PART I – FINANCIAL INFORMATION [Item 1. Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) The company reported a net loss of $7.4 million for Q2 2025 and $15.6 million for the six months ended June 30, 2025, with cash of $32.4 million and total assets of $38.7 million, and increased liabilities to $21.2 million due to new warrant liabilities [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets increased to $38.7 million, driven by cash and cash equivalents of $32.4 million, while total liabilities rose to $21.2 million due to $11.8 million in warrant liabilities, resulting in total stockholders' equity of $17.5 million Condensed Consolidated Balance Sheet Highlights (in thousands of US dollars) | Account | June 30, 2025 (Unaudited) | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $32,429 | $30,321 | | Total current assets | $38,439 | $36,610 | | **Total assets** | **$38,665** | **$36,862** | | **Liabilities & Equity** | | | | Warrant liabilities | $11,800 | $— | | Total current liabilities | $20,192 | $11,295 | | **Total liabilities** | **$21,192** | **$11,295** | | Series A preferred stock | $— | $18,843 | | **Total stockholders' equity** | **$17,473** | **$6,724** | [Condensed Consolidated Statements of Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For Q2 2025, net loss was $7.4 million, a slight decrease from Q2 2024 due to a $0.9 million positive change in warrant liabilities, while the six-month net loss increased to $15.6 million due to higher operating expenses Financial Performance Summary (in thousands of US dollars, except per share data) | Metric | Q2 2025 | Q2 2024 | Six Months 2025 | Six Months 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 | | Loss from operations | ($8,906) | ($8,328) | ($18,835) | ($16,036) | | **Net loss** | **($7,420)** | **($7,765)** | **($15,614)** | **($14,871)** | | **Net loss per share (basic and diluted)** | **($0.12)** | **($0.30)** | **($0.32)** | **($0.59)** | [Condensed Consolidated Statements of Cash Flows](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2025, net cash used in operations was $19.3 million, offset by $21.4 million from financing activities, leading to a $2.1 million net cash increase and an ending balance of $32.4 million Cash Flow Summary for the Six Months Ended June 30 (in thousands of US dollars) | Category | 2025 | 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** | **$2,108** | **$(9,092)** | | **Cash and cash equivalents at end of period** | **$32,429** | **$41,409** | - Financing activities in the first six months of 2025 were driven by **$5.98 million** from the issuance of common stock and pre-funded warrants, and **$15.52 million** from the issuance of warrants in the March 2025 offerings[20](index=20&type=chunk) [Notes to Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail the company's focus on gene therapies for IRDs, key events including the October 2024 acquisition and March 2025 financing, and confirm sufficient cash to fund operations for at least the next twelve months - The company is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders, with its pipeline including OPGx-LCA5 for Leber congenital amaurosis and OPGx-BEST1 for Best Disease[22](index=22&type=chunk)[24](index=24&type=chunk) - As of June 30, 2025, the company had **$32.4 million** in cash and cash equivalents, which management believes is sufficient to fund planned expenditures for at least the next twelve months[32](index=32&type=chunk) - In March 2025, the company raised approximately **$21.5 million** in gross proceeds through an underwritten public offering and a private placement, issuing common stock, pre-funded warrants, and warrants[102](index=102&type=chunk)[99](index=99&type=chunk) - On May 5, 2025, all **14,145.374 shares** of Series A Preferred Stock were converted into **14,145,374 shares** of common stock following stockholder approval[98](index=98&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) Management reported increased license and collaboration revenue to $2.9 million for Q2 2025, with rising G&A expenses due to public company costs, stable R&D, and $32.4 million in cash, sufficient for the next twelve months [Overview and Recent Developments](index=35&type=section&id=Overview%20and%20Recent%20Developments) The company is advancing its gene therapy pipeline, with OPGx-LCA5 showing positive data and receiving RMAT designation, and secured a $2.0 million non-dilutive funding agreement for the MERTK program - The most advanced gene therapy program, OPGx-LCA5, has shown clinical proof-of-concept with visual improvement