Ocugen (NASDAQ: OCGN) advances eye gene therapies and extends cash runway
Rhea-AI Filing Summary
Ocugen, Inc. reported fourth quarter and full year 2025 results and a broad business update focused on its gene therapy pipeline. For 2025, total revenue was $4,413 thousand while operating expenses reached $67,329 thousand, leading to a net loss of $67,846 thousand, wider than 2024. Cash, cash equivalents and restricted cash fell to roughly the high teens in millions of dollars, and total stockholders’ equity turned negative.
Clinically, enrollment is complete in the 140-patient Phase 3 liMeliGhT trial of OCU400 for retinitis pigmentosa, with topline data planned in the first quarter of 2027 to support a Biologics License Application. OCU410 for geographic atrophy showed preliminary Phase 2 results including a 46% reduction in lesion growth versus control at 12 months and a 60% slower rate of ellipsoid zone loss in treated eyes. Management is targeting three BLA submissions for OCU400, OCU410ST and OCU410 between 2026 and 2028. To bolster liquidity, Ocugen completed a $22.5 million underwritten registered direct offering in January 2026 and notes it may receive up to an additional $30 million if previously issued warrants are exercised, which together underpin guidance for a cash runway into at least the fourth quarter of 2026.
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Insights
Ocugen pairs strong eye-disease data with higher losses and equity funding reliance.
Ocugen is positioning three ophthalmology gene therapies for Biologics License Applications from
At the same time, 2025 operating expenses of
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
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FORM
CURRENT REPORT
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Item 2.02 Results of Operations and Financial Condition.
On March 4, 2026, Ocugen, Inc. (the “Company”) issued a press release announcing certain financial results for the fourth quarter and the fiscal year ended December 31, 2025. The Company has scheduled a conference call and webcast for 8:30 a.m. Eastern Time on March 4, 2026, to discuss these financial results and business updates. The Company will use presentation materials in connection with the conference call and webcast, which presentation materials will be posted on the Company’s website at www.ocugen.com. Copies of the press release and presentation materials are furnished herewith as Exhibit 99.1 and Exhibit 99.2, respectively, to this Current Report on Form 8-K (this “Report”) and incorporated herein by reference.
The information disclosed under Item 2.02 of this Report, including Exhibit 99.1 and Exhibit 99.2, 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, and shall not be deemed to be incorporated by reference in any Company filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits
| Exhibit No. | Description |
| 99.1 | Press Release of Ocugen, Inc. dated March 4, 2026. |
| 99.2 | Earnings Release Presentation issued March 4, 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 4, 2026
| OCUGEN, INC. | ||
| By: | /s/ Shankar Musunuri | |
| Name: Shankar Musunuri | ||
| Title: Chairman, Chief Executive Officer, & Co-Founder | ||
Exhibit 99.1
Ocugen Provides Business Update with Fourth Quarter and Full Year 2025 Financial Results
Conference Call and Webcast Today at 8:30 a.m. ET
| · | Enrollment for the OCU400 Phase 3 liMeliGhT clinical trial—the first and largest gene therapy registrational trial for broad retinitis pigmentosa patients—was completed. Topline Phase 3 data expected in the first quarter 2027, advancing OCU400 towards potential approval in 2027 |
| · | OCU410ST Phase 2/3 pivotal confirmatory trial nearing enrollment completion. Interim data expected in the third quarter 2026, followed by topline Phase 2/3 data in the second quarter 2027 in advance of the BLA submission. |
| · | OCU410 positive preliminary Phase 2 data announced in January. Full Phase 2 data expected in March 2026. |
| · | First regional licensing agreement for OCU400 in 2025 initiates strategic partnership strategy ahead of commercialization. |
| · | Rounded out executive leadership team with top talent in business development, commercial, finance, and operations to encompass all required expertise for upcoming growth |
Malvern, Pa, March 4, 2026 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported fourth quarter and full year 2025 financial results along with a general business update.
“Considerable development across all our modifier gene therapy programs, notable licensing and financing agreements to strengthen our financial position, and meaningful appointments to our leadership team made 2025 a transformative year for Ocugen,” said Dr. Shankar Musunuri, Chairman, CEO, Co-founder of Ocugen. “We are poised to leverage upcoming catalysts and advance the business as we near the first of our three BLA filings.”
