Ocugen, Inc.
For the period ended Jun 30, 2025. Filed Aug 4, 2025.
View on SEC.govAbout this filing
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, 2025 · Filed Aug 4, 2025
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 2025-03-31; filed 2025-05-09.
0001628280-25-024093
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-04.
0001628280-25-037310
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 81 previous paragraphs and 111 current paragraphs.
48 unchanged; 24 reworded; 35 added; 6 removed; 3 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 (35)
Current report
OCU410ST- We initiated dosing in GARDian3 pivotal confirmatory trial for OCU410ST in July. The OCU410ST Phase 2/3 pivotal confirmatory trial represents our second late-stage clinical program. We plan to submit a BLA for OCU410ST in 2027 in alignment with the Company's strategic goal of filing three BLAs over the next three years. We also believe our modifier gene therapy platform has the potential to address multifactorial retinal diseases including dry age-related macular degeneration ("dAMD"), which affects millions of patients in the United States alone. In May 2025, we announced that the FDA has granted Rare Disease Designation (RPDD) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 ("RP19"), and cone-rod dystrophy 3 ("CORD3"). In November 2024, the EMA granted orphan medicinal product designation ("OMPD") for OCU410ST for the treatment of ABCA4-associated retinopathies (>1200 mutations) including Stargardt disease, RP 19, 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 Stargardt disease (ABCA4-associated retinopathies).
Current report
OCU200 is a multicenter, open-label, dose-ranging study with four cohorts in the dose escalation phase and one cohort in the combination therapy phase. The study follows an accelerated 3+3 sequential dosing design. In the dose-escalation phase, up to 12 subjects will be enrolled if no dose-limiting toxicities are observed. Enrollment may expand to a maximum of 24 subjects if exactly one DLT occurs in each cohort of three subjects. In the combination therapy cohort, 3 to 6 subjects will receive OCU200 at the maximum tolerated dose in combination with Lucentis. Each subject will receive two intravitreal injections of OCU200, administered six weeks apart. The total study population will include up to 30 subjects.
Current report
Recent Events
Current report
Planned Merger of Regenerative Medicine Cell Therapy Platform (including NeoCart) business with Carisma Therapeutics Inc.
Current report
On June 22, 2025, we and OrthoCellix, Inc., a Delaware corporation and our wholly-owned subsidiary to which we have contributed the assets related to our NeoCart product candidate (“OrthoCellix”), entered into an Agreement and Plan of Merger (the “Merger Agreement”), by and among Ocugen, OrthoCellix, Carisma Therapeutics Inc., a Delaware corporation (“Carisma”) and Azalea Merger Sub, Inc., a Delaware corporation and a wholly owned subsidiary of Carisma (“Merger Sub”).
Current report
Pursuant to the Merger Agreement, subject to the satisfaction or waiver of the conditions set forth in the Merger Agreement, Merger Sub will merge with and into OrthoCellix (the “Merger”), with OrthoCellix continuing as a wholly owned subsidiary of Carisma and the surviving company of the Merger. Subject to the terms and conditions of the Merger Agreement, at the effective time of the Merger (the “Effective Time”), each share of common stock, par value $0.00001 per share, of OrthoCellix (“OrthoCellix Common Stock”), issued and outstanding (other than shares of OrthoCellix Common Stock (a) held as treasury stock, (b) owned, directly or indirectly, by Carisma or Merger Sub immediately prior to the Effective Time or (c) as to which appraisal rights have been properly exercised in accordance with Delaware law) shall be converted into and become exchangeable for the right to receive a number of shares of Carisma Common Stock, based on a ratio calculated in accordance with the Merger Agreement (the “Exchange Ratio”).
Current report
Under the terms of the Merger Agreement, upon the closing of the proposed transactions and after giving effect to the contemplated $25.0 million private financing with Ocugen and other select investors, whereby Ocugen will purchase not less than $5.0 million of shares of Carisma’s common stock, par value $0.001 per share (“Carisma Common Stock”), which is expected to close concurrently with the completion of the merger (the “Concurrent Financing”), It is estimated that Ocugen will own more than 50% of the combined company, on a fully diluted basis, and will continue to consolidate OrthoCellix. The percentage of the combined company that each company’s former stockholders will own after completion of the merger is subject to adjustment based on Carisma’s net cash at the closing and the proceeds from the Concurrent Financing, among other adjustments, in each case as described in the Merger Agreement.
