Ocugen, Inc.
For the period ended Sep 30, 2026. Filed Nov 4, 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.
Sep 30, 2026 · Filed Nov 4, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious 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 OperationsCurrent report: 10-Q
Period ended 2026-09-30; filed 2026-11-04.
0001628280-26-069835
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 111 previous paragraphs and 110 current paragraphs.
52 unchanged; 27 reworded; 30 added; 28 removed; 4 previous and 1 current paragraphs rearranged.
Tables are not compared: 5 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 (30)
Current report
Overview
Current report
We are a pioneering biotechnology company focused on developing and then commercializing novel gene therapies to address blindness diseases with high unmet medical needs globally.
Current report
Ocugen’s modifier gene therapy platform underpins all of our late-stage clinical development programs and has the potential to address significant unmet medical needs across large global patient populations through a gene-agnostic approach. Unlike traditional gene therapies and gene-editing technologies that target a single gene mutation, our modifier gene therapy platform is designed to address the underlying disease biology and potentially restore balance across multiple gene networks – potentially enabling us to address large, underserved patient populations rather than narrow single-gene subsets.
Current report
We are currently advancing late-stage clinical development programs for inherited retinal diseases and other causes of blindness that we believe affect millions worldwide, including retinitis pigmentosa (“RP”), Stargardt disease, and geographic atrophy (“GA”), an advanced form of dry age-related macular degeneration (“AMD”).
Current report
Our pipeline:
Current report
OCU400
Current report
Our long-term, 3-year Phase 1/2 data for OCU400 were assessed in evaluable subjects and built on prior 2-year results suggesting consistent and clinically meaningful results – approximately 2-line low luminance visual acuity ("LLVA") improvement in treated eyes compared with untreated fellow eyes in 8 evaluable subjects across multiple genotypes. Additionally, OCU400 appears to have a favorable durability, safety and tolerability profile with no reported treatment-related serious adverse events or adverse events of interest. To date, we have completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400 (N=140 subjects), reflecting strong interest from investigators and patients; and we believe we have gained alignment with the U.S. Food and Drug Administration (“FDA”) on the study design, including primary and secondary endpoints. Topline Phase 3 data is expected in the first quarter of 2027, with plans to submit a biologics license application ("BLA") shortly after.
Current report
In September 2026, OCU400 was granted provisional approval and priority designation from the Longevity and Regenerative Therapies Act (“LARTA”) Board, the regulatory agency responsible for reviewing and approving longevity and regenerative therapy programs within the Commonwealth of The Bahamas. In connection with LARTA’s approval, Ocugen will supply
Current report
OCU400 through an expanded access program (“EAP”), with the goal of treating the first RP patient within 90 days, following full LARTA approval.
Current report
OCU410ST
Current report
OCU410ST is an investigational product being developed for the treatment of Stargardt disease, a rare inherited retinal disease (“IRD”) associated with 1,200+ mutations of the ABCA4 gene. OCU410ST delivers the RORA(Retinoic Acid Receptor ("RAR") Related Orphan Receptor A)gene and is designed to act independently of the specific ABCA4 mutation.
Current report
We completed enrollment in the Phase 2/3 pivotal confirmatory trial (N=63 subjects) early, in just nine months, and expect topline Phase 3 results in the second quarter of 2027, followed by a BLA submission in mid-2027.
Current report
As previously disclosed in our Current Report on Form 8-K filed with the SEC on September 8, 2026, after completing a pre-specified interim analysis of atrophic lesion size in the Phase 2/3 clinical trial of OCU410ST in Stargardt disease, the independent Data Monitoring Committee (“DMC”) recommended continuing the study according to the protocol except with a modification to obtain 8 months of follow up on lesion size in the entire study population. We are currently planning to continue the study to month 12 to analyze full data sets from all enrolled subjects.
Current report
OCU410
Current report
OCU410 is an investigational product being developed for the treatment of GA, secondary to dry AMD, a leading cause of severe central vision loss and legal blindness worldwide.
