Ocugen, Inc.
For the period ended Jun 30, 2026. Filed Aug 6, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2026 · Filed Aug 6, 2026
OCGN$0.93+13.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2026-03-31; filed 2026-05-08.
0001628280-26-032454
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-06-30; filed 2026-08-06.
0001628280-26-054057
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 95 previous paragraphs and 111 current paragraphs.
43 unchanged; 25 reworded; 39 added; 21 removed; 6 previous and 4 current paragraphs rearranged.
Tables are not compared: 3 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (39)
Current report
tOverview
Current report
Additional Phase 1/2 data include:
Current report
In July 2026, we announced that the FDA granted RMAT designation to the Company’s investigational product OCU410 for the treatment of GA, secondary to dAMD.
Current report
In August 2026, we have received FDA clearance for the Phase 3 registrational trial (ArMaDa3) for GA secondary to dry age-related macular degeneration, anchored by positive 12-month Phase 2 ArMaDa data (statistically significant 31% reduction in GA lesion growth (patient population: lesion size ≥2.5 mm 2 and ≤17.5 mm 2) versus control, p<0.05, at the optimal dose planned for Phase 3). We plan to initiate the Phase 3 study late in the third quarter of 2026.
Current report
Research and development expense increased by $2.3 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025. The increase was primarily due to increases of $1.1 million related to personnel costs, $0.5 million related to OCU410ST clinical trial expenses, and $0.5 million related to shared research and development expenses.
Current report
General and administrative expense increased by $0.5 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025. The increase was primarily due to an increase of $0.8 million related to personnel costs and $0.5 million related to professional services. This is offset by a decrease of $0.4 million in commercial expenses, $0.2 million in corporate business fees, and $0.2 million in employee related expenses.
Current report
Interest expense, net increased by $3.2 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025. The increase was primarily due a $1.4 million increase related to the coupon interest on our debt and on the Convertible Senior Notes due 2034 ("Convertible Notes") issued in May due semi-annually and amortization of the issuance cost of $1.8. million.
Current report
Loss on extinguishment of debt
Current report
The loss on extinguishment of debt of $2.4 million for the three months ended June 30, 2026, was a result of the payoff of our Avenue Capital Loan.
Current report
Change in fair value of derivative liability
Current report
The change in fair value of the derivative liability for the three months ended June 30, 2026, resulted in a $1.9 million loss related to the derivative liability associated with the Convertible Notes.
Current report
Other (expense)/income, net
Current report
Other (expense)/income, net changed by $(0.2) million with other expense of $(0.1) million for the three months ended June 30, 2026 compared to other income of $0.1 million for the three months ended June 30, 2025, primarily due to exchange rate fluctuation.
Current report
Comparison of the Six Months Ended June 30, 2026 and 2025
Current report
Collaborative arrangement revenue
Current report
Research and development expense
Current report
Research and development expense increased by $4.0 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025. The increase was primarily due to an increase of $2.6 million related to personnel costs, and an increase of $1.7 million in OCU410ST clinical trial expenses.
Current report
General and administrative expense
Current report
General and administrative expense increased by $2.1 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025. The increase was primarily due to increases of $1.3 million in stock based compensation and $0.8 million in personnel related expenses.
Current report
Interest income
Current report
The decrease in interest income for the six months ended June 30, 2026 compared to the six months ended June 30, 2025 was minimal, driven by a lower average cash balance for the quarter ending March 31, 2026.
Current report
Interest expense
Current report
Interest expense, net increased by $3.3 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025. The increase was primarily due to a $1.4 million increase related to the coupon interest on our debt and on the Convertible Notes issued in May due semi-annually and amortization of the issuance cost of $1.9 million.
Current report
Loss on extinguishment of debt
Current report
The loss on extinguishment of debt of $2.4 million for the six months ended June 30, 2026, was a result of the payoff of our Avenue Capital Loan.
Current report
Change in fair value of derivative liability
Current report
The change in fair value of the derivative liability for the six months ended June 30, 2026, was a $1.9 million loss on the fair value of its derivative liability recorded in association with the Convertible Notes.
Current report
Other (expense)/income, net
Current report
Other (expense)/income, net changed by $(0.4) million with other expense of $(0.2) million for the six months ended June 30, 2026 compared to other income of $0.2 million for the six months ended June 30, 2025, primarily due to exchange rate fluctuation.
Current report
Cash provided by financing activities was $115.4 million for the six months ended June 30, 2026 compared to cash used in financing activities of $1.2 million for the six months ended June 30, 2025. During the six months ended June 30, 2026, cash provided by financing activities increased primarily due to the issuance of Convertible Notes in May 2026, proceeds from the equity offering in January 2026 and exercise of warrants, offset by the repayment of our Avenue Capital Loan and other cost related to the issuance of our Convertible Notes. During the six months ended June 30, 2025, cash used in financing activities primarily consisted of the repayment of EB-5 loans.
