Opus Genetics Stock Price, News & Analysis

Opus Genetics (Nasdaq: IRD) has secured up to $2 million in non-dilutive funding from the Retinal Degeneration Fund (RD Fund) to advance its OPGx-MERTK gene therapy program. The funding will support preclinical development for treating retinitis pigmentosa caused by MERTK gene variants, a condition currently without approved treatments. This partnership between Opus and the RD Fund, the venture philanthropy arm of the Foundation Fighting Blindness, combines industry expertise with philanthropic support to accelerate therapeutic development. The funding will help advance the program toward IND-enabling studies and extends Opus' operational runway into the second half of 2026.

Opus Genetics (NASDAQ: IRD) announced positive Phase 3 trial results for Phentolamine Ophthalmic Solution 0.75%, designed to treat night driving impairment in keratorefractive surgery patients. The LYNX-2 trial met its primary endpoint, with 17.3% of treated patients achieving ≥15-letter improvement in mesopic low contrast distance visual acuity compared to 9.2% in the placebo group (p<0.05). The drug, which reduces pupil diameter through a sympatholytic mechanism, demonstrated significant benefits for patients experiencing vision disturbances like glare and halos under low-light conditions. The study, conducted under FDA Special Protocol Assessment and Fast-Track Designation, showed consistent safety profile with previous trials and no tachyphylaxis over 6 weeks. Opus has a global licensing agreement with Viatris for commercialization rights in the U.S.

Opus Genetics (NASDAQ: IRD) reported Q1 2025 financial results and corporate updates. The company highlighted positive 12-month data from Phase 1/2 trial of OPGx-LCA5 for Leber congenital amaurosis 5, showing sustained treatment benefits. A pediatric cohort study is ongoing with initial data expected in Q3 2025. The company's OPGx-BEST1 program is advancing towards Phase 1/2 trials with early data expected in Q1 2026. Financially, Opus raised $21.5 million through a public offering and private placement, with potential for additional $21.4 million from warrants. Q1 2025 ended with cash position of $41.8 million, expected to fund operations into Q2 2026. The company reported revenue of $4.4 million, with a net loss of $8.2 million ($0.24 per share). Their pipeline includes seven AAV-based gene therapy assets and Phentolamine Ophthalmic Solution 0.75%, currently in Phase 3 trials for presbyopia and dim light vision disturbances.

Opus Genetics (Nasdaq: IRD) announced upcoming presentations of its inherited retinal disease (IRD) gene therapy programs at three scientific conferences in May 2025. The company will present data on:

1. OPGx-MERTK: A gene therapy for MERTK-related Retinitis Pigmentosa (affecting 3% of RP cases), showing effective preservation of photoreceptors and retinal function in rat studies.

2. OPGx-BEST1: A treatment for BEST1-related macular degeneration, demonstrating restoration of RPE-photoreceptor interface in canine models. A Phase 1/2 trial is planned for 2025, with preliminary data expected by Q1 2026.

3. Virtual reality-guided testing methodologies for assessing visual function and defining clinically meaningful improvements in trials.

Opus Genetics (Nasdaq: IRD) has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for its gene therapy candidate OPGx-LCA5, targeting Leber Congenital Amaurosis (LCA) caused by LCA5 gene mutations. The designation is based on promising early data from an ongoing Phase 1/2 open-label, dose-escalation trial evaluating the therapy's safety and potential efficacy in patients with severe vision loss. The RMAT designation offers benefits including expedited development, early FDA interactions, and guidance on efficient development and manufacturing. Additionally, Opus has been invited to participate in the FDA's Initial Comprehensive Multidisciplinary RMAT Meeting and join the Chemistry, Manufacturing and Controls Development and Readiness Pilot program, further supporting their development strategy.

Opus Genetics (NASDAQ: IRD) has reported promising 12-month results from its Phase 1/2 study of OPGx-LCA5, a gene therapy for inherited retinal degeneration. The trial, involving three adult patients (ages 19, 26, and 34), demonstrated sustained benefits observed at six months persisting through one year. Key improvements included:

Patients showed enhanced object recognition in the Multi-Luminance orientation and Mobility Test (MLoMT), visual acuity improvements averaging 3.5 lines, and increased retinal sensitivity. The treatment was well-tolerated with no serious adverse events. Two additional adolescent patients have been treated with promising preliminary data.

The company plans to initiate a registration trial in 2026 following FDA discussions. Opus is also advancing its pipeline with six additional inherited retinal disease treatments, including the BEST-1 program scheduled for Phase 1/2 later this year.

Opus Genetics (Nasdaq: IRD) has reported encouraging one-month clinical data from its first pediatric patient treated with OPGx-LCA5 gene therapy in a Phase 1/2 trial for LCA5-related inherited retinal disease. The 16-year-old patient showed meaningful vision improvement and no drug-related adverse events after a single subretinal injection.

A second pediatric patient has been dosed, with the three-patient pediatric cohort expected to complete enrollment in Q2 2025. Initial data from all pediatric patients is anticipated in Q3 2025. The company previously reported positive six-month results in adult patients, with visual improvements observed in all three adult participants.

Following an FDA Type D meeting, Opus received feedback on its proposed registrational trial design. The company plans to conduct a single-arm, adaptive pivotal study with approximately 19 patients, using the multi-luminance orientation and mobility test (MLoMT) as the primary endpoint. The pivotal trial could potentially begin in Q1 2026.

Opus Genetics (Nasdaq: IRD) has filed definitive proxy materials for its 2025 Annual Meeting scheduled for April 30, addressing a contested board election. The company, formerly Ocuphire Pharma, underwent a significant transformation in 2023-2024, including:

- Appointment of Dr. George Magrath as CEO after terminating former CEO Mina Sooch
- Strategic shift from Ocuphire's legacy assets to gene therapy focus
- Acquisition of Legacy Opus Genetics in October 2024
- Recent completion of $21.5M capital raise led by Perceptive Advisors and Nantahala Capital

The board is urging stockholders to vote FOR all nine company nominees on the BLUE proxy card, opposing former CEO Mina Sooch's attempt to replace the majority of directors. The board also recommends approval of converting preferred stock to common stock to advance the company's gene therapy strategy for inherited retinal diseases (IRDs).

Opus Genetics (Nasdaq: IRD), a clinical-stage ophthalmic biotech company focused on gene therapies for inherited retinal diseases, has announced its participation in the upcoming RBC Capital Markets Ophthalmology Conference.

CEO Dr. George Magrath will lead a fireside chat on Thursday, April 3, 2025, from 12:15 p.m. to 12:45 p.m. ET. The conference will be held virtually, and company management will be available for one-on-one meetings with interested parties.

Investors can arrange meetings through their conference representatives or by emailing ir@ocuphire.com. A webcast link, if available, will be accessible on the Events page of Opus Genetics' Investor website.