Current report
OCU410, which has been granted RMAT designation by the FDA, and ATMP classification by the EMA, initiated its global Phase 3 trial, ArMaDa3, and dosed the first subject in the third quarter of 2026. The initiation of dosing followed the successful completion of a Type B End-of-Phase 2 (“EOP2”) meeting with the Center for Biologics Evaluation and Research (“CBER”) of the FDA in June 2026, resulting in alignment on Phase 3 design elements, including primary and secondary endpoints, dose, adaptive design, and a single pivotal trial pathway to potentially support a BLA submission.
Current report
Additional Development Programs
Current report
Research and development expense increased by $1.4 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. The increase was primarily due to $1.5 million of expenses related to OCU410 and OCU410ST which were driven by an increase in clinical and milestone payments for the three months ended September 30, 2026. This increase is also related to $0.9 million of additional personnel related costs due to higher headcount during the three months ended September 30, 2026. These increases were partially offset by a $0.6 million decrease in OCU400 related expenses, driven by a decrease in clinical activities and a $0.4 million decrease in other costs related to research and development consulting during the three months ended September 30, 2026.
Current report
General and administrative expense decreased by $1.0 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. The decrease was primarily due to external professional services fees.
Current report
Interest expense increased by $1.8 million for the three months ended September 30, 2026 compared to thethree months ended September 30, 2025. This increase was primarily attributable to interest incurred on our Convertible Notes which were issued during the second quarter of 2026.
Current report
The change in fair value of the derivative liability increased by $16.6 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. This increase was due to the remeasurement of the derivative liability related to the Convertible Notes issued during the second quarter of 2026.
Current report
Other (expense) income, net decreased by $1.4 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. This decrease was primarily due to a one-time contract termination fee which occurred in the third quarter of 2025.
Current report
Comparison of the Nine Months Ended September 30, 2026 and 2025
Current report
Research and development expense increased by $5.4 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The increase was primarily due to $3.6 million of additional personnel related costs due to higher headcount for the nine months ended September 30, 2026. The increase also relates to $3.1 million of expenses related to OCU410 and OCU410ST, which were driven by an increase in clinical and milestone payments for the nine months ended September 30, 2026. These were partially offset by $1.3 million in other research and development expenses, primarily related to a decrease in OCU400 expenses, which was driven by a reduction in clinical activities for the nine months ended September 30, 2026.
Current report
General and administrative expense increased by $1.1 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The increase was primarily due to $2.7 million of additional personnel expenses related to an increase in headcount and corporate related fees for the nine months ended September 30, 2026. This increase was partially offset by a decrease of $1.6 million in professional service fees.
Current report
Interest income increased by $0.4 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The increase was primarily due to a higher average cash balance and short-term investments during the nine months ended September 30, 2026 as compared to the nine months ended September 30, 2025.
Current report
Interest expense increased $5.0 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The increase was primarily attributable to interest incurred on our Convertible Notes which were issued during the second quarter of 2026.
Current report
The change in fair value of the derivative liability increased by $14.7 million during the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. This increase was due to the remeasurement of the derivative liability related to the Convertible Notes issued during the second quarter of 2026.
Current report
Other (expense) income, net decreased by $0.9 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The decrease was primarily due to a one-time contract termination fee which occurred in the third quarter of 2025.
Current report
Cash used in investing activities was $59.8 million for the nine months ended September 30, 2026, and primarily consisted of purchases of short-term investments using proceeds from the issuance of the Convertible Notes. Cash used in investing activities was $0.3 million for the nine months ended September 30, 2025, which was mainly attributable to the purchase of property and equipment.
Removed paragraphs (28)
Previous report
tOverview
Previous report
We are a biotechnology company focused on discovering, developing, and commercializing novel gene therapies that improve health and offer hope for patients across the globe.
Previous report
Our technology pipeline includes:
Previous report
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 report
Additional Phase 1/2 data include:
Previous report
▪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 report
▪Approximately 2-line gain (N=8) observed across multiple mutation types in treated eyes compared to untreated eyes at 3 years.
Previous report
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.
Previous report
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 report
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 report
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 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.
Previous 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.
Previous report
Other Programs —
Previous 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.
Previous 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.
Previous 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.
Previous 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.
Previous 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.
