Ocugen(OCGN) - 2025 Q2 - Quarterly Results

Business Update and Second Quarter 2025 Financial Results Overview Business Highlights and Strategic Direction Ocugen reported Q2 2025 financial results and business updates, emphasizing late-stage gene therapy trials, partnerships, and restructuring for three BLA filings within three years - Initiated dosing in OCU410ST Phase 2/3 GARDian3 pivotal confirmatory clinical trial[1] - Actively dosing patients in OCU400 Phase 3 liMeliGhT clinical trial and on track for 2026 BLA filing[1] - Proposed OrthoCellix reverse merger aims to unlock NeoCart/regenerative cell therapy value and focus capital on modifier gene therapy platform[1][2] - Signed binding term sheet for exclusive Korean rights to OCU400 with upfront and near-term development milestone payments totaling up to $11 million[1][3] Strategic Business Developments OrthoCellix Reverse Merger Ocugen announced a proposed reverse merger with OrthoCellix and Carisma Therapeutics to establish a Nasdaq-listed regenerative cell therapy company focused on NeoCart® technology for articular knee cartilage defects - Proposed reverse merger with OrthoCellix and Carisma Therapeutics to create a Nasdaq-listed, late clinical-stage regenerative cell therapy company focused on orthopedic diseases[2] - The combined company will develop NeoCart® technology for articular knee cartilage defects, which has RMAT designation and FDA concurrence for a single, confirmatory Phase 3 clinical trial[2] OCU400 Korean Licensing Agreement Ocugen secured a binding term sheet for exclusive Korean rights to OCU400, including upfront and milestone fees, sales milestones, and a 25% royalty on net sales, with Ocugen manufacturing commercial supply - Signed a binding term sheet for exclusive Korean rights to OCU400 with a well-established leader in the pharmaceutical and healthcare sector in Korea[3] - The license agreement includes upfront and milestone fees, sales milestones of $1 million for every $15 million of net sales in Korea, and a royalty of 25% on net sales[3] - Ocugen will manufacture commercial supply of OCU400 under terms of a supply agreement[3] Corporate Leadership and Advocacy Ocugen strengthened its leadership team and hosted the NSCEB and U.S. Rep. Chrissy Houlahan, leading to the announcement of a bipartisan BIOTech Caucus to increase awareness and support for biotechnology - Made important appointments to the Board of Directors, Retina Scientific Advisory Board, and Leadership Team to provide scientific and strategic know-how[2] - Hosted the National Security Commission on Emerging Biotechnology (NSCEB) and U.S. Rep. Chrissy Houlahan at its manufacturing facility, highlighting biotech innovation[9] - Rep. Houlahan subsequently announced the bipartisan BIOTech Caucus to build greater awareness and understanding of biotechnology among lawmakers[9] Clinical Pipeline Progress Modifier Gene Therapy Platform Ocugen's novel first-in-class modifier gene therapy platform is advancing with OCU400 on track for 2026 BLA/MAA filings, OCU410ST initiating Phase 2/3 trials, and OCU410 showing encouraging Phase 1 and interim Phase 2 data - The modifier gene therapy platform is a novel first-in-class platform designed to address complex diseases potentially caused by imbalances in multiple gene networks[10][15] OCU410ST for Stargardt Disease OCU410ST for Stargardt disease received Rare Pediatric Disease Designation from the FDA, and dosing commenced in July for the Phase 2/3 GARDian3 pivotal confirmatory clinical trial, building on positive Phase 1 data - FDA granted Rare Pediatric Disease Designation (RPDD) to OCU410ST in May, underscoring the urgent need to address Stargardt disease, which has no FDA-approved treatment[4][10] - Initiated dosing in the OCU410ST Phase 2/3 GARDian3 pivotal confirmatory clinical trial in July after FDA clearance in June[1][5][10] - Phase 1 GARDian trial demonstrated 48% slower lesion growth at 12-month follow-up and a statistically significant (p=0.031) and clinically meaningful improvement of nearly 2-line/9-letter gain in BCVA in treated eyes[5] OCU410 for Geographic Atrophy (GA) OCU410 for Geographic Atrophy has shown positive preliminary efficacy and safety data from its Phase 1 trial, including slower GA lesion growth and visual acuity gains, supported by encouraging interim Phase 2 results - 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)[6] - Phase 1 data showed 23% slower geographic atrophy (GA) lesion growth in treated eyes versus fellow eyes and a 2-line/10-letter gain in visual acuity[6][10] - Preliminary results from