UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended September 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from to ________ Commission File Number: 001-34079 Opus Genetics, Inc. (Exact name of Registrant as specified in its charter) Delaware 11-3516358 (State or other jur ...

Exhibit 99.1 Opus Genetics Announces Financial Results for Third Quarter 2025 and Provides Corporate Update - Positive 3-month pediatric and 18-month adult clinical data from OPGx-LCA5 Phase 1/2 trial support the potential for restoring cone-mediated vision - - Successful FDA RMAT meeting provides the potential for an accelerated regulatory pathway to approval for OPGx-LCA5 - - OPGx-BEST1 gene therapy program underway with recruitment ongoing in Phase 1/2 trial for the treatment of BEST1 disease - - Supplem ...

[PART I – FINANCIAL INFORMATION](index=3&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=3&type=section&id=Item%201.%20Financial%20Statements) This section presents Opus Genetics, Inc.'s unaudited condensed consolidated financial statements, including the balance sheets, statements of comprehensive loss, changes in Series A Preferred Stock and stockholders' equity, and cash flows, along with detailed notes explaining significant accounting policies, mergers, commitments, financings, and other financial disclosures for the periods ended June 30, 2025, and December 31, 2024 [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheets Data | Metric (in thousands) | June 30, 2025 | December 31, 2024 | | :-------------------- | :------------ | :---------------- | | Cash and cash equivalents | $32,429 | $30,321 | | Total current assets | $38,439 | $36,610 | | Total assets | $38,665 | $36,862 | | Warrant liabilities | $11,800 | $— | | Total current liabilities | $20,192 | $11,295 | | Total liabilities | $21,192 | $11,295 | | Total stockholders' equity | $17,473 | $6,724 | - Total assets increased by **$1.8 million** from December 31, 2024, to June 30, 2025, primarily driven by an increase in cash and cash equivalents[12](index=12&type=chunk) - Warrant liabilities significantly increased to **$11.8 million** as of June 30, 2025, from zero at December 31, 2024, due to new financings[12](index=12&type=chunk) - Total stockholders' equity increased by **$10.7 million**, from $6.7 million to $17.5 million, reflecting recent financing activities[12](index=12&type=chunk) [Condensed Consolidated Statements of Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) Condensed Consolidated Statements of Comprehensive Loss Data | Metric (in thousands, except per share) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :------------------------------------ | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | License and collaborations revenue | $2,882 | $1,112 | $7,252 | $2,823 | | General and administrative | $5,766 | $3,354 | $12,112 | $8,024 | | Research and development | $6,022 | $6,086 | $13,975 | $10,835 | | Total operating expenses | $11,788 | $9,440 | $26,087 | $18,859 | | Loss from operations | $(8,906) | $(8,328) | $(18,835) | $(16,036) | | Fair value change in warrant and other derivative liabilities | $917 | $— | $3,722 | $— | | Financing costs | $35 | $— | $(1,337) | $— | | Net loss | $(7,420) | $(7,765) | $(15,614) | $(14,871) | | Basic and diluted net loss per share | $(0.12) | $(0.30) | $(0.32) | $(0.59) | - License and collaborations revenue increased significantly by **159%** for the three months ended June 30, 2025, and by **157%** for the six months ended June 30, 2025, compared to the prior year periods[14](index=14&type=chunk) - Net loss decreased for the three months ended June 30, 2025, to **$7.4 million** from $7.8 million in the prior year, but increased for the six months ended June 30, 2025, to **$15.6 million** from $14.9 million[14](index=14&type=chunk) [Condensed Consolidated Statements of Changes in Series A Preferred Stock and Stockholders' Equity](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Series%20A%20Preferred%20Stock%20and%20Stockholders'%20Equity) Condensed Consolidated Statements of Changes in Series A Preferred Stock and Stockholders' Equity Data | Metric (in thousands, except shares) | Balance at Dec 31, 2024 | Issuance of common stock and warrants | Stock-based compensation | Conversion of preferred stock | Net and comprehensive loss | Balance at June 30, 2025 | | :----------------------------------- | :---------------------- | :------------------------------------ | :----------------------- | :---------------------------- | :------------------------- | :----------------------- | | Series A Preferred Stock (Shares) | 14,145.374 | — | — | (14,145.374) | — | — | | Series A Preferred Stock (Amount) | $18,843 | — | — | $(18,843) | — | $— | | Common Stock (Shares) | 31,574,657 | 13,749,160 | 186,919 | 14,145,374 | — | 59,908,055 | | Common Stock (Amount) | $3 | $2 | — | $1 | — | $6 | | Additional Paid-In Capital | $145,719 | $6,387 | $913 | $18,842 | — | $172,079 | | Accumulated Deficit | $(138,998) | — | — | — | $(15,614) | $(154,612) | | Total Stockholders' Equity | $6,724 | $6,389 | $913 | $18,843 | $(15,614) | $17,473 | - All Series A Preferred Stock was converted into **14,145,374 shares** of common stock by June 30, 2025, following stockholder approval[17](index=17&type=chunk) - Additional paid-in capital increased significantly due to common stock and warrant issuances, and the conversion of preferred stock[16](index=16&type=chunk)[17](index=17&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Condensed Consolidated Statements of Cash Flows Data | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :-------------------- | :--------------------------- | :--------------------------- | | Net cash used in operating activities | $(19,263) | $(13,008) | | Net cash used in investing activities | $— | $— | | Net cash provided by financing activities | $21,371 | $3,916 | | Net increase (decrease) in cash and cash equivalents | $2,108 | $(9,092) | | Cash and cash equivalents at end of period | $32,429 | $41,409 | - Net cash used in operating activities increased to **$19.3 million** for the six months ended June 30, 2025, from $13.0 million in the prior year, primarily due to a higher net loss and changes in operating assets and liabilities[20](index=20&type=chunk)[260](index=260&type=chunk) - Net cash provided by financing activities significantly increased to **$21.4 million** for the six months ended June 30, 2025, from $3.9 million in the prior year, driven by proceeds from the March 2025 Offering and Private Placement, and the RDF Agreement[20](index=20&type=chunk)[263](index=263&type=chunk) - The company experienced a net increase in cash and cash equivalents of **$2.1 million** for the six months ended June 30, 2025, compared to a net decrease of $9.1 million in the same period last year[20](index=20&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) [1. Company Description and Summary of Significant Accounting Policies](index=9&type=section&id=1.%20Company%20Description%20and%20Summary%20of%20Significant%20Accounting%20Policies) This section outlines Opus Genetics, Inc.'s business as a clinical-stage biopharmaceutical company focusing on gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. It details the Opus Acquisition, key pipeline assets like OPGx-LCA5 and OPGx-BEST1, and the Viatris License Agreement for RYZUMVI. The section also covers critical accounting policies, including liquidity, revenue recognition, fair value measurements, and recent accounting pronouncements - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders[22](index=22&type=chunk) - The company acquired Private Opus on October 22, 2024, expanding its pipeline to include adeno-associated virus (AAV) based gene therapies for Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[23](index=23&type=chunk)[24](index=24&type=chunk) - RYZUMVI® (Phentolamine Ophthalmic Solution 0.75%) was approved by the FDA in September 2023 for pharmacologically induced mydriasis and commercially launched in April 2024 under the Viatris License Agreement[25](index=25&type=chunk) - As of June 30, 2025, the Company had **$32.4 million** in cash and cash equivalents, which management believes will be sufficient to fund operations for at least twelve months[32](index=32&type=chunk) Fair Value of Financial Instruments (in thousands) | Description | As of June 30, 2025 (Total) | As of December 31, 2024 (Total) | | :---------------------- | :-------------------------- | :------------------------------ | | Short-term investments | $— | $2 | | Warrant liabilities | $11,800 | $— | | Other derivative liabilities | $— | $2 | [2. Mergers](index=15&type=section&id=2.%20Mergers) This section details the Opus Acquisition, where the Company acquired Private Opus, issuing common and Series A Preferred Stock as consideration. It also references the 2020 Rexahn Merger and the associated Contingent Value Rights (CVR) Agreement, which entitles former Rexahn stockholders to future payments based on certain licensing revenues - On October 22, 2024, the Company acquired Private Opus for a total consideration of **$25.8 million**, issuing 5,237,063 shares of common stock and 14,145.374 shares of Series A Preferred Stock[65](index=65&type=chunk) - The Series A Preferred Stock was convertible into **1,000 shares** of common stock per share, with stockholder approval obtained on April 30, 2025[65](index=65&type=chunk) - The Rexahn Merger in November 2020 included a Contingent Value Rights (CVR) Agreement, entitling former Rexahn stockholders to **75-90%** of certain future payments from BioSense, HaiChang, and other IP deals over a fifteen-year term[66](index=66&type=chunk)[67](index=67&type=chunk)[69](index=69&type=chunk) [3. Commitments and Contingencies](index=16&type=section&id=3.%20Commitments%20and%20Contingencies) This section outlines various contractual obligations, including sublicense and license agreements for intellectual property related to drug development, and facility and equipment leases. Most future milestone obligations under these agreements were not deemed probable as of June 30, 2025, and thus no liabilities were recorded - The Company has several license agreements with potential development, regulatory, and commercial milestone payments, including with Apexian Pharmaceuticals (up to **$31 million**), University of Pennsylvania (up to **$2.6 million** for LCA5/RDH12, de minimis for LCA5 VR), and Iveric (up to **$111.7 million** for BEST1/RHO, and **$76.4 million** to Penn/UF for BEST1, **$93.5 million** to Penn/UF for RHO)[70](index=70&type=chunk)[71](index=71&type=chunk)[72](index=72&type=chunk)[74](index=74&type=chunk)[75](index=75&type=chunk)[76](index=76&type=chunk) - As of June 30, 2025, none of the future obligations under these license agreements were considered probable, and therefore no liabilities were recorded[71](index=71&type=chunk)[72](index=72&type=chunk)[74](index=74&type=chunk)[76](index=76&type=chunk)[77](index=77&type=chunk) - The Company relocated its headquarters to Durham, NC, on January 1, 2025, with a monthly base rent of approximately **$3,000**, amended to **$5,500** in July 2025 to include laboratory space, expiring September 30, 2025[78](index=78&type=chunk)[177](index=177&type=chunk) - Rent expense for all leases amounted to **$65,000** and **$9,000** for the three months ended June 30, 2025 and 2024, respectively, and **$132,000** and **$18,000** for the six months ended June 30, 2025 and 2024, respectively[82](index=82&type=chunk) [4. Supplemental Balance Sheet Information](index=18&type=section&id=4.%20Supplemental%20Balance%20Sheet%20Information) This section provides a detailed breakdown of the Company's accrued expenses and other liabilities as of June 30, 2025, and December 31, 2024 Accrued Expenses and Other Liabilities Data | Accrued Expenses and Other Liabilities (in thousands) | June 30, 2025 | December 31, 2024 | | :------------------------------------ | :------------ | :---------------- | | Payroll | $1,327 | $1,481 | | Professional services | $3,233 | $1,608 | | Research and development services and supplies | $2,102 | $4,452 | | Other | $265 | $606 | | Total | $6,927 | $8,147 | - Accrued professional services significantly increased from **$1.6 million** to **$3.2 million**, while research and development services and supplies decreased from **$4.5 million** to **$2.1 million**[84](index=84&type=chunk) [5. Related Party Transactions](index=18&type=section&id=5.%20Related%20Party%20Transactions) This section details transactions with related parties, including consulting agreements with Dr. Pepose and Dr. Jean Bennett, subscription agreements with Dr. George Magrath and Cam Gallagher, and strategic partnerships with Foundation Fighting Blindness (FFB) and its Retinal Degeneration Fund (RDF) for funding and collaboration on gene therapy programs - Consulting expenses for Dr. Pepose amounted to **$119,000** and **$238,000** for the three and six months ended June 30, 2025, respectively[88](index=88&type=chunk) - Dr. Jean Bennett received a restricted stock unit award of **100,000 shares** vesting on October 22, 2025, for consulting services[90](index=90&type=chunk) - The Company entered into a 2025 Letter Agreement with FFB and JCHR to collaborate on studies for RDH12 or BEST1 genes, with an obligation to make two payments of **$300,000** each, one paid by June 30, 2025, and the other due by January 31, 2027[92](index=92&type=chunk)[93](index=93&type=chunk) - A funding agreement (RDF Agreement) with Foundation Fighting Blindness Retinal Degeneration Fund provides up to **$2.0 million** in nondilutive funding for the MERTK Program, with **$1.0 million** disbursed in June 2025[94](index=94&type=chunk) [6. Series A Preferred Stock](index=20&type=section&id=6.%20Series%20A%20Preferred%20Stock) This section details the authorization and issuance of Series A Preferred Stock in connection with the Opus Acquisition and its subsequent conversion into common stock following stockholder approval - **14,145.374 shares** of Series A Preferred Stock were issued upon the close of the Opus Acquisition on October 22, 2024[98](index=98&type=chunk) - On May 5, 2025, all Series A Preferred Stock was converted into **14,145,374 shares** of common stock after stockholder approval at the 2025 Annual Meeting[98](index=98&type=chunk) [7. Financings](index=20&type=section&id=7.%20Financings) This section details the Company's financing activities, including the March 2025 public offering and private placement, the termination of the Lincoln Park Purchase Agreement, the At-The-Market (ATM) program, and prior Registered Direct Offering (RDO) and Pre-Merger Financing, along with the associated warrant activities - The March 2025 Offering and Private Placement generated combined gross proceeds of approximately **$21.5 million**, before deducting **$1.8 million** in underwriting discounts and offering expenses[102](index=102&type=chunk) - The March 2025 Warrants and March 2025 Private Placement Warrants were classified as warrant liabilities due to certain volatility provisions and had a fair value of **$14.7 million** and **$0.8 million**, respectively, at issuance[103](index=103&type=chunk)[106](index=106&type=chunk)[108](index=108&type=chunk)[109](index=109&type=chunk) - The Lincoln Park Purchase Agreement, which allowed the Company to sell up to **$50 million** of common stock, was terminated on April 3, 2025, having generated **$5.2 million** in net proceeds through June 30, 2025[112](index=112&type=chunk)[115](index=115&type=chunk)[117](index=117&type=chunk) - A new At-The-Market (ATM) equity issuance program was established on January 13, 2025, with Leerink Partners LLC, replacing the prior program. Since inception, ATM programs have sold **8,006,791 shares** for **$26.8 million** in gross proceeds[118](index=118&type=chunk)[119](index=119&type=chunk) Warrant Activity Summary | Metric | Outstanding and exercisable at Dec 31, 2024 | Issued | Exercised | Forfeited/Cancelled | Outstanding and exercisable at June 30, 2025 | | :----------------------------------- | :------------------------------------------ | :---------- | :-------- | :------------------ | :------------------------------------------- | | Warrants (Number) | 7,204,299 | 22,229,102 | — | — | 29,433,401 | | Weighted Average Exercise Price | $4.82 | $0.96 | $— | $— | $1.91 | | Weighted Average Remaining Term (Years) | 1.00 | 5.00 | — | — | 3.70 | [8. Stock-based Compensation](index=25&type=section&id=8.%20Stock-based%20Compensation) This section details the Company's stock-based compensation expenses, equity incentive plans (Inducement Plan, 2020 Plan, 2018 Plan), and activity related to stock options and restricted stock units (RSUs), including grants, exercises, forfeitures, and vesting schedules Stock-Based Compensation Expense (in thousands) | Category | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | General and administrative | $614 | $526 | $1,243 | $1,301 | | Research and development | $282 | $280 | $566 | $490 | | Total stock-based compensation | $896 | $806 | $1,809 | $1,791 | - The 2020 Equity Incentive Plan's evergreen provision added **1,578,733 shares** on January 1, 2025[131](index=131&type=chunk) Stock Option Activity Summary | Metric | Number of Options (June 30, 2025) | Weighted Average Exercise Price | | :----------------------------------- | :-------------------------------- | :------------------------------ | | Outstanding at December 31, 2024 | 5,073,736 | $2.68 | | Granted | 2,059,829 | $0.94 | | Exercised | (5,000) | $0.90 | | Forfeited/Cancelled | (473,630) | $1.92 | | Outstanding at June 30, 2025 | 6,654,935 | $2.19 | Restricted Stock Unit (RSU) Activity Summary | Metric | Number of Shares (June 30, 2025) | | :------------------------------- | :------------------------------- | | Non-vested at December 31, 2024 | 1,393,230 | | Granted | 1,455,361 | | Forfeited | (231,788) | | Vested | (381,534) | | Non-vested at June 30, 2025 | 2,235,269 | - Unrecognized stock-based compensation cost was **$6.1 million** as of June 30, 2025, expected to be recognized over a weighted average period of **1.7 years**[145](index=145&type=chunk) [9. Apexian Sublicense Agreement](index=28&type=section&id=9.%20Apexian%20Sublicense%20Agreement) This section details the Apexian Sublicense Agreement, under which the Company obtained exclusive worldwide rights for a Ref-1 Inhibitor program, including APX3330. It outlines potential milestone payments for development, regulatory, and sales achievements, as well as single-digit royalty payments on net sales, none of which were deemed probable as of June 30, 2025 - The Apexian Sublicense Agreement grants exclusive worldwide rights for a Ref-1 Inhibitor program, with APX3330 as the lead compound for ophthalmic and diabetes mellitus conditions[146](index=146&type=chunk) - Potential milestone payments include up to **$11 million** for development and regulatory milestones and up to **$20 million** for sales milestones, plus single-digit percentage royalties on net sales[147](index=147&type=chunk) - As of June 30, 2025, none of the milestone or royalty payments under this agreement were triggered or deemed probable[148](index=148&type=chunk) [10. License and Collaboration Agreements](index=28&type=section&id=10.