UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☒ Annual report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. For the Fiscal Year Ended December 31, 2024 or ☐ Transition report pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. For the transition period from _____ to _____ Commission File No. 001-34079 Securities registered pursuant to Section 12(b) of the Act: Opus Genetics, Inc. (Exact name of registrant as specified in its c ...

Core Insights - Opus Genetics is focused on developing innovative gene therapy treatments for inherited retinal diseases (IRDs) and has made significant progress in its transformation and pipeline [1][2] - The company raised $21.5 million through a public offering and private placement, enhancing its cash position to approximately $50.7 million, which supports key milestones for its gene therapy candidates [2][9] - The acquisition of Opus Genetics has strengthened the company's pipeline, which now includes seven adeno-associated virus (AAV)-based gene therapy assets targeting specific IRDs [4][17] Financial Performance - For the year ended December 31, 2024, Opus reported a net loss of $57.5 million, or $2.15 per share, compared to a net loss of $10.0 million, or $0.46 per share, in 2023 [15][25] - License and collaborations revenue decreased to $11.0 million in 2024 from $19.0 million in 2023, primarily due to the absence of a milestone payment received in 2023 [12][25] - General and administrative expenses increased to $18.2 million in 2024 from $12.0 million in 2023, attributed to transaction costs related to the acquisition and other operational expenses [13][25] Pipeline and Clinical Trials - The lead gene therapy candidate, OPGx-LCA5, is in a Phase 1/2 trial and has shown positive results, with visual improvement observed in all adult patients after one year [9][18] - OPGx-BEST1 is expected to enter clinical trials in 2025, targeting IRDs associated with mutations in the BEST1 gene [9][18] - The LYNX-2 Phase 3 trial for Phentolamine Ophthalmic Solution 0.75% has completed enrollment, with topline data expected in mid-2025 [9][18] Strategic Direction - The company aims to achieve several key milestones in 2025, including up to four clinical trial data readouts and the initiation of new trials for its gene therapy candidates [2][10] - A Type D meeting with the FDA was held to discuss the regulatory path for OPGx-LCA5, indicating proactive engagement with regulatory authorities [9][18] - The company is also seeking a strategic partner to advance the late-stage development of APX3330, a novel oral REF-1 inhibitor for diabetic retinopathy [9][18]

Core Insights - Opus Genetics, Inc. is presenting results from the LYNX-1 Phase 3 study of Phentolamine Ophthalmic Solution 0.75% at the World Cornea Congress IX, focusing on its potential to treat dim light disturbances in patients, particularly those who have undergone keratorefractive surgery [1][3][4] Study Results - The LYNX-1 Phase 3 study met its primary endpoint, showing that 13% of participants treated with Phentolamine gained 15 or more letters of mesopic low contrast distance visual acuity (mLCVA) at Day 8 compared to 3% in the placebo group (p<0.05) [5] - At Day 15, 21% of Phentolamine-treated participants gained 15 or more letters of mLCVA compared to 3% in the placebo group (p<0.01) [5] - Patient-reported outcomes for glare, halos, and starbursts were significantly lower in the Phentolamine group at Day 15 (p<0.01) [5] - In a subset analysis of post-LASIK participants, 29% of those treated with Phentolamine gained 15 or more letters of mLCVA at Day 8 compared to 9% in the placebo group [5] - The positive results from the LYNX-1 study support the ongoing Phase 3 LYNX-2 trial, with topline results expected in mid-2025 [5] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases and other ophthalmic disorders [4] - The company's pipeline includes AAV-based investigational gene therapies targeting mutations in genes associated with various retinal diseases, with the most advanced program addressing mutations in the LCA5 gene [4] - Phentolamine Ophthalmic Solution 0.75% is being investigated for its ability to reduce pupil size and improve vision in patients with reduced mesopic vision following keratorefractive surgery [6]