Enrollment is now complete for the OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP). As a one-year clinical trial, topline data will be available in the first quarter of 2027. These data are anticipated to support the Biologics License Application (BLA) filing for OCU400 and potential approval in 2027. The liMeliGhT clinical trial enrolled 140 patients who were randomized 2:1 into the treatment group (2.5×1010 vg per eye 250 µL) and untreated control group across mutations (RHO and gene-agnostic arms). The target population included patients with early- to late-stage disease among a broad RP population, including pediatrics (3+ years). The primary endpoint is 12-month change in visual function assessed by LDNA (luminance dependent navigation assessment) with improvement in Lux Level from baseline to 12 months. The OCU400 Phase 3 liMeliGhT clinical trial is the only broad RP gene-agnostic trial and the largest known Phase 3 orphan gene therapy trial.
The OCU410ST Phase 2/3 GARDian clinical trial for Stargardt disease (ST) remains ahead of schedule in preparation for the 2027 BLA filing. In January, the Company announced publication of Phase 1 GARDian1 trial results for OCU410ST in EYE. The study supports the favorable safety, tolerability and efficacy profile of OCU410ST and its potential to provide clinically meaningful functional and structural benefits in ST patients. OCU410ST holds the potential to address the unmet medical need that remains for approximately 100,000 Stargardt patients in the U.S. and Europe who have no treatment option available.
Recently, Ocugen announced positive preliminary 12-month data (~50% of patients evaluated to date) 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). Key findings from Phase 2 include 46% lesion growth reduction (medium + high dose vs. control; p=0.015; N=23) at 12 months and 50% responder rate with patients achieving >50% lesion size reduction vs. control. A subgroup analysis of patients with a baseline GA size ≥7.5 mm²—representing advanced atrophy—demonstrated a 57% reduction in lesion growth in treated eyes for medium dose and a 56% reduction in high dose compared with control eyes. This reduction in lesion size in medium and high doses suggests OCU410 may be more effective in patients with substantial disease burden.
The latest OCU410 data set also included encouraging 12-month Phase 1 findings where OCU410-treated eyes demonstrated 60% slower loss of the ellipsoid zone (a structural and functional exploratory endpoint) compared to untreated fellow eyes. The 60% reduction in ellipsoid zone (EZ) loss rate indicates that OCU410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eye of the same patient.
“With approximately 2 to 3 million GA patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. Current therapies have notable limitations, and there are no treatments approved for GA in Europe, as existing FDA-approved options fail to demonstrate meaningful functional outcomes,” said Dr. Musunuri. “OCU410 is therefore well-positioned to address this critical unmet need, and we look forward to reporting full data from the OCU410 Phase 2 clinical trial this month and initiating Phase 3 in 2026.”
The licensing agreement with Kwangdong Pharmaceutical, Co., Ltd. for the exclusive Korean rights to OCU400—with upfront fees and near-term development milestone payments, along with royalties—was a critical step in Ocugen’s business development strategy, affirming a regional partnership approach for OCU400 that preserves the Company’s rights to larger geographies while also generating a potential return for shareholders.
To extend the cash runway into the fourth quarter of 2026, in January 2026 the Company secured $22.5 million in gross proceeds through an underwritten registered direct offering of common stock led by RTW Investments, with additional participation from new and existing investors. This raise follows the $20 million registered direct offering of common stock and warrants with Janus Henderson Investors in August 2025. The Company may receive up to $30 million of additional gross proceeds from the August 2025 registered direct offering if the warrants are exercised in full.
“I am proud of our accomplishments in 2025, as they accelerate our drive to achieve even more significant clinical and pre-commercial objectives in 2026,” said Dr. Musunuri. “With a full bench of experienced leadership across the organization, I am confident that we have the resources and know-how to take Ocugen to the next level.”
