Core Viewpoint - Ocugen, Inc. has entered into a securities purchase agreement with Janus Henderson Investors to raise capital through a registered direct offering of common stock and warrants, aiming to enhance its financial position for ongoing gene therapy developments for blindness diseases [1][3]. Group 1: Offering Details - The company will sell 20,000,000 shares of common stock at a price of $1.00 per share, with warrants to purchase an additional 20,000,000 shares at an exercise price of $1.50 per share [1][3]. - The gross proceeds from the offering are expected to be approximately $20 million, with potential additional proceeds of up to $30 million if the warrants are fully exercised [3]. - The offering is expected to close on or about August 11, 2025, subject to customary closing conditions [3]. Group 2: Company Background - Ocugen, Inc. is a biotechnology leader focused on gene therapies for blindness diseases, utilizing a modifier gene therapy platform to address complex diseases caused by imbalances in multiple gene networks [5]. - The company is developing programs for inherited retinal diseases and other blindness conditions affecting millions globally, including retinitis pigmentosa and Stargardt disease [5].

[Disclosure Regarding Forward-Looking Statements](index=4&type=section&id=DISCLOSURE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) [Forward-Looking Statements Overview](index=4&type=section&id=Forward-Looking%20Statements%20Overview) The report includes forward-looking statements on strategy and operations, cautioning investors about inherent risks - The report contains forward-looking statements regarding strategy, future operations, financial position, revenues, costs, prospects, plans, and objectives[10](index=10&type=chunk) - Statements involve known and unknown risks, uncertainties, and other important factors that may cause **actual results to differ materially**[10](index=10&type=chunk) - Key forward-looking statements include estimates for expenses, future revenues, capital requirements, and progress of product candidates **OCU400, OCU410, OCU410ST**[11](index=11&type=chunk) - Investors should not place undue reliance on forward-looking statements, as the company does not assume any obligation to update them, except as required by law[12](index=12&type=chunk)[13](index=13&type=chunk) [PART I—FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%E2%80%94FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for the periods ended June 30, 2025 [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20as%20of%20June%2030%2C%202025%20and%20December%2031%2C%202024) Condensed Consolidated Balance Sheets | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | Cash | $27,013 | $58,514 | | Total current assets | $32,883 | $61,682 | | Total assets | $53,594 | $82,442 | | Total current liabilities | $17,989 | $21,588 | | Total liabilities | $50,542 | $52,810 | | Total stockholders' equity | $3,052 | $29,632 | - **Cash decreased significantly** from $58.5 million at December 31, 2024, to **$27.0 million** at June 30, 2025[19](index=19&type=chunk) - Total stockholders' equity decreased from **$29.6 million to $3.1 million**, primarily due to accumulated deficit[19](index=19&type=chunk) [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20three%20and%20six%20months%20ended%20June%2030%2C%202025%20and%202024) Condensed Consolidated Statements of Operations and Comprehensive Loss | Metric | Three months ended June 30, 2025 (in thousands) | Three months ended June 30, 2024 (in thousands) | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :--- | :--- | :--- | :--- | :--- | | Collaborative arrangement revenue | $1,373 | $1,141 | $2,854 | $2,155 | | Total operating expenses | $15,168 | $16,590 | $31,150 | $29,820 | | Net loss | $(14,739) | $(15,280) | $(30,089) | $(27,204) | | Net loss per common share — basic and diluted | $(0.05) | $(0.06) | $(0.10) | $(0.11) | - Collaborative arrangement revenue increased by **$0.2 million (19.9%)** for the three months ended June 30, 2025, and by **$0.7 million (32.4%)** for the six months ended June 30, 2025[21](index=21&type=chunk)[154](index=154&type=chunk)[160](index=160&type=chunk) - Net loss for the six months ended June 30, 2025, was **$(30.1) million**, an increase from $(27.2) million in the prior year, primarily due to increased R&D and interest expenses[21](index=21&type=chunk)[161](index=161&type=chunk)[163](index=163&type=chunk) [Condensed Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity%20for%20the%20three%20and%20six%20months%20ended%20June%2030%2C%202025%20and%202024) Condensed Consolidated Statements of Stockholders' Equity | Metric | December 31, 2024 (in thousands) | June 30, 2025 (in thousands) | | :--- | :--- | :--- | | Common Stock Shares | 291,489,058 | 292,313,561 | | Additional Paid-in Capital | $366,938 | $370,474 | | Accumulated Deficit | $(340,221) | $(370,310) | | Total Stockholders' Equity | $29,632 | $3,052 | - Accumulated deficit increased by approximately **$30.1 million** from December 31, 2024, to June 30, 2025, reflecting the net loss incurred during the period[22](index=22&type=chunk) - Total stockholders' equity decreased significantly from **$29.6 million to $3.1 million** over the six-month period[22](index=22&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20six%20months%20ended%20June%2030%2C%202025%20and%202024) Condensed Consolidated Statements of Cash Flows | Cash Flow Activity | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :--- | :--- | :--- | | Net cash used in operating activities | $(30,087) | $(20,506) | | Net cash used in investing activities | $(190) | $(2,865) | | Net cash used in financing activities | $(1,184) | $(100) | | Net decrease in cash and restricted cash | $(31,497) | $(23,463) | | Cash and restricted cash at end of period | $27,325 | $15,999 | - Net cash used in operating activities increased to **$30.1 million** for the six months ended June 30, 2025, from $20.5 million in the prior year, primarily due to higher net loss[27](index=27&type=chunk)[170](index=170&type=chunk)[171](index=171&type=chunk) - Cash used in financing activities increased to **$1.2 million**, mainly due to the repayment of part of the EB-5 loan[27](index=27&type=chunk)[173](index=173&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) [Note 1. Nature of Business and Going Concern](index=11&type=section&id=1.