Opus Genetics Highlights Significant Progress in 2025 and Upcoming Catalysts in 2026
Opus Genetics highlighted its progress achieved during 2025 and upcoming catalysts in 2026. OPGx-BEST1: First participant dosed in Phase 1/2 trial with enrollment ongoing. Initial data expected this quarter at Macula Society with 3-month results from the entire Cohort 1 expected in mid-2026. OPGx-BEST1 is potentially eligible for multiple regulatory designations which the Company expects to file for in 2026. OPGx-LCA5: Enrollment ongoing in run-in period for planned, adaptive pivotal Phase 3 trial. Dosing with OPGx-LCA5 in the Phase 3 trial expected in the second half of 2026. Pre-Clinical Gene Therapy Pipeline: Multiple partnerships with patient advocacy organizations are increasing awareness in the IRD community and providing non-dilutive funding for pre-clinical testing of Opus Genetics gene therapy pipeline. Phentolamine Ophthalmic Solution 0.75%: Supplemental New Drug Application submitted for the treatment of presbyopia, with an anticipated regulatory decision by the end of 2026. LYNX-3, the second pivotal Phase 3 trial in keratorefractive participants with visual disturbances under mesopic, low-contrast conditions, is ongoing with topline results expected in the first half of 2026.
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- Insider Purchase: Cam Gallagher of Opus Genetics acquired 164,000 shares of IRD at $1.97 each on Wednesday, totaling an investment of $323,693, reflecting confidence in the company's future prospects.
- Investment Return: Gallagher's investment is currently up approximately 18.1%, based on today's trading high of $2.33, indicating a positive market response and potential growth outlook for IRD.
- Market Performance: Opus Genetics shares rose about 8.7% on Friday, reflecting optimistic sentiment among investors regarding the company's outlook, potentially driven by Gallagher's purchase.
- CEO Acquisition: Additionally, CEO Dwayne L. Hyzak purchased 3,712 shares of MSC Income Fund at $13.43 each for a total of $49,852, demonstrating ongoing confidence in the fund's performance.
- Equity Incentive Plan: Opus Genetics approved an equity award of 50,000 stock options to a new employee under its 2021 Inducement Plan, aimed at attracting top talent and strengthening its team to drive innovation in gene therapy.
- Option Details: The options have an exercise price equal to the fair market value of the company's common stock on the grant date, vesting over four years with 25% vesting after one year and the remaining 75% in quarterly installments, ensuring long-term employee retention aligned with company goals.
- Compliance Assurance: This equity award complies with Nasdaq Listing Rule 5635(c)(4), ensuring that the company adheres to transparent compliance procedures while attracting talent, thereby enhancing investor confidence.
- Company Background: Opus Genetics focuses on developing gene therapies to restore vision for patients with inherited retinal diseases, with a pipeline of seven AAV-based programs, showcasing the company's strong R&D capabilities and market potential in the biopharmaceutical sector.

- Conference Presentation: Opus Genetics CEO George Magrath will present at the J.P. Morgan Healthcare Conference on January 15, 2026, showcasing the company's latest advancements in gene therapy, which is expected to attract investor interest.
- Pipeline Development: The company is developing seven AAV-based programs, including treatments for LCA5 and BEST1-related retinal degeneration, aimed at addressing the underlying genetic causes of severe retinal disorders, potentially offering new therapeutic options for patients.
- Therapeutic Progress: Opus Genetics is also advancing Phentolamine Ophthalmic Solution, targeting pharmacologically induced mydriasis, with additional indications for presbyopia and low-light visual disturbances in late-stage development, indicating the company's diversified approach in the ophthalmology sector.
- Company Overview: Based in Research Triangle Park, NC, Opus Genetics focuses on developing gene therapies to restore vision for patients with inherited retinal diseases, demonstrating its innovative potential in the biopharmaceutical industry.

- Leadership Presentation: Opus Genetics CEO George Magrath will speak at the J.P. Morgan Healthcare Conference on January 15, 2026, showcasing the company's latest advancements in gene therapy, which is expected to attract investor interest and enhance the company's visibility.
- Technical Focus: Opus Genetics specializes in developing gene therapies for inherited retinal diseases, with a pipeline that includes seven AAV-based programs aimed at providing one-time treatments to address the underlying genetic causes of severe retinal disorders, potentially transforming patient quality of life.
- Product Development: The company is advancing Phentolamine Ophthalmic Solution, which is not only approved for pharmacologically induced mydriasis but also in late-stage development for presbyopia and low-light visual disturbances, indicating its diversified approach in the ophthalmic sector.
- Market Positioning: By focusing on addressing the root causes of severe retinal diseases, Opus Genetics is well-positioned to capture a share in the rapidly growing biopharmaceutical market as demand for gene therapies increases.
Clinical Trial Update: Opus Genetics' shares rose after the company announced it will continue its Phase 1/2 BEST1 clinical trial for the eye disease therapy OPGx-BEST1 without modifications, following a safety review by an Independent Data Monitoring Committee.
Safety Review Findings: The Independent Data Monitoring Committee recommended the continuation of the trial, which targets patients with Best disease, based on a four-week safety data analysis from the first participant.
Company Background: Opus Genetics was formed after Ocuphire Pharma acquired it in an all-stock deal last year, focusing on gene therapy for eye diseases.
CEO's Confidence: CEO George Magrath expressed confidence in the program's advancement following the positive safety review and recommendation from the IDMC.

- Clinical Trial Progress: Opus Genetics' BEST1 clinical trial (BIRD-1) received a positive recommendation from the Independent Data Monitoring Committee, allowing for the continued recruitment of additional participants, indicating the company's potential in treating inherited retinal diseases.
- Safety Assessment: The first participant showed good safety results after one month, supporting the company's ability to proceed with dosing more participants, thereby enhancing confidence in the treatment's efficacy.
- Market Demand: BEST1-associated retinal diseases affect approximately 9,000 patients in the U.S., and Opus Genetics' research aims to address this substantial unmet medical need, potentially offering hope for vision restoration.
- Technological Platform Advantage: The company leverages its proprietary AAV gene therapy platform to deliver a functional BEST1 gene directly to retinal pigment epithelial cells, showcasing its innovative capabilities and competitive edge in the gene therapy market.