Previous report
Comparison of the Six Months Ended June 30, 2026 and 2025
Previous 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.
Previous 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.
Previous 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.
Previous 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.
Previous report
Loss on extinguishment of debt
Previous 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.
Previous 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.
Previous report
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.
Reworded paragraphs (27)
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 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.
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 2026 Quarterly Reports, as applicable, and "Disclosure Regarding Forward-Looking Statements" section in 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
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.
Current wording
OCU400 is an investigational product being developed for early to advanced cases of RP, spanning pediatric to adult populations, including clinical and/or genetic diagnosis with both syndromic and non-syndromic forms of the disease.
Previous 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.
Current wording
OCU400 has been granted Regenerative Medicine Advanced Therapy (“RMAT”) designation and Orphan Drug Designation (“ODD”) by the FDA, and Orphan Medicinal Product Designation (“OMPD”) and Advanced Therapy Medicinal Product (“ATMP”) classification by the European Medicines Agency (“EMA”). Orphan designations provide certain incentives, including potential market exclusivity upon approval. The 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
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.
Current wording
The OCU410ST Phase 1 clinical trial was an open-label, fellow-eye controlled study with safety as the primary endpoint. Observed efficacy in evaluable subjects (N=6) demonstrated that atrophic lesions grew slower by 54% at 12 months for evaluable treated eyes compared to untreated 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 eyes. Additionally, 100% of evaluable treated eyes (N=6) 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 eyes at 12 months, demonstrating preservation or stabilization in photoreceptor integrity. The untreated eyes showed expected growth in atrophy. There have been no reported treatment-related serious adverse events or adverse events of interest to date.
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. 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").
Current wording
OCU410ST has been granted ODD and Rare Pediatric Disease Designation (“RPDD”) by the FDA, and OMPD designation and ATMP classification by the EMA. 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 and provided initial feedback that a single U.S. based trial may be acceptable to support a Marketing Authorization Application ("MAA") in the EU.
Previous wording
In March 2026 we announced positive 12-month topline data from the Phase 2 ArMaDa clinical trial. Key findings from Phase 2 include:
Current wording
In March 2026 we announced 12-month topline data from the Phase 2 ArMaDa2 clinical trial. Key findings include a 31% reduction in lesion growth in the optimal dose (“medium”) group compared to control (p< 0.05); 27% slower rate of EZ loss compared to control, indicating structural preservation of photoreceptors, which is believed to correlate with visual function; and 55% of treated patients demonstrating ≥30% lesion size reduction vs. control.
Previous wording
Novel Biologic Therapy for Retinal Diseases — 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.
Current wording
Novel Biologic Therapy for Retinal Diseases — OCU200 is an investigational, novel recombinant fusion protein consisting of two human proteins, tumstatin and transferrin. OCU200 is designed 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.
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 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.
Current wording
Inhaled Mucosal Vaccine Platform— Our next-generation, investigational 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 the Phase 1 clinical trial for OCU500 in the second quarter of 2026.
Previous 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.
Current wording
Collaborative arrangement revenue increased by $0.2 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. The increase was due to our quarterly assessment of the amount of co-development services provided by us to our business partner in the collaboration agreement.
Previous 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.
Current wording
Interest income increased by $0.4 million for the three months ended September 30, 2026 compared to the three months ended September 30, 2025. The increase was primarily due to a higher average cash balance and short-term investments for the three months ended September 30, 2026.
Previous wording
The following table summarizes the results of our operations for the six months ended June 30, 2026 and 2025 (in thousands):
Current wording
The following table summarizes the results of our operations for the nine months ended September 30, 2026 and 2025 (in thousands):
Previous wording
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 wording
The following table summarizes our research and development expenses by product candidate for the nine months ended September 30, 2026 and 2025 (in thousands):
Previous wording
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.
Current wording
Collaborative arrangement revenue increased by $0.4 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. The increase was due to our quarterly assessment of the amount of co-development services provided by us to our business partner in the collaboration agreement.
Previous wording
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 wording
The loss on extinguishment of debt increased by $2.4 million for the nine months ended September 30, 2026 compared to the nine months ended September 30, 2025. This increase was a result of the payoff of our Avenue Capital Loan which occurred during the second quarter of 2026.
