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]

Company Overview - Ocugen, Inc. is a pioneering biotechnology leader focused on gene therapies for blindness diseases, utilizing a breakthrough modifier gene therapy platform to address significant unmet medical needs for large patient populations [3]. Upcoming Events - Ocugen will host a conference call and live webcast to discuss its first quarter 2025 financial results and provide a business update on May 9, 2025, at 8:30 a.m. ET [1]. - A pre-market earnings announcement will be issued on the same day, with dial-in numbers provided for U.S. and international callers [2]. Product Development - The company is developing programs for inherited retinal diseases and blindness diseases affecting millions globally, including retinitis pigmentosa, Stargardt disease, and geographic atrophy [3].

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]

MALVERN, Pa., April 08, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ:OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen will present at the 2025 Cell & Gene Meeting on the Mediterranean being held April 15-17, 2025 at the Rome Cavalieri in Rome, Italy. "I look forward to sharing more on how Ocugen's modifier gene therapy platform can potentially chan ...

MALVERN, Pa., April 08, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-founder of Ocugen will present at the 2025 Cell & Gene Meeting on the Mediterranean being held April 15-17, 2025 at the Rome Cavalieri in Rome, Italy. “I look forward to sharing more on how Ocugen’s modifier gene therapy platform can potentially cha ...

OCU200 has a very favorable safety and tolerability profileNo serious adverse events related to the study drug have been reportedDosing of the second cohort has been approved MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing ...

Financial Data and Key Metrics Changes - Research and development expenses for Q4 2024 were $8.3 million, up from $7.8 million in Q4 2023. For the full year, R&D expenses decreased to $32.1 million from $39.6 million in 2023 [34] - General and administrative expenses for Q4 2024 were $6.3 million, compared to $5.2 million in Q4 2023. For the full year, G&A expenses decreased to $26.7 million from $32.0 million in 2023 [34] - Net loss for Q4 2024 was approximately $13.9 million or $0.05 per share, compared to a net loss of approximately $11 million or $0.04 per share in Q4 2023. Full year net loss was $54.1 million or $0.20 per share, compared to a net loss of $63.1 million in 2023 or $0.26 per share [35] - Cash and restricted cash totaled $58.8 million as of December 31, 2024, compared to $39.5 million at the end of 2023, providing a cash runway into Q1 2026 [36] Business Line Data and Key Metrics Changes - OCU400, targeting retinitis pigmentosa, has shown a statistically significant improvement in low-luminance visual acuity (LLVA) with a p-value of 0.005% at two years, demonstrating long-term durability [14][15] - OCU410ST received orphan medicinal product designation from the EMA and demonstrated a 100% stabilization or improvement in visual function in treated eyes, with a p-value of 0.02% [17][18] - OCU410, targeting geographic atrophy, showed a 44% slower lesion growth at nine months compared to untreated eyes, with no serious adverse events reported [24][26] Market Data and Key Metrics Changes - Approximately 300,000 people in the US and EU are affected by retinitis pigmentosa, with OCU400 addressing a significant unmet medical need [13] - There are no approved treatments for Stargardt disease, affecting about 100,000 patients in the US and Europe, highlighting the potential market for OCU410ST [17] - Geographic atrophy affects approximately 2 to 3 million patients in the US and EU, with 8 million patients globally, indicating a large market opportunity for OCU410 [23] Company Strategy and Development Direction - The company aims to file three Biologics License Applications (BLAs) in the next three years: OCU400 in 2026, OCU410ST in 2027, and OCU410 in 2028, reflecting a bold ambition supported by FDA alignment [11] - The company is focused on advancing its gene therapy platform and has secured $65 million in financing to support its clinical programs [12] - The strategy includes leveraging the ATMP classification for OCU400 and OCU410ST to expedite regulatory review and enhance interactions with the EMA [14][17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving BLA targets due to a strong track record and ongoing clinical progress across all programs [56] - The company is optimistic about the potential of its therapies to address significant unmet medical needs and improve patient outcomes [11][27] - Management highlighted the importance of safety and tolerability profiles in differentiating their products from existing therapies [64] Other Important Information - The company is actively enrolling patients in clinical trials for its therapies, with plans to complete enrollment for OCU400 in the first half of 2025 [16] - The investigational new drug application for OCU500, an inhaled mucosal vaccine for COVID-19, has been cleared by the FDA, with a Phase 1 trial expected to start in Q2 2025 [30][31] Q&A Session Summary Question: Does the runway factor in newly launching studies like OCU410ST Phase 2/3? - Yes, it is already budgeted [42] Question: When can we expect data from the DME program? - Safety and efficacy reports for OCU200 are expected towards the end of the year, focusing on BCVA and dose response [44][47] Question: What gives confidence in achieving the aggressive BLA filing targets? - The track record of starting gene therapy programs and the ongoing progress across all three programs provide confidence [54][56] Question: How will OCU410 compete with existing therapies? - The safety and tolerability profile of OCU410 is a significant advantage, as current therapies have concerning safety profiles [61][64] Question: Will there be grant revenue associated with the OCU500 Phase 1 trial? - NIAID is sponsoring the program, and the company has completed its obligations; funding will not be recorded as revenue [78][81] Question: What is the manufacturing strategy for OCU410ST? - Commercial-scale manufacturing will be used for both Phase 1 and Phase 2/3 studies [85] Question: Will the study include both US and ex-US sites? - The study will primarily be conducted in the US, with Canada as the only ex-US site [87]