in adult patients and initial positive data in pediatric patients[189](index=189&type=chunk) - On May 6, 2025, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to OPGx-LCA5, potentially expediting its development and review[199](index=199&type=chunk) - In June 2025, the company entered into a funding agreement with the Foundation Fighting Blindness Retinal Degeneration Fund (RDF) for up to **$2.0 million** in non-dilutive funding for its MERTK program[196](index=196&type=chunk) [Results of Operations](index=40&type=section&id=Results%20of%20Operations) Q2 2025 revenue increased to $2.9 million, driven by Viatris reimbursements, while G&A expenses rose by $2.4 million due to public company and legal costs, and R&D remained stable, leading to a higher loss from operations Comparison of Three Months Ended June 30 (in thousands of US dollars) | Item | 2025 | 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 | $(64) | | **Loss from operations** | **$(8,906)** | **$(8,328)** | **$(578)** | Comparison of Six Months Ended June 30 (in thousands of US dollars) | Item | 2025 | 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 | $3,140 | | **Loss from operations** | **$(18,835)** | **$(16,036)** | **$(2,799)** | - The increase in G&A expenses in Q2 2025 was primarily due to higher public company related costs (**$1.1 million**), legal costs (**$0.4 million**), patent costs (**$0.3 million**), and payroll costs (**$0.4 million**)[221](index=221&type=chunk) - R&D spending for the six months ended June 30, 2025, shifted significantly, with spending on IRD programs increasing by **$4.1 million** and on PS by **$4.4 million**, while spending on APX3330 decreased by **$6.5 million**[229](index=229&type=chunk) [Liquidity and Capital Resources](index=44&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2025, the company had $32.4 million in cash, sufficient for the next twelve months, bolstered by $21.5 million from March 2025 financings, and terminated its equity line with Lincoln Park - The company's principal source of liquidity as of June 30, 2025, was cash and cash equivalents of **$32.4 million**[233](index=233&type=chunk) - In March 2025, the company raised combined gross proceeds of approximately **$21.5 million** from an underwritten public offering and a private placement[237](index=237&type=chunk) - The common stock purchase agreement with Lincoln Park Capital was terminated by the company effective April 3, 2025[246](index=246&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 is not applicable as the company qualifies as a smaller reporting company - Disclosure is 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) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2025, with no material changes in internal control over financial reporting during the quarter - Based on an evaluation as of June 30, 2025, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective[300](index=300&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended June 30, 2025, that have materially affected, or are reasonably likely to materially affect, the company's internal controls[301](index=301&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=54&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any legal proceedings expected to have a material effect on its business or financial results - The company is not currently a party to any legal proceedings that management believes 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) No material changes to risk factors except for a new risk concerning potential delisting from Nasdaq if the company fails to meet the minimum bid price requirement - A new risk factor has been added regarding the potential delisting from the Nasdaq Capital Market if the company's common stock fails to comply with the continued listing standards, specifically the **$1.00** minimum bid price requirement[304](index=304&type=chunk)[305](index=305&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) The company reported no unregistered sales of equity securities during the period - None[308](index=308&type=chunk) [Item 5. Other Information](index=55&type=section&id=Item%205.%20Other%20Information) No 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 directors or officers adopted or terminated a Rule 10b5-1 trading arrangement during the quarter[311](index=311&type=chunk) [Item 6. Exhibits](index=55&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including funding agreements, officer certifications, and XBRL data files - Exhibits filed include a Funding Agreement with the Foundation Fighting Blindness Retinal Degeneration Fund and a Funding and License Agreement with Eyes on the Future and RDH12 Fund for Sight[312](index=312&type=chunk)