Current report
As part of the proposed transaction, Ocugen is expected to enter into a transition services agreement with OrthoCellix pursuant to which Ocugen will provide, or cause its affiliates to provide, OrthoCellix with certain services to help facilitate an orderly transition of the NeoCart program. Ocugen is also expected to enter a Manufacturing and Supply Agreement with OrthoCellix for the manufacturing of NeoCart.
Current report
To complete the Merger, Carisma’s stockholders must approve the transactions contemplated under the Merger Agreement and Ocugen, as the sole stockholder of OrthoCellix, must adopt the Merger Agreement and approve the Merger and the related transactions contemplated by the Merger Agreement. Additionally, each of the other closing conditions set forth in the Merger Agreement must be satisfied or waived. The Merger will become effective upon the filing of the Certificate of Merger (as defined in the Merger Agreement) or at such later date as is agreed by Carisma and OrthoCellix and specified in the Certificate of Merger. Neither Carisma nor OrthoCellix can predict the exact timing of the consummation of the Merger. After the Merger is complete, Carisma will change its name to OrthoCellix and will operate as a public company. As noted above, it is expected that Ocugen will control OrthoCellix and will consolidate OrthoCellix into its financial statements. Subject to the conditions above, the Merger is expected to close in the fourth quarter of 2025.
Current report
Term Sheet with Korean Partner
Current report
In June 2025, we entered into a binding term sheet to negotiate and enter into a licensing agreement with a well-established leader in the pharmaceutical and healthcare sector in Korea, for exclusive Korean rights to OCU400. Pursuant to the term sheet, under the license agreement we would receive upfront license fees and near-term development milestones equaling up to $11 million and be entitled to sales milestones of $1 million for every $15 million of net sales in Korea in addition to a royalty of 25% on net sales of OCU400 generated by our partner. Additionally, the term sheet contemplates that we would manufacture commercial supply of OCU400 under terms of a supply agreement. We are in the process of negotiating the license agreement with our partner, however, we cannot provide assurance that a definitive agreement for the license will be executed on the timeline we expect or at all, or that, if executed, it will be on terms agreed to in the term sheet.
Current report
Modifier Gene Therapy Platform
Current report
In May 2025, we announced that the FDA has granted RPDD for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, RP19, and CORD3. Previously, OCU410ST received Orphan Drug designations for the treatment of ABCA4-associated retinopathies from the FDA and the EMA.
Current report
In June 2025, we announced that the FDA has cleared the IND amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST. We initiated dosing in GARDian3 pivotal confirmatory trial for OCU410ST in July. The OCU410ST Phase 2/3 pivotal confirmatory trial represents our second late-stage clinical program.
Current report
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.
Current report
The estimated 10-yr economic burden of vision loss diseases in the US is approximately $1.34 trillion. STGD and GA or dry AMD are major contributors to vision loss.
Current report
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.
Current report
Positive preliminary efficacy and safety data from the OCU410 Phase 1 ArMaDa clinical trial at 12 months demonstrated no drug-related serious adverse events (SAEs), 23% slower geographic atrophy (GA) lesion growth in treated eyes versus fellow eyes after a single injection, and 2-line/10-letter gain in visual acuity in treated eyes when compared to untreated fellow eyes. Preliminary results from ongoing Phase 2 clinical trial (N=31), 6-month interim analysis, demonstrated a 27% slower lesion growth and preservation of retinal tissue. These data support the potential for OCU410 to provide a one-time treatment for life for the 2-3 million people in the U.S. & EU combined who suffer from GA.
