Opus Genetics reported third-quarter results alongside progress across its gene-therapy pipeline for inherited retinal diseases. Its lead programs advanced toward later-stage testing, while a third program moved closer to clinical trials.
The company also said FDA approval of phentolamine ophthalmic solution 0.75% for presbyopia triggered a milestone payment to Opus. It expects its cash runway to extend into 2029.
Key details
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OPGx-BEST1: In the low-dose first cohort of its Phase 1/2 BIRD-1 trial, all five participants showed clinically meaningful improvement in visual function, and four showed structural improvement in the retina. Among the four participants evaluable for retinal sensitivity, three improved. The company reported a favorable safety profile, with no serious adverse events or dose-limiting toxicities.
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Opus said it has discussed a potential pivotal-trial endpoint with the FDA: improvement of at least 3 decibels at five or more prespecified locations on a retinal-sensitivity test, alongside a patient-reported outcome. This is a potential endpoint, not a final trial-design decision.
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The company completed dosing of 11 participants in the higher-dose second cohort. It expects to report topline results in the first quarter of 2027, including three-month data from that cohort and six-month data for three first-cohort participants with Best vitelliform macular dystrophy. Opus anticipates a potential pivotal Phase 3 trial could begin in the second half of 2027.
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OPGx-LCA5: The registrational Phase 3 trial is fully enrolled. Opus expects to begin dosing participants in the fourth quarter of 2026.
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OPGx-RDH12: The FDA cleared the program’s investigational new drug application, allowing clinical testing to proceed. Opus plans to begin dosing in a Phase 1/2 trial in the fourth quarter of 2026.
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Phentolamine ophthalmic solution 0.75%: FDA approval for presbyopia triggered a milestone payment to Opus. The announcement did not specify the payment amount.
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Cash and development plans: Opus expects to have cash to fund operations into 2029, which it says will support multiple upcoming clinical milestones and potential opportunities to obtain priority review vouchers. It did not provide further details about those voucher opportunities in the announcement.
The updates mark progress toward larger trials for OPGx-BEST1 and OPGx-LCA5, while OPGx-RDH12 joins the company’s clinical pipeline. The stated cash runway provides a timeframe for advancing those programs, though the announcement gives no quarterly financial figures here.