%20Nature%20of%20Business) The company's recurring net losses and insufficient cash raise substantial doubt about its ability to continue as a going concern - Ocugen is a biotechnology company focused on discovering, developing, and commercializing novel gene therapies[29](index=29&type=chunk) - The company incurred net losses of **$30.1 million** and **$27.2 million** for the six months ended June 30, 2025 and 2024, respectively, and has an accumulated deficit of **$370.3 million**[30](index=30&type=chunk) - Current cash of **$27.0 million** is insufficient to fund operations for the next 12 months, indicating substantial doubt about the company's ability to continue as a going concern[30](index=30&type=chunk)[32](index=32&type=chunk) - Ocugen plans to merge its NeoCart business with Carisma Therapeutics Inc, with Ocugen expected to own **over 50%** of the combined company[33](index=33&type=chunk)[36](index=36&type=chunk)[38](index=38&type=chunk) [Note 2. Summary of Significant Accounting Policies](index=12&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines significant accounting policies, including a revision to EPS calculation for Series B Preferred Stock - The company revised its financial statements to treat Series B Convertible Preferred Stock as a second class of common stock for EPS purposes[40](index=40&type=chunk)[41](index=41&type=chunk) Impact of Revision on Net Loss Attributable to Common Shareholders (Three Months Ended June 30, 2024) | Metric | As Previously Reported (in thousands) | Adjustment (in thousands) | As Adjusted (in thousands) | | :--- | :--- | :--- | :--- | | Net loss attributable to common shareholders | $(10,292) | $(4,967) | $(15,259) | | Net loss per share attributable to common shareholders | $(0.04) | | $(0.06) | - The company adopted ASU 2023-07 on segment reporting in fiscal year 2024 with no impact on reportable segments[69](index=69&type=chunk)[70](index=70&type=chunk)[71](index=71&type=chunk)[72](index=72&type=chunk) Cash and Restricted Cash Reconciliation (in thousands) | Category | As of June 30, 2025 | As of June 30, 2024 | | :--- | :--- | :--- | | Cash | $27,013 | $15,697 | | Restricted cash | $312 | $302 | | Total cash and restricted cash | $27,325 | $15,999 | [Note 3. License and Development Agreements](index=18&type=section&id=3.%20License%20and%20Development%20Agreements) Ocugen has a co-development and commercialization agreement with CanSinoBIO for its modifier gene therapy product candidates - Ocugen has a co-development and commercialization agreement with CanSinoBIO for modifier gene therapy product candidates **OCU400, OCU410, and OCU410ST**[73](index=73&type=chunk) - CanSinoBIO is responsible for manufacturing in its territory (China, Hong Kong, Macau, Taiwan), while Ocugen holds exclusive rights elsewhere[73](index=73&type=chunk) - Collaborative arrangement revenue was approximately **$2.9 million** for the six months ended June 30, 2025, compared to $2.2 million for the same period in 2024[78](index=78&type=chunk) - The contract liability related to these arrangements was **$6.6 million** as of June 30, 2025, down from $8.8 million as of June 30, 2024[79](index=79&type=chunk) [Note 4. Fair Value Measurements](index=19&type=section&id=4.%20Fair%20Value%20Measurements) The fair value of the company's debt instruments approximates their carrying value based on Level 2 inputs - The fair value of borrowings under the EB-5 Loan Agreement and Loan and Security Agreement approximates their carrying value, using **Level 2 inputs**[80](index=80&type=chunk) [Note 5. Property and Equipment](index=19&type=section&id=5.%20Property%20and%20Equipment) Net property and equipment decreased to $15.4 million at June 30, 2025, from $16.6 million at year-end 2024 Property and Equipment, Net | Category | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | Furniture and fixtures | $455 | $433 | | Machinery and equipment | $3,235 | $3,192 | | Leasehold improvements | $16,089 | $16,089 | | Total property and equipment, net | $15,445 | $16,554 | - Total property and equipment, net, decreased by approximately **$1.1 million** from December 31, 2024, to June 30, 2025[81](index=81&type=chunk) [Note 6. Operating Leases](index=20&type=section&id=6.%20Operating%20Leases) The company's future minimum lease payments total $6.3 million, with a present value of $4.8 million - The company leases office, laboratory, and manufacturing space, with its Malvern, Pennsylvania GMP facility lease including an option to extend for up to five years[82](index=82&type=chunk) Future Minimum Base Rent Payments | For the years ending December 31, | Amount (in thousands) | | :--- | :--- | | Remainder of 2025 | $642 | | 2026 | $1,259 | | 2027 | $1,197 | | 2028 | $1,216 | | 2029 | $985 | | 2030 | $387 | | Thereafter | $661 | | Total | $6,347 | | Less: present value adjustment | $(1,549) | | Present value of minimum lease payments | $4,798 | [Note 7. Accrued Expenses and Other Current Liabilities](index=20&type=section&id=7.