[Corporate Update and Pipeline Highlights](index=1&type=section&id=Corporate%20Update%20and%20Pipeline%20Highlights) The company received key regulatory designations, reported positive clinical trial results, and secured non-dilutive funding - The FDA granted **Regenerative Medicine Advanced Therapy (RMAT) designation** for OPGx-LCA5, a gene therapy for Leber Congenital Amaurosis (LCA), signaling potential for accelerated development[2](index=2&type=chunk) - **Positive 12-month Phase 1/2 clinical data** for OPGx-LCA5 in adults and encouraging early pediatric data support its potential for vision restoration[2](index=2&type=chunk) - Reported **positive topline results** from two Phase 3 trials (VEGA-3 and LYNX-2) for Phentolamine Ophthalmic Solution 0.75% in presbyopia and night vision disturbances[2](index=2&type=chunk) - The OPGx-BEST1 program for inherited retinal disease is on track to enter a **Phase 1/2 trial in the second half of 2025**[2](index=2&type=chunk) - Secured **non-dilutive funding** from patient advocacy groups to advance multiple early-stage gene therapy programs, including OPGx-RDH12 and OPGx-MERTK[2](index=2&type=chunk)[6](index=6&type=chunk) [Pipeline Updates](index=2&type=section&id=Pipeline%20Updates) This section details clinical and preclinical progress across the company's key therapeutic programs [OPGx-LCA5 – Gene Therapy for Leber Congenital Amaurosis (LCA)](index=2&type=section&id=OPGx-LCA5%20%E2%80%93%20Gene%20Therapy%20for%20Leber%20Congenital%20Amaurosis%20(LCA)) The OPGx-LCA5 program received RMAT designation and demonstrated sustained vision improvements in clinical trials - The FDA granted **Regenerative Medicine Advanced Therapy (RMAT) designation**, providing a pathway for accelerated development and review[6](index=6&type=chunk) - Twelve-month clinical data from the adult cohort in the Phase 1/2 trial showed **sustained improvements in visual function**, including gains in visual acuity and better mobility testing scores[6](index=6&type=chunk) - Initial one-month post-treatment data in the pediatric cohort indicated **vision improvement with no drug-related adverse events**[6](index=6&type=chunk) [OPGx-BEST1 – Gene Therapy for BEST1-Related IRD](index=2&type=section&id=OPGx-BEST1%20%E2%80%93%20Gene%20Therapy%20for%20BEST1-Related%20IRD) The OPGx-BEST1 program is advancing toward a Phase 1/2 trial supported by positive preclinical data - Preclinical data presented in May showed **restoration of the retinal pigment epithelium-photoreceptor interface** in a canine model using AAV-mediated gene delivery[6](index=6&type=chunk) - The company remains on track to submit an IND application and initiate a **Phase 1/2 trial in the second half of 2025**[6](index=6&type=chunk) [OPGx-RDH12 and OPGx-MERTK – Advancing with Non-Dilutive Support](index=2&type=section&id=OPGx-RDH12%20and%20OPGx-MERTK%20%E2%80%93%20Advancing%20with%20Non-Dilutive%20Support) Early-stage gene therapies OPGx-RDH12 and OPGx-MERTK are advancing with non-dilutive funding from patient groups - A partnership with the Global RDH12 Alliance provides up to **$1.6 million in non-dilutive funding** to advance OPGx-RDH12 for RDH12-LCA[6](index=6&type=chunk) - Received up to **$2 million in non-dilutive funding** from the Retinal Degeneration Fund to advance OPGx-MERTK for retinitis pigmentosa caused by MERTK mutations[6](index=6&type=chunk) [Phentolamine Ophthalmic Solution 0.75% – Advancing Toward sNDA Submissions](index=3&type=section&id=Phentolamine%20Ophthalmic%20Solution%200.75%25%20%E2%80%93%20Advancing%20Toward%20sNDA%20Submissions) Positive Phase 3 trial results for Phentolamine Ophthalmic Solution support planned sNDA submissions - The VEGA-3 Phase 3 trial for presbyopia met its primary endpoint, with **27.2% of treated patients achieving a ≥15-letter gain** in near visual acuity compared to 11.5% on placebo (p<0.0001)[12](index=12&type=chunk) - The LYNX-2 Phase 3 trial for keratorefractive patients with night vision disturbances also **met its primary and secondary endpoints**, showing significant gains in mesopic low contrast vision[12](index=12&type=chunk) [Outlook and Upcoming Milestones](index=3&type=section&id=Upcoming%20Expected%20Data%20Readouts%20and%20Program%20Advancements) The company outlines key clinical and regulatory milestones expected in the second half of 2025 - Key upcoming milestones for the second half of 2025 include: - Reporting three-month pediatric data from