Financial Data and Key Metrics Changes - The company's cash, cash equivalents, and restricted cash totaled $27.3 million as of June 30, 2025, down from $58.8 million as of December 31, 2024 [22] - Total operating expenses for the three months ended June 30, 2025, were $15.2 million, including $8.4 million in research and development expenses and $6.8 million in general and administrative expenses, compared to $16.6 million in the same period of 2024 [22][23] Business Line Data and Key Metrics Changes - The OCU400 Phase three Limelight clinical trial for retinitis pigmentosa is actively recruiting patients in the United States and Canada, with a target for BLA and MAA filings in 2026 [7] - The OCU410 ST clinical trial for Stargardt disease has achieved key milestones, including the first patient dosing in July 2025 [12] - Preliminary data for OCU410 ST and OCU410 shows favorable safety and efficacy with improved structural and functional outcomes [8] Market Data and Key Metrics Changes - The OCU400 therapy has the potential to address over 100 different mutations associated with retinitis pigmentosa, which currently lacks approved treatment options for approximately 298,000 patients in the US and Europe [10] - Stargardt disease affects around 100,000 individuals in the US and Europe combined, with an estimated one million people globally, and there is currently no FDA-approved treatment available [11] Company Strategy and Development Direction - The company aims to file three biological licensing applications and market authorization applications in the next three years, focusing on providing one-time therapies for significant unmet medical needs [6] - A regional partnership for OCU400 has been signed with a well-established leader in the pharmaceutical sector in Korea, allowing the company to retain rights in larger geographies [16] - The proposed reverse merger with OrthoCelix is intended to create a NASDAQ-listed late clinical stage regenerative cell therapy company, focusing on orthopedic diseases [17] Management's Comments on Operating Environment and Future Outlook - Management expressed enthusiasm about the progress of their modified gene therapy platform and the positive response from the FDA regarding their clinical trials [6][12] - The company is actively exploring strategic partnerships to enhance its financial position and drive long-term strategy [23] Other Important Information - The Data and Safety Monitoring Board reported no serious adverse events related to OCU400, recommending the continuation of the study [11] - Leadership changes were made to strengthen the company's internal expertise, including the appointment of a new Chief Development Officer and Executive Vice President of Commercial and Business Development [20] Q&A Session Summary Question: Are there any other deals that you might be looking to execute? - The company is continuously looking for potential partnership opportunities, including regional partnerships for all gene therapy programs [27] Question: How many sites are included in the Stargardt Phase three trial? - The trial has 15 centers activated, and the company does not anticipate challenges in enrolling Stargardt patients due to the lack of approved products [32] Question: What does a 27% lesion growth reduction in GA mean for patients? - A 27% reduction in lesion growth is expected to significantly help patients maintain their visual function over time [34] Question: Was there a futility analysis during the DSMB review? - There was no futility analysis; the review was purely for safety [40] Question: When will the interim analysis for geographic atrophy be updated? - The interim analysis data is expected in the fourth quarter, providing structural and functional outcomes [41] Question: What is the regulatory path for OCU400 in Korea? - The company expects to use US FDA approval to gain approval in Korea without needing further clinical trials [63]

Financial Data and Key Metrics Changes - Research and development expenses for Q1 2025 were $9.5 million, up from $6.8 million in Q1 2024, indicating a significant increase in investment in R&D [21] - General and administrative expenses for Q1 2025 were $6.5 million, slightly up from $6.4 million in the same period of 2024 [22] - The net loss for Q1 2025 was approximately $15.3 million, or $0.05 net loss per share, compared to a net loss of approximately $11.9 million, or $0.05 net loss per share, in Q1 2024 [22] - Cash and restricted cash totaled $38.1 million as of March 31, 2025, down from $58.8 million as of December 31, 2024, indicating a decrease in liquidity [22] Business Line Data and Key Metrics Changes - The company is advancing three gene therapies through clinical trials, with a focus on modifier gene therapy, which targets diseases with large patient populations [7][9] - OCU400 has shown positive two-year long-term safety and efficacy data, demonstrating a statistically significant improvement in visual function [12] - OCU410 is designed to address multiple pathways in dry AMD, with a goal of providing a one-time therapy for millions suffering from the condition [17] Market Data and Key Metrics Changes - OCU400 has the potential to treat approximately 300,000 RP patients in the U.S. and EU, and 1.6 million patients globally, using a gene-agnostic approach [11] - The company is planning to initiate a Phase III clinical trial for OCU410 in 2026, targeting a BLA submission in 2028 [10] Company Strategy and Development Direction - The company aims to file three biologics license applications (BLA) in the next three years, focusing on modifier gene therapies that offer potential cures for life [7] - The strategy includes building manufacturing capabilities in-house to support future commercial launches, with plans for a second site in the U.S. [31][32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress of their gene therapy programs and the significance of upcoming milestones, emphasizing the mission to address unmet medical needs for patients facing vision loss [58] - The company is actively engaging with regulatory agencies to align clinical trial designs and expedite the approval process for their therapies [39] Other Important Information - The company is in discussions regarding developmental funding for its vaccine technology for influenza [20] - The investigational new drug application for OCU500 is in effect, with plans for a Phase I clinical trial to begin in Q2 2025 [20] Q&A Session Summary Question: Enrollment status for the Limelight study and filing targets - Management confirmed that enrollment for the OCU400 Phase III trial is on track for the first half of 2025, with a BLA submission expected a year from now [28] Question: Manufacturing capabilities for filing and commercialization - Management stated that process validations at commercial scale are on target for completion this year, with plans to bring manufacturing in-house after initial commercial launch [30][31] Question: Update on the OCU200 program - Management indicated that a clinical update, including preliminary efficacy and safety data, is expected before the end of the year [34] Question: Clarification on European regulations for OCU400 - Management clarified that no additional trials are required in Europe if approval is obtained in the U.S., as the trial has sufficient representation for global mutations [55]

