Core Insights - Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases (IRDs) [8] - The company’s gene therapy OPGx-LCA5 is currently in a Phase 1/2 trial aimed at restoring vision for individuals with a rare genetic form of blindness caused by mutations in the LCA5 gene [2][8] - The recent feature on Good Morning America highlights the potential of gene therapy to change lives, bringing national attention to the advancements in treating inherited blindness [3][5] Company Overview - Opus Genetics is based in Research Triangle Park, NC, and is developing AAV-based gene therapies targeting various inherited retinal diseases, including Leber congenital amaurosis (LCA) and bestrophinopathy [8] - The company’s pipeline includes lead candidates OPGx-LCA5 and OPGx-BEST1, with ongoing clinical trials for both therapies [8] - In addition to gene therapies, Opus Genetics is advancing a partnered therapy, Phentolamine Ophthalmic Solution 0.75%, which is being studied in Phase 3 programs for presbyopia and other visual disturbances [8]

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]

Momentum investing is all about the idea of following a stock's recent trend, which can be in either direction. In the "long context," investors will essentially be "buying high, but hoping to sell even higher." And for investors following this methodology, taking advantage of trends in a stock's price is key; once a stock establishes a course, it is more than likely to continue moving in that direction. The goal is that once a stock heads down a fixed path, it will lead to timely and profitable trades.Whil ...

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]