%20License%20and%20Collaboration%20Agreements) This section details key license and collaboration agreements, including the Viatris License Agreement for PS Products (RYZUMVI), the BioSense License and Assignment Agreement for RX-3117, the termination of the Processa License Agreement, and the SBIR Grant Agreement for the RHO product. It outlines revenue recognition, milestone payments, and royalties associated with these agreements - The Viatris License Agreement granted Viatris exclusive rights to develop and commercialize PS Products (including RYZUMVI) worldwide, except for certain Asian countries. The Company received a **$35 million** upfront payment and a **$10 million** milestone payment for FDA approval of PS[149](index=149&type=chunk)[152](index=152&type=chunk) - Revenue recognized under the Viatris License Agreement was **$2.9 million** and **$7.3 million** for the three and six months ended June 30, 2025, respectively, primarily from reimbursement of R&D services and, to a lesser extent, royalty payments[155](index=155&type=chunk) - The BioSense License and Assignment Agreement offers potential milestone payments up to **$84.5 million** and tiered royalties for RX-3117 in the BioSense Territory, but no payments were probable as of June 30, 2025[162](index=162&type=chunk) - The Processa License Agreement for RX-3117 was terminated by Processa, effective October 25, 2025, meaning no future payments will be received after this date[166](index=166&type=chunk) - The Company recognized **$173,000** and **$197,000** of revenue from an SBIR grant for the RHO product development for the three and six months ended June 30, 2025, respectively[168](index=168&type=chunk) [11. Net Loss per Share](index=31&type=section&id=11.%20Net%20Loss%20per%20Share) This section explains the calculation of basic and diluted net loss per share, noting that potential common shares from Series A and RDO warrants, stock options, and RSUs were excluded from diluted EPS calculations as their inclusion would be anti-dilutive due to the net loss - Basic and diluted net loss per share are computed by dividing net loss by the weighted average number of common shares outstanding, with common stock equivalents considered if dilutive[169](index=169&type=chunk) Anti-Dilutive Potential Common Shares | Potential Common Shares | June 30, 2025 | June 30, 2024 | | :---------------------- | :------------ | :------------ | | Series A and RDO warrants | 29,433,401 | 7,204,299 | | Stock options | 6,654,935 | 4,962,489 | | RSUs | 2,235,269 | 1,167,090 | - No incremental common stock equivalents were included in diluted loss per share calculations because their effect would have been anti-dilutive given the reported net loss[169](index=169&type=chunk) [12. Income Taxes](index=31&type=section&id=12.%20Income%20Taxes) This section states that the Company's effective tax rate was zero percent for the periods presented due to a full valuation allowance on net deferred income tax assets. It also notes the Company is assessing the impact of the recently signed One Big, Beautiful Bill (OBBB) on tax policy, particularly regarding R&D expense capitalization - The effective tax rate for the three and six months ended June 30, 2025 and 2024, was **zero percent**[171](index=171&type=chunk) - A full valuation allowance has been established on the Company's net deferred income tax assets due to uncertainty of future taxable income[171](index=171&type=chunk) - The Company is currently assessing the impact of the One Big, Beautiful Bill (OBBB), signed July 4, 2025, which makes significant changes to tax policy, especially regarding the capitalization of research and development expenses[171](index=171&type=chunk) [13. Deferred Compensation Plan](index=31&type=section&id=13.%20Deferred%20Compensation%20Plan) This section details the Company's 401(k) plan, which became effective October 1, 2021, and includes matching contributions for eligible employees - The Company offers a 401(k) plan to all employees, with matching contributions of **100%** on the first **3%** deferred and an additional **50%** on the next **2%** deferred[173](index=173&type=chunk) 401(k) Matching Contributions (in thousands) | Period | Contributions | | :------------------------- | :------------ | | 3 Months Ended June 30, 2025 | $34 | | 3 Months Ended June 30, 2024 | $43 | | 6 Months Ended June 30, 2025 | $111 | | 6 Months Ended June 30, 2024 | $101 | [14. Subsequent Events](index=31&type=section&id=14.%20Subsequent%20Events) This section reports events occurring after June 30, 2025, including a funding and license agreement for the RDH12 Program and an amendment to the headquarters lease to include laboratory space - On July 22, 2025, the Company entered into the RDH12 Agreement with Eyes on the Future and RDH12 Fund for Sight, providing up to **$1.6 million** in funding for the RDH12 Program[174](index=174&type=chunk) - The RDH12 Agreement includes diligence obligations and a non-exclusive, global, royalty-free license to the Funding Parties if certain milestones are not met or a 'License Trigger Event' occurs[175](index=175&type=chunk) - On July 1, 2025, the headquarters lease was amended to include laboratory space, increasing the monthly base rent to approximately **$5,500**, with the lease expiring on September 30, 2025[177](index=177&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=33&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the Company's financial condition and results of operations, including an overview of its business, pipeline, recent developments, and strategic outlook. It analyzes revenue and expense trends, liquidity, capital resources, and critical accounting policies, offering insights into past performance and future expectations [Overview](index=35&type=section&id=Overview) Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases and small molecule therapies for ophthalmic disorders. Following the Opus Acquisition, the pipeline expanded to include AAV-based gene therapies like OPGx-LCA5 (Phase 1/2 ongoing, RMAT designation) and OPGx-BEST1 (IND submission expected H2 2025), alongside RYZUMVI (FDA approved, commercialized) and APX3330 (seeking strategic partner). The Company's strategic outlook involves advancing its pipeline, exploring out-licensing, and financing operations through equity, debt, and collaborations - Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders[186](index=186&type=chunk) - The Opus Acquisition on October 22, 2024, expanded the pipeline to include AAV-based gene therapies for Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[188](index=188&type=chunk) - OPGx-LCA5, the most advanced gene therapy, is in an ongoing Phase 1/2 clinical trial, showing visual improvement in adult patients and initial positive data in pediatric patients, and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA[189](index=189&type=chunk)[199](index=199&type=chunk) - OPGx-BEST1 is a gene therapy candidate for Best Disease, with an Investigational New Drug (IND) application and Phase 1/2 trial expected in the second half of 2025[190](index=190&type=chunk) - RYZUMVI (Phentolamine Ophthalmic Solution 0.75%) was FDA-approved in September 2023 and commercialized in April 2024. The Company plans to file a Supplemental New Drug Application (sNDA) for presbyopia in H2 2025[192](index=192&type=chunk)[193](index=193&type=chunk) - APX3330, a small molecule inhibitor for diabetic retinopathy, is seeking a strategic partner due to capital requirements and developmental timelines[194](index=194&type=chunk) - Net loss was **$7.4 million** and **$15.6 million** for the three and six months ended June 30, 2025, respectively, with an accumulated deficit of **$154.6 million** as of June 30, 2025[204](index=204&type=chunk) [Financial Operations Overview](index=38&type=section&id=Financial%20Operations%20Overview) This section details the Company's financial operations, categorizing revenue primarily from license and collaborations (Viatris License Agreement) and expenses into general and administrative (personnel, legal, business development) and research and development (clinical trials, manufacturing, license fees). It also covers fair value changes in warrant liabilities, financing costs, other income (interest, grants), and income tax provisions - License and collaborations revenue is primarily derived from the Viatris License Agreement, including a one-time payment, milestone payment, and reimbursement for R&D services, with RYZUMVI royalties also contributing[207](index=207&type=chunk) - General and administrative expenses include personnel costs, insurance, legal fees, business development, and professional services[211](index=211&type=chunk) - Research and development expenses cover clinical trials, nonclinical studies, regulatory activities, manufacturing, and license fees. R&D expenses for PS are fully reimbursed by Viatris[212](index=212&type=chunk)[213](index=213&type=chunk) - Fair value changes in warrant and other derivative liabilities primarily relate to the March 2025 Warrants and Private Placement Warrants[215](index=215&type=chunk) - Financing costs consist of issuance costs attributed to the March 2025 Warrants and March 2025 Private Placement Warrants[216](index=216&type=chunk) - Other income, net, includes interest from cash, gains/losses from equity investments, and grant reimbursements[217](index=217&type=chunk) [Results of Operations](index=40&type=section&id=Results%20of%20Operations) [Comparison of Three Months Ended June 30, 2025 and 2024](index=40&type=section&id=Comparison%20of%20Three%20Months%20Ended%20June%2030,%202025%20and%202024) For the three months ended June 30, 2025, license and collaborations revenue increased significantly due to higher PS R&D services. General and administrative expenses rose due to public company costs, legal fees, and payroll. Research and development expenses saw a slight decrease, with lower manufacturing and consulting costs offset by higher clinical, toxicology, and payroll expenses. The period also saw a fair value change benefit in warrant liabilities and financing costs Comparison of Three Months Ended June 30, 2025 and 2024 Data | Metric (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | Change | | :-------------------- | :--------------------------- | :--------------------------- | :----- | | License and collaborations revenue | $2,882 | $1,112 | $1,770 | | General and administrative | $5,766 | $3,354 | $2,412 | | Research and development | $6,022 | $6,086 | $(64) | | Net loss | $(7,420) | $(7,765) | $345 | - License and collaborations revenue increased by **$1.8 million**, or **159%**, primarily due to increased PS research and development services[220](index=220&type=chunk) - General and administrative expenses increased by **$2.4 million**, or **72%**, mainly due to public company costs, legal fees, patent costs, and payroll[221](index=221&type=chunk) - Research and development expenses decreased slightly by **$0.1 million**, as lower manufacturing and consulting costs were largely offset by higher clinical, toxicology, and payroll-related costs[222](index=222&type=chunk) - A fair value change in warrant and other derivative liabilities resulted in a **$0.9 million** benefit for the three months ended June 30, 2025, compared to zero in the prior year[223](index=223&type=chunk) [Comparison of Six Months Ended June 30, 2025 and 2024](index=42&type=section&id=Comparison%20of%20Six%20Months%20Ended%20June%2030,%202025%20and%202024) For the six months ended June 30, 2025, license and collaborations revenue significantly increased due to higher PS R&D services. General and administrative expenses rose due to public company, legal, and patent-related costs. Research and development expenses increased, driven by higher clinical and toxicology costs for PS and IRD programs, and payroll, partially offset by decreased APX3330-related expenses. The period also saw a substantial fair value change benefit in warrant liabilities and financing costs Comparison of Six Months Ended June 30, 2025 and 2024 Data | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change | | :-------------------- | :--------------------------- | :--------------------------- | :----- | | License and collaborations revenue | $7,252 | $2,823 | $4,429 | | General and administrative | $12,112 | $8,024 | $4,088 | | Research and development | $13,975 | $10,835 | $3,140 | | Net loss | $(15,614) | $(14,871) | $(743) | - License and collaborations revenue increased by **$4.4 million**, or **157%**, primarily due to increased PS research and development services[227](index=227&type=chunk) - General and administrative expenses increased by **$4.1 million**, or **51%**, mainly due to public company costs, legal support, patent fees, and professional services[228](index=228&type=chunk) - Research and development expenses increased by **$3.1 million**, or **29%**, driven by higher clinical costs for PS trials and IRD programs, toxicology costs, and payroll, partially offset by decreased APX3330-related expenses[229](index=229&type=chunk) - A fair value change in warrant and other derivative liabilities resulted in a **$3.7 million** benefit for the six months ended June 30, 2025, compared to zero in the prior year[230](index=230&type=chunk) - Financing costs of **$1.3 million** were incurred for the six months ended June 30, 2025, primarily due to issuance costs for the March 2025 Warrants and Private Placement Warrants[231](index=231&type=chunk) [Liquidity and Capital Resources](index=44&type=section&id=Liquidity%20and%20Capital%20Resources) This section details the Company's liquidity position, historical funding sources, and future capital requirements. As of June 30, 2025, the Company had $32.4 million in cash and cash equivalents, expected to fund operations for at least twelve months. Recent financings include the March 2025 Offering and Private Placement ($21.5 million gross proceeds) and $1.0 million from the RDF Agreement. The Lincoln Park Purchase Agreement was terminated. The Company anticipates needing additional capital through equity, debt, or collaborations to fund its increasing R&D expenses and achieve profitability - As of June 30, 2025, the Company had **$32.4 million** in cash and cash equivalents, which is believed to be sufficient to fund operations for at least twelve months[233](index=233&type=chunk) - Historical funding sources include **$89.7 million** from equity offerings, **$8.5 million** from convertible notes, **$35.0 million** upfront payment and **$10.0 million** milestone payment from the Viatris License Agreement, and **$1.0 million** from the RDF Agreement[234](index=234&type=chunk) - The March 2025 Offering and Private Placement generated approximately **$21.5 million** in combined gross proceeds, before deducting underwriting discounts and offering expenses[237](index=237&type=chunk) - The Lincoln Park Purchase Agreement, which provided for up to **$50 million** in equity line financing, was terminated on April 3, 2025, having generated **$5.2 million** in net proceeds[246](index=246&type=chunk)[269](index=269&type=chunk) - The At-The-Market (ATM) program has sold **8,006,791 shares** of common stock since its inception, generating **$26.8 million** in gross proceeds through June 30, 2025[247](index=247&type=chunk)[268](index=268&type=chunk) - Future capital requirements are expected to increase due to ongoing clinical trials, nonclinical studies, and development of new product candidates. The Company expects to finance these needs through equity, debt, or collaborations, which may result in stockholder dilution or restrictive covenants[209](index=209&type=chunk)[270](index=270&type=chunk)[271](index=271&type=chunk) [Critical Accounting Policies and Estimates](index=52&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section highlights the Company's critical accounting policies that require significant management estimates and judgments, specifically focusing on license and collaborations revenue recognition under ASC 606 and the accounting for income tax assets and liabilities, including the full valuation allowance on net deferred tax assets - License and collaborations revenue is recognized in accordance with ASC 606, requiring allocation of transaction price to performance obligations based on estimated stand-alone selling prices using a discounted cash flow approach and probability assessments[293](index=293&type=chunk) - A full valuation allowance has been provided on the Company's net deferred tax assets due to the uncertainty of future taxable income and other factors impacting their realizability[294](index=294&type=chunk) [Recent Accounting Pronouncements](index=53&type=section&id=Recent%20Accounting%20Pronouncements) This section refers to Note 1 for a discussion of recently issued accounting pronouncements, including ASU 2023-07 on Segment Reporting and ASU 2023-09 on Income Taxes, and their impact on the Company's financial statements - The Company adopted ASU 2023-07 (Segment Reporting) on January 1, 2024, with no material impact on consolidated financial statements[63](index=63&type=chunk) - ASU 2023-09 (Income Taxes) is effective for fiscal years beginning after December 15, 2024, and is expected to augment income tax disclosures related to rate reconciliation and jurisdictional detail[64](index=64&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=53&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section states that quantitative and qualitative disclosures about market risk are not applicable for smaller reporting companies - Quantitative and Qualitative Disclosures About Market Risk are not applicable for smaller reporting companies[296](index=296&type=chunk) [Item 4. Controls and Procedures](index=53&type=section&id=Item%204.%20Controls%20and%20Procedures) This section confirms that the Company's disclosure controls and procedures were evaluated and deemed effective as of June 30, 2025. It also states that there were no material changes in internal control over financial reporting during the quarter - The Company's disclosure controls and procedures were evaluated and concluded to be effective as of June 30, 2025[300](index=300&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2025, that materially affected or are reasonably likely to materially affect the Company's internal control over financial reporting[301](index=301&type=chunk) [PART II – OTHER INFORMATION](index=54&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=54&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the Company is not currently a party to any legal proceedings that are likely to materially affect its business or financial results, though it acknowledges that litigation can have an adverse impact - The Company is not currently a party to any legal proceedings that are likely to materially affect its business or financial results[303](index=303&type=chunk) [Item 1A. Risk Factors](index=54&type=section&id=Item%201A.%20Risk%20Factors) This section updates the Company's risk factors, primarily highlighting the risk of potential delisting from the Nasdaq Capital Market if it fails to comply with the Minimum Bid Price Requirement, and the potential adverse effects of such an event or a reverse stock split - The Company's common stock has recently traded below the Nasdaq Minimum Bid Price Requirement of **$1.00** per share, posing a risk of delisting if compliance is not regained[305](index=305&type=chunk) - Potential delisting could lead to decreased liquidity, increased volatility, damage to reputation, and negatively impact the ability to raise capital[307](index=307&type=chunk) - The Company may consider a reverse stock split to regain compliance, which could also negatively affect the stock price[306](index=306&type=chunk)[307](index=307&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=55&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section states that there were no unregistered sales of equity securities or use of proceeds to report for the period - None[308](index=308&type=chunk) [Item 3. Defaults Upon Senior Securities](index=55&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section states that there were no defaults upon senior securities to report for the period - None[309](index=309&type=chunk) [Item 4. Mine Safety Disclosures](index=55&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section states that mine safety disclosures are not applicable to the Company - Not applicable to the Company[310](index=310&type=chunk) [Item 5. Other Information](index=55&type=section&id=Item%205.%20Other%20Information) This section reports that none of the Company's directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the quarter ended June 30, 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or officers during the quarter ended June 30, 2025[311](index=311&type=chunk) [Item 6. Exhibits](index=55&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed as part of the Form 10-Q, including funding and license agreements, and certifications - Key exhibits include the Funding Agreement with Foundation Fighting Blindness Retinal Degeneration Fund (Exhibit 10.1) and the Funding and License Agreement with Eyes on the Future and RDH12 Fund for Sight (Exhibit 10.2)[312](index=312&type=chunk) - Certifications of the Principal Executive Officer and Principal Financial Officer pursuant to Sections 302 and 906 of the Sarbanes-Oxley Act of 2002 are also included (Exhibits 31.1, 31.2, 32.1)[312](index=312&type=chunk) [SIGNATURES](index=56&type=section&id=SIGNATURES) This section contains the authorized signatures of Opus Genetics, Inc.'s Chief Executive Officer and Head of Financial Quality Assurance, affirming the filing of the report - The report is duly signed by George Magrath, Chief Executive Officer and Director, and Amy Rabourn, Head of Financial Quality Assurance, on August 13, 2025[318](index=318&type=chunk)[319](index=319&type=chunk)

