Core Points - The conference call is focused on Opus Genetics and their LCA5 data presentation [1] - Participants are informed that the webcast will allow video and audio access, while phone participants will need to view the replay later [1][2] - The call is being recorded for future reference [1] Company Information - The host of the conference call is Jenny Kobin from Opus Investor Relations [2]

Core Insights - Opus Genetics announced positive three-month data from its Phase 1/2 clinical trial for OPGx-LCA5, a gene therapy for Leber congenital amaurosis type 5 (LCA5), showing potential to restore vision in pediatric patients [1][2][8] Clinical Trial Results - The pediatric cohort showed an average improvement of 0.3 logMAR in visual acuity, surpassing improvements seen in adults [3] - All three pediatric participants demonstrated significant improvements in Full-Field Stimulus Testing, with greater than one log unit improvement in cone sensitivity to red and blue light [4] - Participants in the Multi-Luminance Orientation and Mobility Test identified more objects at three months compared to baseline, with two participants showing greater improvement in the treated eye [5] - Microperimetry data indicated early signs of improved fixation stability in one participant, suggesting functional retinal recovery [6] Safety and Tolerability - OPGx-LCA5 has been well-tolerated among all six participants (three adults and three pediatric), with no serious ocular adverse events or dose-limiting toxicities reported [7][10] Future Plans - The company plans to meet with the U.S. FDA in Q4 2025 to discuss the trial results and next steps for the LCA5 program [2][8] Background Information - OPGx-LCA5 targets LCA5, an ultra-rare inherited retinal disease caused by mutations in the LCA5 gene, affecting approximately 200 patients [16] - The therapy utilizes an adeno-associated virus 8 (AAV8) vector to deliver a functional LCA5 gene to the outer retina, with no approved therapies currently available for LCA5-related conditions [14][16]

Core Viewpoint - Opus Genetics has initiated the first patient dosing in LYNX-3, a pivotal Phase 3 clinical trial for Phentolamine Ophthalmic Solution 0.75%, aimed at treating chronic night driving impairment in keratorefractive patients with reduced mesopic vision [1][2]. Group 1: Clinical Trial Details - LYNX-3 is the second Phase 3 trial for this indication, designated as Fast Track by the FDA, and follows a Special Protocol Assessment [2]. - The trial will enroll approximately 200 adults with decreased visual acuity in low-light conditions post-keratorefractive surgery, including LASIK and PRK [5]. - Participants will be randomized to receive either Phentolamine Ophthalmic Solution 0.75% or placebo for about two weeks [5]. Group 2: Treatment Mechanism and Benefits - Phentolamine Ophthalmic Solution 0.75% is a non-selective α1/α2 adrenergic antagonist designed to reduce pupil size in low-light conditions, potentially improving visual quality by decreasing the impact of peripheral light rays [3][7]. - The primary endpoint of the trial is the percentage of participants achieving a ≥15-letter improvement in mesopic low-contrast visual acuity at Day 15 [6]. - Key secondary endpoints include patient-reported outcomes related to night driving and visual disturbances such as glare and halos [6]. Group 3: Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on gene therapies for inherited retinal diseases and small-molecule therapies for ophthalmic disorders [9]. - The company is advancing Phentolamine Ophthalmic Solution 0.75% in two Phase 3 programs, with one approved indication for drug-induced mydriasis and positive data for presbyopia [4][9].

Company Overview - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [3] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Lead gene therapy candidates include OPGx-LCA5, currently in a Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, targeting BEST1-related retinal degeneration [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 reduced low light vision [3] Corporate Update - George Magrath, MD, CEO, and Ash Jayagopal, Ph.D., MBA, Chief Scientific and Development Officer, will present a corporate update at the H.C. Wainwright 27th Annual Global Investment Conference from September 8-10, 2025 [1] - The presentation will be available on demand starting September 8, 2025, at 7:00 a.m. ET [1] Investor Relations - Recent investor event replays are accessible on the Opus Genetics website under the Investors section [2]

