RESEARCH TRIANGLE PARK, N.C., Nov. 20, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), announced today that management will participate in the following investor conferences. BTIG 5th Annual Ophthalmology Day (Virtual): Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, will participate ...

Core Insights - Opus Genetics has initiated the dosing of the first participant in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [2][5] - The OPGx-BEST1 therapy is a one-time subretinal injection aimed at restoring function to retinal pigment epithelium cells affected by mutations in the BEST1 gene [3][6] - Initial data from the trial is expected in Q1 2026, which will evaluate the safety, tolerability, and preliminary efficacy of the treatment [7] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases [10] - The company’s pipeline includes seven AAV-based programs, with OPGx-BEST1 targeting BEST1-related retinal degeneration [10] - The company is based in Research Triangle Park, NC, and is also advancing other therapies for various ocular conditions [10] Clinical Trial Details - The Phase 1/2 trial, known as BIRD-1, is an adaptive, open-label, dose-exploring study evaluating OPGx-BEST1 in participants with Best Vitelliform Macular Dystrophy or Autosomal-Recessive Bestrophinopathy [7] - The trial will explore biological activity through functional and anatomical endpoints, including changes in visual function and retinal structure [7] - The study is being conducted by a team of experts from the Retina Foundation of the Southwest and Retina Consultants of Texas [4]

Core Insights - Opus Genetics has initiated the first dosing in its OPGx-BEST1 Phase 1/2 clinical trial for Best disease, marking a significant milestone for the inherited retinal disease community [1][4] - The trial aims to evaluate the safety, tolerability, and preliminary efficacy of a one-time subretinal injection of OPGx-BEST1, targeting the underlying genetic cause of Best disease [6][9] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases [9] - The company is advancing multiple AAV-based programs, including OPGx-LCA5 and OPGx-BEST1, with a pipeline targeting various genetic mutations associated with severe retinal disorders [9] Clinical Trial Details - The OPGx-BEST1 trial is a multi-center, adaptive, open-label study designed to explore the safety and efficacy of the gene therapy in patients with Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB) [6] - Initial data from the trial is expected in the first quarter of 2026, with the study focusing on functional and anatomical endpoints related to visual function and retinal structure [6] Disease Background - Best disease, caused by mutations in the BEST1 gene, leads to progressive vision loss and affects an estimated 9,000 patients in the United States [2][8] - The BEST1 gene is crucial for producing bestrophin, a protein that regulates chloride ion movement in retinal cells, and its mutations can result in severe retinal conditions [7][8]

Core Insights - Opus Genetics, Inc. reported a quarterly loss of $0.12 per share, better than the Zacks Consensus Estimate of a loss of $0.14, and improved from a loss of $0.29 per share a year ago, resulting in an earnings surprise of +14.29% [1] - The company generated revenues of $3.08 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 11.36%, although this represents a decline from $3.87 million in the same quarter last year [2] - Opus Genetics shares have increased approximately 65.6% year-to-date, significantly outperforming the S&P 500's gain of 16.4% [3] Earnings Outlook - 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 next quarter is -$0.12 on revenues of $3.75 million, and for the current fiscal year, it is -$0.56 on revenues of $13.67 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Opus Genetics belongs, is currently ranked in the top 34% of over 250 Zacks industries, indicating a favorable outlook compared to lower-ranked industries [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors or through tools like the Zacks Rank [5][6]