[Executive Summary & Corporate Update](index=1&type=section&id=Executive%20Summary%20%26%20Corporate%20Update) Opus Genetics reported Q2 2025 financial results and provided a corporate update, highlighting significant clinical and regulatory milestones across its gene therapy and small molecule programs [Introduction](index=1&type=section&id=Introduction) Opus Genetics announced its financial results for the second quarter ended June 30, 2025, and provided a corporate update, highlighting significant clinical and regulatory milestones across its gene therapy and small molecule programs - Positive 12-month Phase 1/2 clinical data in adult cohort and early pediatric clinical data support potential for meaningful vision restoration with **OPGx-LCA5**[2](index=2&type=chunk) - FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for **OPGx-LCA5**, providing a pathway for accelerated development and review[2](index=2&type=chunk) - Positive topline results reported from **VEGA-3** and **LYNX-2 Phase 3 trials** with Phentolamine Ophthalmic Solution 0.75%[2](index=2&type=chunk) - **OPGx-BEST1** is on track to enter Phase 1/2 trial in H2 2025 for the treatment of bestrophin-1 related inherited retinal disease[2](index=2&type=chunk) [CEO Commentary](index=1&type=section&id=CEO%20Commentary) CEO George Magrath highlighted significant progress across the pipeline, including the RMAT designation for OPGx-LCA5, sustained functional vision improvements in adult patients, and early efficacy signs in pediatric cohorts. He also noted the advancement of OPGx-BEST1 and the positive Phase 3 readouts for Phentolamine, emphasizing the company's focus on delivering transformative treatments - **RMAT designation** for OPGx-LCA5 program underscores the strength of clinical data and the urgent need for effective gene therapies[3](index=3&type=chunk) - **Sustained functional vision improvements** observed in adult patients and early signs of efficacy in the pediatric cohort for OPGx-LCA5[3](index=3&type=chunk) - Advancement of **OPGx-BEST1** toward the clinic and nomination of two additional development candidates (**RDH12, MERTK**) highlight the breadth of the IRD pipeline[3](index=3&type=chunk) - **Positive readouts** from two Phase 3 Phentolamine trials represent a major step toward bringing a new treatment option to patients with vision challenges[3](index=3&type=chunk) [Pipeline Updates](index=2&type=section&id=Pipeline%20Updates) Opus Genetics provided updates on its gene therapy programs, including RMAT designation for OPGx-LCA5, preclinical progress for OPGx-BEST1, and non-dilutive funding for OPGx-RDH12 and OPGx-MERTK, alongside positive Phase 3 results for Phentolamine Ophthalmic Solution [OPGx-LCA5 Program](index=2&type=section&id=OPGx-LCA5%20Program) The OPGx-LCA5 program received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, signifying its potential for accelerated development. Clinical data from the Phase 1/2 trial showed sustained visual function improvements in adult participants over 12 months, and initial one-month pediatric data indicated vision improvement with no drug-related adverse events - FDA granted **RMAT designation** for OPGx-LCA5 for the treatment of Leber Congenital Amaurosis (LCA) due to genetic variations in the LCA5 gene[6](index=6&type=chunk) - **Twelve-month clinical data** from adult participants in the Phase 1/2 trial demonstrated sustained improvements in visual function, including visual acuity gains and improved mobility testing scores[6](index=6&type=chunk) - Initial pediatric data at **one-month post-treatment** showed vision improvement with no drug-related adverse events[6](index=6&type=chunk) [OPGx-BEST1 Program](index=2&type=section&id=OPGx-BEST1%20Program) Preclinical data for OPGx-BEST1 demonstrated restoration of the retinal pigment epithelium-photoreceptor interface in a canine model. The program is on track for Investigational New Drug (IND) submission and initiation of a Phase 1/2 trial in the second half of 2025 - **Preclinical data** presented at the American Ophthalmological Society (AOS) demonstrated restoration of the retinal pigment epithelium-photoreceptor interface in a canine model of BEST1 using AAV-mediated gene delivery[6](index=6&type=chunk) - Investigational New Drug (IND) submission and **Phase 1/2 trial initiation** remain on track for the second half of 2025[6](index=6&type=chunk) [OPGx-RDH12 and OPGx-MERTK Programs](index=2&type=section&id=OPGx-RDH12%20and%20OPGx-MERTK%20Programs) Opus Genetics secured non-dilutive funding to advance its OPGx-RDH12 and OPGx-MERTK programs. The OPGx-RDH12 program received up to $1.6 million from the Global RDH12 Alliance, while OPGx-MERTK received up to $2 million from the Retinal Degeneration Fund. Preclinical data for OPGx-MERTK showed preservation of retinal function in animal models - Partnership with the Global RDH12 Alliance provides up to **$1.6 million in non-dilutive funding** to accelerate development of OPGx-RDH12 for Leber congenital amaurosis (RDH12-LCA)[6](index=6&type=chunk) - Non-dilutive funding up to **$2 million** received from the Retinal Degeneration Fund to advance OPGx-MERTK, targeting retinitis pigmentosa caused by MERTK mutations[6](index=6&type=chunk) - **Preclinical OPGx-MERTK data** presented at the American Society of Gene & Cell Therapy (ASGCT) in May showed preservation of retinal function in animal models[6](index=6&type=chunk) [Phentolamine Ophthalmic Solution 0.75% Program](index=3&type=section&id=Phentolamine%20Ophthalmic%20Solution%200.75%25%20Program) The VEGA-3 Phase 3 trial for presbyopia met its primary and multiple secondary endpoints, demonstrating significant gains in near visual acuity and a favorable safety profile. The LYNX-2 Phase 3 trial also met its endpoints for night vision disturbances in keratorefractive patients, showing improved mesopic low contrast vision and reduced night-driving symptoms. An sNDA submission for the presbyopia indication is planned for the second half of 2025 - **VEGA-3 Phase 3 trial** met its primary and multiple secondary endpoints in presbyopia, with **27.2% of treated patients** achieving a ≥15-letter gain in near visual acuity (vs. 11.5% on placebo, p<0.0001)[12](index=12&type=chunk) - **LYNX-2 Phase 3 trial** met its primary and multiple secondary endpoints in keratorefractive patients with night vision disturbances, showing statistically significant gains in mesopic low contrast vision and improvements in night-driving related symptoms[12](index=12&type=chunk) - Supplemental New Drug Application (**sNDA**) submission for presbyopia indication planned for the **second half of 2025**[12](index=12&type=chunk) [Additional Medical Meeting Presentations](index=3&type=section&id=Additional%20Medical%20Meeting%20Presentations) Opus Genetics presented on the use of virtual reality-guided functional testing to support meaningful clinical endpoints in inherited retinal disease (IRD) trials at the Retinal Imaging Biomarkers Summit - **Virtual reality-guided functional testing** to support meaningful clinical endpoints in IRD trials was presented at the Retinal Imaging Biomarkers Summit[8](index=8&type=chunk) [Upcoming Milestones & Data Readouts](index=3&type=section&id=Upcoming%20Milestones%20%26%20Data%20Readouts) Opus Genetics anticipates several key milestones in the second half of 2025, including reporting three-month pediatric data for OPGx-LCA5, initiating enrollment for the OPGx-BEST1 Phase 1/2 trial, submitting the Phentolamine sNDA for presbyopia, and initiating enrollment for the Phentolamine LYNX-3 Phase 3 trial - Report **three-month pediatric data** from OPGx-LCA5 Phase 1/2 trial in **Q3 2025**[12](index=12&type=chunk) - Initiate enrollment in **Phase 1/2 trial for OPGx-BEST1** in **H2 2025**[12](index=12&type=chunk) - Submit **Phentolamine sNDA for presbyopia** in **H2 2025**[12](index=12&type=chunk) - Initiate enrollment in **Phentolamine LYNX-3 Phase 3 trial** in **H2 2025**, targeting reduced low light vision and nighttime visual disturbances[12](index=12&type=chunk) [Financial Results for the Second Quarter Ended June 30, 2025](index=3&type=section&id=Financial%20Results%20for%20the%20Second%20Quarter%20Ended%20June%2030%2C%202025) Opus Genetics reported its financial performance for Q2 2025, detailing cash position, revenue growth, operating expenses, and a reduced net loss, reflecting strategic investments and collaboration reimbursements [Cash Position](index=3&type=section&id=Cash%20Position) As of June 30, 2025, Opus Genetics held $32.4 million in cash and cash equivalents, which is projected to fund its operations into the second half of 2026 Cash and Cash Equivalents | Metric | June 30, 2025 (in thousands) | | :----- | :--------------------------- | | Cash and cash equivalents | $32,429 | - Based on current operating plans, the Company expects its existing cash resources will fund operations into the **second half of 2026**[9](index=9&type=chunk) [Revenue](index=3&type=section&id=Revenue) License and collaboration revenue significantly increased to $2.9 million for Q2 2025, up from $1.1 million in the prior-year period. This growth was primarily driven by reimbursement for R&D services under the Company's collaboration with Viatris, Inc License and Collaboration Revenue (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | Revenue | $2,882 | $1,112 | +159.2% | - Revenue in both periods was driven by the Company's collaboration with Viatris, Inc., primarily from reimbursement of R&D services[10](index=10&type=chunk) [Operating Expenses](index=3&type=section&id=Operating%20Expenses) Operating expenses for Q2 2025 saw an increase in G&A due to legal and business development costs, while R&D expenses remained stable, largely offset by reimbursements [General and Administrative (G&A) Expenses](index=3&type=section&id=General%20and%20Administrative%20(G%26A)%20Expenses) G&A expenses increased to $5.8 million in Q2 2025 from $3.4 million in Q2 2024, primarily due to higher costs associated with legal and patent-related expenses, payroll, and business development activities. Stock-based compensation also saw a slight increase General and Administrative Expenses (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | G&A Expenses | $5,766 | $3,354 | +71.9% | | Stock-based compensation | $600 | $500 | +20.0% | - The increase was mainly due to higher costs associated with legal and patent-related expenses, payroll, and business development activities[11](index=11&type=chunk) [Research and Development (R&D) Expenses](index=4&type=section&id=Research%20and%20Development%20(R%26D)%20Expenses) R&D expenses slightly decreased to $6.0 million for Q2 2025, compared to $6.1 million in the prior-year period. This minor reduction was mainly attributable to lower manufacturing and consulting costs, partially offset by increased clinical trial, toxicology, and payroll-related expenses. R&D expenses for Phentolamine Ophthalmic Solution 0.75% were fully reimbursed Research and Development Expenses (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | R&D Expenses | $6,022 | $6,086 | -1.0% | | Stock-based compensation | $300 | $300 | 0.0% | - The slight decrease was primarily due to lower manufacturing and consulting costs, partially offset by increased clinical trial, toxicology, and payroll-related expenses[13](index=13&type=chunk) - R&D expenses related to Phentolamine Ophthalamine Solution 0.75% were fully reimbursed under the Viatris License Agreement[13](index=13&type=chunk) [Net Loss and Earnings Per Share (EPS)](index=4&type=section&id=Net%20Loss%20and%20Earnings%20Per%20Share%20(EPS)) Opus Genetics reported a net loss of $7.4 million, or $(0.12) per basic and diluted share, for Q2 2025, an improvement from a net loss of $7.8 million, or $(0.30) per share, in Q2 2024 Net Loss and EPS (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY) | | :----- | :--------------------- | :--------------------- | :----------- | | Net Loss | $(7,420) | $(7,765) | -4.4% | | Basic and Diluted EPS | $(0.12) | $(0.30) | +60.0% | - The number of shares used in per share calculations significantly increased from **25,827,265** in Q2 2024 to **63,376,392** in Q2 2025[21](index=21&type=chunk) [Condensed Consolidated Financial Statements](index=6&type=section&id=Condensed%20Consolidated%20Financial%20Statements) The condensed consolidated financial statements for Q2 2025 show an increase in total assets and stockholders' equity, alongside a significant rise in liabilities primarily due to warrant liabilities and a long-term funding agreement [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, Opus Genetics reported total assets of $38.7 million, an increase from $36.9 million at December 31, 2024. Total liabilities significantly increased to $21.2 million from $11.3 million, primarily due to the introduction of warrant liabilities and a long-term funding agreement. Total stockholders' equity also increased to $17.5 million from $6.7 million Condensed Consolidated Balance Sheets (as of June 30, 2025 vs. December 31, 2024) | Metric (in thousands) | June 30, 2025 | December 31, 2024 | Change | | :-------------------- | :------------ | :---------------- | :----- | | Cash and cash equivalents | $32,429 | $30,321 | +$2,108 | | Total current assets | $38,439 | $36,610 | +$1,829 | | Total assets | $38,665 | $36,862 | +$1,803 | | Warrant liabilities | $11,800 | $— | +$11,800 | | Total current liabilities | $20,192 | $11,295 | +$8,897 | | Total liabilities | $21,192 | $11,295 | +$9,897 | | Total stockholders' equity | $17,473 | $6,724 | +$10,749 | - The significant increase in total liabilities was driven by the introduction of **warrant liabilities ($11.8 million)** and a new **long-term funding agreement ($1.0 million)** with a related party[19](index=19&type=chunk) [Condensed Consolidated Statements of Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For the three months ended June 30, 2025, Opus Genetics reported a comprehensive loss of $7.4 million, an improvement from $7.8 million in the prior-year period. This was influenced by increased license and collaboration revenue and a fair value change in warrant liabilities, despite higher operating expenses Condensed Consolidated Statements of Comprehensive Loss (Q2 2025 vs. Q2 2024) | Metric (in thousands) | Q2 2025 | Q2 2024 | Change | | :-------------------- | :------ | :------ | :----- | | License and collaborations revenue | $2,882 | $1,112 | +$1,770 | | Total operating expenses | $11,788 | $9,440 | +$2,348 | | Loss from operations | $(8,906) | $(8,328) | -$(578) | | Fair value change in warrant and other derivative liabilities | $917 | $— | +$917 | | Net loss | $(7,420) | $(7,765) | +$345 | | Comprehensive loss | $(7,420) | $(7,765) | +$345 | - For the six months ended June 30, 2025, the net loss was **$(15,614) thousand**, compared to **$(14,871) thousand** for the same period in 2024[21](index=21&type=chunk) [About Opus Genetics](index=4&type=section&id=About%20Opus%20Genetics) Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders. Its pipeline includes AAV-based gene therapies like OPGx-LCA5 and OPGx-BEST1, and the partnered Phentolamine Ophthalmic Solution 0.75% for presbyopia and night vision disturbances - Opus Genetics is a **clinical-stage biopharmaceutical company** developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders[15](index=15&type=chunk) - The Company's pipeline features **AAV-based gene therapies** targeting inherited retinal diseases including Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[15](index=15&type=chunk) - Lead gene therapy candidates are **OPGx-LCA5** (in Phase 1/2 trial for LCA5-related mutations) and **OPGx-BEST1** (targeting BEST1-related retinal degeneration)[15](index=15&type=chunk) - Opus is also advancing **Phentolamine Ophthalmic Solution 0.75%**, a partnered therapy being studied in two Phase 3 programs for presbyopia and reduced low light vision and nighttime visual disturbances[15](index=15&type=chunk) [Forward-Looking Statements & Contacts](index=5&type=section&id=Forward-Looking%20Statements%20%26%20Contacts) This section provides a standard disclaimer regarding forward-looking statements, cautioning readers about inherent risks and uncertainties that could cause actual results to differ materially from projections. It also includes contact information for investor relations and media inquiries - This press release contains **forward-looking statements** subject to certain risks and uncertainties that could cause actual business, prospects, and results of operations to differ materially[16](index=16&type=chunk) - Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of the press release[16](index=16&type=chunk) - Contact information for investors (**Jenny Kobin, Remy Bernarda**) and media (**Kimberly Ha**) is provided[17](index=17&type=chunk)