Current report
▪the timing and terms of the conversion of Convertible Notes under the Indenture;
Current report
We have incurred recurring operating losses, generated negative cash flows from operations, and expects to continue to incur significant expenditures to support the research, development, and potential commercialization of its product candidates. We have funded our operations to date through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds. We have incurred net losses of approximately $44.1 million and $30.1 million for the six months ended June 30, 2026 and 2025, respectively. We have incurred net losses of approximately $24.9 million and $14.7 million for the three months ended June 30, 2026 and 2025, respectively. As of June 30, 2026, we had an accumulated deficit of $452.1 million and cash and cash equivalents totaling $100.1 million.
Current report
In January 2026, we raised approximately $20.7 million in net proceeds through an underwritten registered direct offering of shares of common stock. In March 2026, investors partially exercised outstanding warrants, resulting in $14.2 million in net proceeds. In May 2026, we completed a private offering of $130.0 million aggregate principal amount of Convertible Notes, generating net proceeds of approximately $112.5 million after discounts, commissions, and offering expenses. We used approximately $32.7 million of the net proceeds from this offering to fully repay and terminate the Avenue Capital Loan, with the remaining proceeds available for general corporate purposes.
Current report
Management believes these financing transactions strengthened our financial position and, based on our current cash, cash equivalents, and anticipated operating plans, provide us with increased flexibility to fund our operations and strategic priorities. However, management’s going concern assessment requires consideration of all known and reasonably knowable conditions and events through one year from the issuance date of these condensed consolidated financial statements, including our recurring operating losses, expected negative cash flows from operations, future clinical and commercialization expenditures, and obligations and uncertainties related our financing arrangements, including potential settlement and share reservation considerations associated with the convertible notes.
Current report
After evaluating these conditions and management’s plans, we have concluded that there is substantial doubt about our ability to continue as a going concern within one year after the date these condensed consolidated financial statements are issued. Management continues to evaluate and pursue plans to mitigate these conditions, which may include raising additional capital, managing the timing and scope of operating expenditures, pursuing strategic partnerships or licensing arrangements, and other financing or corporate transactions. There can be no assurance that such plans will be successfully implemented, that additional financing will be available on acceptable terms, or at all, or that we will be able to execute our strategic plans within the required timeframe. If we are unable to obtain additional funding or otherwise successfully execute our plans when needed, we may be required to delay, reduce, or eliminate certain research and development programs and, commercialization activities, consider various strategic alternatives, including a merger or sale, or cease our operations.
Current report
Derivative Liability
Current report
During the quarter ended June 30,2026, the Company adopted an accounting policy for derivative liabilities in connection with the issuance of convertible notes containing embedded conversion options that require bifurcation and separate accounting as derivative instruments under ASC 815, Derivatives and Hedging. As this is the first time the Company has entered into transactions containing such features, the adoption of this accounting policy relates to a new type of transaction and does not represent a change in accounting principle.
Current report
The Company evaluates the terms of its convertible instruments at issuance and at each reporting date to determine whether embedded conversion features require bifurcation from the host debt instrument. When bifurcation is required, the embedded conversion option is initially recognized as a derivative liability at fair value on the issuance date. The debt host is initially recorded at the residual amount after allocating the proceeds to the derivative liability. The derivative liability is subsequently remeasured at fair value at each reporting date, with changes in fair value recognized in the condensed consolidated statements of operations. The debt discount resulting from the initial allocation to the derivative liability is amortized to interest expense over the contractual term of the convertible notes using the effective interest method.
Current report
The fair value of the derivative liability is determined using appropriate valuation techniques that incorporate observable market data, when available, and significant unobservable inputs, as necessary. Accordingly, the derivative liability is generally classified within Level 3 of the fair value hierarchy.
Removed paragraphs (21)
Previous report
Overview
Previous report
Additional data include:
Previous report
We expect to begin a rolling BLA submission in the third quarter of 2026 and plan to complete the BLA submission by the second quarter of 2027. We announced OCU400 Phase 3 liMeliGhT enrollment completion (N=140 subjects), reflecting strong interest from investigators and patients.
Previous report
In January 2026, we announced publication of Phase 1 GARDian1 Trial results for OCU410ST. The study supports favorable safety and tolerability and clinically meaningful functional and structural benefits in patients with ABCA4-associated retinopathies including Stargardt disease.
Previous report
•Subgroup analysis (subjects with baseline GA lesions ≥5 mm 2 and ≤17.5 mm 2) demonstrates 33% reduction in lesion growth compared to control in medium dose OCU410 with similar reductions in the high dose group
Previous report
We expect to meet with FDA/EMA to align on the Phase 3 study design and initiate the Phase 3 study by the third quarter of 2026.
Previous report
Novel Modifier Gene Therapy Platform
Previous report
In February 2025, we announced that alignment has been reached with the FDA to move forward with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST which can be the basis of a BLA submission. The GARDian Phase 2/3 clinical trial randomized 63 subjects. The primary endpoint in the clinical trial is change in atrophic lesion size. Secondary endpoints include visual acuity as measured by best corrected visual acuity and LLVA compared to untreated controls. One-year data will be utilized for the BLA filing. The Phase 2/3 pivotal confirmatory trial has adaptive design with sample size re-estimation. OCU410ST is intended for early to advanced cases of ST. The masked interim analysis for the OCU410ST Phase 2/3 GARD ian3 trial in Stargardt disease is on track as planned for mid-2026 for 24 subjects (16 treated, 8 controls) who have completed 8 months in the trial.