Current report
Regenerative Cell Therapy Platform
Current report
Research and development expense decreased by $0.5 million for the three months ended June 30, 2025 compared to the three months ended June 30, 2024. The decrease was primarily due to $(0.7) million related to OCU500, which is driven by a decrease in preclinical activities and GMP manufacturing of Phase 1 clinical trial material.
Current report
General and administrative expense decreased by $0.9 million for the three months ended June 30, 2025 compared to the three months ended June 30, 2024. The decrease was primarily due to $1.1 million in professional services fees and $0.2 million related to reduced stock based compensation.
Current report
Interest (expense) income, net decreased by $1.2 million for the three months ended June 30, 2025 compared to the three months ended June 30, 2024. The decrease was primarily due to $1.3 million in interest expense on our long term debt, which was not leveraged until three months ended December 31, 2024.
Current report
Other income (expense), net
Current report
Other income (expense), net decreased by $0.1 million for the three months ended June 30, 2025 compared to the three months ended June 30, 2024. The decrease was primarily due to $0.1 million in one time IRS refund.
Current report
Comparison of the Six Months Ended June 30, 2025 and 2024
Current report
Collaborative arrangement revenue
Current report
Research and development expense
Current report
Research and development expense increased by $2.2 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. The increase was primarily due to $2.3 million related to increased headcount related expenses.
Current report
General and administrative expense
Current report
General and administrative expense decreased by $0.9 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. The decrease was primarily due to $0.9 million in professional service fees and $0.4 million related to reduced stock based compensation. This was offset by an increase of $0.4 million to our commercial related activities for OCU400.
Current report
Interest (expense) income, net
Current report
Interest (expense) income, net decreased by $2.5 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. The decrease was primarily due to $2.5 million in interest expense on our long term debt, which was not leveraged until three months ended December 31, 2024.
Current report
Other income (expense), net
Current report
Other income (expense), net decreased by $0.2 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. The decrease was primarily due to $0.2 million in a one time IRS refund, and unrealized gain on currency.
Current report
On July 4, 2025, the One Big Beautiful Bill Act (“OBBBA”) was enacted, introducing amendments to U.S. tax laws with various effective dates from 2025 to 2027. The Company is currently assessing the implications of these tax law changes and does not expect that they will have a material impact on its Financial Statements in the current year.
Removed paragraphs (6)
Previous report
In February, dosing was complete in the Phase 2 portion of the OCU410 Phase 1/2 ArMaDa clinical trial for GA, an advanced stage of dAMD. In evaluable subjects, OCU410 12-month data demonstrates a 4-line (23-letter) gain in the visual acuity and 41% slower GA lesion growth in treated eyes versus untreated fellow eyes after single injection. OCU410 unique mechanism of action targets multiple pathways associated with dAMD 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.
Previous report
Subsequently, 12-month data was presented that demonstrates 4-line (23-letter) gain in the visual acuity when compared to untreated fellow eyes and 41% slower GA lesion growth in treated eye versus fellow eyes after single injection.
Previous report
Regenerative Medicine Cell Therapy Platform
Previous report
Research and development expense increased by$2.7 million for the three months ended March 31, 2025 compared to the three months ended March 31, 2024. The increase was primarily due to $1.3 millionin employee-related expenses due to an increase in number of employees and $0.7 million related to OCU410/410ST clinical activities for Phase 1/2 clinical trial; and $0.3 million related to OCU400, which is driven by an increase in clinical activities for Phase 3 clinical trial.
Previous report
General and administrative expense decreased by an immaterial amount for the three months ended March 31, 2025 compared to the three months ended March 31, 2024.
Previous report
Interest expense, net increased by$1.2 million for the three months ended March 31, 2025 compared to the three months ended March 31, 2024. The increase was primarily due to an interest expense related to the Loan and Security Agreement which was entered in November 2024.
Reworded paragraphs (24)
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, 2024, included in our 2024 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 2024 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, 2024, included in our 2024 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 2024 Annual Report, First Quarter 10-Q, 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.
Previous wording
We are a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics and vaccines that improve health and offer hope for patients across the globe.