[PART I – FINANCIAL INFORMATION](index=4&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) This part details Opus Genetics, Inc.'s unaudited condensed consolidated financial statements, including balance sheets, income, and cash flow statements, with explanatory notes [Item 1. Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) This section presents Opus Genetics, Inc.'s unaudited condensed consolidated financial statements, including balance sheets, income, and cash flow statements, with detailed notes [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This table presents the Company's financial position, detailing assets, liabilities, and equity as of March 31, 2025, and December 31, 2024 Condensed Consolidated Balance Sheets (in thousands) | Metric | March 31, 2025 (Unaudited) | December 31, 2024 | | :-------------------------------- | :-------------------------- | :------------------ | | **Assets** | | | | Cash and cash equivalents | $41,792 | $30,321 | | Total current assets | $47,928 | $36,610 | | Total assets | $48,167 | $36,862 | | **Liabilities and Stockholders' Equity** | | | | Warrant liabilities | $12,715 | — | | Total current liabilities | $24,251 | $11,295 | | Total liabilities | $24,251 | $11,295 | | Series A preferred stock | $18,843 | $18,843 | | Total stockholders' equity | $5,073 | $6,724 | | Total liabilities, series A preferred stock, and stockholders' equity | $48,167 | $36,862 | - Cash and cash equivalents increased by **$11.5 million** from December 31, 2024, to March 31, 2025[11](index=11&type=chunk) - Total current liabilities significantly increased from **$11.3 million** to **$24.3 million**, primarily due to the introduction of **$12.7 million** in warrant liabilities[11](index=11&type=chunk) [Condensed Consolidated Statements of Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) This table details the Company's financial performance, including revenue, expenses, and net loss for the three months ended March 31, 2025, and 2024 Condensed Consolidated Statements of Comprehensive Loss (in thousands, except per share amounts) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | License and collaborations revenue | $4,370 | $1,711 | | General and administrative expenses | $6,346 | $4,670 | | Research and development expenses | $7,953 | $4,749 | | Total operating expenses | $14,299 | $9,419 | | Loss from operations | $(9,929) | $(7,708) | | Financing costs | $(1,372) | — | | Fair value change in warrant liabilities | $2,805 | — | | Other income, net | $302 | $602 | | Net loss | $(8,194) | $(7,106) | | Basic and diluted net loss per share | $(0.24) | $(0.29) | | Number of shares used in per share calculations (Basic and Diluted) | 33,884,920 | 24,520,475 | - License and collaborations revenue increased by **$2.7 million (155.4%)** from **$1.7 million** in Q1 2024 to **$4.4 million** in Q1 2025[13](index=13&type=chunk) - Net loss increased by **$1.1 million (15.3%)** from **$(7.1 million)** in Q1 2024 to **$(8.2 million)** in Q1 2025[13](index=13&type=chunk) - Basic and diluted net loss per share improved from **$(0.29)** in Q1 2024 to **$(0.24)** in Q1 2025, despite a higher net loss, due to an increase in the number of shares outstanding[13](index=13&type=chunk) [Condensed Consolidated Statements of Changes in Series A Preferred Stock and Stockholders' Equity](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Changes%20in%20Series%20A%20Preferred%20Stock%20and%20Stockholders%27%20Equity) This table outlines changes in Series A Preferred Stock and stockholders' equity, reflecting issuances, repurchases, stock-based compensation, and net loss Changes in Stockholders' Equity (in thousands) | Metric | Balance at Dec 31, 2024 | Issuance of Common Stock & Warrants | Issuance Costs | Stock-based Compensation | Share Repurchases | Exercise of Stock Options | Net Loss | Balance at Mar 31, 2025 | | :-------------------------- | :---------------------- | :---------------------------------- | :------------- | :----------------------- | :------------------ | :----------------------- | :--------- | :---------------------- | | Series A Preferred Stock Amount | $18,843 | — | — | — | — | — | — | $18,843 | | Common Stock Shares | 31,574,657 | 13,749,160 | — | 186,919 | (31,913) | 5,000 | — | 45,483,823 | | Common Stock Amount | $3 | $2 | — | — | — | — | — | $5 | | Additional Paid-In Capital | $145,719 | $6,387 | $(728) | $913 | $(36) | $5 | — | $152,260 | | Accumulated Deficit | $(138,998) | — | — | — | — | — | $(8,194) | $(147,192) | | Total Stockholders' Equity | $6,724 | $5,980 | $(728) | $913 | $(36) | $5 | $(8,194) | $5,073 | - Total stockholders' equity decreased from **$6.7 million** at December 31, 2024, to **$5.1 million** at March 31, 2025, primarily due to the net loss of **$8.2 million**, partially offset by proceeds from common stock and warrant issuances[16](index=16&type=chunk) - Common stock shares outstanding increased significantly from **31,574,657** to **45,483,823**, largely due to the issuance of common stock and pre-funded warrants in connection with the March 2025 offering and private placement[16](index=16&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This table summarizes the Company's cash inflows and outflows from operating, investing, and financing activities for the three months ended March 31, 2025, and 2024 Condensed Consolidated Statements of Cash Flows (in thousands) | Activity | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(8,994) | $(5,716) | | Net cash used in investing activities | — | — | | Net cash provided by financing activities | $20,465 | $2,376 | | Net increase (decrease) in cash and cash equivalents | $11,471 | $(3,340) | | Cash and cash equivalents at beginning of period | $30,321 | $50,501 | | Cash and cash equivalents at end of period | $41,792 | $47,161 | - Net cash used in operating activities increased from **$(5.7 million)** in Q1 2024 to **$(9.0 million)** in Q1 2025[19](index=19&type=chunk) - Net cash provided by financing activities significantly increased from **$2.4 million** in Q1 2024 to **$20.5 million** in Q1 2025, primarily due to proceeds from the March 2025 offering and private placement[19](index=19&type=chunk) - The company experienced a net increase in cash and cash equivalents of **$11.5 million** in Q1 2025, a reversal from a net decrease of **$(3.3 million)** in Q1 2024[19](index=19&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) This section provides detailed explanations of significant accounting policies, mergers, commitments, financings, and other financial details [1. Company Description and Summary of Significant Accounting Policies](index=8&type=section&id=1.%20Company%20Description%20and%20Summary%20of%20Significant%20Accounting%20Policies) Opus Genetics, Inc. is a clinical-stage ophthalmic biotechnology company focused on gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders. The company completed the Opus Acquisition in October 2024, integrating Private Opus's AAV-based gene therapy portfolio. Key accounting policies include revenue recognition for license and collaboration agreements, fair value measurements for financial instruments, and the treatment of warrant liabilities - Opus Genetics, Inc. is a clinical-stage ophthalmic biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders[20](index=20&type=chunk) - On October 22, 2024, the Company acquired Private Opus, integrating its adeno-associated virus (AAV) based gene therapy portfolio[21](index=21&type=chunk)[22](index=22&type=chunk) - The company's pipeline includes OPGx-LCA5 for LCA5-associated IRD (Phase 1/2 ongoing) and OPGx-BEST1 for BEST1 gene mutations (preclinical)[22](index=22&type=chunk) - Phentolamine Ophthalmic Solution 0.75% (RYZUMVI®) was approved by the FDA in September 2023 and commercially launched in April 2024 through a license agreement with Viatris[23](index=23&type=chunk) - As of March 31, 2025, the Company had **$41.8 million** in cash and cash equivalents, believed to be sufficient for at least twelve months[29](index=29&type=chunk) Fair Value Measurements (in thousands) | Description | As of March 31, 2025 (Total) | Level 1 | Level 2 | Level 3 | | :------------------------ | :--------------------------- | :------ | :------ | :------ | | **Assets:** | | | | | | Short-term investments | $1 | $1 | — | — | | **Liabilities:** | | | | | | Warrant liabilities | $12,715 | — | — | $12,715 | | Other derivative liabilities | $2 | — | — | $2 | | **Description** | **As of December 31, 2024 (Total)** | **Level 1** | **Level 2** | **Level 3** | | **Assets:** | | | | | | Short-term investments | $2 | $2 | — | — | | **Liabilities:** | | | | | | Other derivative liabilities | $2 | — | — | $2 | [2. Mergers](index=15&type=section&id=2.%20Mergers) This section details the Opus Acquisition completed on October 22, 2024, where the Company issued common and Series A preferred stock as consideration. It also references the prior Rexahn Merger from November 2020, which included a Contingent Value Rights (CVR) Agreement for former Rexahn stockholders, with no new payments or probable milestones accrued as of March 31, 2025 - On October 22, 2024, the Company completed the Opus Acquisition, issuing **5,237,063** shares of common stock and **14,145.374** shares of Series A Preferred Stock to Private Opus security holders for a total consideration of **$25.8 million**[61](index=61&type=chunk) - The transaction was accounted for as an asset acquisition, with intellectual property associated with IRD therapies comprising over **90%** of Private Opus's assets[61](index=61&type=chunk) - The Rexahn Merger, completed on November 5, 2020, included a Contingent Value Rights (CVR) Agreement entitling former Rexahn stockholders to future payments based on BioSense, HaiChang, and Parent IP Deal revenues[63](index=63&type=chunk)[64](index=64&type=chunk)[68](index=68&type=chunk) - As of March 31, 2025, no payments subject to the CVR Agreement had been received beyond those reported in Q2 and Q3 2021, and no new milestones were considered probable[64](index=64&type=chunk) [3. Commitments and Contingencies](index=16&type=section&id=3.%20Commitments%20and%20Contingencies) This section outlines various contractual obligations and potential liabilities, primarily stemming from license agreements for intellectual property and facility leases. Most future milestone and royalty payments under these agreements were not deemed probable as of March 31, 2025, and thus no liabilities were recorded, except for a de minimis payment for the LCA5 VR License and $800,000 for the FFB strategic partnership - The Company has various license agreements (Apexian, Penn LCA5/RDH12, Iveric BEST1/RHO, Penn/UF BEST1, LCA5 VR, Penn/UF RHO, MEEI) with potential milestone and royalty payments, but none were deemed probable as of March 31, 2025, except for de minimis payments[65](index=65&type=chunk)[66](index=66&type=chunk)[67](index=67&type=chunk)[69](index=69&type=chunk)[70](index=70&type=chunk)[71](index=71&type=chunk)[72](index=72&type=chunk)[73](index=73&type=chunk) - The maximum potential aggregate milestone payments under the Iveric Agreement (BEST1 and RHO programs) are **$111.7 million**, and under the Penn and University of Florida BEST1 License Agreement are **$76.4 million**[67](index=67&type=chunk)[70](index=70&type=chunk) - The Company relocated its headquarters to Durham, NC, on January 1, 2025, with a monthly base rent of approximately **$3 thousand**, expiring September 30, 2025[74](index=74&type=chunk) - Rent expense for all leases amounted to **$67 thousand** for the three months ended March 31, 2025, significantly up from **$9 thousand** in the prior year period[76](index=76&type=chunk) - The Company is required to fund two additional installments totaling **$0.8 million** for the FFB strategic partnership, reflected in accrued expenses and other liabilities[86](index=86&type=chunk) [4. Supplemental Balance Sheet Information](index=20&type=section&id=4.%20Supplemental%20Balance%20Sheet%20Information) This section provides a breakdown of accrued expenses and other liabilities, showing changes in payroll, professional services, research and development services and supplies, and other categories between December 31, 2024, and March 31, 2025 Accrued Expenses and Other Liabilities (in thousands) | Category | March 31, 2025 | December 31, 2024 | | :-------------------------------- | :------------- | :---------------- | | Payroll | $515 | $1,481 | | Professional services | $4,070 | $1,608 | | Research and development services and supplies | $4,141 | $4,452 | | Other | $380 | $606 | | **Total** | **$9,106** | **$8,147** | - Total accrued expenses and other liabilities increased by **$1.0 million** from **$8.1 million** at December 31, 2024, to **$9.1 million** at March 31, 2025[79](index=79&type=chunk) - Professional services accrued expenses more than doubled, increasing from **$1.6 million** to **$4.1 million**[79](index=79&type=chunk) [5. Related Party Transactions](index=20&type=section&id=5.%20Related%20Party%20Transactions) This section details consulting agreements with Dr. Jay Pepose and Dr. Jean Bennett, including compensation and stock-based awards. It also notes subscription agreements with the CEO and Chairman in connection with a private offering and a strategic partnership with FFB for natural history studies - Consulting expenses for Dr. Jay Pepose amounted to **$119 thousand** for Q1 2025, up from **$99 thousand** in Q1 2024[82](index=82&type=chunk) - Dr. Jean Bennett was granted a restricted stock unit award for **100,000** shares vesting on October 22, 2025, in connection with her Board appointment and consulting agreement[84](index=84&type=chunk) - The Company's CEO, Dr. George Magrath, and Chairman, Cam Gallagher, entered into subscription agreements for shares of common stock and warrants in a March 2025 private offering[83](index=83&type=chunk)[89](index=89&type=chunk) - The Company is obligated to provide **$2.0 million** in funding to FFB for natural history studies, with **$0.8 million** remaining to be funded as of March 31, 2025[85](index=85&type=chunk)[86](index=86&type=chunk) [6. Series A Preferred Stock](index=22&type=section&id=6.%20Series%20A%20Preferred%20Stock) This section describes the Series A Non-Voting Convertible Preferred Stock issued during the Opus Acquisition. It highlights that stockholders approved the conversion of these shares into common stock on April 30, 2025, leading to the conversion of all Series A Preferred Stock into 14,145,374 shares of common stock on May 5, 2025 - **14,145.374** shares of Series A Preferred Stock were issued during the Opus Acquisition on October 22, 2024[87](index=87&type=chunk) - Stockholders approved the conversion of each Series A Preferred Stock share into **1,000** shares of common stock on April 30, 2025[87](index=87&type=chunk) - All Series A Preferred Stock was converted into **14,145,374** shares of common stock on May 5, 2025[87](index=87&type=chunk) [7. Financings](index=22&type=section&id=7.%20Financings) This section details the Company's financing activities, including the March 2025 Offering and Private Placement which raised $21.5 million gross proceeds and involved the issuance of common stock, March 2025 Warrants, March 2025 Private Placement Warrants, and Pre-Funded Warrants. It also covers the termination of the Lincoln Park Purchase Agreement, ongoing At-The-Market (ATM) programs, a Registered Direct Offering (RDO), and pre-merger financings - The March 2025 Offering and Private Placement, closed on March 24, 2025, generated approximately **$21.5 million** in combined gross proceeds[90](index=90&type=chunk) - The March 2025 Financings included the issuance of **12,219,736** shares of common stock, **8,832,895** pre-funded warrants, and **22,239,102** warrants (March 2025 Warrants and Private Placement Warrants)[88](index=88&type=chunk)[89](index=89&type=chunk) - The fair value of March 2025 Warrants at issuance was **$14.7 million**, and March 2025 Private Placement Warrants was **$0.8 million**, both classified as warrant liabilities[95](index=95&type=chunk)[98](index=98&type=chunk) - The Lincoln Park Purchase Agreement, which allowed the Company to sell up to **$50 million** of common stock, was terminated effective April 3, 2025[101](index=101&type=chunk)[106](index=106&type=chunk) - A new At-The-Market (ATM) program with Leerink Partners LLC was established on January 13, 2025, for up to **$40 million** in common stock sales, replacing a prior ATM program[107](index=107&type=chunk)[108](index=108&type=chunk) Warrant Activity Summary (in thousands, except share amounts) | Metric | Warrants (Shares) | Weighted Average Exercise Price | | :-------------------------------- | :---------------- | :------------------------------ | | Outstanding and exercisable at Dec 31, 2024 | 7,204,299 | $4.82 | | Issued (Q1 2025) | 22,229,102 | $0.96 | | Outstanding and exercisable at Mar 31, 2025 | 29,433,401 | $1.91 | [8. Stock-based Compensation](index=26&type=section&id=8.%20Stock-based%20Compensation) This section details the Company's stock-based compensation expenses and equity incentive plans. It covers the Inducement Plan and the 2020 Equity Incentive Plan, outlining the grants of stock options, restricted stock units (RSUs), and common stock for services, along with their associated expenses and vesting schedules Stock-based Compensation Expense (in thousands) | Category | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------- | :-------------------------------- | :-------------------------------- | | General and administrative | $628 | $775 | | Research and development | $285 | $210 | | **Total stock-based compensation** | **$913** | **$985** | - Total stock-based compensation decreased from **$0.99 million** in Q1 2024 to **$0.91 million** in Q1 2025[116](index=116&type=chunk) - The 2020 Equity Incentive Plan automatically increased by **1,578,733** shares on January 1, 2025, due to its evergreen provision[118](index=118&type=chunk) - Stock options granted in Q1 2025 totaled **1,262,685** with a weighted average fair value of **$0.65** per share, compared to **762,080** options at **$2.16** per share in Q1 2024[120](index=120&type=chunk)[121](index=121&type=chunk) - RSUs granted in Q1 2025 totaled **748,833** with a weighted average fair value of **$0.95** per unit, compared to **313,364** RSUs at **$2.69** per unit in Q1 2024[124](index=124&type=chunk) - Unrecognized stock-based compensation cost was **$6.8 million** as of March 31, 2025, expected to be recognized over a weighted average period of **2.0 years**[129](index=129&type=chunk) [9. Apexian Sublicense Agreement](index=30&type=section&id=9.%20Apexian%20Sublicense%20Agreement) This section details the Apexian Sublicense Agreement, under which the Company obtained exclusive worldwide patent and intellectual property rights for a Ref-1 Inhibitor program, including APX3330. The agreement includes potential milestone payments up to $11 million for development/regulatory and $20 million for sales, plus single-digit royalties on net sales. As of March 31, 2025, none of these payments were triggered or deemed probable - The Apexian Sublicense Agreement grants exclusive worldwide rights for a Ref-1 Inhibitor program, including APX3330, for ophthalmic and diabetes mellitus conditions[130](index=130&type=chunk) - Potential milestone payments include up to **$11 million** for development/regulatory achievements and up to **$20 million** for sales milestones, plus single-digit percentage royalties on net sales[131](index=131&type=chunk) - As of March 31, 2025, none of the milestone or royalty payments under this agreement were triggered or considered probable[132](index=132&type=chunk) [10. License and Collaboration Agreements](index=30&type=section&id=10.%20License%20and%20Collaboration%20Agreements) This section outlines key license and collaboration agreements, primarily focusing on the Viatris License Agreement for PS Products (RYZUMVI®), which generated $4.4 million in revenue in Q1 2025. It also covers agreements with BioSense and Processa for RX-3117, and an SBIR grant for the RHO product, noting that most future milestone payments under these agreements are constrained due to uncertainty - The Viatris License Agreement grants Viatris exclusive rights to develop and commercialize PS Products (including RYZUMVI®) in the Viatris Territory, with the Company eligible for up to **$130 million** in additional milestone payments and tiered royalties[133](index=133&type=chunk)[135](index=135&type=chunk) - Revenue recognized under the Viatris License Agreement was **$4.4 million** for Q1 2025, up from **$1.7 million** in Q1 2024, primarily from reimbursement of ongoing research and development services[138](index=138&type=chunk)[142](index=142&type=chunk) - The Company has agreements with BioSense and Processa for the RX-3117 drug compound, with potential milestone payments up to **$84.5 million** and tiered royalties, but none were deemed probable as of March 31, 2025[144](index=144&type=chunk)[147](index=147&type=chunk) - A Small Business Innovation Research (SBIR) grant of **$0.9 million** was received in September 2024 for the RHO product development, with **$24 thousand** recognized as other income in Q1 2025[149](index=149&type=chunk) [11. Net Loss per Share](index=33&type=section&id=11.