Previous report
The latest data from the OCU410ST Phase 1 clinical trial demonstrates that atrophic lesions grew slower by 54% at 12 months for evaluable treated subjects when compared to untreated fellow eyes. In the secondary endpoint- Best Corrected Visual Acuity (BCVA), treated eyes showed an improvement with 1-line (6ETDRS Letter) gain in the visual acuity when compared to untreated fellow eyes. Additionally, 100% of evaluable treated eyes demonstrated stabilization or improvement vs. untreated eyes in visual function.").
Previous report
In January 2026, the Company announced publication of Phase 1 GARDian1 Trial results for OCU410ST. 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.
Previous report
Inhaled Mucosal Vaccine Platform
Previous report
We are party to the WU License Agreement with Washington University, pursuant to which we licensed the rights to develop, manufacture, and commercialize a mucosal COVID-19 vaccine for the prevention of COVID-19 in the Mucosal Vaccine Territory. In addition, we internally developed technology related to the flu and COVID-19's vaccine design and filed intellectual property. We are developing a next-generation, inhalation-based mucosal vaccine platform based on a novel ChAd vector, which includes OCU500, a COVID-19 vaccine. NIAID intends to initiate a Phase 1 clinical trial for OCU500 in the second quarter of 2026.
Previous report
Research and development expense increased by $1.7 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. The increase was primarily due to $1.5 million in employee-related expenses due to an increase in the number of employees and $1.4 million related to OCU410/410ST clinical activities for confirmatory Phase 2/3 clinical trial. This is offset by decrease in $(0.5) million related to OCU400, which is due to reduced manufacturing service fee, and a decrease in ($0.4) million related to consulting expense as compared to the quarter ended March 31, 2025.
Previous report
General and administrative expense increased by $1.7 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. This increase was primarily due to the increase in the number of employees and related personnel costs.
Previous report
Interest expense, net increased by $0.1 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. The increase was primarily due to an increase in interest expense related to the Loan and Security Agreement which was entered in November 2024.
Previous report
Venture Opportunities Fund II, L.P. and Avenue Venture Opportunities Fund, L.P. as lenders and Avenue Capital Management II, L.P. as administrative agent and collateral agent (the “Avenue Loan Agreement”), including payment of the related prepayment fee and expenses, and terminate the Avenue Loan Agreement and all related loan documents. See Note 16—Subsequent Events in the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q for additional details regarding the issuance of the notes.
Previous report
Cash provided by financing activities was $35.0 million for the three months ended March 31, 2026 compared to cash used in financing activities of $1.2 million for the three months ended March 31, 2025. In January 2026, the Company completed an underwritten registered direct offering of 15.0 million shares of common stock at an offering price of $1.50 per share of common stock, resulting in gross proceeds of $22.5 million. The Company received net proceeds of approximately $20.7 million, after deducting commissions and other offering expenses. During the three months ended March 31, 2026, the holder exercised warrants to purchase 10 million shares of common stock for aggregate net proceeds of approximately $14.2 million. The warrants were exercised at an exercise price of $1.50 per share.
Previous report
During the three months ended March 31, 2025, cash used by financing activities was $1.2 million primarily consisted of a payment related to the EB-5 Loan Agreement.
Previous report
As of March 31, 2026, we had cash and cash equivalents of approximately $31.9 million. This amount will not meet our capital requirements over the next 12 months. Due to the inherent uncertainty involved in making estimates and the risks associated with the research, development, and commercialization of biotechnology products, this estimate may be based on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us.
Previous report
We are subject to risks and uncertainties frequently encountered by companies in the biotech industry, and while we intend to continue research, development, and commercialization efforts for our product candidates, we will require significant additional funding. If we are unable to obtain additional funding in the future and/or our research, development, and commercialization efforts require higher than anticipated capital, there will be a negative impact on our financial viability. We will continue to explore options to fund our operations through public and private placements of equity and/or debt, payments from potential strategic research and development arrangements, sales of assets, licensing and/or collaboration arrangements with pharmaceutical companies or other institutions, funding from the government, particularly for the development of our novel inhaled mucosal vaccine platform, or funding from other third parties. Such financing and funding may not be available at all, or on terms that are favorable to us. While management believes that we have a plan to fund operations, our plan may not be successfully implemented. If we cannot obtain the necessary funding, we will need to delay, scale back, or eliminate some or all of our research and development programs and commercialization efforts; consider other various strategic alternatives, including a merger or sale; or cease operations. If we cannot expand our operations or otherwise capitalize on our business opportunities because we lack sufficient capital, our business, financial condition, and results of operations could be materially adversely affected.
Previous report
As a result of these factors, together with the anticipated continued spending that will be necessary to continue to research, develop, and commercialize our product candidates, there is substantial doubt about our ability to continue as a going concern within one year after the date that the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q are issued.