Current wording
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 wording
•Modifier Gene Therapy Platform— Based on the use of nuclear hormone receptors ("NHRs"), we believe our 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. We also believe our modifier gene therapy platform has the potential to address multifactorial retinal diseases including dry age-related macular degeneration ("dAMD"), which affects millions of patients in the United States alone, using OCU410, and Stargardt disease, which is a rare genetic disease, using OCU410ST. We are actively recruiting patients in the United States and Canada in the Phase 3 liMeliGhT clinical trial for OCU400 for the treatment of RP and are on track to complete enrollment in the first half of 2025. 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. The EMA also granted eligibility to submit the OCU400 MAA via the centralized procedure as an ATMP based on the current study design and statistical analysis plan. 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 October 2024, the Data and Safety Monitoring Board ("DSMB") for the OCU410ST GARDian clinical trial approved enrollment for the second phase of the Phase 1/2 clinical trial. 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 Biologics License Application ("BLA") submission. Additionally, the EMA granted orphan medicinal product designation ("OMPD") for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, RP 19 ("RP19"), and cone-rod dystrophy 3 ("CORD3"). In March 2025, OCU410 and OCU410ST received ATMP classification from the EMA.
Current 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. We are actively recruiting patients in the United States and Canada in the Phase 3 liMeliGhT clinical trial for OCU400 for the treatment of RP and are on track to complete enrollment in support of our target BLA and MAA filings in 2026. 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. In June 2025, Data and Safety Monitoring Board (DSMB) for Phase 3 OCU400-301 convened for evaluable subjects. No Serious Adverse Events (SAEs) related to OCU400 were reported. The recommendation was to continue study dosing as planned. The EMA also granted eligibility to submit the OCU400 MAA via the centralized procedure as an ATMP based on the current study design and statistical analysis plan.
Previous wording
•Novel Biologic Therapy for Retinal Diseases— OCU200 is a novel 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. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and we are actively recruiting patients. In March 2025, the DSMB approved continuation of dosing in the second cohort and we intend to complete the Phase 1 clinical trial in the second half of 2025.
Current wording
•Novel Biologic Therapy for Retinal Diseases— OCU200 is a novel 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. The first patient was dosed in the OCU200 Phase 1 clinical trial in January 2025, and we are actively recruiting patients. In June 2025, the DSMB approved continuation of dosing in the third cohort and we intend to complete the Phase 1 clinical trial in the second half of 2025.
Previous wording
•Regenerative Medicine Cell Therapy Platform— Our Phase 3-ready regenerative cell therapy platform technology, which includes NeoCart (autologous chondrocyte-derived neocartilage), is being developed for the repair of knee cartilage injuries in adults. We received concurrence from the FDA on the confirmatory Phase 3 trial design and have completed renovating an existing facility into a current GMP facility to support clinical study and initial commercial launch. This facility is needed to generate patient-specific NeoCart implant from chondrocytes derived from knee biopsy. We intend to initiate the Phase 3 trial contingent on adequate availability of funding and/or based on potential future partnership.
Current wording
•Regenerative Medicine Cell Therapy Platform— Our Phase 3-ready regenerative cell therapy platform technology, which includes NeoCart (autologous chondrocyte-derived neocartilage), is being developed for the repair of knee cartilage injuries in adults. We received concurrence from the FDA on the confirmatory Phase 3 trial design and have completed renovating an existing facility into a current GMP facility to support clinical study and initial commercial launch. This facility is needed to generate patient-specific NeoCart implant from chondrocytes derived from knee biopsy. For more information, see “Management's Discussion and Analysis of Financial Condition and Results of Operations. --Recent Events.”
Previous wording
•Inhaled Mucosal Vaccine Platform — Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, we announced that the Investigational New Drug ("IND") application is in effect and 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 2025. We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms.
Current wording
•Inhaled Mucosal Vaccine Platform— Our next-generation, inhaled mucosal vaccine platform includes OCU500, a COVID-19 vaccine; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. We have completed IND-enabling studies and GMP manufacturing of clinical trial material for OCU500. In January 2025, we announced that the Investigational New Drug ("IND") application is in effect and 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 third quarter of 2025. We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms.