Financial Performance and Capital Requirements - The company incurred net losses of approximately $63.1 million and $54.1 million for the years ended December 31, 2023, and 2024, respectively, with an accumulated deficit of $340.2 million as of December 31, 2024[229]. - The cash balance as of December 31, 2024, was approximately $58.5 million, which is insufficient to meet capital requirements over the next 12 months[229]. - The company estimates that its cash and cash equivalents will fund operations into the first quarter of 2026, indicating a need for significant additional capital[229]. - The company anticipates an increase in expenses for fiscal year 2025 compared to fiscal year 2024 due to ongoing clinical activities and increased headcount[232]. - The company may need to raise additional capital to support its product development activities, which could lead to dilution for existing shareholders[230]. - The company has $30.0 million of outstanding principal borrowings under the Loan and Security Agreement as of December 31, 2024[247]. - The company has incurred significant net operating losses, with United States federal net operating loss carryforwards of approximately $263.9 million as of December 31, 2024[254]. - The company is actively seeking funding through various means, including public and private placements, government grants, and collaborations[244]. Product Development and Regulatory Challenges - The company has not generated any revenue from product sales to date and may never attain profitability without sufficient revenues from product candidates[231]. - The company faces significant risks related to the success of its product candidates, including regulatory approval and market acceptance[225]. - The company relies on third parties for conducting clinical trials, which may not perform satisfactorily, potentially delaying development[225]. - The company is focused on developing product candidates from its modifier gene therapy platform, which is subject to an uncertain regulatory environment[263]. - Regulatory approval processes for gene therapy products are lengthy and unpredictable, potentially delaying commercialization and revenue generation[268]. - The company is at risk of delays in clinical trials due to various factors, including regulatory approvals and patient enrollment challenges[270]. - The company must navigate changing regulatory requirements that could lengthen the approval process and increase development costs[265]. - The company must establish agreements with distributors and maintain patent protections to ensure successful commercialization of its product candidates[265]. - The company currently has no product candidates approved for sale in any jurisdiction, including international markets, and lacks experience in obtaining regulatory approval outside the United States[298]. Market and Competitive Landscape - The biotechnology industry is characterized by intense competition, with numerous companies focusing on gene therapies and regenerative medicine[331]. - Competitors may obtain regulatory approval more rapidly, potentially establishing a strong market position before the company can enter the market[336]. - Acceptance of product candidates among physicians and patients may be limited, affecting revenue generation even if approved[339]. - Market acceptance of product candidates is crucial for generating significant revenues and achieving profitability[341]. - Potential market opportunities for product candidates may be smaller than estimated, adversely affecting revenue and business sustainability[342]. - Successful commercialization depends on timely coverage and adequate reimbursement from third-party payors, which is influenced by various factors including efficacy and cost-effectiveness[343]. Manufacturing and Supply Chain Risks - The company does not own manufacturing facilities and faces risks related to production delays, quality control, and compliance with stringent regulations, which could impact revenue generation[363]. - The company relies on third-party manufacturers for clinical and commercial supplies, which may affect future profit margins if agreements are not established on commercially favorable terms[361]. - The number of available, qualified third-party manufacturers is limited, and finding alternatives could lead to delays and increased expenses in product development and commercialization[372]. - The company may encounter shortages in materials necessary for production, which could hinder the ability to meet demand for approved products and result in lost revenues[371]. Legal and Compliance Issues - The company is currently facing numerous securities class action lawsuits, which could divert management's attention and potentially harm its business[383]. - The company may be subject to substantial penalties if it fails to comply with federal and state healthcare laws, adversely affecting its financial condition and results of operations[385]. - Legislative changes, including cost-containment programs and price controls, could negatively impact the company's business and results of operations[387]. - The company may face criminal and civil penalties if it fails to comply with anti-corruption laws, which could adversely impact its financial condition and operations[403]. Intellectual Property and Patent Risks - The patent prosecution process is expensive and time-consuming, and the company may not be able to maintain necessary patent protections for its technologies[404]. - The company may face challenges in identifying patentable aspects of its research and development output, impacting its ability to secure patent protection[406]. - The company’s patent rights may be challenged in courts or patent offices, leading to potential loss of exclusivity or narrowing of patent claims[409]. - The company’s ability to develop and market its product candidates is contingent upon not infringing third-party intellectual property rights, which poses a significant risk[417].