%20Accrued%20Expenses%20and%20Other%20Current%20Liabilities) Accrued expenses and other current liabilities decreased to $12.9 million, driven by lower deferred revenue and employee-related expenses Accrued Expenses and Other Current Liabilities | Category | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--- | :--- | :--- | | Research and development | $250 | $160 | | Clinical | $388 | $740 | | Professional fees | $1,153 | $977 | | Employee-related | $1,671 | $2,433 | | Deferred revenue relating to collaborative arrangements | $6,603 | $8,368 | | Other | $2,834 | $2,822 | | Total accrued expenses and other current liabilities | $12,899 | $15,500 | - Deferred revenue from collaborative arrangements decreased by **$1.8 million**, and employee-related accrued expenses decreased by **$0.8 million**[84](index=84&type=chunk) [Note 8. Debt](index=20&type=section&id=8.%20Debt) The company's debt includes an EB-5 Loan and a Loan and Security Agreement with a variable interest rate - The EB-5 Loan Agreement had **$1.5 million** principal outstanding as of June 30, 2025, down from $2.5 million at December 31, 2024[90](index=90&type=chunk) - The Loan and Security Agreement, entered in November 2024, provided **$29.2 million** in net proceeds, with a **$30.0 million** principal outstanding as of June 30, 2025[91](index=91&type=chunk) - The Loan and Security Agreement matures on November 1, 2028, with the first 24 months being interest-only, and bears interest at a variable rate (greater of prime + 4.25% or 12.25%)[90](index=90&type=chunk) - Lenders have the right to convert up to **$6.0 million** of outstanding principal into common stock at 80% of the trading price on the conversion date[90](index=90&type=chunk) Scheduled Debt Maturities (in thousands) | For the years ending December 31, | Total Maturities | | :--- | :--- | | 2025 | $0 | | 2026 | $1,250 | | 2027 | $16,000 | | 2028 | $13,750 | | 2029 and thereafter | $500 | | Total Debt | $31,500 | [Note 9. Equity](index=22&type=section&id=9.%20Equity) The company raised $32.3 million in net proceeds from a public offering and increased its authorized capital stock - In July 2024, Ocugen issued and sold **30.4 million shares** of common stock at $1.15 per share in a public offering, generating **$32.3 million** in net proceeds[94](index=94&type=chunk) - An additional **$2.4 million** in net proceeds was received in August 2024 from the underwriter's exercise of its option to purchase 2.3 million shares[94](index=94&type=chunk) - In July 2024, the company's authorized capital stock was increased to **400 million shares** (390 million common, 10 million preferred)[95](index=95&type=chunk) - The Series B Convertible Preferred Stock, previously issued to Bharat Biotech, was redeemed in May 2024 through a Stock Forfeiture Agreement[98](index=98&type=chunk) [Note 10. Warrants](index=23&type=section&id=10.%20Warrants) As of June 30, 2025, the company had 0.6 million warrants outstanding with a weighted average exercise price of $6.23 - As of June 30, 2025, **0.6 million warrants** were outstanding[98](index=98&type=chunk) - The outstanding warrants had a weighted average exercise price of **$6.23 per share** and expire between 2026 and 2027[98](index=98&type=chunk) [Note 11. Stock-Based Compensation](index=23&type=section&id=11.%20Stock-Based%20Compensation) Stock-based compensation expense was $3.7 million for the six months ended June 30, 2025, with $12.5 million unrecognized Stock-Based Compensation Expense | Expense Category | Three months ended June 30, 2025 (in thousands) | Three months ended June 30, 2024 (in thousands) | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :--- | :--- | :--- | :--- | :--- | | General and administrative | $1,104 | $1,333 | $2,203 | $2,649 | | Research and development | $741 | $565 | $1,527 | $1,010 | | Total | $1,845 | $1,898 | $3,730 | $3,659 | - As of June 30, 2025, the company had **$12.5 million** of unrecognized stock-based compensation expense, expected to be recognized over a weighted-average period of **2.2 years**[99](index=99&type=chunk) Stock Option Activity (June 30, 2025) | Metric | Number of Shares | Weighted Average Exercise Price | | :--- | :--- | :--- | | Options outstanding at Dec 31, 2024 | 16,197,148 | $2.01 | | Granted | 7,585,836 | $0.90 | | Exercised | (158,164) | $0.44 | | Forfeited | (391,490) | $2.36 | | Expired | (21,573) | $0.93 | | Options outstanding at June 30, 2025 | 23,211,757 | $1.65 | PSUs Unvested Activity (June 30, 2025) | Metric | Number of Shares | Weighted Average Grant-Date Fair Value | | :--- | :--- | :--- | | PSUs unvested at Dec 31, 2024 | 872,352 | $1.71 | | Granted | 3,314,445 | $1.58 | | PSUs unvested at June 30, 2025 | 4,186,797 | $1.61 | [Note 12. Net Loss Per Share of Common Stock](index=25&type=section&id=12.%20Net%20Loss%20Per%20Share%20of%20Common%20Stock) Net loss per common share was $(0.10) for the six months ended June 30, 2025, with potentially dilutive securities excluded Net Loss Per Share Calculation | 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 | | :--- | :--- | :--- | :--- | :--- | | Net loss attributable to common shareholders | $(14,739) | $(15,259) | $(30,089) | $(27,157) | | Weighted shares used in calculating net loss per common share | 292,067,192 | 257,353,857 | 292,032,072 | 257,293,247 | | Net loss per share attributable to common shareholders | $(0.05) | $(0.06) | $(0.10) | $(0.11) | - Potentially dilutive securities totaling **28.9 million** as of June 30, 2025 were excluded from diluted EPS calculation due to their antidilutive effect[109](index=109&type=chunk) [Note 13. Commitments and Contingencies](index=26&type=section&id=13.