Reworded paragraphs (25)
Previous wording
You should read the following discussion and analysis of our financial condition and results of operations in conjunction with the financial statements and the notes thereto included elsewhere in this Quarterly Report on Form 10-Q and with our audited financial statements for the year ended December 31, 2025, included in our 2025 Annual Report. Some of the information contained in this discussion and analysis, including information with respect to our plans and strategy for our business and related financing, include forward-looking statements that involve risks, uncertainties, and assumptions. These statements are based on our beliefs and expectations about future outcomes and are subject to risks and uncertainties that could cause our actual results to differ materially from anticipated results. Except as required by law, we undertake no obligation to publicly update these forward-looking statements, whether as a result of new information, future events, or otherwise. You should read the "Risk Factors" section included in our 2025 Annual Report and the "Risk Factors" and "Disclosure Regarding Forward-Looking Statements" sections of this Quarterly Report on Form 10-Q for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.
Current wording
You should read the following discussion and analysis of our financial condition and results of operations in conjunction with the financial statements and the notes thereto included elsewhere in this Quarterly Report on Form 10-Q and with our audited financial statements for the year ended December 31, 2025, included in our 2025 Annual Report. Some of the information contained in this discussion and analysis, including information with respect to our plans and strategy for our business and related financing, include forward-looking statements that involve risks, uncertainties, and assumptions. These statements are based on our beliefs and expectations about future outcomes and are subject to risks and uncertainties that could cause our actual results to differ materially from anticipated results. Except as required by law, we undertake no obligation to publicly update these forward-looking statements, whether as a result of new information, future events, or otherwise. You should read the "Risk Factors" section included in our 2025 Annual Report and First Quarter 10-Q and "Disclosure Regarding Forward-Looking Statements" section of this Quarterly Report on Form 10-Q for a discussion of important factors that could cause actual results to differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.
Previous wording
We are developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, ST; and multifactorial diseases such as dAMD. Our modifier gene therapy platform is based on the use of NHRs, which have the potential to achieve homeostasis — the basic biological processes in the retina to restore a healthy state from a diseased state. Unlike single gene replacement therapies, which only target one genetic mutation, our modifier gene therapy platform, through its use of NHRs, represents a unique, gene-agnostic approach designed to address not just the mutated gene but provide a molecular "reset" of health and survival of gene networks. OCU400, our lead product candidate in our modifier gene therapy platform, has received ODD from the FDA for RP and LCA, a RMAT designation for the treatment of RP associated with NR2E3 and RHO mutations from the FDA, and OMPD from the EC, based on the recommendation of the EMA, for RP and LCA. These broad ODD, RMAT, and OMPD designations further support the broad (gene-agnostic) therapeutic potential of OCU400 to treat RP associated with mutations in multiple genes.
Current wording
We are developing a modifier gene therapy platform designed to fulfill unmet medical needs related to retinal diseases, including inherited retinal degenerations ("IRDs"), such as retinitis pigmentosa ("RP"), Stargardt disease ("ST"), and multifactorial diseases such as dry age-related macular degeneration ("dAMD"). Our modifier gene therapy platform is based on the use of nuclear hormone receptors ("NHRs"), which have the potential to achieve homeostasis — the basic biological processes in the retina to restore a healthy state from a diseased state. Unlike single gene replacement therapies, which only target one genetic mutation, our modifier gene therapy platform, through its use of NHRs, represents a gene-agnostic approach designed to address not just the mutated gene but provide a molecular "reset" of health and survival of gene networks.
Previous wording
OCU400- Based on the use of nuclear hormone receptors ("NHRs"), we believe our novel modifier gene therapy platform has the potential to address major blindness diseases, including rare genetic diseases such as RP (OCU400), with a gene-agnostic approach. OCU400 is intended for early to advanced cases of RP including clinical and/or genetic diagnosis with both syndromic and non-syndromic forms of the disease. In January 2025, we announced positive two-year data for multiple mutations from the Phase 1/2 clinical trial for OCU400. In February 2025, we announced that the European Commission ("EC") has provided a positive opinion from the European Medicines Agency's ("EMA") Committee for Advanced Therapies for OCU400 Advanced Therapy Medicinal Product ("ATMP") classification. We have completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400. Positive long-term, 3-year Phase 1/2 data for OCU400 were recently assessed in evaluable subjects and builds on prior 2-year results showing consistent clinically meaningful, approximately 2-line LLVA gain across mutations. OCU400 maintained a favorable durability, safety and tolerability profile with no new treatment-related serious adverse events or adverse events of interest emerged.
Current wording
OCU400- our first modifier gene therapy product is based on the use of NHRs, and we believe our novel modifier gene therapy platform has the potential to address major blindness diseases, including rare genetic diseases such as RP, with a gene-agnostic approach. OCU400 is intended for early to advanced cases of RP including clinical and/or genetic diagnosis with both syndromic and non-syndromic forms of the disease. In January 2025, we announced positive two-year data for multiple mutations from the Phase 1/2 clinical trial for OCU400. In February 2025, we announced that the European Commission ("EC") has provided a positive opinion from the European Medicines Agency's ("EMA") Committee for Advanced Therapies for OCU400 Advanced Therapy Medicinal Product ("ATMP") classification. Positive long-term, 3-year Phase 1/2 data for OCU400 were assessed in evaluable subjects and built on prior 2-year results showing consistent clinically meaningful, approximately 2-line low luminance visual acuity ("LLVA") gain across mutations. OCU400 maintained a favorable durability, safety and tolerability profile with no new treatment-related serious adverse events or adverse events of interest emerged.