Financial Data and Key Metrics Changes - Research and development expenses for Q1 2025 were $9.5 million, up from $6.8 million in Q1 2024, indicating a significant increase in investment in R&D [22] - General and administrative expenses for Q1 2025 were $6.5 million, slightly up from $6.4 million in the same period of 2024 [23] - The net loss for Q1 2025 was approximately $15.3 million, or $0.05 net loss per share, compared to a net loss of approximately $11.9 million, or $0.05 net loss per share, in Q1 2024 [23] - Cash and restricted cash totaled $38.1 million as of March 31, 2025, down from $58.8 million as of December 31, 2024, indicating a decrease in liquidity [23] Business Line Data and Key Metrics Changes - The OCU400 program has shown positive two-year long-term safety and efficacy data, demonstrating a statistically significant improvement in visual function with a p-value of 0.005 [12] - The OCU410 program is on track for a Phase III clinical trial initiation in 2026, with a BLA submission expected in 2028 [10] - The OCU200 program for diabetic macular edema is currently in Phase I clinical trials, with plans to complete the trial in the second half of 2025 [21] Market Data and Key Metrics Changes - OCU400 has the potential to treat approximately 300,000 RP patients in the U.S. and EU, and 1.6 million patients globally, using a gene-agnostic approach [11] - OCU410 is designed to address multiple pathways implicated in the pathogenesis of dry AMD, targeting 2-3 million people in the U.S. and Europe, and 8 million globally [18] Company Strategy and Development Direction - The company aims to file three biologics license applications (BLA) in the next three years, focusing on modifier gene therapies that offer potential cures for large patient populations [8] - The strategy includes bringing manufacturing in-house after initial commercial launch, with plans for a second site in the U.S. [32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress of their gene therapy programs and the importance of upcoming milestones, emphasizing the mission to provide one-time therapies for significant unmet medical needs [60] - The company is actively engaging with regulatory agencies to ensure alignment on clinical trial designs and submissions [41] Other Important Information - The company is exploring strategic partnerships for funding its vaccine technology for flu [22] - The EMA has granted eligibility for the OCU400 MAA submission, recognizing its potential to address serious unmet medical needs in Europe [14] Q&A Session Summary Question: Enrollment status for the Limelight study and filing targets - Management confirmed that enrollment is on track for the first half of 2025, with a BLA submission expected a year from now [28] Question: Manufacturing capabilities for filing and commercialization - Management stated that process validations at commercial scale are on target for completion this year, with plans to bring manufacturing in-house after initial launch [31][32] Question: Update on the OCU200 program - Management indicated that a clinical update, including preliminary efficacy and safety data, is expected before the end of the year [36] Question: Clarification on European regulations for OCU400 - Management clarified that no additional trials are required in Europe if approval is obtained in the U.S., as the trial has sufficient representation for global mutations [57] Question: Impact of changes at government agencies on clinical trials - Management reported no negative impact from recent changes at government agencies, maintaining strong engagement with NIAID for the clinical study plan [44] Question: Interim look for the Limelight study - Management confirmed that there is no interim look in the study, and full data will be available once the CSR is finalized [48]