Business Updates
Novel Modifier Gene Therapy Platform—Targeting Three BLA Filings in the Next Three Years
| · | OCU400 – Completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400 and are on track to file the rolling BLA in the third quarter of 2026. Subjects will be followed for a year after dosing for primary endpoint analyses. Positive long-term, 3-year Phase 1/2 durable, safety and tolerability data demonstrates sustained clinically meaningful, approximately 2-line LLVA gain, reinforcing durable gene-agnostic benefit. |
| · | OCU410ST – The Phase 2/3 GARDian3 pivotal confirmatory trial is progressing ahead of schedule with anticipated enrollment completion in the first quarter of 2026. Interim data is expected in the third quarter of 2026. |
| · | OCU410 – In January 2026, Ocugen announced positive preliminary 12-month data for Phase 2 subjects from the ArMaDa clinical trial for GA secondary to dAMD. The complete data set for the ArMaDa trial is expected to be available in March 2026. |
Other Programs
| · | OCU200 – No serious adverse events (SAEs) or adverse events (AEs) related to OCU200 reported to date across the dose-escalation cohorts and trial enrollment is expected to be completed by the first quarter of 2026. |
| · | OCU500 – NIAID intends to initiate the OCU500 Phase 1 clinical trial in the second quarter of 2026. |
| · | NeoCart – Created OrthoCellix as a wholly-owned subsidiary of Ocugen for the regenerative cell therapy assets with a goal of obtaining independent financing. |
Financial Results
| · | Fourth quarter — Research and development expenses for the three months ended December 31, 2025, were $10.7 million compared to $8.3 million for the three months ended December 31, 2024. General and administrative expenses for the three months ended December 31, 2025, were $6.1 million compared to $6.3 million for the three months ended December 31, 2024. Ocugen reported a $0.06 net loss per common share for the three months ended December 31, 2025, compared to a $0.05 net loss per common share for the three months ended December 31, 2024. |
| · | Full year — Research and development expenses for the year ended December 31, 2025, were $39.8 million compared to $32.1 million for the year ended December 31, 2024. General and administrative expenses for the year ended December 31, 2025, were $27.6 million compared to $26.7 million for the year ended December 31, 2024. Ocugen reported a $0.23 net loss per common share for the year ended December 31, 2025, compared to a $0.20 net loss per common share for the year ended December 31, 2024. |
| · | Ocugen’s cash and restricted cash, totalled $18.9 million as of December 31, 2025, compared to $58.8 million as of December 31, 2024. The Company estimates that additional proceeds from the $22.5 million financing in January 2026 will enable it to fund its operations into the fourth quarter of 2026. If the Janus Henderson warrants are fully exercised this year, it is expected that cash runway will be extended into the second quarter of 2027. The Company had 312.4 million shares of common stock outstanding as of December 31, 2025. |
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s leadership team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.
Attendees are invited to participate on the call or webcast using the following details:
Dial-in
Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 3029428
Webcast: Available on the events section of the Ocugen investor site
A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.
About Ocugen, Inc.
Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. 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, 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, including the timing of enrollment and data readouts, the ability to initiate new clinical programs, Ocugen’s financial condition and expected cash runway into the fourth quarter of 2026, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, statements regarding potential market size and commercial possibilities of Ocugen’s product candidates, and Ocugen’s projections under its license agreement with Kwangdong Pharmaceutical Co., Ltd., 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 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
(Tables to follow)
OCUGEN, INC.
CONSOLIDATED BALANCE SHEETS
(in thousands)
(Unaudited)
| As of December 31, | ||||||||
| 2025 | 2024 | |||||||
| Assets | ||||||||
| Current assets | ||||||||
| Cash | $ | 18,571 | $ | 58,514 | ||||
| Prepaid expenses and other current assets | 5,769 | 3,168 | ||||||
| Total current assets | 24,340 | 61,682 | ||||||
| Property and equipment, net | 14,392 | 16,554 | ||||||
| Restricted cash | 316 | 307 | ||||||
| Other assets | 4,468 | 3,899 | ||||||
| Total assets | $ | 43,516 | $ | 82,442 | ||||
| Liabilities and stockholders' equity | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 6,202 | $ | 4,243 | ||||
| Accrued expenses and other current liabilities | 14,733 | 15,500 | ||||||
| Operating lease obligations | 858 | 519 | ||||||
| Current portion of long term debt | 1,250 | 1,326 | ||||||