Previous wording
◦Visual function benefits were consistently observed over 3 years, with 88% (7/8) of evaluable treated subjects showing improvement or preservation versus untreated fellow eyes.
Current wording
▪Visual function benefits were consistently observed over 3 years, with 88% (7/8) of evaluable treated subjects showing improvement or preservation versus untreated fellow eyes; and,
Previous wording
Topline Phase 3 data is expected in the first quarter of 2027, advancing OCU400 towards potential approval in the fourth quarter of 2027 as a treatment option for early- to late-stage RP.
Current wording
We have completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400 (N=140 subjects), reflecting strong interest from investigators and patients. Topline Phase 3 data is expected in the first quarter of 2027, advancing OCU400 towards potential approval in the fourth quarter of 2027 as a treatment option for early- to late stage RP. The U.S. Food and Drug Administration ("FDA") acknowledged the regulatory eligibility to rolling BLA submission by virtue of our RMAT designation for OCU400, but, under the FDA’s general policy, we would need to submit topline Phase 3 data in a pre-BLA meeting prior to the FDA agreeing to a rolling submission schedule.
Previous wording
OCU410 and OCU410ST are being developed utilizing the RORA (RAR Related Orphan Receptor A) gene for the treatment of GA secondary to dAMD and ST, respectively. OCU410 is a potential one-time, curative therapy with a single sub-retinal injection that targets multiple pathways associated with AMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA, require multiple injections per year, and have safety considerations. OCU410ST has received ODD from the FDA and OMPD from the EMA for the treatment of ABCA4-associated retinopathies (>1200 mutations) including ST, RP19, and cone-rod dystrophy 3 (CORD3), and has the potential to be the first approved therapy to treat ST.
Current wording
OCU410ST/OCU410 utilizes a first-in-class modifier gene therapy approach by delivering the human RORA (Retinoic Acid Receptor ("RAR") Related Orphan Receptor A) gene to diseased retinal tissue via subretinal AAV5 delivery. RORA modulates lipid metabolism, oxidative stress, and inflammation key drivers of retinal degeneration that restores retinal homeostasis by offering a unique four-way disease-modifying potential. OCU410 is a potential one-time therapy with a single sub-retinal injection that targets multiple pathways associated with AMD pathogenesis, in contrast to products currently approved or under development that treat only one cause of GA that require multiple injections per year, and some safety limitations. Currently, there is significant economic burden of vision loss diseases in the United States. ST and GA are major contributors to vision loss. We believe OCU410 has the potential to reduce treatment costs, prevent vision-related disability, and ease the broader healthcare and societal burden driven by structural and functional vision loss. OCU410ST has received OMPD from the EMA for the treatment of ABCA4-associated retinopathies (>1200 mutations) including ST, retinitis pigmentosa 19 ("RP19"), and cone-rod dystrophy 3 ("CORD3"), and has the potential to be the first approved therapy to treat ST.
Previous wording
OCU410ST- We initiated dosing in GARDian3 pivotal confirmatory trial for OCU410ST in July 2025. The OCU410ST Phase 2/3 pivotal confirmatory trial represents our second late-stage clinical program. We plan to submit a BLA for OCU410ST by mid-2027 in alignment with our strategic goal of filing three BLAs by 2028. In November 2024, the EMA granted orphan medicinal product designation ("OMPD") for OCU410ST for the treatment of ABCA4-associated retinopathies (>1200 mutations) including ST, RP 19, and CORD3. In May 2025, we announced that the FDA granted Rare Pediatric Disease Designation ("RPDD") for OCU410ST for the treatment of ABCA4-associated retinopathies including ST, retinitis pigmentosa 19 ("RP19"), and cone-rod dystrophy 3 ("CORD3"). In June 2025, we announced that the FDA has cleared the Investigational New Drug ("IND") amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all ST
Current wording
OCU410ST- We initiated dosing in GARDian3 pivotal confirmatory trial for OCU410ST in July 2025. The OCU410ST Phase 2/3 pivotal confirmatory trial represents our second late-stage clinical program. We plan to submit a BLA for OCU410ST by mid-2027 in alignment with our strategic goal of filing three BLAs by 2028. OCU410ST has received ODD from the FDA. In November 2024, the EMA granted OMPD for OCU410ST for the treatment of ABCA4-associated retinopathies (>1200 mutations) including ST, RP 19, and CORD3. In May 2025, we announced that the FDA granted Rare Pediatric Disease Designation ("RPDD") for OCU410ST for the treatment of ABCA4-associated retinopathies including ST, RP19, and CORD3. In June 2025, we announced that the FDA has cleared the Investigational New Drug ("IND") amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all ST (ABCA4-associated retinopathies). In August 2025, we announced that the Committee for Medicinal Products for Human Use ("CHMP") of the EMA reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for ST and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application ("MAA").