%20Commitments%20and%20Contingencies) The company is involved in legal proceedings, including a dismissed securities class action and several stockholder derivative lawsuits - Commitments include license and development agreements, lease obligations, and debt agreements[110](index=110&type=chunk) - A securities class action lawsuit filed in April 2024 was **dismissed with prejudice** in July 2025, though plaintiffs have 30 days to appeal[111](index=111&type=chunk) - Multiple stockholder derivative lawsuits are stayed pending resolution of the securities class action motion to dismiss[112](index=112&type=chunk) - The company believes the lawsuits are without merit and intends to vigorously defend, with no current assessment of likely outcome or material loss[114](index=114&type=chunk) [Note 14. Segment Reporting](index=27&type=section&id=14.%20Segment%20Reporting) Ocugen operates as a single operating and reportable segment focused on gene therapies - Ocugen operates as **one operating and reportable segment** focused on gene therapies, with revenue from the CanSinoBIO co-development agreement[115](index=115&type=chunk) - The CEO, as CODM, reviews financial information and uses net loss at the consolidated level for resource allocation and performance assessment[116](index=116&type=chunk) Segment Profit or Loss and Significant Expenses (Six Months Ended June 30, in thousands) | Metric | 2025 | 2024 | | :--- | :--- | :--- | | Collaborative arrangement revenue | $2,854 | $2,155 | | Total research and development | $17,932 | $15,728 | | General and administrative | $13,218 | $14,092 | | Loss from operations | $(28,296) | $(27,665) | | Segment and consolidated net loss | $(30,089) | $(27,204) | [Note 15. Subsequent Events](index=28&type=section&id=15.%20Subsequent%20Events) The company received and subsequently regained compliance with the Nasdaq minimum bid price requirement in July 2025 - On July 1, 2025, Ocugen received a Nasdaq notice for non-compliance with the **minimum bid price requirement** ($1.00 per share)[120](index=120&type=chunk) - The company **regained compliance** by July 28, 2025, by maintaining a minimum closing bid price of $1.00 or more for ten consecutive business days[120](index=120&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=29&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial results, pipeline progress, and liquidity challenges, emphasizing the need for additional funding [Overview](index=29&type=section&id=Overview) Ocugen is a biotechnology company developing a pipeline of gene therapies, biologic therapies, and vaccines - **OCU400** is in a Phase 3 trial for Retinitis Pigmentosa, with BLA/MAA filings anticipated in 2026[123](index=123&type=chunk)[137](index=137&type=chunk)[140](index=140&type=chunk) - **OCU410ST** initiated dosing in a pivotal trial for Stargardt disease, targeting a BLA submission in 2027[124](index=124&type=chunk)[143](index=143&type=chunk) - **OCU410** completed dosing in a Phase 1/2 trial for geographic atrophy, showing positive preliminary data[125](index=125&type=chunk)[148](index=148&type=chunk) - **OCU200** dosed its first patient in a Phase 1 trial for DME, DR, and wet AMD, with completion expected in H2 2025[126](index=126&type=chunk)[149](index=149&type=chunk)[150](index=150&type=chunk) - **NeoCart** is a Phase 3-ready regenerative medicine cell therapy for knee cartilage injuries[131](index=131&type=chunk)[151](index=151&type=chunk) - **OCU500**, an inhaled mucosal vaccine for COVID-19, is expected to begin a Phase 1 trial in Q3 2025[131](index=131&type=chunk)[152](index=152&type=chunk) [Recent Events](index=30&type=section&id=Recent%20Events) The company announced a planned merger of its NeoCart business and a licensing term sheet for OCU400 in Korea - Ocugen entered into a Merger Agreement on June 22, 2025, to merge its **NeoCart** product candidate business with Carisma Therapeutics Inc[128](index=128&type=chunk) - Ocugen is estimated to own **more than 50%** of the combined company and will continue to consolidate OrthoCellix, with the merger expected to close in Q4 2025[130](index=130&type=chunk)[134](index=134&type=chunk) - Ocugen entered a binding term sheet for exclusive Korean rights to OCU400, expecting up to **$11 million** in upfront/near-term milestones and a **25% royalty** on net sales[135](index=135&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Net loss decreased for the three-month period but increased for the six-month period ended June 30, 2025 Financial Performance (Three Months Ended June 30, in thousands) | Metric | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | Collaborative revenue | $1,373 | $1,141 | $232 | | Research and development | $8,402 | $8,902 | $(500) | | General and administrative | $6,766 | $7,688 | $(922) | | Net loss | $(14,739) | $(15,280) | $541 | - Research and development expense decreased by **$0.5 million** for the three months ended June 30, 2025, due to a decrease in OCU500 preclinical activities[155](index=155&type=chunk) - General and administrative expense decreased by **$0.9 million** for the three months, mainly due to reduced professional services fees[156](index=156&type=chunk) Financial Performance (Six Months Ended June 30, in thousands) | Metric | 2025 | 2024 | Change | | :--- | :--- | :--- | :--- | | Collaborative revenue | $2,854 | $2,155 | $699 | | Research and development | $17,932 | $15,728 | $2,204 | | General and administrative | $13,218 | $14,092 | $(874) | | Net loss | $(30,089) | $(27,204) | $(2,885) | - Research and development expense increased by **$2.2 million** for the six months, primarily due to increased headcount-related expenses[161](index=161&type=chunk) - Interest (expense) income, net, decreased by **$2.5 million** for the six months, due to interest expense on long-term debt leveraged in Q4 2024[163](index=163&type=chunk) [Liquidity and Capital Resources](index=36&type=section&id=Liquidity%20and%20Capital%20Resources) The company's current cash is insufficient to fund operations for the next 12 months, raising going concern doubts - As of June 30, 2025, Ocugen had **$27.0 million in cash**, which is