| Total current liabilities | 23,043 | 21,588 | ||||||
| Non-current liabilities | ||||||||
| Operating lease obligations, less current portion | 3,494 | 3,313 | ||||||
| Long term debt, net | 27,542 | 27,345 | ||||||
| Other non-current liabilities | 1,603 | 564 | ||||||
| Total non-current liabilities | 32,639 | 31,222 | ||||||
| Total liabilities | 55,682 | 52,810 | ||||||
| Stockholders' equity | ||||||||
| Convertible preferred stock | - | - | ||||||
| Common stock | 3,125 | 2,915 | ||||||
| Treasury stock | (48 | ) | (48 | ) | ||||
| Additional paid-in capital | 392,763 | 366,938 | ||||||
| Accumulated other comprehensive income | 61 | 48 | ||||||
| Accumulated deficit | (408,067 | ) | (340,221 | ) | ||||
| Total stockholders' equity | (12,166 | ) | 29,632 | |||||
| Total liabilities and stockholders' equity | $ | 43,516 | $ | 82,442 | ||||
OCUGEN, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
(Unaudited)
| Three months ended December 31, | Year ended December 31, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Collaborative arrangement revenue | $ | (193 | ) | $ | 764 | $ | 4,413 | $ | 4,055 | |||||||
| Total revenue | (193 | ) | 764 | 4,413 | 4,055 | |||||||||||
| Operating expenses | ||||||||||||||||
| Research and development | 10,670 | 8,290 | 39,750 | 32,126 | ||||||||||||
| General and administrative | 6,132 | 6,314 | 27,579 | 26,686 | ||||||||||||
| Total operating expenses | 16,802 | 14,604 | 67,329 | 58,812 | ||||||||||||
| Loss from operations | (16,995 | ) | (13,840 | ) | (62,916 | ) | (54,757 | ) | ||||||||
| Other income (expense) | ||||||||||||||||
| Interest income | 144 | 408 | 922 | 1,251 | ||||||||||||
| Interest expense | (1,331 | ) | (601 | ) | (5,188 | ) | (688 | ) | ||||||||
| Other income (expense) | 476 | 153 | (664 | ) | 140 | |||||||||||
| Other (expense) income, net | (711 | ) | (40 | ) | (4,930 | ) | 703 | |||||||||
| Net loss | $ | (17,706 | ) | $ | (13,880 | ) | $ | (67,846 | ) | $ | (54,054 | ) | ||||
| Net loss attributable to common shareholders— basic and diluted | (17,706 | ) | (13,880 | ) | (67,846 | ) | (54,054 | ) | ||||||||
| Weighted shares used in calculating net loss per common share — basic and diluted | 312,339,265 | 290,924,531 | 300,167,989 | 270,995,121 | ||||||||||||
| Net loss per share attributable to common shareholders — basic and diluted | $ | (0.06 | ) | $ | (0.05 | ) | $ | (0.23 | ) | $ | (0.20 | ) | ||||
| Net loss attributable to Series B Convertible Preferred shareholders — basic and diluted | - | - | - | (44 | ) | |||||||||||
| Weighted shares used in calculating net loss per Series B Convertible Preferred Stock — basic and diluted | - | - | - | 54,745 | ||||||||||||
| Net loss per share attributable to Series B Convertible Preferred shareholders — basic and diluted | $ | - | $ | - | $ | - | $ | (0.80 | ) | |||||||
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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FAQ
What financial results did Ocugen (OCGN) report for full year 2025?
Ocugen reported 2025 total revenue of $4,413 thousand and operating expenses of $67,329 thousand, resulting in a net loss of $67,846 thousand. This loss widened versus 2024 and, together with lower cash, contributed to total stockholders’ equity becoming negative by year-end.
How strong is Ocugen (OCGN)'s cash position and runway after recent financings?
As of December 31, 2025, Ocugen’s cash, cash equivalents and restricted cash were in the high teens in millions of dollars. A $22.5 million underwritten offering completed in January 2026, plus up to $30 million possible from warrant exercises, underpins projected runway into at least Q4 2026.
What progress has Ocugen (OCGN) made with its OCU400 retinitis pigmentosa program?
Ocugen has fully enrolled 140 patients in the Phase 3 liMeliGhT trial of OCU400 for retinitis pigmentosa, randomized 2:1 to treatment versus control. As a one-year study, topline data are planned for the first quarter of 2027 to support a Biologics License Application.
What did the Phase 2 data for OCU410 in geographic atrophy show?
Preliminary Phase 2 data for OCU410 in geographic atrophy showed a 46% lesion growth reduction at 12 months for medium and high doses combined versus control and a 50% responder rate. Treated eyes also demonstrated a 60% slower ellipsoid zone loss rate versus untreated fellow eyes.
What is Ocugen (OCGN)'s plan for future regulatory filings across its pipeline?
Ocugen is targeting three Biologics License Applications in three years. It plans a BLA for OCU410ST in Stargardt disease around 2027, a BLA for OCU400 in retinitis pigmentosa around 2027, and a BLA for OCU410 in geographic atrophy around 2028, subject to trial outcomes.
How did Ocugen’s operating expenses and losses change compared with 2024?
In 2025, research and development expense rose to $39,750 thousand from $32,126 thousand, while general and administrative expense was $27,579 thousand versus $26,686 thousand. These increases contributed to the net loss widening to $67,846 thousand from $54,054 thousand in 2024.
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