Previous wording
The OCU410ST Phase 1 clinical trial demonstrated that atrophic lesions grew slower by 54% at 12 months for evaluable treated subjects when compared to untreated fellow eyes. In the secondary endpoint- Best Corrected Visual Acuity (BCVA), treated-eyes showed an improvement with 1-line (6 ETDRS Letter) gain in the visual acuity when compared to untreated fellow eyes. Additionally, 100% of evaluable treated eyes demonstrated stabilization or improvement vs. untreated eyes in visual function. In evaluable subjects (N=6) the rate of ellipsoid zone (EZ) loss was 116% slower in OCU410ST-treated eyes compared to untreated fellow eyes at 12 months, demonstrating preservation or stabilization in photoreceptor integrity. The untreated eyes showed expected decline in atrophy. In April 2026, we announced early completion of dosing in the Phase 2/3 pivotal confirmatory trial (N=63 subjects). GARDian 3 trial enrollment and dosing completed successfully in less than 9 months. We plan to submit the BLA for OCU410ST by mid-2027.
Current wording
The OCU410ST Phase 1 clinical trial demonstrated that atrophic lesions grew slower by 54% at 12 months for evaluable treated eyes compared to untreated fellow eyes. In the secondary endpoint- Best Corrected Visual Acuity ("BCVA" ), treated-eyes showed an improvement with 1-line (6 ETDRS Letter) gain in the visual acuity when compared to untreated fellow eyes. Additionally, 100% of evaluable treated eyes demonstrated stabilization or improvement vs. untreated eyes in visual function. In evaluable subjects (N=6) the rate of ellipsoid zone ("EZ") loss was 116% slower in OCU410ST-treated eyes compared to untreated fellow eyes at 12 months, demonstrating preservation or stabilization in photoreceptor integrity. The untreated eyes showed expected decline in atrophy. In April 2026, we announced early completion of dosing in the Phase 2/3 pivotal confirmatory trial (N=63 subjects). GARDian 3 trial enrollment and dosing completed successfully in less than 9 months. We plan to submit the BLA for OCU410ST by mid-2027.
Previous wording
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.
Current wording
OCU410- In Phase 1 study, no OCU410-related serious adverse events were observed and no cases of endophthalmitis, retinal detachment, vasculitis, choroidal neovascularization, or ischemic optic neuropathy were reported to date. The Phase 2 clinical trial was built directly on the clean safety profile observed for OCU410 in the Phase 1 study.
Previous wording
OCU410- We completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for OCU410 for the treatment of geographic atrophy ("GA"), an advanced form of dAMD. Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events ("SAEs"), reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity ("LLVA"). In March 2025, OCU410 and OCU410ST received ATMP classification from the EMA.
Current wording
We completed dosing in Phase 2 of the Phase 1/2 ArMaDa clinical trial for OCU410 for the treatment of geographic atrophy ("GA"), an advanced form of dAMD. Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the OCU410 Phase 1/2 ArMaDa clinical trial included: no drug-related serious adverse events ("SAEs"), reduced lesion growth, preservation of retinal tissue. In March 2025, OCU410 and OCU410ST received ATMP classification from the EMA.
Previous wording
•27% slower rate of ellipsoid zone (EZ) loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function
Current wording
▪ 27% slower rate of EZ loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function; and
Previous wording
Inhaled Mucosal Vaccine Platform— Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine. We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. The National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health ("NIH"), intends to initiate a Phase 1 clinical trial for OCU500 in the second quarter of 2026.
Current wording
Inhaled Mucosal Vaccine Platform— Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine. We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. The Company is collaborating with the National Institute of Allergy and Infectious Diseases ("NIAID"), part of the National Institutes of Health, for the Phase 1 clinical trial for OCU500. NIAID is responsible for conducting and funding these studies, including contracting with clinical sites and service providers. The Company supplies investigational product for use in the studies and incurs only those costs for which it is directly responsible. Accordingly, costs funded directly by NIAID are not included in the Company's research and development expenses. NIAID initiated Phase 1 clinical trial for OCU500 in the second quarter of 2026.
Previous wording
We believe the following table provides more transparency as to the type of research and development expenses incurred. The following table summarizes our research and development expenses by product candidate for the three months ended March 31, 2026 and 2025 (in thousands):
Current wording
The following table summarizes our research and development expenses by product candidate for the three months ended June 30, 2026 and 2025 (in thousands):
Previous wording
Collaborative arrangement revenue increased by $0.1 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. The change was due to our quarterly reassessment of the amount of co-development services provided by us to the business partner in the collaboration agreement.
Current wording
Collaborative arrangement revenue increased by $0.1 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025. The increase was due to our quarterly reassessment of the amount of co-development services provided by us to the business partner in the collaboration agreement.
Previous wording
Interest income decreased by $0.2 million for the three months ended March 31, 2026 compared to the three months ended March 31, 2025. The decrease was primarily due to a lower average cash balance for the quarter ending March 31, 2026.
Current wording
Interest income increased by $0.2 million for the three months ended June 30, 2026 compared to the three months ended June 30, 2025. The increase was primarily due to a higher average cash balance for the three months ended June 30, 2026.
Previous wording
As of March 31, 2026, we had $31.9 million in cash and cash equivalents. We have not generated revenue from our product candidates to date, and have primarily funded our operations through the sale of common stock, warrants to purchase common stock, the issuance of convertible notes and debt, and grant proceeds. Since our inception and through March 31, 2026, we have raised an aggregate of $427.8 million to fund our operations, of which $383.1 million was from gross proceeds from the sale of our common stock and warrants, $10.3 million was from the issuance of convertible notes, $33.4 million was from the issuance of debt, $0.8 million was from the royalty agreement and $0.2 million was from grant proceeds.