ongoing Phase 2 clinical trial (N=31), 6-month interim analysis, demonstrated 27% slower lesion growth and preservation of retinal tissue[6][10] OCU400 for Retinitis Pigmentosa (RP) The OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa is actively recruiting patients and remains on track for BLA and MAA submissions in 2026, with EMA eligibility granted and DSMB recommending continued dosing - Patients are actively being recruited in the United States and Canada for the OCU400 Phase 3 liMeliGhT clinical trial[7] - The trial remains on track for BLA and MAA submissions in 2026, and the European Medicines Agency has granted eligibility for MAA submission through the centralized procedure[1][7][10] - Data and Safety Monitoring Board (DSMB) convened and found no SAEs related to OCU400 and recommended to continue study dosing as planned[10] Other Clinical Programs Ocugen is advancing other clinical programs, including OCU500, an inhaled vaccine, with a Phase 1 clinical trial expected to begin in Q3 2025, and OCU200, an ophthalmic biologic, slated to complete its Phase 1 clinical trial in H2 2025 OCU500 Inhaled Vaccines The National Institute of Allergy and Infectious Diseases intends to initiate the Phase 1 clinical trial for OCU500, an inhaled vaccine, in the third quarter of 2025 - The National Institute of Allergy and Infectious Diseases (NIAID) intends to initiate the Phase 1 clinical trial for OCU500 in the third quarter of 2025[8] OCU200 Ophthalmic Biologic For OCU200, an ophthalmic biologic product, the DSMB has approved the continuation of dosing in the third cohort, and the company plans to complete the Phase 1 clinical trial in the second half of 2025 - DSMB approved continuation of dosing in the third cohort for OCU200[11] - The Company intends to complete the Phase 1 clinical trial for OCU200 in the second half of 2025[11] Second Quarter 2025 Financial Performance Key Financial Highlights Ocugen's Q2 2025 financial performance showed decreased cash, cash equivalents, and restricted cash compared to year-end 2024, providing a cash runway into Q1 2026, with reduced operating expenses and net loss per common share Cash, Cash Equivalents, and Restricted Cash (in millions) | Metric | June 30, 2025 | December 31, 2024 | | :----- | :------------ | :---------------- | | Cash, Cash Equivalents, and Restricted Cash | $27.3 million | $58.8 million | - The Company's cash runway is projected into the first quarter of 2026[16][17] Total Operating Expenses (Three Months Ended June 30, in millions) | Expense Type | Q2 2025 | Q2 2024 | | :----------- | :------ | :------ | | Total Operating Expenses | $15.2 million | $16.6 million | | Research and Development | $8.4 million | $8.9 million | | General and Administrative | $6.8 million | $7.7 million | Net Loss Per Common Share (Three Months Ended June 30) | Metric | Q2 2025 | Q2 2024 | | :----- | :------ | :------ | | Net Loss Per Common Share | $0.05 | $0.06 | - 292.2 million shares of common stock outstanding as of June 30, 2025[16] Conference Call and Webcast Details Conference Call and Webcast Information Ocugen hosted a conference call and webcast on August 1, 2025, at 8:30 a.m. ET to discuss financial results and business highlights, with participation details provided and a replay available for approximately 45 days - A conference call and webcast were scheduled for August 1, 2025, at 8:30 a.m. ET to discuss financial results and business highlights[1][13] - Dial-in numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers; Conference ID: 9627149[14] - Webcast available on the events section of the Ocugen investor site, with a replay and archived webcast available for approximately 45 days[14] About Ocugen, Inc. Company Profile and Mission Ocugen, Inc. is a pioneering biotechnology company focused on gene therapies for blindness diseases, utilizing its breakthrough modifier gene therapy platform with a gene-agnostic approach to address complex diseases affecting millions globally - Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases[15] - Utilizes a breakthrough modifier gene therapy platform with a gene-agnostic approach to address significant unmet medical needs for large patient populations[15] - Programs are in development for inherited retinal diseases and blindness diseases affecting millions, including retinitis pigmentosa, Stargardt disease, and geographic atrophy[15] Cautionary Note on Forward-Looking Statements Forward-Looking Statements Disclosure This section serves as a cautionary note, indicating that the press release contains forward-looking statements