Core Viewpoint - Opus Genetics, Inc. has announced a public offering and private placement to raise approximately $21 million for its gene therapy programs targeting inherited retinal diseases [1][3][4]. Group 1: Offering Details - The public offering consists of 21,052,631 shares of common stock and warrants at a price of $0.95, potentially generating gross proceeds of $20 million [5]. - The concurrent private placement includes 1,176,471 shares of common stock and warrants at an offering price of $1.275, with gross proceeds of $1.5 million [6]. - The total potential gross proceeds from both offerings, including warrants, could reach approximately $43 million [4]. Group 2: Use of Proceeds - The net proceeds from the offerings will be utilized to fund clinical development of the lead gene therapy programs, OPGx-LCA5 and OPGx-BEST1, as well as for general corporate purposes and working capital [3]. Group 3: Warrants and Conditions - Warrants issued in the public offering could provide up to $21.4 million in additional capital upon the release of OPGx-BEST1 DUO-1001 Cohort 1 data [4]. - The warrants from the public offering will be exercisable immediately and have an initial exercise price of $0.95, expiring five years from issuance [5]. - The private placement warrants will also be exercisable immediately, with an initial exercise price of $1.15, and similar expiration terms [6]. Group 4: Company Background - Opus Genetics is focused on developing gene therapies for inherited retinal diseases and other ophthalmic disorders, with a pipeline that includes investigational therapies for various genetic mutations [11]. - The company is currently evaluating its most advanced gene therapy program in a Phase 1/2 trial, with plans to initiate a Phase 1/2 study for BEST1 in 2025 [11].

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]

Core Insights - Opus Genetics, Inc. has approved equity awards for two new employees as part of its 2021 Inducement Plan, effective March 13, 2025 [1] - The equity awards consist of options to purchase a total of 205,742 shares at an exercise price of $0.93 per share, with a four-year vesting schedule [2] - Opus Genetics is focused on developing therapies for inherited retinal diseases (IRDs) and other ophthalmic disorders, with a pipeline that includes gene therapies and other investigational treatments [3] Group 1 - The independent Board of Directors approved equity awards as a material inducement for new hires [1] - The options granted will vest over four years, with 25% vesting after one year and the remainder in monthly or quarterly installments [2] - The company is developing AAV-based gene therapies targeting specific genetic mutations associated with various retinal diseases [3] Group 2 - The most advanced program targets mutations in the LCA5 gene, currently in a Phase 1/2 trial with promising early results [3] - Other investigational products include BEST1 gene therapy and Phentolamine Ophthalmic Solution 0.75%, which is in Phase 3 trials for presbyopia [3] - The company has reached an agreement with the FDA for a Phase 3 trial of oral APX3330 for diabetic retinopathy treatment [3]

Core Insights - Opus Genetics, Inc. announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the ARVO 2025 Meeting, showcasing advancements in gene therapies for inherited retinal diseases [1][3] - The company is focusing on its lead candidate OPGx-LCA5, which is currently in a Phase 1/2 trial, with promising 12-month data indicating visual improvement in adult patients [4][3] - Additional presentations will cover pre-clinical results for OPGx-MERTK and OPGx-RDH12, expanding the company's pipeline in treating various retinal disorders [5][7] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies for inherited retinal diseases and other ophthalmic disorders [9] - The company's pipeline includes AAV-based investigational gene therapies targeting mutations in genes associated with bestrophinopathy, Leber congenital amaurosis (LCA), and retinitis pigmentosa [9] - The most advanced program, OPGx-LCA5, is designed to address mutations in the LCA5 gene and is currently undergoing a Phase 1/2 open-label, dose-escalation trial [9] Upcoming Presentations - The presentation titled "Recovery of cone mediated vision in severe ciliopathy after gene augmentation; One year results from a Phase I/II trial of LCA5-LCA (OPGx-LCA5)" will take place on May 4, 2025, highlighting the efficacy of OPGx-LCA5 [4] - Another presentation on OPGx-MERTK will discuss its evaluation in a rat model of retinitis pigmentosa, scheduled for May 8, 2025 [5] - A study on the ocular tolerability of OPGx-RDH12 will be presented on May 5, 2025, focusing on its delivery in cynomolgus primates [6][7] Clinical Trials and Regulatory Status - The ongoing Phase 1/2 trial of OPGx-LCA5 has shown that all three adult patients experienced visual improvement at six months, with new data indicating sustained efficacy for a year [4][3] - The company plans to advance OPGx-LCA5 into a pivotal Phase 3 trial, contingent on continued safety and efficacy [3] - Phentolamine Ophthalmic Solution 0.75% is currently in Phase 3 trials for treating presbyopia and reduced dim light vision, with FDA Fast Track Designation granted for chronic night driving impairment [9]