Current wording
As of June 30, 2026, we had approximately $100.1 million of cash and cash equivalents, along with $0.3 million of restricted cash. To date, we have not generated revenue from our product candidates. Our operations have been financed primarily through the sale of common stock, warrants, convertible notes and other debt instruments, as well as grant funding. Since our inception and through June 30, 2026, we have raised an aggregate $558.4 million to fund our operations, of which $383.7 million was from gross proceeds from the sale of our common stock and warrants, $140.3 million was from the issuance of convertible notes, $33.4 million was from the issuance of indebtedness, $0.8 million was from the royalty agreement and $0.2 million was from grant proceeds.
Previous wording
Since our inception, we have devoted substantial resources to research and development and have incurred significant net losses and may continue to incur net losses in the future. We incurred net losses of approximately $19.2 million and $15.4 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had an accumulated deficit of $427.2 million. In addition, we had accounts payable and accrued expenses and other current liabilities of $14.9 million and indebtedness of $29.2 million.
Current wording
Since our inception, we have devoted substantial resources to research and development and have incurred significant net losses and may continue to incur net losses in the future. We incurred net losses of approximately $(44.1) million and $(30.1) million for the six months ended June 30, 2026 and 2025, respectively. As of June 30, 2026, we had an accumulated deficit of $452.1 million. In addition, we had accounts payable and accrued expenses and other current liabilities of $16.2 million and indebtedness of $116.6 million.
Previous wording
The following table provides a summary of our cash flows for the three months ended March 31, 2026 and 2025 (in thousands):
Current wording
The following table provides a summary of our cash flows for the six months ended June 30, 2026 and 2025 (in thousands):
Previous wording
On May 7, 2026, we completed our previously announced private offering of $115.0 million aggregate principal amount of 6.75% Convertible Senior Notes due 2034 (the “notes”). The notes are general unsecured obligations and bear interest at a rate of 6.75% per year, payable semi-annually in arrears on May 15 and November 15 of each year, beginning on November 15, 2026. The notes mature on May 15, 2034, unless earlier repurchased, redeemed or converted. We used a portion of the proceeds to fully repay our obligations underthe Loan and Security Agreement that the Company is party to with Avenue
Current wording
In May 2026, we completed a private offering of $130.0 million aggregate principal amount of Convertible Notes, generating net proceeds of approximately $112.5 million after discounts, commissions, and offering expenses. We used approximately $32.7 million of the net proceeds from this offering, to fully repay and terminate our Avenue Capital Loan with the remaining proceeds available for general corporate purposes. The Convertible Notes are general unsecured obligations and bear interest at a rate of 6.75% per year payable semi-annually in arrears on May 15 and November 15 of each year, beginning on November 15, 2026. The Convertible Notes mature on May 15, 2034, unless earlier repurchased, redeemed or converted.
Previous wording
Cash used in operating activities was $21.8 million for the three months ended March 31, 2026, and primarily consisted of a net loss of $19.2 million adjusted for non-cash items including stock-based compensation of $3.4 million, depreciation and amortization of $1.0 million, non-cash lease expense of $0.3 million, other non-cash items of $(0.7) million, and a change in net working capital of $6.6 million.
Current wording
Cash used in operating activities was $34.0 million for the six months ended June 30, 2026, and primarily consisted of a net loss of $44.1 million adjusted for non-cash items including amortization of debt issuance cost of $0.6 million, stock-based compensation of $5.2 million, depreciation and amortization of $1.2 million, non-cash lease expense of $0.6 million, non-cash expense from collaborative arrangements, net of $1.7 million, change in fair value of derivative liability $1.9 million, and loss on extinguishment of debt $2.4 million related to repayment of the Avenue Capital Loan.
Previous wording
Cash used in operating activities was $19.4 million for the three months ended March 31, 2025, and primarily consisted of a net loss of $15.4 million adjusted for non-cash items including stock-based compensation of $1.9 million, depreciation and amortization of $0.9 million, non-cash lease expense of $0.3 million, other non-cash items of $0.6 million, and a change in net working capital of $6.5 million.
Current wording
Cash used in operating activities was $30.1 million for the six months ended June 30, 2025, and primarily consisted of a net loss of $30.1 million adjusted for non-cash items including stock-based compensation of $3.7 million, non-cash expense from collaborative arrangements, net of $1.8 million, non-cash lease expense of $0.6 million, and depreciation and amortization of $1.8 million.
Previous wording
Cash used by investing activities was immaterial for the three months ended March 31, 2026, and primarily consisted of payments related to the purchases of property and equipment. Cash used by investing activities was $0.1 million for the three months ended March 31, 2025, and primarily consisted of payments related to the purchases of property and equipment.
Current wording
Cash used in investing activities was $(0.03) million for the six months ended June 30, 2026, and primarily consisted of purchase of fixed assets. Cash used in investing activities was $(0.2) million for the six months ended June 30, 2025, were which primarily consisted of payments related to security deposits and the purchases of property and equipment.
Previous wording
We have commitments under certain licensing and development agreements, lease obligations, debt agreements, and consulting agreements. There have been no material changes to our contractual obligations as reported in our 2025 Annual Report.