Previous wording
The OCU400 Phase 3 liMeliGhT clinical trial is currently underway, with enrollment on track to meet our target BLA and Marketing Authorization Application filings in mid-2026.
Current wording
The OCU400 Phase 3 liMeliGhT clinical trial is currently underway, with enrollment on track to meet our target BLA and Marketing Authorization Application filings in 2026.
Previous wording
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 Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x
Current wording
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 Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5x1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. 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 GARDian3 Phase 2/3 pivotal confirmatory trial has adaptive design with sample size re-estimation. When 24 subjects in the study (16 in treatment group and 8 in control group) complete their 8 months clinical assessments, a masked interim analysis is planned. OCU410ST is intended for early to advanced cases of Stargardt disease.
Previous wording
The latest data from the OCU410ST Phase 1 clinical trial demonstrates atrophic lesions grew slower by 54% at six months and by 103% at 12 months in evaluable treated eyes when compared to untreated eyes. In the secondary endpoint, BCVA, treated eyes demonstrated statistically significant (p=0.02) improvement in visual function when compared to untreated fellow eyes. 6 /6 (100%) evaluable treated eyes demonstrated stabilization or improvement in visual function. Ocugen plans to initiate the Phase 2/3 study by mid-year with a target BLA filing in 2027.
Current wording
The latest data from the OCU410ST Phase 1 clinical trial demonstrates atrophic lesions grew slower by 48% at 12 months for evaluable subjects. In the secondary endpoint-BCVA, treated eyes demonstrated statistically significant (p=0.031) improvement with nearly 2-line (9 letter*) gain in the visual acuity when compared to untreated fellow eyes 100% of evaluable treated eyes demonstrated stabilization or improvement vs. untreated eye in visual function. Ocugen has initiated dosing in Phase 2/3 study with a target BLA filing in 2027.
Previous wording
NeoCart is a Phase 3-ready, regenerative cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process. NeoCart is a three-dimensional tissue-engineered disc of new cartilage that is manufactured by growing the patient's own chondrocytes, the cells responsible for maintaining cartilage health. Current surgical and nonsurgical treatment options for knee cartilage injuries in adults are limited in their efficacy and durability. In prior clinical studies, Phase 2 and Phase 3, NeoCart has shown potential to accelerate healing, reduce pain, and provide regenerative native-like cartilage strength with durable benefits post transplantation. NeoCart was shown to be generally well-tolerated and demonstrated greater clinical efficacy than microfracture surgery at two years after treatment. Based on this clinical benefit, the FDA granted a RMAT designation to NeoCart for the repair of full-thickness lesions of knee cartilage injuries in adults. Additionally, we received concurrence from the FDA on the confirmatory Phase 3 trial design where chondroplasty will be used as a control group. We have completed renovating an existing facility into a GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for personalized Phase 3 trial material. We intend to initiate the Phase 3 trial contingent on adequate availability of funding and/or based on potential future partnership.
Current wording
NeoCart is a Phase 3-ready, regenerative cell therapy technology that combines breakthroughs in bioengineering and cell processing to enhance the autologous cartilage repair process. NeoCart is a three-dimensional tissue-engineered disc of new cartilage that is manufactured by growing the patient's own chondrocytes, the cells responsible for maintaining cartilage health. Current surgical and nonsurgical treatment options for knee cartilage injuries in adults are limited in their efficacy and durability. In prior clinical studies, Phase 2 and Phase 3, NeoCart has shown potential to accelerate healing, reduce pain, and provide regenerative native-like cartilage strength with durable benefits post transplantation. NeoCart was shown to be generally well-tolerated and demonstrated greater clinical efficacy than microfracture surgery at two years after treatment. Based on this clinical benefit, the FDA granted a RMAT designation to NeoCart for the repair of full-thickness lesions of knee cartilage injuries in adults. Additionally, we received concurrence from the FDA on the confirmatory Phase 3 trial design where chondroplasty will be used as a control group. We have completed renovating an existing facility into a GMP facility in accordance with the FDA's regulations in support of NeoCart manufacturing for personalized Phase 3 trial material. For additional information, see Management's Discussion and Analysis of Financial Condition and Results of Operations – Recent Events.”