%20Net%20Loss%20per%20Share) This section explains the calculation of basic and diluted net loss per share, noting that Series A Preferred Stock, warrants, stock options, and RSUs were considered common stock equivalents. However, no incremental common stock equivalents were included in diluted loss per share calculations for the periods presented because their inclusion would have been anti-dilutive due to the net loss - Basic and diluted net loss per share are calculated by dividing net loss by the weighted average number of common shares outstanding, with common stock equivalents (Series A Preferred Stock, warrants, stock options, RSUs) considered for dilution[150](index=150&type=chunk) - No incremental common stock equivalents were included in diluted loss per share for the periods presented because their effect would have been anti-dilutive due to the net loss[150](index=150&type=chunk) Potential Common Shares Not Included in Diluted EPS (Anti-Dilutive) | Instrument | March 31, 2025 | March 31, 2024 | | :---------------- | :------------- | :------------- | | Warrants | 29,433,401 | 7,204,299 | | Stock options | 6,294,455 | 4,827,433 | | RSUs | 2,039,387 | 993,112 | [12. Income Taxes](index=34&type=section&id=12.%20Income%20Taxes) This section states that the effective tax rate for the three months ended March 31, 2025, and 2024 was zero percent. A full valuation allowance has been established against the Company's net deferred income tax assets due to the uncertainty of future taxable income, resulting in no tax benefit recognized for the pre-tax loss - The effective tax rate was **zero percent** for the three months ended March 31, 2025, and 2024[154](index=154&type=chunk) - A full valuation allowance has been established to reduce the Company's net deferred income tax assets, meaning no tax benefit was recognized for the pre-tax loss[154](index=154&type=chunk) [13. Deferred Compensation Plan](index=34&type=section&id=13.%20Deferred%20Compensation%20Plan) This section describes the Company's 401(k) plan, which became effective October 1, 2021. The Company makes matching contributions of 100% on the first 3% of deferred compensation and an additional 50% on the next 2%. Contributions for Q1 2025 and Q1 2024 were $0.1 million each - The Company offers a 401(k) plan to employees, effective October 1, 2021[156](index=156&type=chunk) - Company matching contributions are **100%** on the first **3%** deferred and an additional **50%** on the next **2%**[156](index=156&type=chunk) - The Company contributed **$0.1 million** to the 401(k) plan during each of the three months ended March 31, 2025, and 2024[156](index=156&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=35&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the Company's financial condition and operational results for the three months ended March 31, 2025, compared to the prior year. It covers the strategic shift following the Opus Acquisition, pipeline updates, recent financing activities, and a detailed analysis of revenue, expenses, and cash flows, along with liquidity outlook and critical accounting policies [Forward-Looking Statements](index=35&type=section&id=Forward-Looking%20Statements) This section highlights that the report contains forward-looking statements subject to various risks and uncertainties, which the Company does not commit to update - The report contains forward-looking statements regarding strategic business plans, product candidate applications, FDA discussions, and drug development/commercialization[160](index=160&type=chunk) - These statements are subject to risks and uncertainties, including preliminary clinical data, integration challenges from the Opus Acquisition, difficulties in gene therapy development/manufacturing, clinical trial delays, regulatory changes, and dependence on strategic partners[161](index=161&type=chunk)[162](index=162&type=chunk)[168](index=168&type=chunk) - The Company does not undertake to publicly update any forward-looking statements unless required by law[165](index=165&type=chunk) [Overview](index=37&type=section&id=Overview) This section provides an overview of Opus Genetics, Inc.'s business, focusing on its clinical-stage gene therapy pipeline for inherited retinal diseases and recent product developments - Opus Genetics, Inc. is a clinical-stage ophthalmic biotechnology company focused on gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders[166](index=166&type=chunk) - The Opus Acquisition on October 22, 2024, expanded the pipeline to include AAV-based gene therapies for Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[167](index=167&type=chunk)[169](index=169&type=chunk)[170](index=170&type=chunk) - The most advanced gene therapy program, OPGx-LCA5, is in an ongoing Phase 1/2 clinical trial, showing visual improvement in adult patients and enrolling its first pediatric patient in Q1 2025[172](index=172&type=chunk) - OPGx-LCA5 received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA on May 6, 2025, which may expedite development and review[177](index=177&type=chunk) - RYZUMVI® (Phentolamine Ophthalmic Solution 0.75%) was FDA-approved in September 2023 and commercialized in April 2024 by Viatris, triggering a **$10 million** milestone payment[173](index=173&type=chunk) - The Company is seeking a strategic partner for APX3330, a small-molecule inhibitor for diabetic retinopathy, to redirect resources to gene therapy programs[175](index=175&type=chunk) [Strategic Outlook](index=40&type=section&id=Strategic%20Outlook) This section outlines the Company's future plans, including pipeline advancement, capital raising strategies, and anticipated increases in operational expenses - The Company plans to advance its current pipeline and explore out-licensing or in-licensing opportunities[178](index=178&type=chunk) - Primary activities have been R&D, business/financial planning, personnel recruitment, and capital raising[178](index=178&type=chunk) - Significant revenues are not expected until FDA approval and successful commercialization of LCA5, BEST1, other assets, or PS for additional indications[178](index=178&type=chunk) - Future cash needs are expected to be financed through equity, debt, alternative financings, collaborations, strategic alliances, and licensing arrangements[178](index=178&type=chunk) - Net loss for Q1 2025 was **$8.2 million**, increasing from **$7.1 million** in Q1 2024, with an accumulated deficit of **$147.2 million** as of March 31, 2025[181](index=181&type=chunk) - Expenses are anticipated to increase due to ongoing clinical trials, nonclinical studies, development of new candidates, regulatory approvals, manufacturing, intellectual property, and public company operations[181](index=181&type=chunk)[185](index=185&type=chunk) [Financial Operations Overview](index=41&type=section&id=Financial%20Operations%20Overview) This section provides an overview of the Company's revenue sources, expense categories, financing costs, and other income, along with its income tax policy - License and collaborations revenue is primarily derived from the Viatris License Agreement (license transfer, milestone, R&D expense reimbursement, royalties) and, to a lesser extent, from BioSense and Processa agreements[182](index=182&type=chunk) - General and administrative expenses include personnel costs, insurance, legal fees, business development, and professional fees[185](index=185&type=chunk)[186](index=186&type=chunk) - Research and development expenses cover compensation, clinical trials, regulatory activities, manufacturing, and license fees; budgeted PS development expenses are fully reimbursed by Viatris[187](index=187&type=chunk)[189](index=189&type=chunk) - R&D expenses are expected to increase in later stages of clinical development due to larger and longer trials[190](index=190&type=chunk) - Financing costs consist of issuance costs for March 2025 Warrants and Private Placement Warrants[191](index=191&type=chunk) - Fair value changes in warrant liabilities are due to fluctuations in common stock fair value, volatility, expected term, and interest rates[192](index=192&type=chunk) - Other income, net, includes interest income, gains/losses from equity investments, and grant reimbursements[193](index=193&type=chunk) - A full valuation allowance is maintained on net deferred tax assets due to uncertainty of future taxable income, resulting in no income tax provision[194](index=194&type=chunk) [Results of Operations](index=43&type=section&id=Results%20of%20Operations) This section provides a comparative analysis of the Company's revenue, expenses, and net loss for the three months ended March 31, 2025, and 2024 Operating Results Comparison (in thousands) | Metric | March 31, 2025 | March 31, 2024 | Change | | :-------------------------------- | :------------- | :------------- | :----- | | License and collaborations revenue | $4,370 | $1,711 | $2,659 | | General and administrative | $6,346 | $4,670 | $1,676 | | Research and development | $7,953 | $4,749 | $3,204 | | Total operating expenses | $14,299 | $9,419 | $4,880 | | Loss from operations | $(9,929) | $(7,708) | $(2,221) | | Financing costs | $(1,372) | — | $(1,372) | | Fair value change in warrant liabilities | $2,805 | — | $2,805 | | Other income, net | $302 | $602 | $(300) | | Net loss | $(8,194) | $(7,106) | $(1,088) | - License and collaborations revenue increased by **$2.7 million**, driven by higher reimbursable R&D activity under the Viatris License Agreement[198](index=198&type=chunk) - General and administrative expenses increased by **$1.7 million**, primarily due to higher professional services, corporate legal support, intellectual property legal fees, and investor relations costs[199](index=199&type=chunk) Research and Development Expenses Components (in thousands) | Category | March 31, 2025 | March 31, 2024 | Change | | :-------------------------------- | :------------- | :------------- | :----- | | Phentolamine Ophthalmic Solution 0.75% ("PS") | $4,035 | $1,065 | $2,970 | | IRD programs | $1,923 | — | $1,923 | | APX 3330 | $393 | $2,663 | $(2,270) | | Employee related expenses | $1,409 | $937 | $472 | | **Total research and development expenses** | **$7,953** | **$4,749** | **$3,204** | - Research and development expenses increased by **$3.2 million**, mainly due to higher clinical costs for PS and IRD programs, and increased payroll, partially offset by reduced APX3330 expenses[200](index=200&type=chunk) - Financing costs of **$1.4 million** in Q1 2025 were due to issuance costs for March 2025 Warrants and Private Placement Warrants, with no comparable costs in Q1 2024[201](index=201&type=chunk) - A **$2.8 million** benefit from fair value change in warrant liabilities was recognized in Q1 2025, reflecting fluctuations in common stock fair value and other market factors[202](index=202&type=chunk) - Other income, net, decreased from **$0.6 million** in Q1 2024 to **$0.3 million** in Q1 2025, primarily due to lower interest income[203](index=203&type=chunk)[204](index=204&type=chunk) [Liquidity and Capital Resources](index=47&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the Company's cash position, historical capital sources, recent financing activities, and future capital requirements to fund operations and R&D - As of March 31, 2025, the Company had **$41.8 million** in cash and cash equivalents, expected to fund operations for at least twelve months[205](index=205&type=chunk) - Historical capital sources include **$89.7 million** from equity offerings, **$8.5 million** from convertible notes, and **$45 million** in license/milestone payments from the Viatris License Agreement[206](index=206&type=chunk) - The March 2025 Offering and Private Placement generated approximately **$21.5 million** in gross proceeds[209](index=209&type=chunk) - The Lincoln Park Purchase Agreement, which provided for up to **$50 million** in equity line financing, was terminated effective April 3, 2025[219](index=219&type=chunk)[245](index=245&type=chunk) - The At-The-Market (ATM) program has sold **8,006,791** shares for **$26.8 million** in gross proceeds since inception[220](index=220&type=chunk)[243](index=243&type=chunk) Cash Flows Summary (in thousands) | Activity | March 31, 2025 | March 31, 2024 | | :-------------------------------- | :------------- | :------------- | | Net cash used in operating activities | $(8,994) | $(5,716) | | Net cash provided by investing activities | — | — | | Net cash provided by financing activities | $20,465 | $2,376 | | Net increase (decrease) in cash and cash equivalents | $11,471 | $(3,340) | - Operating cash outflow increased due to higher net loss and changes in operating assets/liabilities[235](index=235&type=chunk)[236](index=236&type=chunk) - Financing cash inflow significantly increased due to the March 2025 financings and ATM proceeds[238](index=238&type=chunk)[239](index=239&type=chunk) - Future capital requirements include funding R&D for gene therapy candidates, potential milestone/royalty obligations, and operational expenses, with reliance on cash on hand, future financings, and collaboration payments[247](index=247&type=chunk)[266](index=266&type=chunk)[267](index=267&type=chunk) [Critical Accounting Policies and Estimates](index=58&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section outlines the Company's critical accounting policies and estimates, particularly for revenue recognition and income tax assets and liabilities - Critical accounting policies involve significant estimates and judgments, including license and collaborations revenue recognition and income tax assets and liabilities[268](index=268&type=chunk)[269](index=269&type=chunk) - Revenue from license and collaboration agreements is recognized by allocating transaction price to performance obligations based on fair value, using discounted cash flow approach and probability assessments[270](index=270&type=chunk) - A full valuation allowance is applied to net deferred tax assets due to uncertainty of future taxable income[271](index=271&type=chunk) [Recent Accounting Pronouncements](index=58&type=section&id=Recent%20Accounting%20Pronouncements) This section refers to Note 1 for a discussion of recently issued accounting pronouncements relevant to the Company's financial statements - Refer to Note 1 for a discussion of recently issued accounting pronouncements[272](index=272&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=59&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section states that quantitative and qualitative disclosures about market risk are not applicable for smaller reporting companies - Quantitative and qualitative disclosures about market risk are not applicable for smaller reporting companies[273](index=273&type=chunk) [Item 4. Controls and Procedures](index=60&type=section&id=Item%204.%20Controls%20and%20Procedures) This section confirms that the Company's disclosure controls and procedures were evaluated and deemed effective as of March 31, 2025. It also states that there were no material changes in internal control over financial reporting during the quarter - The Company's disclosure controls and procedures were evaluated and concluded to be effective as of March 31, 2025[277](index=277&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter ended March 31, 2025[278](index=278&type=chunk) [PART II – OTHER INFORMATION](index=60&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) This section covers legal proceedings, risk factors, equity sales, defaults, mine safety, other information, and exhibits [Item 1. Legal Proceedings](index=60&type=section&id=Item%201.%20Legal%20Proceedings) This section states that the Company is not currently a party to any legal proceedings that are likely to materially affect its business or financial results, while acknowledging that litigation can have an adverse impact - The Company is not currently a party to any legal proceedings likely to materially affect its business or financial results[280](index=280&type=chunk) - Litigation, regardless of outcome, can adversely impact the Company due to defense and settlement costs and diversion of management resources[280](index=280&type=chunk) [Item 1A. Risk Factors](index=60&type=section&id=Item%201A.%20Risk%20Factors) This section highlights a new material risk factor related to the Company's common stock potentially being delisted from the Nasdaq Capital Market due to failure to comply with the Minimum Bid Price Requirement. Such delisting could negatively impact liquidity, volatility, reputation, and access to capital - No material changes to risk factors were disclosed other than those related to Nasdaq listing standards[281](index=281&type=chunk) - The Company's common stock has recently traded below the Nasdaq Minimum Bid Price Requirement of **$1.00** per share, risking delisting[282](index=282&type=chunk) - Potential delisting could lead to decreased liquidity, increased volatility, reputational damage, and impaired ability to raise capital or pursue strategic plans[284](index=284&type=chunk) - The Company may consider a reverse stock split to regain compliance, which could also negatively affect the stock price[283](index=283&type=chunk)[284](index=284&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=62&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section indicates that there were no unregistered sales of equity securities or use of proceeds to report for the period - No unregistered sales of equity securities or use of proceeds to report[286](index=286&type=chunk) [Item 3. Defaults Upon Senior Securities](index=62&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This section states that there were no defaults upon senior securities during the reporting period - No defaults upon senior securities occurred[287](index=287&type=chunk) [Item 4. Mine Safety Disclosures](index=62&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This section states that mine safety disclosures are not applicable to the Company - Mine safety disclosures are not applicable to the Company[288](index=288&type=chunk) [Item 5. Other Information](index=62&type=section&id=Item%205.%20Other%20Information) This section reports that none of the Company's directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the quarter ended March 31, 2025 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or officers during Q1 2025[289](index=289&type=chunk) [Item 6. Exhibits](index=62&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed as part of the Form 10-Q, including various agreements, bylaws, warrants, certifications, and XBRL documents - Exhibits include the Sales Agreement, Amended and Restated Bylaws, Forms of Warrant and Pre-Funded Warrant, Amended and Restated Employment Agreements, and Sarbanes-Oxley Act certifications[290](index=290&type=chunk) [SIGNATURES](index=64&type=section&id=SIGNATURES) This section contains the official signatures of the Company's Chief Executive Officer and Chief Financial Officer - The report was signed on May 15, 2025, by George Magrath, Chief Executive Officer and Director, and Nirav Jhaveri, Chief Financial Officer[294](index=294&type=chunk)[295](index=295&type=chunk)