Current wording
We have commitments under certain licensing and development agreements, lease obligations, debt agreements, and consulting agreements. Except in connection with the issuance of Convertible Notes pursuant to the Indenture in May 2026, as described in this Quarterly Report on Form 10-Q, there have been no material changes to our contractual obligations as reported in our 2025 Annual Report.
Previous wording
•the preparation and submission of INDs with the FDA for current and future product candidates;
Current wording
▪the preparation and submission of Investigational New Drug applications, or INDs, with the FDA for current and future product candidates;
Previous wording
The preparation of financial statements in conformity with GAAP requires us to make judgments, estimates, and assumptions in the preparation of our condensed consolidated financial statements. Actual results could differ from those estimates. There have been no material changes to our critical accounting policies and estimates as reported in our 2025 Annual Report.
Current wording
The preparation of financial statements in conformity with GAAP requires us to make judgments, estimates, and assumptions in the preparation of our condensed consolidated financial statements. Actual results could differ from those estimates. There have been no material changes to our critical accounting policies and estimates as reported in our 2025 Annual Report except as noted below:
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
OCU400 has received Orphan Drug Designations ("ODD") from the FDA for RP and Leber congenital amaurosis ("LCA"), a Regenerative Medicine Advanced Therapy ("RMAT") designation for the treatment of RP associated with NR2E3 and RHO mutations from the FDA, and orphan medicinal product designation ("OMPD") from the European Commission ("EC"), based on the recommendation of the European Medicines Agency ("EMA"), for RP and LCA. These broad ODD, RMAT, and OMPD designations further support the broad (gene-agnostic) therapeutic potential of OCU400 to treat RP associated with mutations in multiple genes. We have successfully completed process performance qualification ("PPQ") batches in preparation for potential approval in the fourth quarter of 2027 as a treatment option for early- to late-stage RP.
Current report — wording carried from elsewhere
The following table summarizes the results of our operations for the six months ended June 30, 2026 and 2025 (in thousands):
Current report — wording carried from elsewhere
The following table summarizes our research and development expenses by product candidate for the six months ended June 30, 2026 and 2025 (in thousands):
Current report — wording carried from elsewhere
Collaborative arrangement revenue increased by $0.2 million for the six months ended June 30, 2026 compared to the six months ended June 30, 2025. The increase was due to our quarterly reassessment of the amount of co-development services provided by us to the business partner in the collaboration agreement.
Rearranged previous paragraphs (6)
Previous report — wording carried elsewhere
(ABCA4-associated retinopathies). In August 2025, we announced that the Committee for Medicinal Products for Human Use ("CHMP") of the EMA reviewed the study design, endpoints and planned statistical analysis of the ongoing pivotal confirmatory OCU410ST Phase 2/3 GARDian3 clinical trial for ST and provided acceptability of a single U.S.-based trial for submission of a Marketing Authorization Application ("MAA").
Previous report — wording carried elsewhere
OCU410ST/OCU410 utilizes a first-in-class modifier gene therapy approach by delivering the human RORA gene to diseased retinal tissue via subretinal AAV5 delivery. RORA modulates lipid metabolism, oxidative stress, and inflammation key drivers of retinal degeneration that restores retinal homeostasis by offering a unique four-way disease-modifying potential.
Previous report — wording carried elsewhere
Currently, there is significant economic burden of vision loss diseases in the US. ST and GA are major contributors to vision loss. OCU410 has the potential to reduce treatment costs, prevent vision-related disability, and ease the broader healthcare and societal burden driven by structural and functional vision loss.
Previous report — wording carried elsewhere
The OCU410ST Phase 1 clinical trial demonstrated that atrophic lesions grew slower by 54% at 12 months for evaluable treated subjects when compared to untreated fellow eyes. In the secondary endpoint, Best Corrected Visual Acuity (BCVA), treated eyes showed an improvement with 1-line (6 ETDRS Letter) gain in the visual acuity when compared to untreated fellow eyes. Additionally, 100% of evaluable treated eyes demonstrated stabilization or improvement vs. untreated eyes in visual function. In evaluable subjects (N=6) ellipsoid zone (EZ) loss rate was 116% slower in OCU410ST-treated eyes compared to untreated fellow eyes at 12 months, demonstrating preservation or stabilization in photoreceptor integrity. The untreated eyes showed expected decline in atrophy. In April 2026, the Company announced early completion of dosing in phase 2/3 pivotal confirmatory trial (N=63 subjects). GARDian 3 trial enrollment and dosing completed successfully in less than 9 months. We plan to submit the BLA for OCU410ST by mid-2027.
Previous report — wording carried elsewhere
Novel Biologic Therapy for Retinal Diseases
Previous report — wording carried elsewhere
OCU200 is a novel recombinant fusion protein consisting of two human proteins, tumstatin and transferrin. OCU200 possesses unique features which potentially enable it to treat vascular complications of diabetic macular edema ("DME"), diabetic retinopathy ("DR"), and wet age-related macular degeneration ("AMD"). Tumstatin is the active component of OCU200 and binds to integrin receptors,which play a crucial role in disease pathogenesis. Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors. OCU200 Phase 1 clinical trial enrollment was completed in the first quarter of 2026.