100% Enrollment Completion Interim data Initiate Rolling BLA Submission Phase 3 T opline D ata Initiate Phase 3 Phase 2 Study Results 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q Phase 3 top line data BLA Submission Ocugen - Fourth Quarter & Full Year 2025 Business Update BLA Completion

 

 

22 Executive Summary Appointments to Executive Leadership Team Ocugen - Fourth Quarter & Full Year 2025 Business Update Abhi Gupta Executive Vice President, Commercial and Business Development Rita Johnson - Greene Chief Financial Officer Paul Staid Executive Vice President, Operations

 

 

Financial Update Three months ended December 31, 2025 Statement of Operations 2024 2025 $8.3 $10.7 Research and development expense 6.3 6.1 General and administrative expense (0.2) (1.2) Interest (expense) income, net $(13.9) $(17.7) Net loss $(0.05) $(0.06) Net loss per share of common stock — basic and diluted December 31, 2024 (audited) December 31, 2025 Balance Sheet Data $58.8 $18.9 Cash and restricted cash $28.7 $28.8 Debt 291,367,558 312,379,972 Shares outstanding Gross proceeds of $22.5 raised in Jan 2026 extends cash runway into Q4 2026 Expected conversion of warrants exercised by Janus Henderson for an additional gross proceeds of $30 million which will extend cash runway into 2Q 2027 Except as otherwise noted, all amounts are in millions, except per share amounts Ocugen - Fourth Quarter & Full Year 2025 Business Update 23

 

 

Questions & Answers

 

 

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