insufficient to fund operations for the next 12 months[165](index=165&type=chunk)[177](index=177&type=chunk)[178](index=178&type=chunk) - Since inception through June 30, 2025, the company raised **$368.9 million**, primarily from equity sales[165](index=165&type=chunk) - In November 2024, Ocugen secured **$29.2 million** in net proceeds from a Loan and Security Agreement[166](index=166&type=chunk) - In July 2024, a public offering generated **$34.7 million** in net proceeds from the sale of 32.7 million common shares[167](index=167&type=chunk) - Net cash used in operating activities was **$30.1 million** for the six months ended June 30, 2025, compared to $20.5 million in the prior year[169](index=169&type=chunk)[170](index=170&type=chunk)[171](index=171&type=chunk) - Future funding requirements are significant and depend on clinical trial progress, regulatory approvals, and commercialization costs[175](index=175&type=chunk)[179](index=179&type=chunk) - The company is exploring various funding strategies, including equity/debt placements and strategic arrangements, but there is no assurance of success[177](index=177&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section states that there are no applicable quantitative and qualitative disclosures about market risk - This item is not applicable[184](index=184&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2025 - As of June 30, 2025, the company's disclosure controls and procedures were deemed **effective** by management[185](index=185&type=chunk) - **No material changes** in internal control over financial reporting occurred during the three months ended June 30, 2025[186](index=186&type=chunk) [PART II—OTHER INFORMATION](index=40&type=section&id=PART%20II%E2%80%94OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=40&type=section&id=Item%201.%20Legal%20Proceedings) This section refers to Note 13 for a discussion of legal proceedings, including class action and derivative lawsuits - For a discussion of legal proceedings, refer to Note 13 in the notes to the condensed consolidated financial statements[188](index=188&type=chunk) [Item 1A. Risk Factors](index=40&type=section&id=Item%201A.%20Risk%20Factors) This section updates risk factors, highlighting new risks related to the planned merger with Carisma Therapeutics Inc - No material changes to risk factors were reported, except as set forth in this section[189](index=189&type=chunk) - Risks associated with the planned merger with Carisma Therapeutics Inc include the possibility of **non-completion**, which could lead to significant costs and negative publicity[190](index=190&type=chunk)[192](index=192&type=chunk)[193](index=193&type=chunk) - Potential lawsuits arising from the proposed merger could **delay or prevent its consummation** and incur significant costs[194](index=194&type=chunk) - **Geopolitical events**, global conflicts, and trade policies could adversely affect business, supply chain, and clinical trial costs[195](index=195&type=chunk)[196](index=196&type=chunk)[197](index=197&type=chunk) - Changes in funding or disruptions at government agencies like the **FDA and SEC** could hinder product development timelines and business operations[198](index=198&type=chunk)[201](index=201&type=chunk)[202](index=202&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=42&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales or un-reported purchases of equity securities occurred during the period - No unregistered sales of equity securities or un-reported purchases of equity securities occurred during the period[203](index=203&type=chunk) [Item 3. Defaults Upon Senior Securities](index=42&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - There were no defaults upon senior securities[204](index=204&type=chunk) [Item 4. Mine Safety Disclosures](index=42&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - This item is not applicable[205](index=205&type=chunk) [Item 5. Other Information](index=42&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted or terminated Rule 10b5-1 trading plans during the quarter - No directors or officers adopted or terminated Rule 10b5-1 or non-Rule 10b5-1 trading plans during the three months ended June 30, 2025[206](index=206&type=chunk) [Item 6. Exhibits](index=43&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the report, including merger agreements, certifications, and XBRL documents - Exhibits include the Third Amendment to the Exclusive License Agreement, the Agreement and Plan of Merger with Carisma Therapeutics Inc, and various certifications[207](index=207&type=chunk) [Signatures](index=44&type=section&id=Signatures) The report is duly signed by the Principal Executive Officer and Principal Financial Officer as of August 4, 2025 - The report is signed by Shankar Musunuri, Ph.D., MBA, Chairman, CEO, & Co-Founder (Principal Executive Officer), and Ramesh Ramachandran, CPA, MBA, CMA, Chief Accounting Officer (Principal Financial Officer)[213](index=213&type=chunk) - Signatures are dated **August 4, 2025**[213](index=213&type=chunk)