Previous wording
We are party to an exclusive license agreement with Washington University in St. Louis, 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 United States, Europe, Japan, South Korea, Australia, China, and Hong Kong. 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; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. Our inhaled mucosal vaccine platform is driven by our conviction to serve a major public health concern, which requires the endorsement and support of government funding in order to develop and ultimately commercialize our vaccine candidates. As these vaccine candidates are being developed to be administered via inhalation, we believe they have the potential to generate rapid local immune response in the upper airways and lungs, where viruses enter and infect the body. We believe this novel delivery route may help reduce or prevent infection and transmission as well as provide protection against new virus variants. In
Current wording
We are party to an exclusive license agreement with Washington University in St. Louis, 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 United States, Canada, Europe, Japan, South Korea, Australia, China, and Hong Kong. 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; OCU510, a seasonal quadrivalent flu vaccine; and OCU520, a combination quadrivalent seasonal flu and COVID-19 vaccine. Our inhaled mucosal vaccine platform is driven by our conviction to serve a major public health concern, which requires the endorsement and support of government funding in order to develop and ultimately commercialize our vaccine candidates. As these vaccine candidates are being developed to be administered via inhalation, we believe they have the potential to generate rapid local immune response in the upper airways and lungs, where viruses enter and infect the body. We believe this novel delivery route may help reduce or prevent infection and transmission as well as provide protection against new virus variants. In January we announced that the IND application is in effect to initiate the Phase 1 clinical trial of OCU500. The NIAID will sponsor and conduct the Phase 1 clinical trial of OCU500 to assess the safety, tolerability, and immunogenicity of OCU500 administered via two different routes, inhalation into the lungs and intranasally as a spray. NIAID intends to initiate the Phase 1 clinical trial of OCU500 in the third quarter of 2025. We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms.
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, 2025 and 2024 (in thousands):
Current wording
The following table summarizes our research and development expenses by product candidate for the three months ended June 30, 2025 and 2024 (in thousands):
Previous wording
Collaborative arrangement revenue increased by $0.5 million for thethree months ended March 31, 2025 compared to the three months ended March 31, 2024. The decrease 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 June 30, 2025 compared to the three months ended June 30, 2024. 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
As of March 31, 2025, we had $37.8 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, 2025, we have raised an aggregate of $368.9 million to fund our operations, of which $325.1 million was from gross proceeds from the sale of our common stock and warrants, $10.3 million was from the issuance of convertible notes, $33.3 million was from the issuance of debt, and $0.2 million was from grant proceeds.
Current wording
As of June 30, 2025, we had $27.0 million in cash. 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 June 30, 2025, we have raised a gross aggregate of $368.9 million to fund our operations, of which $325.1 million was from gross proceeds from the sale of our common stock and warrants, $10.3 million was from the issuance of convertible notes, $33.3 million was from the issuance of debt, and $0.2 million was from grant proceeds.
Previous wording
In November 2024, we entered into a debt financing transaction (the “Loan and Security Agreement”) with Avenue Capital Management II, L.P., as administrative agent and collateral agent (the “Agent”, together with Avenue I and Avenue II, “Avenue”), Avenue Venture Opportunities Fund II, L.P., as a lender (“Avenue 2”), and Avenue Venture Opportunities Fund, L.P., as a lender (“Avenue 1”, and together with Avenue 2, the “Lenders”) for net proceeds of $29.2 million. The Loan and Security Agreement provides for term loans in an aggregate principal amount of up to $30.0 million delivered on November 6, 2024 (the "Term Loans"). The loan has a maturity date of November 1, 2028, of which the first 24 months are interest only, and bears interest at a variable rate per annum equal to the greater of the prime rate as reported in The Wall Street Journal plus 4.25% or 12.25%. Additionally, the Lenders have the right to convert an aggregate amount of up to $6.0 million of the outstanding principal amount into shares of our common stock at a conversion price per share equal to 80% of the trading price on the date of conversion, which shall be at Lenders' option. In the event we elect to prepay the Term Loans in full, Lenders shall have 10 days to elect to exercise its conversion right prior to such prepayment. All conversion rights shall terminate on Term Loans payoff. In connection with the entry into the Loan and Security Agreement, we entered into a Subscription Agreement (the "Subscription Agreement") by and among us and the Lenders, pursuant to which we issued (i) 211,268 shares of common stock to Avenue 1 and (ii) 845,070 shares of common stock to Avenue 2, with an issue date as of November 6, 2024 (the "Equity Grant"). Notwithstanding the foregoing, the aggregate amount of our common stock issued pursuant to this conversion right and the Equity Grant shall not exceed a number of shares equal to 19.9% of our outstanding common stock. The Loan and Security Agreement is collateralized by all of our assets in which the Agent is granted a senior secured lien. We also granted the Lenders a negative pledge on our intellectual property.