```markdown [First Quarter 2025 Corporate Update](index=1&type=section&id=1.%20First%20Quarter%202025%20Corporate%20Update) Opus Genetics announced its Q1 2025 financial results and provided a corporate update, highlighting positive 12-month data from the OPGx-LCA5 Phase 1/2 study, ongoing pediatric enrollment, and the anticipated clinical entry of OPGx-BEST1 [Overview and Key Announcements](index=1&type=section&id=1.1.%20Overview%20and%20Key%20Announcements) Opus Genetics announced its Q1 2025 financial results and provided a corporate update, highlighting positive 12-month data from the OPGx-LCA5 Phase 1/2 study, ongoing pediatric enrollment, and the anticipated clinical entry of OPGx-BEST1 - Opus Genetics announced **Q1 2025 financial results and corporate update**[1](index=1&type=chunk)[2](index=2&type=chunk) - ARVO presentation highlighted **12-month results from Phase 1/2 study** supporting **OPGx-LCA5 gene therapy** potential to restore meaningful vision[1](index=1&type=chunk) - **Pediatric cohort of LCA5 study** ongoing with initial multi-patient data anticipated in **Q3 2025**[1](index=1&type=chunk) - **OPGx-BEST1** is on track for **IND filing** and initiation of **Phase 1/2 trial**, with early data expected in **Q1 2026**[1](index=1&type=chunk) [CEO Commentary](index=1&type=section&id=1.2.%20CEO%20Commentary) CEO George Magrath emphasized a strong start to 2025 with significant progress across both the inherited retinal disease (IRD) gene therapy platform and the phentolamine eye drop franchise, noting durable efficacy for OPGx-LCA5 and a strengthened cash position from recent financing - **CEO George Magrath** noted a **strong start in 2025** with progress in **IRD platform** and **phentolamine eye drop franchise**[3](index=3&type=chunk) - **Positive 12-month data** from **OPGx-LCA5 Phase 1/2 trial** showed **durable efficacy** in adult patients[3](index=3&type=chunk) - Initial success with **OPGx-LCA5** has the potential to translate to the rest of the pipeline, including **six additional IRD gene therapy candidates**[4](index=4&type=chunk) - Company ended Q1 with a **strong cash position** after completing a successful financing with leading institutional healthcare investors[4](index=4&type=chunk) [Strategic Highlights and Pipeline Progress](index=2&type=section&id=2.%20Strategic%20Highlights%20and%20Pipeline%20Progress) Opus Genetics is advancing its gene therapy and ophthalmic solution pipelines, with significant clinical progress, regulatory designations, and upcoming data readouts across multiple programs [Gene Therapy Programs](index=2&type=section&id=2.1.%20Gene%20Therapy%20Programs) Opus Genetics is advancing its AAV-based gene therapy pipeline, with OPGx-LCA5 showing promising 12-month clinical data in adults, initiating a pediatric cohort, and receiving RMAT designation. OPGx-BEST1 is on track for IND filing and Phase 1/2 trial initiation by Q4 2025 - Opus's pipeline includes a portfolio of **seven adeno-associated virus (AAV)-based gene therapy assets**, each targeting a specific IRD[7](index=7&type=chunk) - Emerging clinical data on **OPGx-LCA5** provide evidence of **clinical proof of concept** and support its potential to restore meaningful vision[7](index=7&type=chunk) - **OPGx-BEST1** is on track to enter the clinic by **Q4 2025**, with preliminary data expected in **Q1 2026**[7](index=7&type=chunk) - **OPGx-LCA5** received **Regenerative Medicine Advanced Therapy (RMAT) designation** from the FDA, offering potential for expedited development and review[7](index=7&type=chunk) [OPGx-LCA5 Program](index=2&type=section&id=2.1.1.%20OPGx-LCA5%20Program) The OPGx-LCA5 program demonstrated sustained efficacy in adult patients, initiated pediatric enrollment with encouraging early safety, and discussed regulatory pathways with the FDA - **One-year open-label data** on adult patients in the **Phase 1/2 clinical trial of OPGx-LCA5** showed **sustained improvements** in subjective and objective measures of efficacy[7](index=7&type=chunk) - Enrollment in a cohort of **three pediatric patients** began in **February 2025**, with preliminary data on the first patient showing an **encouraging early safety profile** and **meaningful improvement in visual function** at one month[7](index=7&type=chunk) - The pediatric cohort is expected to complete enrollment in **Q2 2025**, with initial data from all three patients anticipated in **Q3 2025**[7](index=7&type=chunk) - A **Type D meeting** was held with the **FDA** in **March 2025** to discuss the potential regulatory path for **OPGx-LCA5**, including the design of a potential registrational study[8](index=8&type=chunk) [OPGx-BEST1 Program](index=3&type=section&id=2.1.2.%20OPGx-BEST1%20Program) The OPGx-BEST1 program is advancing towards an IND filing and Phase 1/2 trial initiation by Q4 2025, supported by positive IND-enabling study data - **OPGx-BEST1** is an investigational **Phase 1/2-ready asset** in development for IRDs associated with mutations in the **BEST1 gene**[11](index=11&type=chunk) - **IND-enabling studies of OPGx-BEST1** provided **safety and efficacy data** in support of a first-in-human clinical trial[11](index=11&type=chunk) - Opus plans to file an **IND** and begin a **Phase 1/2 trial by Q4 2025**, with preliminary data expected in **Q1 2026**[11](index=11&type=chunk) [Phentolamine Ophthalamine Solution 0.75% Program](index=3&type=section&id=2.2.%20Phentolamine%20Ophthalmic%20Solution%200.75%25%20Program) The Phentolamine Ophthalmic Solution 0.75% program is progressing with two pivotal Phase 3 trials, LYNX-2 (for dim light vision disturbances) and VEGA-3 (for presbyopia), both having completed enrollment in Q1 2025 with topline data expected mid-year 2025 and H1 2025, respectively. The program has FDA Fast Track designation for LYNX-2 and is funded by Viatris Inc - The **LYNX-2 pivotal Phase 3 trial** for visual loss in low light conditions completed enrollment in **Q1 2025**, with topline data expected **mid-year 2025**[11](index=11&type=chunk) - The **LYNX-2 trial** is covered by a **Special Protocol Assessment (SPA) agreement** with the FDA[11](index=11&type=chunk) - The **FDA** granted **Fast Track designation** for **Phentolamine Ophthalmic Solution 0.75%** for treatment of significant chronic night driving impairment[11](index=11&type=chunk) - The **VEGA-3 pivotal Phase 3 trial** for presbyopia completed enrollment in **Q1 2025**, with topline data expected in the **first half of 2025**[11](index=11&type=chunk) - The development portfolio related to **Phentolamine Ophthalmic Solution 0.75%** is being funded by the Company's partner, **Viatris Inc.**[11](index=11&type=chunk) [Expected Milestones and Growth Drivers](index=3&type=section&id=2.3.%20Expected%20Milestones%20and%20Growth%20Drivers) Key upcoming milestones include initial data from pediatric OPGx-LCA5 patients in Q3 2025, IND filing and Phase 1/2 initiation for OPGx-BEST1 in 2025 with Q1 2026 data, and topline data from both LYNX-2 and VEGA-3 Phase 3 trials for Phentolamine Ophthalmic Solution 0.75% in mid-2025 and H1 2025, respectively - **Initial data** from **three pediatric patients** treated with **OPGx-LCA5** anticipated in **Q3 2025**[11](index=11&type=chunk) - **IND filing** and initiation of a **Phase 1/2 clinical trial for OPGx-BEST1** is planned for **2025**, with preliminary data expected in **Q1 2026**[11](index=11&type=chunk) - **Topline data** from the **LYNX-2 pivotal Phase 3 trial** evaluating **Phentolamine Ophthalmic Solution 0.75%** for visual loss in low light conditions associated with keratorefractive surgery are expected **mid-year 2025**[11](index=11&type=chunk) - **Topline data** from the **VEGA-3 pivotal Phase 3 clinical trial** evaluating **Phentolamine Ophthalmic Solution 0.75%** for the treatment of presbyopia are expected in the **first half of 2025**[11](index=11&type=chunk) [Financing and Funding](index=2&type=section&id=2.4.%20Financing%20and%20Funding) In March 2025, Opus completed a successful public offering and private placement, raising approximately $21.5 million in gross proceeds, with potential for an additional $21.4 million from warrant exercises tied to the BEST1 program, significantly strengthening the company's cash position - In **March 2025**, Opus completed a **successful underwritten public offering** and concurrent **private placement** anchored by leading healthcare investors[7](index=7&type=chunk) - The transaction raised approximately **$21.5 million in gross proceeds**[7](index=7&type=chunk) - Potential for up to **$21.4 million in additional proceeds** upon exercise of warrants that are tied to data release from the **BEST1 program**[7](index=7&type=chunk) [Financial Results for Q1 2025](index=4&type=section&id=3.%20Financial%20Results%20for%20Q1%202025) Opus Genetics reported its Q1 2025 financial performance, highlighting increased cash and cash equivalents, significant growth in license revenue, and higher operating expenses leading to an increased net loss [Financial Highlights](index=4&type=section&id=3.1.%20Financial%20Highlights) Opus Genetics reported $41.8 million in cash and cash equivalents as of March 31, 2025, providing a funding runway into Q2 2026. License and collaborations revenue increased significantly by 158.8% YoY, while both G&A and R&D expenses rose by 34.0% and 69.6% respectively, leading to an increased net loss of $8.2 million for the quarter Cash and Cash Equivalents | Metric | March 31, 2025 | | :---------------------- | :------------- | | Cash and cash equivalents | $41.8 million | | Funding runway | Into Q2 2026 | License and Collaborations Revenue | Metric | Q1 2025 | Q1 2024 | YoY Change | | :------ | :------------- | :------------- | :--------- | | Revenue | $4.4 million | $1.7 million | +158.8% | Operating Expenses (in millions) | Metric | Q1 2025 | Q1 2024 | YoY Change | | :----------- | :------ | :------ | :--------- | | G&A Expenses | $6.3 | $4.7 | +34.0% | | R&D Expenses | $8.0 | $4.7 | +69.6% | Net Loss and EPS | Metric | Q1 2025 | Q1 2024 | | :---------------- | :------------- | :------------- | | Net Loss | $(8.2) million | $(7.1) million | | Basic & Diluted EPS | $(0.24) | $(0.29) | [Condensed Consolidated Balance Sheets](index=9&type=section&id=3.2.%20Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2025, total assets increased to $48.167 million from $36.862 million at December 31, 2024, primarily driven by higher cash and cash equivalents. Total current liabilities significantly increased to $24.251 million due to the introduction of $12.715 million in warrant liabilities Condensed Consolidated Balance Sheets (in thousands) | | As of March 31, 2025 (Unaudited) | As of December 31, 2024 | | :-------------------------------- | :------------------------- | :---------------------- | | **Assets** | | | | Cash and cash equivalents | $41,792 | $30,321 | | Total current assets | $47,928 | $36,610 | | Total assets | $48,167 | $36,862 | | **Liabilities and Stockholders' Equity** | | | | Warrant liabilities | $12,715 | $— | | Total current liabilities | $24,251 | $11,295 | | Total liabilities | $24,251 | $11,295 | | Total stockholders' equity | $5,073 | $6,724 | [Condensed Consolidated Statements of Comprehensive Loss](index=10&type=section&id=3.3.%20Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For Q1 2025, Opus Genetics reported a net loss of $8.194 million, an increase from $7.106 million in Q1 2024. This was primarily due to higher operating expenses (G&A and R&D) and new financing costs, partially offset by a fair value change in warrant liabilities and increased license revenue Condensed Consolidated Statements of Comprehensive Loss (in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | License and collaborations revenue | $4,370 | $1,711 | | Total operating expenses | $14,299 | $9,419 | | Loss from operations | $(9,929) | $(7,708) | | Financing costs | $(1,372) | $— | | Fair value change in warrant liabilities | $2,805 | $— | | Net loss | $(8,194) | $(7,106) | | Basic and diluted EPS | $(0.24) | $(0.29) | [Additional Information](index=5&type=section&id=4.%20Additional%20Information) This section provides an overview of Opus Genetics' mission and pipeline, outlines forward-looking statements and associated risk factors, and lists key contact information [About Opus Genetics](index=5&type=section&id=4.1.%20About%20Opus%20Genetics) Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company focused on developing AAV-based gene therapies for inherited retinal diseases (IRDs) such as LCA, bestrophinopathy, and retinitis pigmentosa, alongside Phentolamine Ophthalmic Solution 0.75% for presbyopia and dim light vision disturbances, currently in Phase 3 trials - **Opus Genetics** is a **clinical-stage ophthalmic biopharmaceutical company** developing therapies to treat patients with IRDs and other ophthalmic disorders[17](index=17&type=chunk) - Pipeline includes **AAV-based investigational gene therapies** addressing gene mutations for different forms of **Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa**[17](index=17&type=chunk) - The pipeline also includes **Phentolamine Ophthalmic Solution 0.75%**, being investigated in **Phase 3 trials** for **presbyopia** and **mesopic (dim) light vision disturbances**[17](index=17&type=chunk) [Forward-Looking Statements and Risk Factors](index=5&type=section&id=4.2.%20Forward-Looking%20Statements%20and%20Risk%20Factors) The report contains forward-looking statements regarding future operations and financial performance, which are subject to various risks and uncertainties. Key risks include the preliminary nature of clinical data, integration challenges from the Opus Acquisition, difficulties in gene therapy development, potential clinical trial delays, regulatory changes, dependence on pipeline success and third parties, intellectual property issues, and the need for additional capital - This press release contains **forward-looking statements** regarding cash runway, potential growth, and clinical trial data, subject to certain risks and uncertainties[19](index=19&type=chunk)[20](index=20&type=chunk) - **Key risks** include **preliminary clinical data**, failure to successfully integrate businesses after the **Opus Acquisition**, difficulties in developing and manufacturing **novel gene therapies**, and potential **clinical trial delays or failures**[21](index=21&type=chunk) - Other risks involve **changes in regulatory requirements**, heavy dependence on **product pipeline**, **intellectual property protection**, and the future need for **substantial additional capital**[21](index=21&type=chunk)[22](index=22&type=chunk)[25](index=25&type=chunk) - Readers are cautioned not to place undue reliance on these **forward-looking statements** and to review detailed **risk factors in SEC filings**[20](index=20&type=chunk)[23](index=23&type=chunk) [Contacts](index=8&type=section&id=4.3.%20Contacts) Contact information for corporate inquiries (Nirav Jhaveri, CFO) and investor relations (Corey Davis, Ph.D. at LifeSci Advisors) is provided Contacts | Role | Name | Email | | :--------------- | :-------------------- | :---------------------- | | Corporate (CFO) | Nirav Jhaveri | ir@ocuphire.com | | Investor Relations | Corey Davis, Ph.D. | cdavis@lifesciadvisors.com | ```

PART I [ITEM 1. BUSINESS](index=6&type=section&id=ITEM%201.%20BUSINESS) Opus Genetics develops gene therapies for inherited retinal diseases, expanding its pipeline and prioritizing gene therapy programs - **Opus Genetics, Inc.** is a clinical-stage ophthalmic biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders[31](index=31&type=chunk) - The **Opus Acquisition** in **October 2024** significantly expanded the product pipeline to include adeno-associated virus (AAV) based gene therapies for Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa[35](index=35&type=chunk)[40](index=40&type=chunk) - **OPGx-LCA5**, the most advanced gene therapy program, targets LCA5 gene mutations. An ongoing **Phase 1/2 clinical trial** has shown visual improvement in three adult patients with late-stage disease (one-year data) with no dose-limiting toxicity or serious adverse events. The program has received **Rare Pediatric Disease Designation** and **Orphan Drug Designation** from the FDA, with the first pediatric patient enrolled in **Q1 2025** and data anticipated in **Q3 2025**[36](index=36&type=chunk)[44](index=44&type=chunk)[55](index=55&type=chunk)[58](index=58&type=chunk) - **OPGx-BEST1** is another gene therapy candidate for **BEST1 gene mutations (Best Disease)**, with preclinical studies supporting a first-in-man clinical trial. Preliminary **Phase 1/2** data is aimed for **Q1 2026**[37](index=37&type=chunk)[44](index=44&type=chunk)[78](index=78&type=chunk)[89](index=89&type=chunk) - **RYZUMVI (Phentolamine Ophthalmic Solution 0.75%)** was approved by the FDA in **September 2023** for pharmacologically induced mydriasis and commercially launched by **Viatris** in **April 2024**. Two **Phase 3** trials (**VEGA-3** for presbyopia, **LYNX-2** for dim light vision disturbances) are ongoing, with topline results expected in **H1 2025** and **mid-2025**, respectively. It also received **Fast Track designation** for chronic night driving impairment[39](index=39&type=chunk)[45](index=45&type=chunk)[47](index=47&type=chunk)[48](index=48&type=chunk)[99](index=99&type=chunk)[107](index=107&type=chunk)[110](index=110&type=chunk)[115](index=115&type=chunk)[119](index=119&type=chunk) - **APX3330**, a small-molecule inhibitor for **non-proliferative diabetic retinopathy (NPDR)**, has completed **Phase 2** and received FDA agreement under SPA for a **Phase 3** program. The company is seeking a strategic partner to advance its clinical development, redirecting existing resources toward gene therapy programs[38](index=38&type=chunk)[42](index=42&type=chunk)[45](index=45&type=chunk)[126](index=126&type=chunk)[130](index=130&type=chunk) - The company relies on **third-party contract manufacturers** for drug substances and products and does not own or operate any manufacturing facilities[143](index=143&type=chunk) - The company faces substantial competition from **major pharmaceutical and biotechnology companies** with greater resources. While no direct competitors are currently known for **OPGx-LCA5**, **BEST1**, **NMNAT1**, or **MerTK gene therapies**, other IRD gene therapies are in development. **RYZUMVI** faces competition for presbyopia and a generic challenge from **Sandoz, Inc. APX3330** competes with **anti-VEGF therapies** and other R&D stage treatments for diabetic retinopathy[185](index=185&type=chunk)[186](index=186&type=chunk)[187](index=187&type=chunk)[188](index=188&type=chunk)[189](index=189&type=chunk)[380](index=380&type=chunk)[381](index=381&type=chunk)[382](index=382&type=chunk)[397](index=397&type=chunk) [Company Overview & Strategy](index=6&type=section&id=Company%20Overview%20%26%20Strategy) This section describes the company's core business, strategic focus, and operational approach [Pipeline Overview](index=9&type=section&id=Pipeline%20Overview) This section details the company's product candidates, their development stages, and therapeutic areas [Market Overview](index=25&type=section&id=Market%20Overview) This section provides an analysis of the target markets for the company's products [Sales and Marketing](index=26&type=section&id=Sales%20and%20Marketing) This section outlines the company's strategies for product sales and market penetration [Manufacturing](index=26&type=section&id=Manufacturing) This section describes the company's manufacturing processes and supply chain arrangements [License Agreements](index=28&type=section&id=License%20Agreements) This section details the company's various licensing and collaboration agreements [Intellectual Property](index=30&type=section&id=Intellectual%20Property) This section covers the company's patents, trademarks, and other proprietary rights [Competition](index=32&type=section&id=Competition) This section analyzes the competitive landscape and key market rivals [Government Regulation and Product Approvals](index=33&type=section&id=Government%20Regulation%20and%20Product%20Approvals) This section explains the regulatory framework and approval processes for the company's products [Healthcare Reform](index=47&type=section&id=Healthcare%20Reform) This section discusses the impact of healthcare reform on the company's business [Healthcare Frauds & Abuse and Compliance Laws and Regulations](index=49&type=section&id=Healthcare%20Frauds%20%26%20Abuse%20and%20Compliance%20Laws%20and%20Regulations) This section addresses compliance with healthcare fraud and abuse laws [Healthcare Reimbursement](index=52&type=section&id=Healthcare%20Reimbursement) This section describes the reimbursement landscape for the company's products [Human Capital Resources](index=54&type=section&id=Human%20Capital%20Resources) This section provides information on the company's employees and human resource management [Available Information](index=54&type=section&id=Available%20Information) This section specifies where public information about the company can be accessed [ITEM 1A. RISK FACTORS](index=54&type=section&id=ITEM%201A.