Core Viewpoint - Ocugen reported strong Q2 2025 results with revenue exceeding analyst estimates, but ongoing cash burn and losses indicate a need for future funding to sustain development [1][5][11] Financial Performance - GAAP revenue for Q2 2025 was $1.373 million, significantly higher than the $0.47 million analyst estimate, and up 27.3% from $1.1 million in Q2 2024 [2][5] - Net loss per share (GAAP) narrowed to $(0.05), better than the expected $(0.06) and last year's $(0.06) [2][5] - Total operating expenses decreased by 8.4% year over year to $15.2 million, with R&D expenses down 5.6% to $8.4 million and general & administrative expenses down 11.7% to $6.8 million [2][5] - Cash, cash equivalents, and restricted cash at the end of the period were $27.3 million, a decline of 53.6% from $58.8 million at the end of 2024 [2][5] Business Focus and Developments - Ocugen is focused on gene therapy for vision-threatening diseases, targeting conditions like retinitis pigmentosa and age-related macular degeneration [3][4] - The company is also advancing an inhaled vaccine platform for respiratory diseases, with a candidate selected for Phase 1 clinical trials [4][9] - Recent strategic moves include a merger of its OrthoCellix subsidiary with Carisma Therapeutics to create a new cell therapy company focused on knee cartilage repair [7] Clinical Progress - The company made significant progress in its clinical trials, including patient enrollment in the Phase 3 "liMeliGhT" trial for OCU400 and the initiation of Phase 2/3 trials for OCU410ST [6] - Interim results from OCU410 showed a 27% slower lesion growth in age-related macular degeneration, supporting future regulatory submissions [6] Partnerships and Licensing - Ocugen secured a binding term sheet for exclusive rights to OCU400 in Korea, which includes up to $11 million in milestone payments and royalties [8] - Licensing agreements are crucial for accessing non-dilutive capital, aiding in the company's financial strategy [8] Future Guidance - Management reiterated expectations to file three major marketing applications by 2028, with the OCU400 BLA filing targeted for 2026 [10] - Existing cash is projected to last into early 2026, necessitating close monitoring of cash burn and funding strategies [11]

[Business Update and Second Quarter 2025 Financial Results Overview](index=1&type=section&id=Business%20Update%20and%20Second%20Quarter%202025%20Financial%20Results%20Overview) [Business Highlights and Strategic Direction](index=1&type=section&id=Business%20Highlights%20and%20Strategic%20Direction) 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](index=1&type=chunk) - Actively dosing patients in **OCU400 Phase 3 liMeliGhT clinical trial** and on track for **2026 BLA filing**[1](index=1&type=chunk) - Proposed OrthoCellix reverse merger aims to unlock NeoCart/regenerative cell therapy value and focus capital on modifier gene therapy platform[1](index=1&type=chunk)[2](index=2&type=chunk) - Signed binding term sheet for exclusive Korean rights to OCU400 with upfront and near-term development milestone payments totaling up to **$11 million**[1](index=1&type=chunk)[3](index=3&type=chunk) [Strategic Business Developments](index=1&type=section&id=Strategic%20Business%20Developments) [OrthoCellix Reverse Merger](index=1&type=section&id=OrthoCellix%20Reverse%20Merger) 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](index=2&type=chunk) - 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](index=2&type=chunk) [OCU400 Korean Licensing Agreement](index=1&type=section&id=OCU400%20Korean%20Licensing%20Agreement) 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](index=3&type=chunk) - 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](index=3&type=chunk) - Ocugen will manufacture commercial supply of OCU400 under terms of a supply agreement[3](index=3&type=chunk) [Corporate Leadership and Advocacy](index=2&type=section&id=Corporate%20Leadership%20and%20Advocacy) 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](index=2&type=chunk) - Hosted the National Security Commission on Emerging Biotechnology (NSCEB) and U.S. Rep. Chrissy Houlahan at its manufacturing facility, highlighting biotech innovation[9](index=9&type=chunk) - Rep. Houlahan subsequently announced the bipartisan BIOTech Caucus to build greater awareness and understanding of biotechnology among lawmakers[9](index=9&type=chunk) [Clinical Pipeline Progress](index=1&type=section&id=Clinical%20Pipeline%20Progress) [Modifier Gene Therapy Platform](index=2&type=section&id=Modifier%20Gene%20Therapy%20Platform) 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](index=10&type=chunk)[15](index=15&type=chunk) [OCU410ST for Stargardt Disease](index=1&type=section&id=OCU410ST%20for%20Stargardt%20Disease) 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](index=4&type=chunk)[10](index=10&type=chunk) - Initiated dosing in the **OCU410ST Phase 2/3 GARDian3 pivotal confirmatory clinical trial** in July after FDA clearance in June[1](index=1&type=chunk)[5](index=5&type=chunk)[10](index=10&type=chunk) - 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](index=5&type=chunk) [OCU410 for Geographic Atrophy (GA)](index=2&type=section&id=OCU410%20for%20Geographic%20Atrophy%20(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](index=6&type=chunk) - 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](index=6&type=chunk)[10](index=10&type=chunk) - 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](index=6&type=chunk)[10](index=10&type=chunk) [OCU400 for Retinitis Pigmentosa (RP)](index=2&type=section&id=OCU400%20for%20Retinitis%20Pigmentosa%20(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](index=7&type=chunk) - 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](index=1&type=chunk)[7](index=7&type=chunk)[10](index=10&type=chunk) - Data and Safety Monitoring Board (DSMB) convened and found **no SAEs related to OCU400** and recommended to continue study dosing as planned[10](index=10&type=chunk) [Other Clinical Programs](index=2&type=section&id=Other%20Clinical%20Programs) 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](index=2&type=section&id=OCU500%20Inhaled%20Vaccines) 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](index=8&type=chunk) [OCU200 Ophthalmic Biologic](index=3&type=section&id=OCU200%20Ophthalmic%20Biologic) 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](index=11&type=chunk) - The Company intends to complete the **Phase 1 clinical trial for OCU200** in the **second half of 2025**[11](index=11&type=chunk) [Second