Current wording
In November 2024, the Company entered into a debt financing transaction (the “Loan and Security Agreement”) with Avenue Capital Management II, L.P., as administrative agent and collateral agent (the “Agent”, together with Avenue I and Avenue II, “Avenue”), Avenue Venture Opportunities Fund II, L.P., as a lender (“Avenue 2”), and Avenue Venture Opportunities Fund, L.P., as a lender (“Avenue 1”, and together with Avenue 2, the “Lenders”) for net proceeds of $29.2 million. The Loan and Security Agreement provides for term loans in an aggregate principal amount of up to $30.0 million delivered on November 6, 2024 (the "Term Loans"). The loan has a maturity date of November 1, 2028, of which the first 24 months are interest only, and bears interest at a variable rate per annum equal to the greater of the Prime Rate plus 4.25% or 12.25%. Additionally, the Lender has the right to convert an aggregate amount of up to $6.0 million of the outstanding principal amount into shares of Common Stock at a conversion price per share equal to a 80% of the trading price on the date of conversion, which shall be at Lenders' option. In the event the Company elects to prepay the Term Loans in full, Lenders shall have 10 days to elect to exercise its conversion right prior to such prepayment. All conversion rights shall terminate on Term Loans payoff. In connection with the entry into the Loan and Security Agreement, we entered into a Subscription Agreement (the "Subscription Agreement") by and among us and the Lenders, pursuant to which we issued (i) 211,268 shares of common stock to Avenue 1 and (ii) 845,070 shares of common stock to Avenue 2, with an issue date as of November 6, 2024 (the "Equity Grant"). Notwithstanding the foregoing, the aggregate amount of our common stock issued pursuant to this conversion right and the Equity Grant shall not exceed a number of shares equal to 19.9% of our outstanding common stock. The Loan and Security Agreement is collateralized by all of our assets in which the Agent is granted a senior secured lien. We also granted the Lenders a negative pledge on our intellectual property.
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 $15.4 million and $11.9 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $355.6 million. In addition, we had accounts payable and accrued expenses and other current liabilities of $16.2 and indebtedness of $27.7 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 $30.1 million and $27.2 million for the six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $370.3 million. In addition, we had accounts payable and accrued expenses and other current liabilities of $17.1 million and indebtedness of $28.0 million.
Previous wording
The following table provides a summary of our cash flows for the three months ended March 31, 2025 and 2024 (in thousands):
Current wording
The following table provides a summary of our cash flows for the six months ended June 30, 2025 and 2024 (in thousands):
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, depreciation and amortization of $1.8 million, non-cash lease expense of $0.6 million, non-cash expense from collaborative arrangements, net of $1.8 million, other non-cash items of $0.1 million, and a change in net working capital of $4.5 million.
Previous wording
Cash used in operating activities was $10.6 million for the three months ended March 31, 2024, and primarily consisted of a net loss of $11.9 million adjusted for non-cash items including stock-based compensation of $1.8 million, depreciation and amortization of $0.2 million, non-cash lease expense of $0.1 million, other non-cash items of $1.0 million, and a change in net working capital of $1.7 million.