Core Insights - Opus Genetics, Inc. has appointed Rob Gagnon as Chief Financial Officer, bringing over two decades of financial and operational leadership experience in the biotech sector [1][2][3] Company Overview - 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 [5] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [5] - Lead gene therapy candidates include OPGx-LCA5, currently in a Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1, targeting BEST1-related retinal degeneration [5] - Opus is also advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and being studied in two Phase 3 programs for presbyopia and visual disturbances [5] Leadership Experience - Rob Gagnon has raised over $2 billion in capital and has experience in guiding companies through IPOs, late-stage clinical development, and M&A [2][3] - Prior to joining Opus, Gagnon served as CFO at Remix Therapeutics and held senior roles at Verastem Oncology, Harvard Bioscience, Clean Harbors, and Biogen [2][3][4] - Gagnon holds an MBA from MIT Sloan School of Management and a BA in Accounting from Bentley College, and is a Certified Public Accountant [4]

Company Overview - Opus Genetics, Inc. is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) and small molecule therapies for other ophthalmic disorders [3] - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Lead gene therapy candidates include OPGx-LCA5, currently in a Phase 1/2 trial for LCA5-related mutations, and OPGx-BEST1 for BEST1-related retinal degeneration [3] - Additionally, the company is advancing Phentolamine Ophthalmic Solution 0.75%, which is approved for one indication and is being studied in two Phase 3 programs for presbyopia and low light vision disturbances [3] Upcoming Presentations - Opus Genetics will present its IRD gene therapy programs at several scientific conferences in September 2025 [1] - Presentation at the Ophthalmology Futures Forum will focus on "Gene & Cell Therapies for Rare & Common Retinal Diseases: Hype Vs Progress" on September 3, 2025 [2] - At the RD 2025 International Symposium, the company will share one-year results from a Phase I/II study of OPGx-LCA5 for inherited retinal degeneration due to biallelic mutations in the LCA5 gene [2] - The LSX World Congress will feature a presentation titled "The Equation for Maturation: Biotech Requirements to Achieve Scale" [2] Key Personnel - Sally Tucker, Ph.D., Senior Vice President Clinical Development, will present at the Ophthalmology Futures Forum and participate in a panel discussion in Paris, France on September 3, 2025 [4] - Ash Jayagopal, Ph.D., Chief Scientific & Development Officer, will present in Prague, Czech Republic on September 15, 2025 [4] - Ben Yerxa, Ph.D., President, will also participate in a panel discussion in Boston, MA on September 17, 2025 [4]

Core Insights - Opus Genetics has received FDA acceptance for its Investigational New Drug (IND) application for OPGx-BEST1, a gene therapy targeting BEST1-related inherited retinal disease (IRD) [1][4] - The Phase 1/2 clinical trial is set to begin in the second half of 2025, focusing on safety, tolerability, and preliminary efficacy of a single subretinal injection [3][4] - BEST1-related IRDs currently lack approved treatments, highlighting the urgency for effective therapies in this area [4] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company specializing in gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [5] - The company's pipeline includes AAV-based gene therapies for conditions such as Leber congenital amaurosis (LCA) and retinitis pigmentosa, with OPGx-LCA5 also in an ongoing Phase 1/2 trial [5] - The company is based in Research Triangle Park, NC, and is committed to advancing multiple therapies for patients with unmet needs [5]

Company Performance - Opus Genetics, Inc. reported a quarterly loss of $0.12 per share, which was better than the Zacks Consensus Estimate of a loss of $0.25, representing an earnings surprise of +52.00% [1] - The company posted revenues of $2.88 million for the quarter ended June 2025, missing the Zacks Consensus Estimate by 12.13%, compared to revenues of $1.11 million a year ago [2] - Over the last four quarters, Opus Genetics has surpassed consensus EPS estimates three times and topped consensus revenue estimates three times [2] Stock Movement and Outlook - Opus Genetics shares have declined approximately 6.7% since the beginning of the year, while the S&P 500 has gained 9.6% [3] - The company's earnings outlook is crucial for investors, as it includes current consensus earnings expectations for upcoming quarters and any recent changes to these expectations [4] - The current consensus EPS estimate for the upcoming quarter is -$0.25 on revenues of $3.44 million, and for the current fiscal year, it is -$0.99 on revenues of $15.66 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Opus Genetics belongs, is currently ranked in the bottom 41% of over 250 Zacks industries, indicating potential challenges for stock performance [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact Opus Genetics' stock performance [5][6]

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)