Core Insights - 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] Company Overview - The company is based in Research Triangle Park, NC, and its pipeline includes AAV-based gene therapies targeting conditions such as Leber congenital amaurosis (LCA), bestrophinopathy, and retinitis pigmentosa [3] - Opus Genetics' lead gene therapy candidates are OPGx-LCA5, currently in an ongoing 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] Upcoming Events - Ash Jayagopal, PhD, MBA, Chief Scientific and Development Officer, will present a corporate update at Chardan's 9th Annual Genetic Medicines Conference on October 21, 2025, at 12:00 p.m. ET [1]

Core Insights - Opus Genetics, Inc. is set to present positive clinical results from its gene therapy and Phentolamine Ophthalmic Solution programs at various medical and industry conferences in October 2025 [1][2] 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 for BEST1-related retinal degeneration [5] - 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 visual disturbances [5] Conference Participation - The company will participate in several key conferences, including: - Cell and Gene Meeting on the Mesa - Fierce Biotech Week - American Academy of Optometry Annual Meeting 2025 - Eyecelerator @ American Academy of Ophthalmology (AAO) [3][4] Presentation Details - Presentation on OPGx-LCA5 will include three-month pediatric and 18-month adult clinical data from the Phase 1/2 trial for Leber congenital amaurosis type 5 [6] - A fireside chat titled "Investing with Intention – How Early Vision and Partnership Shaped Opus Genetics" will feature key executives from the company [6] - A poster presentation on the pivotal Phase 3 trial of Phentolamine Ophthalmic Solution will also take place [6]

Company Overview - Opus Genetics, Inc. (IRD) currently holds a Momentum Style Score of A, indicating strong momentum potential [3] - The company has a Zacks Rank of 2 (Buy), suggesting favorable investment conditions [4] Price Performance - Over the past week, IRD shares have increased by 19.74%, significantly outperforming the Zacks Medical - Biomedical and Genetics industry, which rose by 1.09% [6] - In a longer time frame, IRD's monthly price change is 34.15%, compared to the industry's 3.08% [6] - Over the past quarter, IRD shares have risen by 55.66%, and by 30.95% in the last year, while the S&P 500 has only moved 8.12% and 17.38%, respectively [7] Trading Volume - The average 20-day trading volume for IRD is 358,390 shares, which serves as a useful indicator of market interest [8] Earnings Outlook - In the past two months, one earnings estimate for IRD has moved higher, while none have moved lower, resulting in an increase in the consensus estimate from -$0.99 to -$0.55 [10] - For the next fiscal year, one estimate has also moved upwards with no downward revisions during the same period [10] Conclusion - Considering the strong price performance, positive earnings outlook, and high momentum score, IRD is positioned as a promising investment opportunity [12]

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]