Current wording
Cash used in operating activities was $20.5 million for the six months ended June 30, 2024, and primarily consisted of a net loss of $27.2 million adjusted for non-cash items including stock-based compensation of $3.7 million, non-cash expense from collaborative arrangements, net of $1.7 million, non-cash lease expense of $0.2 million, depreciation and amortization of $0.8 million, other non-cash items of $0.1 million, and a change in net working capital of $0.2 million.
Previous wording
Cash used by investing activities was immaterial for the three months ended March 31, 2025, and primarily consisted of payments related to the purchases of property and equipment. Cash used by investing activities was $2.4 million for the three months ended March 31, 2024, and primarily consisted of payments related to the purchases of property and equipment.
Current wording
Cash used in investing activities was $0.2 million for the six months ended June 30, 2025, and primarily consisted of payments related to the payment of security deposits and purchases of property and equipment. Cash used in by investing activities was $(2.9) million for the six months ended June 30, 2024, were primarily consisted of payments related to the purchases of property and equipment.
Previous wording
Cash used by financing activities was $1.2 million for the three months ended March 31, 2025 compared to cash provided by financing activities of $0.1 million for the three months ended March 31, 2024. During the three months ended March 31, 2025, cash used by financing activities primarily consisted of a payment related to the EB-5 Loan Agreement.
Current wording
Cash used in financing activities was $1.2 million for the six months ended June 30, 2025 compared to cash used in financing activities of $0.1 million for the six months ended June 30, 2024. During the six months ended June 30, 2025, cash reduced by financing activities primarily consisted of the repayment of part of the companies EB-5 loan. During the six months ended June 30, 2024, cash provided financing activities primarily consisted of gross proceeds of $(0.1) million paid related to the sale of restricted stock units.
Previous wording
•the initiation, progress, timing, costs, and results of clinical trials for our product candidates;
Current wording
•the initiation, progress, timing, costs, and results of trials for our product candidates;
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
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.
Current wording
As of June 30, 2025, we had cash of approximately $27.0 million. This amount will not be sufficient to fund our operations 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, we may have based this estimate on assumptions that may prove to be wrong, and our operating plan may change as a result of many factors currently unknown to us. Given this uncertainty, and despite the additional funding from the debt transaction with Avenue Capital, we will need to raise significant additional capital in order to fund our operations until we recognize significant revenue from product sales. Our management continues to evaluate different strategies to obtain the funding required for our future operations. These strategies may include, but are not limited to: 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. Our ability to secure funding is subject to numerous risks and uncertainties, including, but not limited to the impact of the geopolitical turmoil, macroeconomic conditions, and the impact of inflation and as a result, there can be no assurance that these funding efforts will be successful. 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.
Rearranged current paragraphs (4)
Current report — wording carried from elsewhere
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 report — wording carried from elsewhere
The following table summarizes the results of our operations for the six months ended June 30, 2025 and 2024 (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, 2025 and 2024 (in thousands):
Current report — wording carried from elsewhere
Collaborative arrangement revenue increased by $0.7 million for the six months ended June 30, 2025 compared to the six months ended June 30, 2024. 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 (3)
Previous report — wording carried elsewhere
1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. 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.
Previous report — wording carried elsewhere
January we announced that the IND application is in effect to initiate the Phase 1 clinical trial of OCU500. The NIAID will sponsor and conduct the Phase 1 clinical trial of OCU500 to assess the safety, tolerability, and immunogenicity of OCU500 administered via two different routes, inhalation into the lungs and intranasally as a spray. NIAID intends to initiate the Phase 1 clinical trial of OCU500 in the second quarter of 2025. We are continuing discussions with relevant government agencies as well as strategic partners regarding developmental funding for our OCU510 and OCU520 platforms.
Previous report — wording carried elsewhere
As of March 31, 2025, we had cash and cash equivalents of approximately $37.8 million. This amount will not meet our capital requirements over the next 12 months. We believe that our cash and cash equivalents will enable us to fund our operations into the first quarter of 2026. 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.