regarding Ocugen's strategy, clinical programs, financial condition, and potential transactions, which are subject to numerous risks and uncertainties - The press release contains forward-looking statements regarding strategy, business plans, clinical programs, financial condition, and anticipated regulatory filings[17] - Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from current expectations[17] - Risks include that preliminary clinical trial results may not be indicative of final data, and that definitive agreements for the Korean license or OrthoCellix merger may be delayed or not executed[17] Contact Information Media and Investor Relations Contact For media and investor inquiries, Tiffany Hamilton, AVP, Head of Communications, is listed as the primary contact for Ocugen - Contact for inquiries: Tiffany Hamilton, AVP, Head of Communications[18] - Email: Tiffany.Hamilton@ocugen.com[18] Condensed Consolidated Financial Statements Condensed Consolidated Balance Sheets The unaudited condensed consolidated balance sheets present Ocugen's financial position as of June 30, 2025, compared to December 31, 2024, detailing assets, liabilities, and stockholders' equity Condensed Consolidated Balance Sheets (in thousands) | Metric | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Assets | | | | Cash | $27,013 | $58,514 | | Prepaid expenses and other current assets | $5,870 | $3,168 | | Total current assets | $32,883 | $61,682 | | Property and equipment, net | $15,445 | $16,554 | | Restricted cash | $312 | $307 | | Other assets | $4,954 | $3,899 | | Total assets | $53,594 | $82,442 | | Liabilities and Stockholders' Equity | | | | Accounts payable | $4,237 | $4,243 | | Accrued expenses and other current liabilities | $12,899 | $15,500 | | Operating lease obligations | $853 | $519 | | Current portion of long term debt | — | $1,326 | | Total current liabilities | $17,989 | $21,588 | | Operating lease obligations, less current portion | $3,945 | $3,313 | | Long term debt, net | $28,025 | $27,345 | | Other non-current liabilities | $583 | $564 | | Total non-current liabilities | $32,553 | $31,222 | | Total liabilities | $50,542 | $52,810 | | Common Stock | $2,924 | $2,915 | | Treasury stock | $(48) | $(48) | | Additional paid-in capital | $370,474 | $366,938 | | Accumulated other comprehensive income | $12 | $48 | | Accumulated deficit | $(370,31) | $(340,221) | | Total stockholders' equity | $3,052 | $29,632 | | Total liabilities and stockholders' equity | $53,594 | $82,442 | Condensed Consolidated Statements of Operations and Comprehensive Loss The unaudited condensed consolidated statements of operations and comprehensive loss provide a detailed breakdown of Ocugen's revenues, operating expenses, and net loss for the three and six months ended June 30, 2025, compared to the same periods in 2024 Condensed Consolidated Statements of Operations and Comprehensive Loss (in thousands, except per share) | Metric | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :------------------------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Collaborative arrangement revenue | $1,373 | $1,141 | $2,854 | $2,155 | | Total revenue | $1,373 | $1,141 | $2,854 | $2,155 | | Research and development expenses | $8,402 | $8,902 | $17,932 | $15,728 | | General and administrative expenses | $6,766 | $7,688 | $13,218 | $14,092 | | Total operating expenses | $15,168 | $16,590 | $31,150 | $29,820 | | Loss from operations | $(13,795) | $(15,449) | $(28,296) | $(27,665) | | Interest (expense) income, net | $(1,058) | $173 | $(1,972) | $475 | | Other (expense) income, net | $114 | $(4) | $179 | $(14) | | Net loss | $(14,739) | $(15,280) | $(30,089) | $(27,204) | | Foreign currency translation adjustment | $(28) | $3 | $(36) | $8 | | Comprehensive loss | $(14,767) | $(15,277) | $(30,125) | $(27,196) | | Net loss attributable to common shareholders— basic and diluted | $(14,739) | $(15,259) | $(30,089) | $(27,157) | | Weighted shares used in calculating net loss per common share — basic and diluted | 292,067,192 | 257,353,857 | 292,032,072 | 257,293,247 | | Net loss per share attributable to common shareholders — basic and diluted | $(0.05) | $(0.06) | $(0.10) | $(0.11) | | Net loss attributable to Series B Convertible Preferred shareholders — basic and diluted | — | $(21) | — | $(47) | | Weighted shares used in calculating net loss per Series B Convertible Preferred Stock — basic and diluted | — | 54,745 | — | 54,745 | | Net loss per share attributable to Series B Convertible Preferred shareholders — basic and diluted | — | $(0.38) | — | $(0.86) |