Quarter 2025 Financial Performance](index=3&type=section&id=Second%20Quarter%202025%20Financial%20Performance) [Key Financial Highlights](index=3&type=section&id=Key%20Financial%20Highlights) 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](index=16&type=chunk)[17](index=17&type=chunk) 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](index=16&type=chunk) [Conference Call and Webcast Details](index=3&type=section&id=Conference%20Call%20and%20Webcast%20Details) [Conference Call and Webcast Information](index=3&type=section&id=Conference%20Call%20and%20Webcast%20Information) 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](index=1&type=chunk)[13](index=13&type=chunk) - Dial-in numbers: **(800) 715-9871** for U.S. callers and **(646) 307-1963** for international callers; Conference ID: **9627149**[14](index=14&type=chunk) - Webcast available on the events section of the Ocugen investor site, with a replay and archived webcast available for approximately **45 days**[14](index=14&type=chunk) [About Ocugen, Inc.](index=3&type=section&id=About%20Ocugen%2C%20Inc.) [Company Profile and Mission](index=3&type=section&id=Company%20Profile%20and%20Mission) 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](index=15&type=chunk) - Utilizes a breakthrough **modifier gene therapy platform** with a gene-agnostic approach to address significant unmet medical needs for large patient populations[15](index=15&type=chunk) - Programs are in development for inherited retinal diseases and blindness diseases affecting millions, including retinitis pigmentosa, Stargardt disease, and geographic atrophy[15](index=15&type=chunk) [Cautionary Note on Forward-Looking Statements](index=4&type=section&id=Cautionary%20Note%20on%20Forward-Looking%20Statements) [Forward-Looking Statements Disclosure](index=4&type=section&id=Forward-Looking%20Statements%20Disclosure) 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](index=17&type=chunk) - 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](index=17&type=chunk) - 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](index=17&type=chunk) [Contact Information](index=4&type=section&id=Contact%20Information) [Media and Investor Relations Contact](index=4&type=section&id=Media%20and%20Investor%20Relations%20Contact) 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](index=18&type=chunk) - Email: **Tiffany.Hamilton@ocugen.com**[18](index=18&type=chunk) [Condensed Consolidated Financial Statements](index=5&type=section&id=Condensed%20Consolidated%20Financial%20Statements) [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) 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](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) 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) |

Company Performance - Ocugen reported a quarterly loss of $0.05 per share, which was better than the Zacks Consensus Estimate of a loss of $0.06, representing an earnings surprise of +16.67% [1] - The company posted revenues of $1.37 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 1.93%, compared to revenues of $1.14 million a year ago [2] - Over the last four quarters, Ocugen has surpassed consensus EPS estimates two times but has not beaten consensus revenue estimates [2] Stock Movement and Outlook - Ocugen shares have increased by approximately 28% since the beginning of the year, outperforming the S&P 500's gain of 7.8% [3] - The future performance of Ocugen's stock will largely depend on management's commentary during the earnings call and the company's earnings outlook [4][6] - The current consensus EPS estimate for the upcoming quarter is -$0.06 on revenues of $1.2 million, and for the current fiscal year, it is -$0.23 on revenues of $2.86 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Ocugen belongs, is currently in the top 40% of over 250 Zacks industries, indicating a favorable outlook [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Ocugen's stock performance [5]

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 [23] - Total operating expenses for the three months ended June 30, 2025, were $15.2 million, compared to $16.6 million for the same period in 2024 [23] Business Line Data and Key Metrics Changes - The OCU400 Phase three Limelight clinical trial for retinitis pigmentosa is currently recruiting patients in the United States and Canada, with BLA and MAA filings expected in 2026 [9] - The OCU410 ST clinical trial for Stargardt disease has achieved key milestones, including the first patient dosing and rare pediatric disease designation [12][13] - Preliminary data for OCU410 ST shows a 23% slower lesion growth compared to untreated eyes after a single subretinal injection [15] Market Data and Key Metrics Changes - The OCU400 gene 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 [11] - Stargardt disease affects around 100,000 individuals in the US and Europe, with no FDA-approved treatment available [12] Company Strategy and Development Direction - The company aims to file three biological licensing applications and market authorization applications in the next three years [7] - A regional partnership for OCU400 has been signed, with plans to close the definitive agreement by September [16] - The proposed reverse merger with OrthoCelix is intended to create a NASDAQ-listed late clinical stage regenerative cell therapy company [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 recent collaborations [7][9] - The company is actively exploring strategic partnerships to enhance funding and minimize shareholder dilution [56] Other Important Information - The Data and Safety Monitoring Board reported no serious adverse events related to OCU400, allowing the study to continue as planned [12] - Leadership changes were made to strengthen the company's