the OPGx-LCA5 Phase 1/2 trial (Q3 2025) - Initiating enrollment in the Phase 1/2 trial for OPGx-BEST1 (H2 2025) - Submitting the Phentolamine sNDA for presbyopia (H2 2025) - Initiating enrollment in the Phentolamine LYNX-3 Phase 3 trial (H2 2025)[12](index=12&type=chunk) [Second Quarter 2025 Financial Results](index=3&type=section&id=Financial%20Results%20for%20the%20Second%20Quarter%20Ended%20June%2030%2C%202025) The company reports Q2 2025 financial performance, including revenue growth and an updated cash runway [Financial Performance Summary](index=3&type=section&id=Financial%20Performance%20Summary) The company reported a net loss of $7.4 million on revenue of $2.9 million, with a cash runway into H2 2026 - As of June 30, 2025, the company had cash and cash equivalents of **$32.4 million**, which is expected to fund operations into the second half of 2026[9](index=9&type=chunk) Q2 2025 Key Financial Metrics (vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | License & Collaboration Revenue | $2.9 million | $1.1 million | +163.6% | | G&A Expenses | $5.8 million | $3.4 million | +70.6% | | R&D Expenses | $6.0 million | $6.1 million | -1.6% | | Net Loss | $7.4 million | $7.8 million | Improved | | Net Loss per Share | $(0.12) | $(0.30) | Improved | [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets grew to $38.7 million, while liabilities increased to $21.2 million as of June 30, 2025 Balance Sheet Summary (in thousands) | Balance Sheet Item | June 30, 2025 | Dec 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $32,429 | $30,321 | | Total assets | $38,665 | $36,862 | | Total liabilities | $21,192 | $11,295 | | Total stockholders' equity | $17,473 | $6,724 | [Condensed Consolidated Statements of Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) The company's net loss improved to $7.4 million for Q2 2025, compared to $7.8 million in the prior-year period Statement of Comprehensive Loss (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | License and collaborations revenue | $2,882 | $1,112 | | Total operating expenses | $11,788 | $9,440 | | Loss from operations | $(8,906) | $(8,328) | | Net loss | $(7,420) | $(7,765) | | Net loss per share (basic and diluted) | $(0.12) | $(0.30) | | Weighted avg. shares outstanding | 63,376,392 | 25,827,265 | [Company Overview](index=4&type=section&id=About%20Opus%20Genetics) Opus Genetics is a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases - Opus Genetics is a clinical-stage biopharmaceutical company specializing in **gene therapies for inherited retinal diseases (IRDs)** and small molecule therapies for other eye disorders[15](index=15&type=chunk) - The company's lead gene therapy candidates are **OPGx-LCA5** for Leber congenital amaurosis (LCA) and **OPGx-BEST1** for BEST1-related retinal degeneration[15](index=15&type=chunk) - Opus is also advancing a partnered therapy, **Phentolamine Ophthalmic Solution 0.75%**, which is being studied in Phase 3 programs for presbyopia and nighttime visual disturbances[15](index=15&type=chunk)

Core Insights - Opus Genetics has achieved significant progress in its clinical pipeline, including positive clinical data for OPGx-LCA5 and FDA's RMAT designation, indicating strong potential for vision restoration therapies [1][2][5] - The company reported positive topline results from Phase 3 trials for Phentolamine Ophthalmic Solution 0.75%, which could provide new treatment options for patients with vision challenges [1][2][13] - Financial results for Q2 2025 show an increase in collaboration revenue and a net loss reduction compared to the same period in 2024, indicating improved financial performance [9][12][22] Pipeline Updates - OPGx-LCA5, a gene therapy for Leber Congenital Amaurosis, has shown sustained visual function improvements in adult patients, with initial pediatric data indicating positive outcomes [1][5] - OPGx-BEST1 is on track to enter Phase 1/2 trials in H2 2025, targeting bestrophin-1 related inherited retinal disease [1][4][14] - Non-dilutive funding has been secured to support the advancement of multiple early-stage gene therapy programs, including OPGx-RDH12 and OPGx-MERTK [1][13] Financial Performance - As of June 30, 2025, Opus Genetics reported cash and cash equivalents of $32.4 million, expected to fund operations into the second half of 2026 [8] - License and collaboration revenue for Q2 2025 totaled $2.9 million, up from $1.1 million in Q2 2024, driven by collaboration with Viatris, Inc. [9] - General and administrative expenses increased to $5.8 million in Q2 2025 from $3.4 million in Q2 