%20RISK%20FACTORS) The company faces significant risks in acquisition, gene therapy, clinical trials, commercialization, finances, regulation, and IP - Risks related to the **Opus Acquisition** include potential integration failures, the expanded pipeline not improving common stock value, and the possibility of diverting funds for **Series A Preferred Stock** dividends if conversion is not approved by stockholders[315](index=315&type=chunk)[316](index=316&type=chunk)[318](index=318&type=chunk)[319](index=319&type=chunk)[320](index=320&type=chunk)[321](index=321&type=chunk) - Gene therapy product candidates are based on novel technology, making development time and cost difficult to predict. There is a risk of unforeseen adverse events, negative public perception, and complexities in manufacturing[322](index=322&type=chunk)[325](index=325&type=chunk)[327](index=327&type=chunk)[328](index=328&type=chunk) - Clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results. Difficulties in patient enrollment and changes in regulatory requirements could further impede development and regulatory approvals[346](index=346&type=chunk)[350](index=350&type=chunk)[353](index=353&type=chunk)[356](index=356&type=chunk)[358](index=358&type=chunk)[360](index=360&type=chunk) - Commercial success is heavily dependent on the product pipeline. Failure to find strategic partners or inadequate commercialization by partners (like **Viatris** for **RYZUMVI**/PS) could materially harm the business. Achieving market acceptance among physicians, patients, and payors is also a significant challenge[364](index=364&type=chunk)[365](index=365&type=chunk)[368](index=368&type=chunk)[369](index=369&type=chunk)[373](index=373&type=chunk)[388](index=388&type=chunk)[392](index=392&type=chunk) - The company has not generated significant revenue from product sales, expects to incur losses for the foreseeable future, and had an **accumulated deficit** of approximately **$139.0 million** as of **December 31, 2024**. Substantial additional capital will be needed, which could lead to stockholder dilution or require relinquishing rights to technologies[412](index=412&type=chunk)[414](index=414&type=chunk)[415](index=415&type=chunk)[416](index=416&type=chunk)[423](index=423&type=chunk)[426](index=426&type=chunk)[719](index=719&type=chunk)[722](index=722&type=chunk) - The highly regulated biopharmaceutical industry means legislative reform or changes in the regulatory environment could increase the difficulty and cost of obtaining marketing approval, affect pricing, and impact commercial viability. Post-marketing obligations, restrictions, or withdrawal from the market are also risks[433](index=433&type=chunk)[434](index=434&type=chunk)[436](index=436&type=chunk)[437](index=437&type=chunk) - Inadequate patent protection could allow competitors to commercialize similar products. The company is currently involved in a **patent infringement lawsuit** against **Sandoz, Inc.** regarding **RYZUMVI®**, which could be expensive, time-consuming, and unsuccessful, potentially reducing market share and future revenue[397](index=397&type=chunk)[398](index=398&type=chunk)[487](index=487&type=chunk)[490](index=490&type=chunk)[491](index=491&type=chunk)[498](index=498&type=chunk)[503](index=503&type=chunk)[504](index=504&type=chunk)[510](index=510&type=chunk) - The company relies completely on third parties (CROs, manufacturers) for nonclinical and clinical trials, as well as drug supply and manufacturing. Failures by these third parties to meet contractual duties, deadlines, or regulatory requirements could delay or prevent regulatory approval and harm the business[460](index=460&type=chunk)[462](index=462&type=chunk)[465](index=465&type=chunk)[471](index=471&type=chunk)[473](index=473&type=chunk)[476](index=476&type=chunk) - The business and operations are vulnerable to system failures or unplanned events, including cyber incidents, network security breaches, service interruptions, or data corruption, which could materially disrupt operations and adversely affect the business[532](index=532&type=chunk)[546](index=546&type=chunk) [ITEM 1B. UNRESOLVED STAFF COMMENTS](index=95&type=section&id=ITEM%201B.%20UNRESOLVED%20STAFF%20COMMENTS) The company has no unresolved staff comments from the SEC - No unresolved staff comments are applicable to the company[542](index=542&type=chunk) [ITEM 1C. CYBERSECURITY](index=95&type=section&id=ITEM%201C.%20CYBERSECURITY) The company manages cybersecurity risks via NIST frameworks, with no material incidents reported to date - The company has adopted a cybersecurity risk management program based on **National Institute of Standards and Technology (NIST) frameworks**[543](index=543&type=chunk) - The program includes processes to identify, assess, manage, and monitor risks from cybersecurity threats, conducting assessments at least annually and evaluating third-party vendor cybersecurity risks[543](index=543&type=chunk) - As of the report date, no risks from a cybersecurity threat or incident have been identified that are reasonably likely to have a material effect on the business strategy, results of operations, or financial condition[546](index=546&type=chunk) - The cybersecurity risk management program is led by the Company's **Finance organization**, with the **Senior Vice President of Finance** reporting on cybersecurity matters to the **Audit Committee** at least annually and to senior management as appropriate[547](index=547&type=chunk) [ITEM 2. PROPERTIES](index=96&type=section&id=ITEM%202.%20PROPERTIES) The company's headquarters is in Durham, NC, occupying 84 square feet under a lease expiring in September 2025 - The company's headquarters is located in **Durham, North Carolina**[548](index=548&type=chunk) - The headquarters consists of approximately **84 square feet** of office space under a short-term, non-cancellable facility lease that expires on **September 30, 2025**[548](index=548&type=chunk) [ITEM 3. LEGAL PROCEEDINGS](index=96&type=section&id=ITEM%203.%20LEGAL%20PROCEEDINGS) The company is involved in a patent infringement lawsuit against Sandoz regarding RYZUMVI generic approval - The company is subject to litigation and claims arising in the ordinary course of business[549](index=549&type=chunk) - In **March 2025**, the company, in collaboration with its commercialization partner for **RYZUMVI®**, filed a **patent infringement complaint** against **Sandoz, Inc.** in response to **Sandoz's ANDA** filing for a generic version of **RYZUMVI**[549](index=549&type=chunk)[504](index=504&type=chunk) - The complaint seeks equitable relief enjoining **Sandoz** from infringing the specified **RYZUMVI** patents[549](index=549&type=chunk) [ITEM 4. MINE SAFETY DISCLOSURES](index=97&type=section&id=ITEM%204.%20MINE%20SAFETY%20DISCLOSURES) This item is not applicable to the company - **Mine Safety Disclosures** are not applicable to the registrant[550](index=550&type=chunk) PART II [ITEM 5. MARKET FOR REGISTRANT'S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES](index=97&type=section&id=ITEM%205.%20MARKET%20FOR%20REGISTRANT%27S%20COMMON%20EQUITY%2C%20RELATED%20STOCKHOLDER%20MATTERS%20AND%20ISSUER%20PURCHASES%20OF%20EQUITY%20SECURITIES) Common stock trades on Nasdaq, with new shares issued post-acquisition and Series A Preferred Stock convertible pending approval - The company's common stock trades on the **Nasdaq Capital Market** under the symbol "**IRD**"[5](index=5&type=chunk)[551](index=551&type=chunk) - As of **March 27, 2025**, there were **45,483,823 shares** of common stock outstanding and approximately **67 holders of record**[7](index=7&type=chunk)[552](index=552&type=chunk) - In connection with the **Opus Acquisition** on **October 22, 2024**, the company issued **5,237,063 shares** of common stock and **14,145.374 shares** of **Series A Non-Voting Convertible Preferred Stock** to the securityholders of Private Opus[553](index=553&type=chunk)[565](index=565&type=chunk)[770](index=770&type=chunk) - The **Series A Preferred Stock** is convertible into common stock, subject to stockholder approval at the **2025 Annual Meeting**. If conversion is not approved, holders will be entitled to **cumulative quarterly cash dividends** starting **October 15, 2025**[321](index=321&type=chunk)[554](index=554&type=chunk)[556](index=556&type=chunk)[807](index=807&type=chunk) - The company has not paid any cash dividends on its common stock since inception and plans to retain future earnings for business development[538](index=538&type=chunk)[560](index=560&type=chunk) [ITEM 6. [RESERVED]](index=101&type=section&id=ITEM%206.%20%5BRESERVED%5D) This item is reserved and not applicable - This item is reserved and not applicable[562](index=562&type=chunk) [ITEM 7. MANAGEMENT'S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS](index=101&type=section&id=ITEM%207.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) The company discusses its financial condition, operating results, and capital needs, reporting a net loss of $57.5 million for 2024 - **Opus Genetics, Inc.** is a clinical-stage ophthalmic biotechnology company developing gene therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders[564](index=564&type=chunk) - The company acquired Private Opus on **October 22, 2024**, expanding its pipeline to include AAV-based gene therapies for LCA, bestrophinopathy, and retinitis pigmentosa[565](index=565&type=chunk)[566](index=566&type=chunk) - Key pipeline assets include **OPGx-LCA5** (**Phase 1/2** ongoing, pediatric data **Q3 2025**), **OPGx-BEST1** (preliminary **Phase 1/2** data **Q1 2026**), **RYZUMVI** (FDA approved **Sept 2023**, commercialized **April 2024** by **Viatris**), and **APX3330** (**Phase 3** program ready, seeking partner)[569](index=569&type=chunk)[570](index=570&type=chunk)[571](index=571&type=chunk)[572](index=572&type=chunk) Summary of Operating Results (in thousands) | Metric | 2024 | 2023 | Change | | :----------------------------------- | :---------- | :---------- | :---------- | | License and collaborations revenue | $10,992 | $19,049 | $(8,057) | | General and administrative expenses | $18,215 | $11,959 | $6,256 | | Research and development expenses | $26,851 | $17,653 | $9,198 | | Acquired in-process R&D expenses | $28,000 | $0 | $28,000 | | Total operating expenses | $73,066 | $29,612 | $43,454 | | Loss from operations | $(62,074) | $(10,563) | $(51,511) | | Net loss | $(57,532) | $(9,986) | $(47,546) | | Accumulated deficit (as of Dec 31) | $(138,998) | $(81,466) | $(57,532) | - As of **December 31, 2024**, the company had **$30.3 million** in **cash and cash equivalents** and believes this is sufficient to fund operations for at least twelve months[602](index=602&type=chunk)[640](index=640&type=chunk)[737](index=737&type=chunk) - The company's primary funding sources have been **equity offerings** (**$89.7 million**), **convertible notes** (**$8.5 million**), and license/milestone payments from the **Viatris License Agreement** (**$35 million** non-refundable, **$10 million** milestone)[603](index=603&type=chunk) - Subsequent to **December 31, 2024**, the company raised approximately **$21.5 million** in combined gross proceeds from an **underwritten public offering** and a **private placement** in **March 2025**[607](index=607&type=chunk)[891](index=891&type=chunk) - The company anticipates substantial additional capital requirements in the future to fund R&D, regulatory approvals, manufacturing, and commercialization efforts, which may involve equity, debt, or strategic collaborations[423](index=423&type=chunk)[644](index=644&type=chunk)[647](index=647&type=chunk) [Company Overview](index=101&type=section&id=Company%20Overview) This section provides a high-level summary of the company's business and strategic direction [Strategic Outlook](index=102&type=section&id=Strategic%20Outlook) This section outlines the company's future plans and strategic priorities [Financial Operations Overview](index=103&type=section&id=Financial%20Operations%20Overview) This section summarizes the company's financial structure and operational performance [Results of Operations](index=105&type=section&id=Results%20of%20Operations) This section analyzes the company's financial performance over the reporting periods [Liquidity and Capital Resources](index=107&type=section&id=Liquidity%20and%20Capital%20Resources) This section details the company's cash position, funding sources, and capital needs [Contractual Obligations and Commitments](index=113&type=section&id=Contractual%20Obligations%20and%20Commitments) This section outlines the company's significant contractual liabilities and future commitments [Critical Accounting Policies and Estimates](index=114&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section describes the key accounting policies and significant estimates used in financial reporting [Recent Accounting Pronouncements](index=115&type=section&id=Recent%20Accounting%20Pronouncements) This section discusses the impact of recently issued accounting standards [ITEM 7A. QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK](index=115&type=section&id=ITEM%207A.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURES%20ABOUT%20MARKET%20RISK) This item is not applicable to the company - **Quantitative and Qualitative Disclosures About Market Risk** are not applicable to the registrant[666](index=666&type=chunk) [ITEM 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA](index=126&type=section&id=ITEM%208.%20FINANCIAL%20STATEMENTS%20AND%20SUPPLEMENTARY%20DATA) This section presents audited financial statements, including balance sheets, income statements, and cash flows, with notes - The section includes the **Report of Independent Registered Public Accounting Firm**, **Consolidated Balance Sheets**, **Consolidated Statements of Comprehensive Loss**, **Consolidated Statements of Changes in Series A Preferred Stock and Stockholders' Equity**, **Consolidated Statements of Cash Flows**, and **Notes to Consolidated Financial Statements**[671](index=671&type=chunk)[704](index=704&type=chunk) - The consolidated financial statements are prepared in conformity with **U.S. Generally Accepted Accounting Principles (GAAP)**[15](index=15&type=chunk)[708](index=708&type=chunk)[734](index=734&type=chunk) - A **critical audit matter** identified was the accounting for the **Opus Acquisition**, specifically the significant estimation used to determine the fair value of **acquired in-process research and development (IPR&D)** of **$28 million**, due to the complexity of the valuation model and sensitivity of underlying assumptions[713](index=713&type=chunk)[714](index=714&type=chunk) [Report of Independent Registered Public Accounting Firm](index=127&type=section&id=Report%20of%20Independent%20Registered%20Public%20Accounting%20Firm) This section presents the auditor's opinion on the company's financial statements [Consolidated Balance Sheets](index=129&type=section&id=Consolidated%20Balance%20Sheets) This section provides a snapshot of the company's assets, liabilities, and equity at specific dates [Consolidated Statements of Comprehensive Loss](index=130&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) This section details the company's revenues, expenses, and net loss over periods [Consolidated Statements of Changes in Series A Preferred Stock and Stockholders' Equity](index=131&type=section&id=Consolidated%20Statements%20of%20Changes%20in%20Series%20A%20Preferred%20Stock%20and%20Stockholders%27%20Equity) This section tracks changes in the company's equity accounts [Consolidated Statements of Cash Flows](index=132&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) This section reports the company's cash inflows and outflows from operating, investing, and financing activities [Notes to Consolidated Financial Statements](index=133&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section provides detailed explanations and disclosures supporting the financial statements [Note 1. Company Description and Summary of Significant Accounting Policies](index=133&type=section&id=Note%201.%20Company%20Description%20and%20Summary%20of%20Significant%20Accounting%20Policies) This note details Company Description and Summary of Significant Accounting Policies [Note 2. Mergers](index=138&type=section&id=Note%202.%20Mergers) This note details Mergers [Note 3. Commitments and Contingencies](index=140&type=section&id=Note%203.%20Commitments%20and%20Contingencies) This note details Commitments and Contingencies [Note 4. Supplemental Balance Sheet Information](index=143&type=section&id=Note%204.%20Supplemental%20Balance%20Sheet%20Information) This note details Supplemental Balance Sheet Information [Note 5. Related Party Transactions](index=143&type=section&id=Note%205.%20Related%20Party%20Transactions) This note details Related Party Transactions [Note 6. Series A Preferred Stock](index=144&type=section&id=Note%206.%20Series%20A%20Preferred%20Stock) This note details Series A Preferred Stock [Note 7. Stockholders' Equity](index=145&type=section&id=Note%207.%20Stockholders%27%20Equity) This note details Stockholders' Equity [Note 8. Stock-based Compensation](index=148&type=section&id=Note%208.%20Stock-based%20Compensation) This note details Stock-based Compensation [Note 9. Apexian Sublicense Agreement](index=150&type=section&id=Note%209.%20Apexian%20Sublicense%20Agreement) This note details Apexian Sublicense Agreement [Note 10. License and Collaboration Agreements](index=151&type=section&id=Note%2010.%20License%20and%20Collaboration%20Agreements) This note details License and Collaboration Agreements [Note 11. Net loss per share](index=153&type=section&id=Note%2011.%20Net%20loss%20per%20share) This note details Net loss per share [Note 12. Income Taxes](index=154&type=section&id=Note%2012.%20Income%20Taxes) This note details Income Taxes [Note 13. Deferred Compensation Plan](index=155&type=section&id=Note%2013.%20Deferred%20Compensation%20Plan) This note details Deferred Compensation Plan [Note 14. Subsequent Events](index=156&type=section&id=Note%2014.%20Subsequent%20Events) This note details Subsequent Events [ITEM 9. CHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL DISCLOSURE](index=115&type=section&id=ITEM%209.%20CHANGES%20IN%20AND%20DISAGREEMENTS%20WITH%20ACCOUNTANTS%20ON%20ACCOUNTING%20AND%20FINANCIAL%20DISCLOSURE) There have been no changes in or disagreements with accountants on accounting and financial disclosure - There were no changes in or disagreements with accountants on accounting and financial disclosure[668](index=668&type=chunk) [ITEM 9A. CONTROLS AND PROCEDURES](index=115&type=section&id=ITEM%209A.