internal expertise and critical functions [20][21] 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 [28] Question: Can you remind us how many sites are included in the Stargardt Phase three trial? - There are 15 centers activated for the Stargardt trial, and the company does not anticipate challenges in enrolling patients due to the lack of approved products [33][34] Question: Can you talk about the potential interim update for OCU410? - An interim analysis for the geographic atrophy trial is expected in the fourth quarter, providing structural and functional outcomes [43] Question: When will we get feedback from the EMA regarding the pivotal study? - Feedback from the EMA regarding Stargardt is expected by the fourth quarter of this year [46] Question: How does the company plan to bring in additional funds? - The company is working on potential business development opportunities and partnerships to bring in non-dilutive funding [56]

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]

Core Viewpoint - Ocugen, Inc. is advancing its gene therapy programs for blindness diseases, with significant clinical trial progress and strategic partnerships aimed at supporting future Biologics License Application (BLA) filings [2][11]. Business Development - The company is focused on securing strategic partnerships, including a licensing agreement for OCU400 in Korea, which includes sales milestones of $1 million for every $15 million in net sales and a 25% royalty on net sales [3]. - A proposed reverse merger with OrthoCellix and Carisma Therapeutics aims to create a Nasdaq-listed regenerative cell therapy company, enhancing focus on orthopedic diseases and the NeoCart technology [2]. Clinical Trials and Designations - The FDA has granted Rare Pediatric Disease Designation (RPDD) to OCU410ST for Stargardt disease, highlighting the unmet medical need for this condition affecting approximately 100,000 people in the U.S. and Europe [4]. - The OCU410ST Phase 2/3 GARDian3 clinical trial has commenced, building on positive results from the Phase 1 trial, which showed a 48% slower lesion growth in treated eyes [5]. - Preliminary data from the OCU410 Phase 1 ArMaDa trial indicated a 23% slower geographic atrophy lesion growth and a 2-line/10-letter gain in visual acuity [6]. Financial Performance - As of June 30, 2025, the company reported cash and cash equivalents of $27.3 million, down from $58.8 million at the end of 2024, providing a cash runway into the first quarter of 2026 [16]. - Total operating expenses for Q2 2025 were $15.2 million, a decrease from $16.6 million in Q2 2024, with research and development expenses at $8.4 million [16][25]. - The company reported a net loss of $0.05 per common share for Q2 2025, compared to a net loss of $0.06 per common share in the same period of 2024 [16][26].

Core Insights - Ocugen, Inc. is enhancing its Retina Scientific Advisory Board and Executive Leadership Team to strengthen its expertise and guidance as it aims for three Biologics License Applications (BLAs) in the next three years [1][2] Leadership and Advisory Board Updates - The company has appointed three renowned retinal surgeons to its Retina Scientific Advisory Board to provide innovative therapeutic options for patients with serious retinal diseases [2][3][4][5] - New members include Dr. Jeffrey S. Heier, Dr. Peter K. Kaiser, and Dr. Arshad M. Khanani, who bring extensive experience in retinal research and clinical trials [3][4][5][6] - Dr. Lejla Vajzovic continues as the SAB chair, alongside existing members Dr. David S. Boyer and Dr. Carl D. Regillo [6] Strategic Appointments - Vijay Tammara, PhD, has been appointed as Chief Development Officer, bringing over 32 years of regulatory experience and a track record of successful marketing authorizations [8][9] - Abhi Gupta, MBA, has been named Executive Vice President of Commercial and Business Development, with over 20 years of experience in gene therapy and corporate development [10][11] Company Focus and Goals - Ocugen aims to optimize its R&D and clinical efforts while pursuing strategic partnerships and developing a commercial strategy [7] - The company is focused on delivering paradigm-changing gene therapies for inherited retinal diseases and other blindness conditions affecting millions globally [12]

Core Viewpoint - Ocugen, Inc. has initiated dosing for the first patient in its Phase 2/3 GARDian3 clinical trial for OCU410ST, a gene therapy aimed at treating Stargardt disease, marking a significant milestone in addressing the unmet medical needs of patients suffering from this condition [1][2]. Company Overview - Ocugen, Inc. is a biotechnology company focused on developing gene therapies for blindness diseases, aiming to provide innovative solutions for patients globally [9]. Clinical Trial Details - The Phase 2/3 GARDian3 trial will enroll 51 participants diagnosed with Stargardt disease, with 34 receiving a one-time subretinal injection of OCU410ST and 17 in a control group [4]. - The primary objective of the trial is to evaluate the reduction in atrophic lesion size, with key secondary endpoints including improvements in best corrected visual acuity (BCVA) and low luminance visual acuity (LLVA) [4]. Previous Trial Results - The Phase 1 GARDian trial showed promising results, with a 48% slower lesion growth at 12-month follow-up in treated eyes compared to untreated eyes, and a statistically significant improvement in BCVA [2][3]. Future Plans - Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, as part of its strategy to file three BLAs over the next three years [5]. Disease Background - Stargardt disease is the most common form of inherited macular degeneration, leading to progressive vision loss due to the degeneration of photoreceptor cells in the retina [7][8].