2024, primarily due to higher legal and patent-related costs [10] Upcoming Milestones - The company plans to report three-month pediatric data from the OPGx-LCA5 trial in Q3 2025 and initiate enrollment for OPGx-BEST1 in H2 2025 [14] - A supplemental New Drug Application (sNDA) for Phentolamine is expected to be submitted in the second half of 2025 [14] - Enrollment for the LYNX-3 Phase 3 trial targeting nighttime visual disturbances is anticipated to begin in H2 2025 [14]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [1][3] - The company will have its CEO, George Magrath, participate in a fireside chat at the H.C. Wainwright 5th Annual Ophthalmology Virtual Conference on August 13, 2025 [1][2] Company Overview - Opus Genetics is developing AAV-based gene therapies targeting inherited retinal diseases such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - The lead candidate, OPGx-LCA5, is currently in a Phase 1/2 trial for LCA5-related mutations and has shown promising early results [3] - Additional programs include OPGx-BEST1 for BEST1-related retinal degeneration and a Phase 3-ready small molecule therapy for diabetic retinopathy, developed under a Special Protocol Assessment with the FDA [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and dim light vision disturbances [3] - Opus Genetics is headquartered in Research Triangle Park, NC [3]

Core Viewpoint - Opus Genetics has formed a strategic partnership with the Global RDH12 Alliance to advance the development of OPGx-RDH12, a gene therapy targeting RDH12 mutations associated with Leber congenital amaurosis (RDH12-LCA) [1][2][3] Group 1: Collaboration Details - The Global RDH12 Alliance will contribute up to $1.6 million towards the OPGx-RDH12 program, which includes a risk-sharing structure and performance-based milestones [3] - The collaboration aims to co-develop the OPGx-RDH12 program, focusing on clinical and regulatory strategies, with the goal of filing an Investigational New Drug (IND) application with the FDA by late 2025 [3] Group 2: Background on RDH12-LCA - RDH12-LCA is an ultra-rare form of childhood blindness affecting several thousand people globally, characterized by early visual decline and structural retinal changes by age two [5] - The OPGx-RDH12 therapy utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the RDH12 gene directly to retinal photoreceptors, with preclinical studies showing restoration of RDH12 activity [5] Group 3: Organizational Insights - The RDH12 Fund for Sight is a U.S.-based non-profit organization focused on supporting families affected by RDH12-LCA and funding research for treatments [7] - Eyes on the Future is a UK-based non-profit organization dedicated to research into rare eye diseases, specifically targeting RDH12 inherited retinal dystrophies [8] - Opus Genetics is a clinical-stage biopharmaceutical company developing gene and small molecule therapies for vision-threatening eye diseases, with a pipeline that includes therapies for various inherited retinal diseases [9]

Core Insights - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [1][3] - The company granted equity awards to two new non-executive employees as an inducement for employment, consisting of stock options and restricted stock units (RSUs) [1][2] Equity Awards Details - The equity awards include stock options for 240,000 shares and 150,000 RSUs, with the stock options having an exercise price of $0.94, equal to the closing price on the grant date [2] - Stock options vest over four years, with 25% vesting on the one-year anniversary and the remaining 75% vesting quarterly thereafter; RSUs vest in four equal installments on each anniversary of the grant date [2] Company Pipeline - Opus Genetics is developing AAV-based gene therapies targeting inherited retinal diseases such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - The lead candidate, OPGx-LCA5, is in a Phase 1/2 trial for LCA5-related mutations and has shown promising early results [3] - Additional programs include OPGx-BEST1 for BEST1-related retinal degeneration and a Phase 3-ready small molecule therapy for diabetic retinopathy, developed under a Special Protocol Assessment with the FDA [3] - The company is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and dim light vision disturbances [3]