Core Insights - Opus Genetics, Inc. announced that three abstracts on its investigational gene therapy candidates have been accepted for presentation at the ARVO 2025 Meeting, showcasing 12-month data from the first three adult patients in the ongoing Phase 1/2 trial of OPGx-LCA5 [1][3] - The company aims to advance OPGx-LCA5 into a pivotal Phase 3 trial, contingent on continued safety and efficacy results [3] Group 1: OPGx-LCA5 Trial Results - The ongoing Phase 1/2 trial of OPGx-LCA5 has shown that subjective and objective signs of efficacy persisted for one year in the first three adult patients [1][4] - Previous results indicated that OPGx-LCA5 was well tolerated, with all three adult patients demonstrating visual improvement at six months [4] Group 2: Upcoming Presentations - The abstracts accepted for presentation include details on OPGx-LCA5, OPGx-MERTK, and OPGx-RDH12, with specific presentation times and authors listed [2][4][5] - A subset analysis from the completed LYNX-1 Phase 3 trial of Phentolamine Ophthalmic Solution 0.75% will also be presented, focusing on reduced mesopic low contrast vision in post-LASIK subjects [6] Group 3: Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases and other ophthalmic disorders [7] - The company's pipeline includes several investigational gene therapies targeting various genetic mutations associated with retinal diseases, with OPGx-LCA5 being the most advanced candidate [7]

Core Insights - The FDA has granted Fast Track designation for Phentolamine Ophthalmic Solution 0.75% to treat significant chronic night driving impairment in keratorefractive patients with reduced mesopic vision [1][7] - Enrollment in the VEGA-3 Phase 3 trial for presbyopia is complete, while the LYNX-2 trial is expected to complete enrollment in the first half of 2025 [1][2] - Phentolamine Ophthalmic Solution 0.75% aims to provide a non-invasive treatment option for presbyopia and low light vision disturbances post-keratorefractive surgery [2][10] VEGA-3 Phase 3 Program - The VEGA-3 trial involves 545 participants and is designed to assess the improvement in near visual acuity after treatment with Phentolamine Ophthalmic Solution 0.75% [3] - The primary endpoint is a 15-letter improvement in distance-corrected near visual acuity on day eight post-treatment [3] LYNX-2 Phase 3 Program - The LYNX-2 trial targets 200 subjects who have experienced decreased visual acuity in low light conditions after keratorefractive surgery, with over 95% enrollment achieved [5] - The primary endpoint is a gain of 3 lines (or 15 letters) in distance vision improvement on a low contrast chart after 15 days of dosing [5] About Phentolamine Ophthalmic Solution 0.75% - This solution is a non-selective alpha-1 and alpha-2 adrenergic antagonist that reduces pupil size, potentially improving vision in low light conditions [10] - It is being developed as a treatment for both presbyopia and visual disturbances following keratorefractive surgery [10] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on gene therapies for inherited retinal diseases and other ophthalmic disorders [11] - The company is advancing multiple programs, including Phentolamine Ophthalmic Solution 0.75% and gene therapies targeting specific genetic mutations associated with retinal diseases [11]

Core Insights - Opus Genetics has initiated dosing of the first pediatric patient in its Phase 1/2 trial for OPGx-LCA5, a gene therapy for Leber congenital amaurosis (LCA), with initial data expected by Q3 2025 [1] - The trial has shown early clinical proof of concept, with significant visual improvement observed in adult patients as early as one month post-treatment, and new 12-month data will be presented in Q2 2025 [2][3] - An FDA meeting is scheduled for March 2025 to discuss the design and endpoints for a pivotal Phase 3 trial for OPGx-LCA5 [1][3] Company Overview - Opus Genetics is a clinical-stage ophthalmic biotechnology company focused on developing gene therapies for inherited retinal diseases and other ophthalmic disorders [7][8] - The company’s pipeline includes investigational gene therapies targeting various genetic mutations associated with retinal diseases, with OPGx-LCA5 being the most advanced program [8] Clinical Trial Details - The Phase 1/2 trial is designed to evaluate the safety and preliminary efficacy of OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene [4] - Efficacy endpoints include functional vision measurements using Multi-Luminance orientation and Mobility Test (MLoMT), Full-Field Stimulus Testing (FST), and microperimetry [4] Future Developments - The company plans to share new 12-month data from adult patients treated with OPGx-LCA5 at a major medical conference in Q2 2025 [2][5] - The ongoing collaboration with the University of Pennsylvania is crucial for the advancement of the OPGx-LCA5 program [3]