Financial Data and Key Metrics Changes - Research and development expenses for Q1 2025 were $9.5 million, up from $6.8 million in Q1 2024, indicating a 39.7% increase [21] - General and administrative expenses for Q1 2025 were $6.5 million, slightly up from $6.4 million in the same period of 2024 [22] - Net loss for Q1 2025 was approximately $15.3 million, or $0.05 net loss per share, compared to a net loss of approximately $11.9 million, or $0.05 net loss per share in Q1 2024 [22] - Cash and restricted cash totaled $38.1 million as of March 31, 2025, down from $58.8 million as of December 31, 2024, indicating a significant decrease in liquidity [22] Business Line Data and Key Metrics Changes - The OCU400 program has shown positive two-year long-term safety and efficacy data, demonstrating a statistically significant improvement in visual function with a p-value of 0.005 [11] - OCU410 SD has received alignment with the FDA for a Phase II-III pivotal confirmatory trial, which could support a BLA submission in 2027 [8] - The OCU410 program for geographic atrophy (GA) has shown a 41% slower GA lesion growth in treated eyes compared to untreated eyes after a single injection [18] Market Data and Key Metrics Changes - OCU400 has the potential to treat approximately 300,000 RP patients in the U.S. and EU, and 1.6 million patients globally, using a gene-agnostic approach [10] - OCU410 is designed to address multiple pathways implicated in the pathogenesis of dry AMD, targeting 2-3 million people in the U.S. and Europe, and 8 million globally suffering from GA [17] Company Strategy and Development Direction - The company aims to file three biologics license applications (BLA) in the next three years, focusing on modifier gene therapies that offer potential cures for large patient populations [6] - The strategy includes building manufacturing capabilities in-house to support future commercial launches, with plans for a second site in the U.S. [31][32] - The company is exploring strategic partnerships and funding opportunities to enhance its working capital and support its vaccine technology development [22][21] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the progress of their gene therapy programs and the significance of upcoming milestones, emphasizing the mission to provide one-time therapies for life to address unmet medical needs [61] - The management highlighted the importance of ongoing discussions with regulatory agencies like the FDA and EMA to align clinical trial designs and expedite the approval process for their therapies [40] Other Important Information - The company is currently in discussions regarding developmental funding for its vaccine technology for influenza [21] - The investigational new drug application for OCU500 is in effect, with plans to initiate a Phase I clinical trial in Q2 2025 [20] Q&A Session Summary Question: Enrollment status of the Limelight study and filing targets - The enrollment for the OCU400 Phase III trial is on track for the first half of 2025, with a BLA submission expected a year from now [28] Question: Manufacturing capabilities for filing and commercialization - The company is on target to complete process validations at commercial scale this year, with plans to bring manufacturing in-house after initial commercial launch [30][31] Question: Update on the OCU200 program - The clinical trial for OCU200 is expected to be completed in the latter part of this year, with updates on preliminary efficacy and safety to be provided before year-end [34] Question: Clarification on clinical trials in Europe - No additional trials are required in Europe if approval is obtained in the U.S., as the current trial has sufficient representation for global mutations [58]

Core Insights - Ocugen, Inc. is advancing its novel modifier gene therapies for blindness diseases, with a goal of filing three Biologics License Applications (BLA) or Marketing Authorization Applications (MAA) within the next three years [2][6] Financial Performance - As of March 31, 2025, the company's cash and restricted cash totaled $38.1 million, down from $58.8 million as of December 31, 2024 [12] - Total operating expenses for Q1 2025 were $16.0 million, compared to $13.2 million in Q1 2024, with research and development expenses increasing from $6.8 million to $9.5 million [12] - The net loss per common share for Q1 2025 was $0.05, consistent with the loss per share reported in Q1 2024 [12][19] Clinical Development Updates - The Phase 3 liMeliGhT clinical trial for OCU400, targeting retinitis pigmentosa (RP), is open to all eligible patients and aims to treat approximately 300,000 RP patients in the U.S. and EU [3][12] - The company has received FDA alignment to proceed with a Phase 2/3 pivotal trial for OCU410ST for Stargardt disease, which affects around 100,000 individuals in the U.S. and EU [4][12] - In the Phase 2 portion of the OCU410 trial for geographic atrophy (GA), treated subjects showed a 4-line (23-letter) gain in visual acuity and 41% slower GA lesion growth compared to untreated eyes [5][12] Regulatory Progress - All three modifier gene therapies have received Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA), which accelerates the regulatory review process [6][12] - The EMA has provided a positive opinion for the ATMP classification of OCU400, allowing for a centralized MAA submission [12] Future Plans - Ocugen plans to initiate the Phase 2/3 study for OCU410ST by mid-2025, with a target BLA filing in 2027 [4][12] - The company is also preparing to complete the Phase 1 clinical trial for OCU200, its candidate for diabetic macular edema, in the second half of 2025 [9][12]

Core Insights - Ocugen, Inc. is set to present its innovative modifier gene therapy platform at two major conferences, highlighting its ongoing clinical trials for retinal diseases [1][2] - The company aims to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) within the next three years, addressing significant unmet medical needs [2] Conference Participation - Ocugen will participate in the ARVO 2025 Annual Meeting and Retina World Congress, showcasing its research and engaging with the retina community [1][2] - The company will present on its clinical trials for OCU400, OCU410ST, and OCU410, focusing on retinitis pigmentosa, Stargardt disease, and geographic atrophy respectively [1][5][6] Clinical Trials - OCU400 is currently in a Phase 3 clinical trial (LiMeliGhT) for retinitis pigmentosa, while OCU410ST is in a Phase 2/3 pivotal confirmatory trial for Stargardt disease [1][5] - OCU410 is undergoing a Phase 2 clinical trial (ArMaDa) for geographic atrophy secondary to dry age-related macular degeneration [1][6] Company Vision - Ocugen is committed to developing therapies for inherited retinal diseases and aims to provide access to these treatments globally [8][9] - The company emphasizes its gene-agnostic approach, which has the potential to treat multiple retinal diseases with a single product [2][9]