Financial Performance - The company reported that it has not generated significant revenue from product sales and expects to incur losses for the foreseeable future[152]. - The company reported a net loss of $17.5 million for the three months ended September 30, 2025, compared to a net loss of $7.5 million for the same period in 2024[189]. - As of September 30, 2025, the company had an accumulated deficit of $172.1 million[189]. - The net loss for the three months ended September 30, 2025, was $17.5 million, compared to a net loss of $7.5 million in the same period of 2024, an increase of $9.9 million[205]. - For the nine months ended September 30, 2025, net cash used in operating activities was $25.4 million, attributed to a net loss of $33.1 million[256]. - For the nine months ended September 30, 2024, cash used in operating activities was $18.1 million, with a net loss of $22.4 million[258]. Clinical Development - OPGx-LCA5 is currently in a Phase 1/2 clinical trial, with six participants treated, all showing improvements in vision[160]. - The FDA provided constructive feedback on the registration strategy for OPGx-LCA5, acknowledging the significant unmet medical need for LCA5-related blindness[163]. - The Phase 3 trial for OPGx-LCA5 is expected to begin dosing in the second half of 2026, with topline clinical data anticipated approximately one year later[165]. - OPGx-BEST1 received FDA clearance for an IND application, with initial data expected in the first quarter of 2026[167]. - The company has five programs in pre-clinical development, with three currently in IND-enabling studies supported by grants[168]. - The company anticipates increased research and development expenses as it advances clinical trials for LCA5, BEST1, and PS[198]. - The company intends to file a Supplemental New Drug Application for the treatment of presbyopia with PS in Q4 2025[172]. - The FDA granted RMAT designation to OPGx-LCA5, facilitating expedited development and review for the investigational gene therapy[183]. Revenue and Funding - The company raised approximately $23.0 million in a registered direct offering that closed on November 7, 2025[176]. - The August 2025 private placement generated gross proceeds of $3.5 million from the issuance of 3,138,338 shares of common stock[177]. - The November 2025 Registered Direct Offering generated gross proceeds of approximately $23.0 million, with the intention to advance LCA5 and BEST-1 gene therapy programs[228]. - The company raised approximately $94.9 million through various equity offerings and $8.5 million from convertible notes, including a one-time non-refundable cash payment of $35.0 million in Q4 2023 related to the Viatris License Agreement[227]. - The RDF Agreement provides for nondilutive funding of up to $2.0 million for the development of the MERTK Program, with $1.0 million already disbursed[179]. - License and collaborations revenue decreased to $3.1 million for the three months ended September 30, 2025, down from $3.9 million in 2024, a decline of $0.8 million attributed to reduced PS research and development services[206]. - For the nine months ended September 30, 2025, license and collaborations revenue increased to $10.3 million from $6.7 million in 2024, a rise of $3.6 million due to increased PS research and development services[216]. - License and collaborations revenue to date includes a one-time non-refundable payment of $35 million and a milestone payment of $10 million[262]. - The company anticipates recognizing revenue as it earns reimbursement for research and development services under the Viatris License Agreement[262]. - The company expects to satisfy its short-term and long-term obligations through cash on hand, future equity and debt financings, and reimbursement payments[279]. Stock and Market Activity - The market price of the company's common stock is expected to be volatile, with potential risks of delisting from the Nasdaq Capital Market[152]. - The company has sold an aggregate of 9,479,494 shares under the ATM program, resulting in gross proceeds of $28.5 million[264]. - The company established an at-the-market equity offering program with an aggregate offering price of up to $40.0 million[245]. - As of September 30, 2024, 1,538,461 RDO Warrants were still outstanding from a previous offering that raised $15.0 million[247]. - The Series A Warrants, issued on November 19, 2020, have an initial exercise price of $4.4795 per share and 5,665,838 warrants were still outstanding as of September 30, 2025[253]. Expenses - General and administrative expenses increased to $5.0 million in Q3 2025 from $2.9 million in Q3 2024, an increase of $2.1 million primarily due to public company costs and legal fees[207]. - Research and development expenses for Q3 2025 were $6.4 million, down from $9.0 million in Q3 2024, a decrease of $2.6 million mainly due to lower APX 3330 expenses[210]. - General and administrative expenses for the nine months ended September 30, 2025, were $17.1 million compared to $10.9 million in 2024, an increase of $6.2 million driven by various operational costs[217]. - Research and development expenses for the nine months ended September 30, 2025, totaled $20.4 million, slightly up from $19.8 million in 2024, an increase of $0.6 million primarily due to higher IRD program expenses[221]. - The fair value change in warrant and other derivative liabilities was $9.5 million for Q3 2025, reflecting fluctuations in common stock fair value[211]. - The fair value change in warrant and other derivative liabilities for the nine months ended September 30, 2025, was $5.8 million, attributed to the March 2025 Warrants[222]. Corporate Developments - The Opus Acquisition expanded the product pipeline significantly, issuing 5,237,063 shares of common stock and 14,145.374 shares of Series A preferred stock[157]. - The company relocated its headquarters to Durham, North Carolina, and amended its lease for the headquarters on a month-to-month basis[269].