Core Insights - Opus Genetics announced positive topline results from the VEGA-3 trial, which evaluated Phentolamine Ophthalmic Solution 0.75% for treating presbyopia, showing significant improvement in near vision for participants [1][2][3] Group 1: Trial Results - The VEGA-3 trial met its primary endpoint, with 27.2% of participants achieving a ≥15-letter improvement in binocular distance-corrected near visual acuity (DCNVA) compared to 11.5% in the placebo group (p<0.0001) [2][7] - Key secondary efficacy endpoints were also met, indicating rapid and sustained improvement in near visual acuity [6] - Significant patient-reported functional benefits were observed, with satisfaction in near vision reported upon awakening (p<0.0001) [7] Group 2: Study Design - VEGA-3 is a multicenter, randomized, double-masked, placebo-controlled Phase 3 study that enrolled 545 participants across 40 sites in the United States [4] - Participants were randomized in a 3:2 ratio to receive either Phentolamine Ophthalmic Solution 0.75% or placebo, administered once daily in the evening [4] Group 3: Safety Profile - The safety profile of Phentolamine Ophthalmic Solution 0.75% was consistent with previous clinical trials, with no treatment-related serious adverse events reported [6][15] - Common treatment-emergent adverse events included conjunctival hyperemia, instillation site irritation, and dysgeusia, all predominantly mild [15] Group 4: Future Plans - The positive results from the VEGA-3 trial support the submission of an application to the U.S. Food and Drug Administration (FDA), planned for the second half of 2025 [3] - Opus Genetics is also advancing Phentolamine Ophthalmic Solution 0.75% in two Phase 3 programs for presbyopia and dim light vision disturbances [11][13]

Core Insights - Opus Genetics, Inc. has secured up to $2 million in non-dilutive funding from the Retinal Degeneration Fund to advance its OPGx-MERTK program aimed at treating retinitis pigmentosa caused by MERTK gene mutations [1][2] - The funding will support the preclinical development of OPGx-MERTK, which is expected to progress towards Investigational New Drug (IND) enabling studies [2] - Opus anticipates that this funding will extend its cash runway into the second half of 2026, ensuring operational continuity [3] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene and small molecule therapies for inherited retinal diseases (IRDs) [5] - The company's pipeline includes AAV-based gene therapies targeting various IRDs, with its lead candidate OPGx-LCA5 currently in a Phase 1/2 trial [5] - Opus is also advancing therapies for diabetic retinopathy and presbyopia, showcasing a diverse approach to vision-threatening conditions [5] Program Details - The OPGx-MERTK program utilizes a modern AAV vector to target MERTK-associated IRD, which affects approximately 600 individuals in the U.S. [4] - The funding from the RD Fund highlights the collaboration between venture philanthropy and industry to accelerate the development of treatments for rare diseases [2]

Core Insights - Opus Genetics announced positive topline results from the LYNX-2 Phase 3 clinical trial for Phentolamine Ophthalmic Solution 0.75%, targeting chronic night driving impairment in keratorefractive patients with reduced mesopic vision [1][4][6] Group 1: Clinical Trial Results - The LYNX-2 study met its primary endpoint, showing that 17.3% of patients treated with Phentolamine achieved a ≥15-letter improvement in mesopic low contrast distance visual acuity (mLCVA) at Day 15, compared to 9.2% in the placebo group (p<0.05) [3][12] - Patient-reported outcomes indicated significant improvements in night-driving vision, particularly in difficulties seeing the road due to oncoming headlights and glare during dawn or dusk [4][12] - The study involved 199 patients who had undergone keratorefractive surgery and reported decreased visual acuity under low-light conditions, treated over a 6-week period [5][6] Group 2: Mechanism and Safety Profile - Phentolamine Ophthalmic Solution 0.75% works by reducing pupil diameter through a sympatholytic mechanism, avoiding risks associated with older agents [2][10] - The safety profile of Phentolamine was consistent with previous studies, with no new safety signals identified and no evidence of tachyphylaxis observed over the 6-week period [6][7] Group 3: Market Potential and Partnerships - The therapy addresses a significant unmet need, as there are currently no FDA-approved treatments for this condition, potentially offering meaningful benefits to keratorefractive patients [4][10] - Opus Genetics has a global licensing agreement with Viatris for the commercialization of Phentolamine Ophthalmic Solution 0.75% in the U.S. [8]

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 March 31, 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 | | --- | --- | | ( ...