Previous 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.
Current wording
As of September 30, 2026, we had $24.6 million in cash and cash equivalents, along with $0.3 million of restricted cash and $59.6 million of short-term investments. To date, we have not generated revenue from our product candidates. Since our inception and through September 30, 2026, we have funded our operations through gross proceeds from the sale of our common stock and warrants, from the issuance of convertible notes and additional indebtedness, from royalty agreements, and 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 $(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.
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 $47.7 million and $50.1 million for the nine months ended September 30, 2026 and 2025, respectively. As of September 30, 2026, we had an accumulated deficit of
Previous wording
The following table provides a summary of our cash flows for the six months ended June 30, 2026 and 2025 (in thousands):
Current wording
The following table provides a summary of our cash flows for the nine months ended September 30, 2026 and 2025 (in thousands):
Previous 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.
Current wording
Cash used in operating activities was $49.7 million for the nine months ended September 30, 2026, and primarily consisted of a net loss of $47.7 million adjusted for non-cash items including stock-based compensation of $7.2 million, depreciation and amortization of $1.7 million, non-cash lease expense of $0.9 million, non-cash expense from collaborative arrangements, net of $3.5 million, other non-cash items of $0.1 million, and a change in net working capital of $0.7 million.
Previous 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.
Current wording
Cash used in operating activities was $43.0 million for the nine months ended September 30, 2025, and primarily consisted of a net loss of $50.1 million adjusted for non-cash items including stock-based compensation of $5.7 million, non-cash expense from collaborative arrangements, net of $2.7 million, non-cash lease expense of $0.9 million, depreciation and amortization of $2.8 million, other non-cash items of $0.1 million, and a change in net working capital of $0.3 million.
Previous wording
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 wording
Cash provided by financing activities was $115.5 million for the nine months ended September 30, 2026 compared to cash provided by financing activities of $17.3 million for the nine months ended September 30, 2025. During the nine months ended September 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 costs related to the issuance of our Convertible Notes. During the nine months ended September 30, 2025, cash provided by financing activities primarily consisted of gross proceeds of $18.3 million related to the sale of common stock and warrants, partially offset by a repayment of $1.0 million of the EB-5 Loan.
Previous wording
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 wording
We have incurred recurring operating losses, generated negative cash flows from operations, and expect to continue to incur significant expenditures to support the research, development, and potential commercialization of our 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$47.7 million and$50.1 million for thenine months ended September 30, 2026 and 2025, respectively. We have incurred net losses of approximately$3.6 million and$20.1 million for thethree months ended September 30, 2026 and 2025, respectively. As of September 30, 2026, we had an accumulated deficit of$455.7 million and cash and cash equivalents and short-term investments totaling$84.3 million.
Previous wording
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 wording
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 unaudited 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.
Previous wording
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 wording
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 unaudited 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.
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 except as noted below:
Current wording
The preparation of financial statements in conformity with GAAP requires us to make judgments, estimates, and assumptions in the preparation of our unaudited 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:
Previous wording
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 wording
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.
Previous wording
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 wording
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 unaudited 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.
Previous wording
For a discussion of recently adopted accounting pronouncements, see Note 2 in the notes to the condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Current wording
For a discussion of recently adopted accounting pronouncements, see Note 2 in the notes to the unaudited condensed consolidated financial statements included in this Quarterly Report on Form 10-Q.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
$455.7 million. In addition, we had accounts payable and accrued expenses and other current liabilities of $17.8 million and indebtedness of $102.0 million.
Rearranged previous paragraphs (4)
Previous report — wording carried elsewhere
• 31% reduction in lesion growth in the optimal dose (medium) group compared to control (p< 0.05)
Previous report — wording carried elsewhere
▪ 27% slower rate of EZ loss compared to control, indicating structural preservation of photoreceptors, which correlates with visual function; and
Previous report — wording carried elsewhere
▪ 55% of treated patients demonstrated ≥30% lesion size reduction vs. control
Previous report — wording carried elsewhere
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.