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 - The company will host a conference call and live webcast to discuss its second quarter 2025 financial results and provide a business update on August 1, 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] Business Focus - Ocugen's modifier gene therapies aim to address complex diseases potentially caused by imbalances in multiple gene networks, with current programs in development for inherited retinal diseases and blindness diseases affecting millions globally, including retinitis pigmentosa, Stargardt disease, and geographic atrophy [3]

Core Viewpoint - Carisma Therapeutics Inc. and OrthoCellix, Inc. have announced a definitive merger agreement to combine in an all-stock transaction, focusing on the development of OrthoCellix's NeoCart® technology for knee cartilage defects and planning to initiate a Phase 3 clinical trial endorsed by the FDA [1][2][4]. Company Overview - Carisma Therapeutics is a biotechnology company specializing in macrophage engineering for therapies targeting fibrosis and cancer [11]. - OrthoCellix is a clinical-stage company developing regenerative cell therapies for orthopedic diseases, with its lead program being the NeoCart® technology aimed at repairing knee cartilage defects [10][3]. NeoCart® Technology - NeoCart® is an autologous cartilage implant technology that utilizes patient cells to repair articular cartilage defects, combining a fortified 3D scaffold with patented bioprocessing technology to produce functional cartilage [3][10]. - The technology has received Regenerative Medicine Advanced Therapy (RMAT) designation and FDA concurrence for a single, confirmatory Phase 3 clinical trial, which is anticipated to launch by the end of 2025 [4][6]. Merger Details - Under the merger agreement, OrthoCellix will merge into a wholly-owned subsidiary of Carisma, with OrthoCellix continuing as a subsidiary and the surviving entity [5][6]. - The merger will result in OrthoCellix stockholders owning approximately 90% of the combined company, while existing Carisma stockholders will own about 10%, subject to adjustments based on Carisma's net cash and financing proceeds [6][8]. Financial Aspects - Carisma plans to enter into subscription agreements for private financing with Ocugen and other investors to support the Phase 3 trial of NeoCart® without additional costs to Ocugen [5][6]. - The merger has been unanimously approved by the boards of both companies and is expected to close in the second half of 2025, pending customary closing conditions [8].

Company Overview - Ocugen Inc. is a clinical-stage biotechnology company focused on developing modifier gene therapies for rare inherited retinal diseases that lead to progressive vision loss [1]. Pipeline and Focus - The company is particularly concentrating on therapies for diseases that cause vision impairment, indicating a specialized approach within the biotechnology sector [1].

Core Viewpoint - Ocugen, Inc. has received FDA clearance to initiate a Phase 2/3 pivotal confirmatory trial for OCU410ST, a gene therapy candidate for Stargardt disease, highlighting the urgency for treatment options for patients with this condition [1][2]. Company Overview - Ocugen, Inc. is a biotechnology company focused on developing gene therapies for blindness diseases, utilizing a unique modifier gene therapy platform [10]. Clinical Trial Details - The Phase 2/3 trial will enroll 51 participants with Stargardt disease, with 34 receiving a subretinal injection of OCU410ST and 17 in a control group [3]. - The primary objective is to evaluate the reduction in atrophic lesion size, with secondary endpoints including improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) [3]. Regulatory Designations - OCU410ST has received Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation from the FDA, emphasizing the need for innovative treatments for ABCA4-associated retinopathies [1][4]. Patient Population - Approximately 100,000 patients in the U.S. and Europe, and 1 million globally, are affected by Stargardt disease, which is characterized by complex genetic mutations [4]. Clinical Data - Positive results from the Phase 1 GARDian trial indicated a 48% slower lesion growth at 12 months in treated eyes compared to untreated eyes, and a statistically significant improvement in visual function [6][7]. Future Plans - Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, aiming to file three BLAs over the next three years [5].

Core Insights - Ocugen, Inc. is set to present at the 2025 BIO International Convention, showcasing its innovative gene therapies for blindness diseases [1][2] - The company has recently executed a binding term sheet for exclusive rights to OCU400 in Korea, indicating a strategic partnership aimed at enhancing its market presence [3] - Ocugen's modifier gene therapy platform has achieved significant milestones, including FDA alignment for a pivotal trial and nearing completion of enrollment for the OCU400 Phase 3 clinical trial, with plans to file a Biologics License Application by mid-2026 [3] Company Overview - Ocugen, Inc. specializes in gene therapies for blindness diseases, focusing on inherited retinal diseases and conditions affecting millions globally, such as retinitis pigmentosa and Stargardt disease [6] - The company's modifier gene therapy platform is designed to address complex diseases caused by imbalances in multiple gene networks, distinguishing it from traditional gene therapies [6] Conference Details - Dr. Shankar Musunuri, Chairman and CEO, will present on June 16, 2025, and participate in panel discussions on optimizing clinical outcomes and navigating the regulatory landscape during the convention [4][5]

Core Viewpoint - Ocugen, Inc. has signed a binding term sheet to negotiate a licensing agreement for exclusive rights to its gene therapy OCU400 in Korea, aimed at treating retinitis pigmentosa (RP) [1][4]. Group 1: Licensing Agreement Details - The licensing agreement will provide Ocugen with upfront fees and near-term development milestones totaling up to $11 million [2][8]. - Ocugen will receive sales milestones of $1 million for every $15 million in net sales in Korea, along with a royalty of 25% on net sales generated by its partner [2][8]. - The company will manufacture the commercial supply of OCU400 under a supply agreement [2]. Group 2: Market Opportunity - There are approximately 15,000 individuals in South Korea affected by RP, presenting a significant market opportunity for the partner to lead in gene therapy [3]. - The regional licensing strategy aligns with Ocugen's goal to partner with established companies to maximize patient reach while generating returns for shareholders [4]. Group 3: Development Timeline - Ocugen is advancing OCU400 through Phase 3 clinical development, with a target for filing a Biologics License Application by mid-2026 [5].

Core Insights - Ocugen, Inc. has received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for OCU410ST, aimed at treating ABCA4-associated retinopathies, including Stargardt disease [1][2] - The designation highlights the urgent need for therapeutic options for Stargardt patients, as there are currently no FDA-approved treatments available [2] - Approximately 100,000 individuals in the U.S. and Europe are affected by Stargardt disease, which primarily impacts children [2] Regulatory and Developmental Aspects - If the Priority Review Voucher (PRV) program is reauthorized, Ocugen may receive a PRV, which can be sold for about $100 million or used for expedited review of another product [3] - The company plans to initiate a Phase 2/3 pivotal confirmatory trial for OCU410ST in the coming weeks, with a target for Biologics License Application (BLA) filing in 2027 [4] Product and Technology Overview - OCU410ST employs an AAV delivery platform to deliver the RORA gene, representing a modifier gene therapy approach that addresses multiple pathophysiological pathways linked to Stargardt disease [5] - Stargardt disease is characterized by retinal degeneration and vision loss, primarily affecting the macula, leading to decreased central vision while some peripheral vision may be preserved [6][7] Company Background - Ocugen, Inc. is a biotechnology leader focused on gene therapies for blindness diseases, with a gene-agnostic approach that targets complex diseases caused by imbalances in multiple gene networks [8]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K ___________________________________________________________ CURRENT REPORT Pursuant to Section 13 OR 15 (d) of The Securities Exchange Act of 1934 Date of Report (Date of Earliest Event Reported): May 9, 2025 OCUGEN, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 001-36751 04-3522315 (State or Other Jurisdiction of Incorporation) (Commission File Number) (Address, including zip code, and telephone number, ...

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]