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]

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 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 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 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]