Financial Performance - Opus Genetics reported a net loss of $17.5 million, or $(0.25) per share, for Q3 2025, compared to a net loss of $7.5 million, or $(0.29) per share, in Q3 2024[14]. - Comprehensive loss for the nine months ended September 30, 2025, was $33,068 million, compared to $22,397 million for the same period in 2024[21]. - Loss from operations for the nine months ended September 30, 2025, was $27,146 million, compared to $24,045 million in the same period of 2024[21]. - Basic and diluted net loss per share for Q3 2025 was $0.25, an improvement from $0.29 in Q3 2024[21]. - The number of shares used in basic and diluted net loss per share calculations for Q3 2025 was 70,636,887, significantly higher than 26,145,080 in Q3 2024[21]. - Fair value change in warrant and other derivative liabilities resulted in a loss of $9,525 million for Q3 2025[21]. - Other income for Q3 2025 was $450 million, slightly down from $483 million in Q3 2024[21]. Revenue and Expenses - Revenue from license and collaborations totaled $3.1 million in Q3 2025, down from $3.9 million in the same period in 2024, primarily due to lower reimbursement for research and development services[11]. - License and collaborations revenue for Q3 2025 was $3,079 million, a decrease from $3,867 million in Q3 2024[21]. - Total operating expenses for Q3 2025 were $11,390 million, slightly down from $11,876 million in Q3 2024[21]. - General and Administrative expenses increased to $5.0 million in Q3 2025 from $2.9 million in Q3 2024, driven by higher legal, payroll, and professional service costs[12]. - Research and Development expenses decreased to $6.4 million in Q3 2025 from $9.0 million in Q3 2024, attributed to lower clinical research and manufacturing costs[13]. - Research and development expenses for the nine months ended September 30, 2025, were $20,384 million, up from $19,817 million in the same period of 2024[21]. Cash and Funding - As of September 30, 2025, Opus Genetics had cash and cash equivalents of $30.8 million, with an additional $23.0 million raised post-quarter through equity offerings, expected to fund operations into the second half of 2027[9]. - Total current liabilities increased to $29.1 million as of September 30, 2025, compared to $11.3 million at the end of 2024, largely due to warrant liabilities[19]. Clinical Developments - The company plans to submit a Supplemental New Drug Application for Phentolamine Ophthalmic Solution 0.75% by year-end 2025[10]. - Positive clinical data from the OPGx-LCA5 Phase 1/2 trial showed significant improvements in cone-mediated vision in pediatric participants over three months[7]. - The FDA accepted the Investigational New Drug application for OPGx-BEST1, with initial data expected in Q1 2026[7]. - The company is advancing its OPGx-RDH12 program with up to $1.6 million in non-dilutive funding from the Global RDH12 Alliance[10].

Core Insights - Opus Genetics has reported positive clinical data from its OPGx-LCA5 Phase 1/2 trial, indicating potential for restoring cone-mediated vision in patients with inherited retinal diseases [1][6] - The company successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA, which may facilitate an accelerated regulatory pathway for OPGx-LCA5 [1][6] Pipeline Updates - The OPGx-LCA5 program is focused on gene therapy for Leber Congenital Amaurosis (LCA) and has shown significant improvements in visual function in both pediatric and adult participants [3][6] - Recruitment is ongoing for the OPGx-BEST1 gene therapy program, targeting BEST1-related inherited retinal diseases [4][5] - The company plans to submit a Supplemental New Drug Application for Phentolamine Ophthalmic Solution 0.75% for presbyopia treatment by the end of 2025 [5][8] Financial Results - As of September 30, 2025, Opus Genetics reported cash and cash equivalents of $30.8 million, with an additional $23.0 million raised through a direct equity offering [10] - License and collaborations revenue for Q3 2025 was $3.1 million, a decrease from $3.9 million in Q3 2024, primarily due to lower research and development service reimbursements [11] - General and Administrative expenses increased to $5.0 million in Q3 2025 from $2.9 million in Q3 2024, attributed to higher legal, payroll, and professional service costs [12] - Research and Development expenses decreased to $6.4 million in Q3 2025 from $9.0 million in Q3 2024, reflecting lower costs in clinical research and manufacturing [13] Net Loss - The net loss for Q3 2025 was $17.5 million, or $(0.25) per share, compared to a net loss of $7.5 million, or $(0.29) per share, in Q3 2024 [15][20]