%20CONTROLS%20AND%20PROCEDURES) Disclosure controls and internal control over financial reporting were effective as of December 31, 2024 - The company's **disclosure controls and procedures** were evaluated as effective as of **December 31, 2024**[669](index=669&type=chunk) - Management concluded that the company's **internal control over financial reporting** was effective as of **December 31, 2024**, based on criteria established in the **Internal Control—Integrated Framework (2013)** issued by **COSO**[673](index=673&type=chunk) - No changes in **internal control over financial reporting** occurred during the quarter ended **December 31, 2024**, that materially affected, or are reasonably likely to materially affect, **internal control over financial reporting**[674](index=674&type=chunk) [ITEM 9B. OTHER INFORMATION](index=116&type=section&id=ITEM%209B.%20OTHER%20INFORMATION) No directors or officers adopted or terminated Rule 10b5-1 trading arrangements in Q4 2024 - No director or officer adopted or terminated a "**Rule 10b5-1 trading arrangement**" or "non-Rule 10b5-1 trading arrangement" during the fiscal quarter ended **December 31, 2024**[675](index=675&type=chunk) [ITEM 9C. DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS](index=116&type=section&id=ITEM%209C.%20DISCLOSURE%20REGARDING%20FOREIGN%20JURISDICTIONS%20THAT%20PREVENT%20INSPECTIONS) This item is not applicable to the company - **Disclosure regarding foreign jurisdictions that prevent inspections** is not applicable to the registrant[676](index=676&type=chunk) PART III [ITEM 10. DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE](index=116&type=section&id=ITEM%2010.%20DIRECTORS%2C%20EXECUTIVE%20OFFICERS%20AND%20CORPORATE%20GOVERNANCE) Information on directors, executive officers, and corporate governance is incorporated by reference from the proxy statement - Information required by Item 10 is incorporated by reference to the sections of the **2025 Proxy Statement** under "**Corporate Governance**" and "**Proposal No. 1 – Election of Directors**," "**Executive Officers**"[679](index=679&type=chunk) - The company has adopted an **Insider Trading Compliance Policy** governing the purchase, sale, and/or other dispositions of its securities by directors, officers, and employees[681](index=681&type=chunk) [ITEM 11. EXECUTIVE COMPENSATION](index=117&type=section&id=ITEM%2011.%20EXECUTIVE%20COMPENSATION) Information regarding executive compensation is incorporated by reference from the proxy statement - Information required by Item 11 is incorporated by reference to the sections of the **2025 Proxy Statement** under "**Executive Compensation**" and "**Proposal No. 1 – Election of Directors – Non-Employee Director Compensation**"[682](index=682&type=chunk) [ITEM 12. SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS](index=117&type=section&id=ITEM%2012.%20SECURITY%20OWNERSHIP%20OF%20CERTAIN%20BENEFICIAL%20OWNERS%20AND%20MANAGEMENT%20AND%20RELATED%20STOCKHOLDER%20MATTERS) Information on security ownership is incorporated by reference from the proxy statement - Information required by Item 12 is incorporated by reference to the sections of the **2025 Proxy Statement** under "**Security Ownership of Certain Beneficial Owners and Management**" and "**Executive Compensation – Securities Authorized for Issuance under Equity Compensation Plans**"[682](index=682&type=chunk) [ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE](index=117&type=section&id=ITEM%2013.%20CERTAIN%20RELATIONSHIPS%20AND%20RELATED%20TRANSACTIONS%2C%20AND%20DIRECTOR%20INDEPENDENCE) Information on related transactions and director independence is incorporated by reference from the proxy statement - Information required by Item 13 is incorporated by reference to the sections of the **2025 Proxy Statement** under "**Certain Relationships and Related-Party Transactions**" and "**Corporate Governance**"[683](index=683&type=chunk) [ITEM 14. PRINCIPAL ACCOUNTANT FEES AND SERVICES](index=117&type=section&id=ITEM%2014.%20PRINCIPAL%20ACCOUNTANT%20FEES%20AND%20SERVICES) Information on principal accountant fees and services is incorporated by reference from the proxy statement - Information required by Item 14 is incorporated by reference to the sections of the **2025 Proxy Statement** under the caption "**Proposal No. 2 – Ratification of Independent Registered Public Accounting Firm**"[683](index=683&type=chunk) PART IV [ITEM 15. EXHIBITS AND FINANCIAL STATEMENT SCHEDULES](index=117&type=section&id=ITEM%2015.%20EXHIBITS%20AND%20FINANCIAL%20STATEMENT%20SCHEDULES) This section lists all exhibits and financial statement schedules filed as part of the Annual Report on Form 10-K - The section lists financial statements, corporate documents (**Restated Certificate of Incorporation**, **Certificate of Amendment**, **Certificate of Designation**, **Amended and Restated Bylaws**), and various forms of warrants[685](index=685&type=chunk)[687](index=687&type=chunk) - Key agreements listed include the **Merger Agreement**, **Sublicense Agreement**, **Lease Agreements**, **Equity Incentive Plans**, **Contingent Value Rights Agreement**, **Sales Agreements**, **Purchase Agreement**, **Registration Rights Agreement**, and multiple **License and Collaboration Agreements** (**Viatris**, Processa, University of Pennsylvania, Iveric, Massachusetts Eye and Ear Infirmary)[687](index=687&type=chunk)[689](index=689&type=chunk)[691](index=691&type=chunk)[694](index=694&type=chunk)[695](index=695&type=chunk)[697](index=697&type=chunk)[699](index=699&type=chunk) - Also included are various employment and consulting agreements for key personnel, an **Insider Trading Compliance Policy**, and certifications from executive officers[681](index=681&type=chunk)[689](index=689&type=chunk)[691](index=691&type=chunk)[694](index=694&type=chunk)[695](index=695&type=chunk)[697](index=697&type=chunk)[699](index=699&type=chunk) [ITEM 16. FORM 10-K SUMMARY](index=125&type=section&id=ITEM%2016.%20FORM%2010-K%20SUMMARY) No Form 10-K summary is provided in this report - No **Form 10-K summary** is provided in this report[702](index=702&type=chunk)

Financial Performance - The company reported a net loss of approximately $56.8 million for the year ended December 31, 2024[6] - Net cash used in operating activities for the year ended December 31, 2024 was approximately $25.6 million[6] Cash Position - The cash and cash equivalents balance as of December 31, 2024 was approximately $30.3 million[6]

Merger Agreement Details - The merger agreement involves OCUPHIRE PHARMA, INC. and OPUS GENETICS INC., with the effective date set for October 22, 2024[1]. - The first merger will result in OPUS GENETICS INC. becoming a wholly owned subsidiary of OCUPHIRE PHARMA, INC.[11]. - Following the first merger, OPUS GENETICS INC. will merge into ORANGE MERGER SUB II, LLC, with the latter being the surviving entity[12]. - The transactions are intended to qualify as a "reorganization" under Section 368(a) of the Code[13]. - The Parent Board has unanimously determined that the contemplated transactions are fair and in the best interests of its stockholders[14]. - The aggregate merger consideration to be paid by Parent for all outstanding shares of Company Capital Stock at Closing will include 5,237,063 shares of Parent Common Stock and 14,145.374 shares of Parent Convertible Preferred Stock, representing 42% of the outstanding capital stock of Parent immediately following the Merger[30]. - The First Merger will result in the Company continuing as the surviving corporation, and the Second Merger will lead to the formation of a new entity named "OPUSTX LLC" as the surviving entity[24][29]. - The First Merger is set to be consummated remotely via electronic exchange of documents on the Closing Date, with the Second Merger to occur no earlier than the tenth Business Day after the Closing Date[26]. - All directors and officers of Parent and certain Company stockholders are executing lock-up agreements and support agreements to facilitate the merger[20][21]. - The Company stockholders have irrevocably approved the merger and waived any rights to receive payment of the fair value of their capital stock under the Delaware General Corporation Law[18]. - The merger will result in the issuance of shares of Parent Common Stock and Parent Convertible Preferred Stock to Company stockholders, with each Parent Preferred Stock Payment Share convertible into 1,000 shares of Parent Common Stock[30]. - The First Effective Time will occur upon the filing of the First Certificate of Merger with the Secretary of State of Delaware, while the Second Effective Time will follow the filing of the Second Certificate of Merger[26][29]. - The certificate of incorporation of Parent will be amended to change its name to "Opus Genetics, Inc." following the First Effective Time[27]. - The merger is structured to ensure that no fractional shares of Parent Common Stock will be issued, with cash payments made for any fractional shares[33]. - The Company will transmit a Stockholder Notice to each stockholder who did not execute the Stockholder Written Consent immediately following the execution of the Agreement[19]. - At the First Effective Time, each share of common stock of First Merger Sub will be converted into one share of common stock of the First Step Surviving Corporation[34]. - The Preferred Stock Exchange Ratio will be adjusted to reflect any changes in outstanding shares due to stock dividends or other similar changes[35]. - At the Second Effective Time, each share of common stock of the First Step Surviving Corporation will be canceled without any conversion or payment[36]. - All holders of Company Capital Stock will cease to have rights as stockholders immediately prior to the First Effective Time[37]. - The Exchange Fund will include Parent Common Stock, Parent Convertible Preferred Stock, and cash for fractional shares[38]. - Dissenting Shares will not convert into the Merger Consideration but will receive the appraised value under the DGCL[44]. - Company Options will be canceled without payment at the First Effective Time[46]. - Company Convertible Notes will be converted into 6,223,538.51 shares of Series Seed-1 Preferred Stock at the First Effective Time[47]. Company Financials and Operations - The Company is duly incorporated and has all necessary corporate power to conduct its business[51]. - The Company has no subsidiaries and does not own any capital stock or equity interests in other entities[53]. - The Company has 77,341,000 authorized shares of Common Stock, with 6,548,709 shares issued and outstanding[63]. - The Company has 65,236,000 authorized shares of Preferred Stock, with 56,053,000 designated as Series Seed Preferred Stock and 33,382,450 shares issued and outstanding[63]. - The Company maintains accurate books and records reflecting its assets and liabilities, ensuring reasonable assurance over financial reporting[73]. - The Company has reserved 5,576,465 shares of Common Stock for issuance under the 2021 Stock Option Plan, with 21,500 shares already issued[66]. - The Company has not declared or paid any dividends or repurchased any shares of its capital stock since the date of the Unaudited Interim Balance Sheet[76]. - The Company has not identified any significant deficiencies or material weaknesses in its internal accounting controls since inception[75]. - The Company has not conducted any mergers, consolidations, or similar transactions except for the Contemplated Transactions[76]. - The Company has not incurred or guaranteed any indebtedness for borrowed money since the date of the Unaudited Interim Balance Sheet[76]. - The Company Financials fairly present the financial position and operating results as of the specified dates[72]. - The Company does not hold any shares of its capital stock in its treasury[63]. - The Company has no undisclosed liabilities as of the date hereof, except for those disclosed in the Unaudited Interim Balance Sheet[81]. - The Company owns all tangible properties and assets used in its business, free and clear of any encumbrances, except for permitted encumbrances[82]. - The Company does not own any real property and has valid leasehold interests in all leased properties[83]. - The Company has identified all material registered intellectual property (IP) and has no pending or threatened challenges to its validity[85]. - The Company exclusively owns or has exclusively licensed all material IP necessary for its business operations[86]. - The Company has not experienced any data breaches or security violations related to sensitive data since its inception[92]. - The Company has a list of material contracts in effect, including those requiring payments exceeding $50,000[94]. - The Company has not breached any material contracts that would allow other parties to cancel or seek damages, ensuring all material contracts are valid and enforceable[98]. - The Company holds all required Governmental Authorizations necessary for its operations, and no permits have been revoked or suspended[101]. - There are no pending or threatened investigations against the Company regarding compliance with drug regulations, indicating a strong legal standing[103]. - All clinical studies conducted by the Company have adhered to applicable regulations, with no studies terminated for safety or noncompliance reasons[105]. - The Company has complied with all laws related to patient health information, including HIPAA, and has not received any allegations of noncompliance[109]. - Manufacturing operations for product candidates have been conducted in accordance with good manufacturing practices, with no significant compliance issues reported[111]. - The Company has made available all material correspondence with regulatory bodies, ensuring transparency in its compliance efforts[112]. - As of the date of the Agreement, there are no pending legal proceedings that could interfere with the contemplated transactions[113]. - The Company has timely filed all required Tax Returns and all such returns are correct and complete in all material respects[117]. - All amounts of Taxes due and owing by the Company have been fully and timely paid, and unpaid Taxes did not materially exceed the reserve for Tax liability[118]. - There are no pending or ongoing audits, assessments, or other actions for any material amount of Taxes of the Company[120]. - The Company has not incurred any Liability for Taxes outside the Ordinary Course of Business since the date of the Unaudited Interim Balance Sheet[118]. - The Company has not participated in any transaction that constitutes a "listed transaction" under the Code[127]. Employee and Labor Compliance - The Company has provided a true and correct list of all current employees and independent contractors, including compensation details[144]. - The Company is in compliance with all applicable Laws regarding labor and employment practices[147]. - There are no pending audits or investigations involving any Company Benefit Plan[137]. - The Company has not been a party to any collective bargaining agreement or similar labor organization[145]. - The Company has not made any election under Section 965(h) of the Code[123]. - The Company has not implemented any "plant closing" or "mass layoff" since its inception, ensuring compliance with the WARN Act[148]. - The Company has been in material compliance with all COVID-19-related laws and regulations, including those from the Occupational Safety and Health Administration and the Centers for Disease Control[148]. Environmental and Regulatory Compliance - The Company has complied with all applicable Environmental Laws and has not received any notices of non-compliance since its inception[149]. - The Company has delivered accurate and complete copies of all material insurance policies, and no cancellations or denials of coverage have been reported since its inception[151]. - There are no undisclosed transactions with affiliates that would require disclosure under Item 404 of Regulation S-K[153]. - The Company has provided all material environmental reports and assessments to the Parent prior to the date of the Agreement[149]. - The Company has not been subject to any investigations regarding potential violations of Anti-Bribery Laws[155]. Parent Company Financials - Each holder of Company Preferred Stock is an accredited investor as defined in Regulation D[157]. - The Company has made no representations or warranties outside of those expressly set forth in the Agreement[158]. - The Company has obtained all necessary corporate approvals for the execution and delivery of the Agreement[168]. - As of the Reference Date, Parent has 125,000,000 authorized shares of common stock, with 26,198,444 shares issued and outstanding[174]. - Parent has 10,000,000 authorized shares of preferred stock, none of which have been issued[174]. - Parent has outstanding options for the purchase of 4,759,037 shares and warrants for 7,204,299 shares of common stock[177][178]. - Parent's financial statements comply with GAAP and fairly present its consolidated financial position as of the respective dates[184]. - Parent has maintained a system of internal control over financial reporting designed to provide reasonable assurance regarding the reliability of financial reporting[190]. - As of the date of the Agreement, there are no unresolved comments from the SEC regarding Parent's SEC Documents[187]. - Parent has not received any comment letters from the SEC regarding delisting or maintenance of listing on Nasdaq since December 31, 2022[187]. - Parent's cash and cash equivalents as of the close of business on the Business Day preceding the date of the Agreement have not materially changed[193]. - Parent has not been classified as a "shell company" under Section 12b-2 of the Exchange Act[194]. - All distributions and repurchases of Parent Common Stock have been undertaken in compliance with applicable laws and regulations[181]. - Parent and its Subsidiaries have conducted business only in the Ordinary Course of Business since the date of the Parent Balance Sheet[195]. - No dividends were declared or paid, and no shares were repurchased or reacquired, except for stock plan exercises[195]. - Parent has not incurred or guaranteed any indebtedness for borrowed money since the date of the Parent Balance Sheet[197]. - No material transactions outside the Ordinary Course of Business have been entered into, except in connection with the Contemplated Transactions[197]. - Parent has not made any expenditures exceeding $100,000 individually or $300,000 in the aggregate[199]. - As of the date hereof, Parent has no undisclosed liabilities that would be material to the Parent[200]. - Parent has not obtained a loan under the Paycheck Protection Program under the CARES Act[200].

Core Insights - The RD Fund has announced the all-stock acquisition of Opus Genetics, Inc. by Ocuphire Pharma, creating a new entity named Opus Genetics focused on gene therapies for inherited retinal diseases (IRDs) [1][3] - The combined company will trade on Nasdaq under the ticker symbol "IRD" [1] - This acquisition marks the first RD Fund portfolio company to enter public markets, validating the venture philanthropy model [3] Company Overview - Opus Genetics was launched in 2021 by the RD Fund to develop gene therapies for IRDs, with initial seed financing from the RD Fund [1][2] - The RD Fund aims to accelerate the approval of therapies for retinal diseases and provide alternative revenue sources for the Foundation Fighting Blindness [4] Financial and Development Highlights - Opus has developed a pipeline of early-stage preclinical assets, including advancing OPGx-LCA5 into a Phase 1/2 trial, which showed safety and visual improvement in early onset retinal degeneration [2] - The RD Fund has raised over $915 million to support research for preventing and treating retinal degenerative diseases [5]