PART I—FINANCIAL INFORMATION [Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited condensed consolidated financial statements for Q1 2025, including balance sheets, statements of operations, and cash flows, highlighting the company's going concern status Condensed Consolidated Balance Sheet Highlights (in thousands) | | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash | $37,800 | $58,514 | | Total current assets | $43,964 | $61,682 | | Total assets | $64,460 | $82,442 | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $16,886 | $21,588 | | Total liabilities | $48,546 | $52,810 | | Total stockholders' equity | $15,914 | $29,632 | | Total liabilities and stockholders' equity | $64,460 | $82,442 | Condensed Consolidated Statements of Operations (in thousands, except per share data) | | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :--- | :--- | :--- | | Collaborative arrangement revenue | $1,481 | $1,014 | | Research and development | $9,529 | $6,826 | | General and administrative | $6,453 | $6,404 | | Loss from operations | ($14,501) | ($12,216) | | Net loss | ($15,350) | ($11,924) | | Net loss per share — basic and diluted | ($0.05) | ($0.05) | Condensed Consolidated Statements of Cash Flows (in thousands) | | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($19,357) | ($10,595) | | Net cash used in investing activities | ($102) | ($2,352) | | Net cash used in financing activities | ($1,245) | ($145) | | Net (decrease) in cash and restricted cash | ($20,712) | ($13,087) | | Cash and restricted cash at end of period | $38,109 | $26,375 | - The company has incurred recurring net losses and its cash of **$37.8 million** as of March 31, 2025, is not sufficient to fund operations for the next 12 months, raising substantial doubt about its ability to continue as a going concern[29](index=29&type=chunk)[31](index=31&type=chunk) - Revenue from the co-development and commercialization agreement with CanSinoBIO was approximately **$1.5 million** for Q1 2025, compared to **$1.0 million** in Q1 2024[69](index=69&type=chunk)[74](index=74&type=chunk) - As of March 31, 2025, the company has total debt of **$31.5 million**, primarily from a Loan and Security Agreement and an EB-5 Loan Agreement, with significant maturities in 2027 and 2028[86](index=86&type=chunk)[88](index=88&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Q1 2025 financial results, highlighting progress across key technology platforms and a wider net loss of **$15.4 million** due to increased R&D expenses, reinforcing liquidity challenges and going concern doubts - The company provided key updates on its clinical pipeline, including progress in Phase 3 for **OCU400 (RP)**, Phase 2 for **OCU410 (dAMD)**, FDA alignment for **OCU410ST (Stargardt)** pivotal trial, and initiation of Phase 1 for **OCU200 (DME/DR/wet AMD)** and **OCU500 (COVID-19 Vaccine)**[117](index=117&type=chunk)[118](index=118&type=chunk) Results of Operations Comparison (in thousands) | | Three months ended March 31, 2025 | Three months ended March 31, 2024 | Change | | :--- | :--- | :--- | :--- | | Collaborative arrangement revenue | $1,481 | $1,014 | $467 | | Research and development | $9,529 | $6,826 | $2,703 | | General and administrative | $6,453 | $6,404 | $49 | | Loss from operations | ($14,501) | ($12,216) | ($2,285) | | Net loss | ($15,350) | ($11,924) | ($3,426) | - R&D expenses increased by **$2.7 million** year-over-year, primarily due to a **$1.3 million** increase in employee-related costs and higher clinical trial activity for **OCU410/410ST ($0.7 million)** and **OCU400 ($0.3 million)**[137](index=137&type=chunk) - As of March 31, 2025, the company had **$37.8 million** in cash and cash equivalents, which management believes will only fund operations into Q1 2026, insufficient for the next 12 months[140](index=140&type=chunk)[152](index=152&type=chunk) - The company requires significant additional funding, exploring options like equity/debt placements, strategic arrangements, and government funding, as inability to secure it raises substantial doubt about its going concern ability[153](index=153&type=chunk)[154](index=154&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company states this item is not applicable, indicating no significant exposure to market risks requiring quantitative and qualitative disclosure - The company states this section is not applicable[159](index=159&type=chunk) [Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - Based on an evaluation as of March 31, 2025, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures are effective[160](index=160&type=chunk) - There were no changes in internal control over financial reporting during the quarter that have materially affected, or are reasonably likely to materially affect, the company's internal control over financial reporting[161](index=161&type=chunk) PART II—OTHER INFORMATION [Legal Proceedings](index=36&type=section&id=Item%201.%20Legal%20Proceedings) The company is defending against a securities class action lawsuit and stockholder derivative lawsuits, believing them without merit and unable to assess the financial impact - The company is defending against a securities class action lawsuit and multiple stockholder derivative lawsuits related to alleged violations of the Exchange Act[107](index=107&type=chunk)[109](index=109&type=chunk) - Management believes the lawsuits are without merit, and as the outcome cannot be determined and a loss is not considered probable or reasonably estimable, no accrual has been recorded[110](index=110&type=chunk) [Risk Factors](index=36&type=section&id=Item%201A.%20Risk%20Factors) This section updates risk factors, emphasizing new or heightened risks from geopolitical events and potential disruptions at U.S. government agencies like the FDA and SEC - Geopolitical issues, including global conflicts, sanctions, and tariffs, could disrupt the supply chain, increase costs for international clinical trials, and adversely affect the business[165](index=165&type=chunk)[166](index=166&type=chunk) - Changes in funding, staffing, or potential shutdowns at government agencies like the FDA and SEC could hinder their ability to review and approve new products in a timely manner, negatively impacting the company's business[167](index=167&type=chunk)[171](index=171&type=chunk) - Uncertainty surrounding the new U.S. presidential administration's policies for the FDA and other regulatory agencies could create new challenges or a more costly environment for therapeutic development[172](index=172&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=37&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities or issuer purchases of its own equity securities during the quarter ended March 31, 2025 - The company confirms there were no unregistered sales of equity securities or issuer purchases of equity securities during the period that were not previously reported[173](index=173&type=chunk) [Defaults Upon Senior Securities](index=37&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports no defaults upon its senior securities during the reporting period - The company reports "None"[174](index=174&type=chunk) [Mine Safety Disclosures](index=37&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) The company states this item is not applicable to its business operations - The company states this section is "Not applicable"[174](index=174&type=chunk) [Other Information](index=37&type=section&id=Item%205.%20Other%20Information) This section discloses a Certificate of Correction for Series C Preferred Stock voting rights and confirms no Rule 10b5-1 trading plan adoptions or terminations by directors or officers - A Certificate of Correction was filed to clarify voting rights of the Series C Preferred Stock, all shares of which were redeemed as of June 28, 2024, and none are outstanding[175](index=175&type=chunk) - During the three months ended March 31, 2025, no directors or officers adopted or terminated a Rule 10b5-1 trading plan or a non-Rule 10b5-1 trading arrangement[178](index=178&type=chunk) [Exhibits](index=39&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Quarterly Report on Form 10-Q, including a Certificate of Correction, various agreements, and required CEO/CAO certifications - The report lists all exhibits filed, including a Certificate of Correction (Exhibit 3.1), various agreements, and required CEO/CAO certifications (Exhibits 31.1, 31.2, 32.1)[179](index=179&type=chunk)

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]

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 ...