Core Insights - Opus Genetics has successfully completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the FDA regarding its gene therapy candidate OPGx-LCA5 for Leber congenital amaurosis (LCA) caused by mutations in the LCA5 gene [1][2][8] - The FDA provided constructive feedback on Opus's registration strategy, including Chemistry, Manufacturing and Controls (CMC), and pivotal trial design, acknowledging the significant unmet medical need for individuals with LCA5-related blindness [2][3] - The company plans to advance its ongoing trial using an adaptive design that includes a Phase 3 portion, which will avoid the need for a separate registrational trial [3][4] Clinical Development - Six late-stage participants have been treated in the Phase 1/2 trial of OPGx-LCA5, with all experiencing clinically meaningful improvements in vision [3][9] - The Phase 3 trial will include a run-in period to evaluate the natural history of each participant, serving as their own control [5][4] - Efficacy and safety will be assessed using various measures, including visual acuity and microperimetry, with dosing anticipated in the second half of 2026 [5][8] Financial Position - Opus Genetics has secured recent financing of $23 million, which will support the advancement of its LCA5 program and fund current operating plans into the second half of 2027 [6][8] Regulatory Environment - The FDA's introduction of the Rare Disease Evidence Principles (RDEP) review process aims to facilitate the approval of drugs for rare diseases, and Opus believes its LCA5 program meets the eligibility criteria for this process [7][8] - OPGx-LCA5 has the potential to be the first gene therapy and one-time treatment for LCA type 5, addressing a significant unmet medical need [8][11] Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small-molecule therapies for other ophthalmic disorders [12] - The company's pipeline includes AAV-based gene therapies targeting conditions such as LCA and bestrophinopathy, with OPGx-LCA5 currently in a Phase 1/2 clinical trial [12][9]

Core Viewpoint - Opus Genetics has announced a registered direct offering to raise approximately $23 million to fund its gene therapy clinical programs for inherited retinal diseases and other ophthalmic disorders [1][2]. Group 1: Financing Details - The financing was led by Perceptive Advisors and Balyasny Asset Management, with participation from Nantahala Capital [1]. - The offering includes the sale of 3,827,751 shares of common stock at a price of $2.09 per share and pre-funded warrants to purchase up to 7,177,033 shares at a purchase price of $2.0899 per warrant [3]. - The offering is expected to close on November 7, 2025, subject to customary closing conditions [3]. Group 2: Use of Proceeds - The net proceeds from the offering will be used to advance the LCA5 and BEST-1 gene therapy clinical programs, as well as for working capital and general corporate purposes [2]. - The company expects its cash resources to fund operations into the second half of 2027, excluding potential proceeds from callable warrants or future milestone payments [2]. Group 3: Company Overview - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases and small molecule therapies for other ophthalmic disorders [6]. - The company's pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA) and bestrophinopathy, with lead candidates OPGx-LCA5 and OPGx-BEST1 currently in clinical trials [6].

Core Viewpoint - The market anticipates Opus Genetics, Inc. will report a year-over-year increase in earnings despite lower revenues for the quarter ending September 2025, with actual results being crucial for stock price movement [1][2]. Earnings Expectations - The consensus EPS estimate for Opus Genetics is a loss of $0.14 per share, reflecting a year-over-year improvement of +51.7%. Revenues are projected at $2.77 million, down 28.4% from the previous year [3]. - The consensus EPS estimate has been revised down by 4.55% over the last 30 days, indicating a reassessment by analysts [4]. Earnings Surprise Prediction - The Zacks Earnings ESP model indicates that the Most Accurate Estimate matches the Zacks Consensus Estimate, resulting in an Earnings ESP of 0%, suggesting no recent differing analyst views [12]. - The stock holds a Zacks Rank of 3, making it challenging to predict an earnings beat conclusively [13]. Historical Performance - In the last reported quarter, Opus Genetics was expected to post a loss of $0.25 per share but actually reported a loss of -$0.12, achieving a surprise of +52.00%. Over the last four quarters, the company has beaten consensus EPS estimates three times [14][15]. Industry Context - Certara, Inc., a peer in the Zacks Medical - Biomedical and Genetics industry, is expected to report earnings of $0.11 per share for the same quarter, indicating a year-over-year decline of -15.4%. Its revenue is projected to be $105.22 million, up 11% from the previous year [19][20]. The consensus EPS estimate for Certara has been revised up by 77.3% in the last 30 days, but a lower Most Accurate Estimate results in an Earnings ESP of -4.76% [20][21].