Opus Genetics reports Q3 2026 results, multiple IRD gene therapy milestones
Cohort 2 dosing wraps in the BEST1 program, the LCA5 Phase 3 trial is fully enrolled, and an RDH12 IND clearance adds a third clinical-stage gene therapy as cash runway extends into 2029.
Opus Genetics, Inc. (Nasdaq: IRD) reported financial results for the third quarter ended September 30, 2026, alongside a string of pipeline updates on November 10, 2026.
For OPGx-BEST1, the company said it saw clinically meaningful improvement in visual function across all five participants in low-dose Cohort 1, with retinal sensitivity improvement by microperimetry in 75% of evaluable participants, and aligned with the FDA on a potential pivotal endpoint of at least 3 dB microperimetry improvement in at least 5 prespecified loci alongside a patient-reported outcome.1 The company finished dosing all 11 participants in the high-dose Cohort 2, and said topline data, including 3-month Cohort 2 results and 6-month data from the three Cohort 1 participants with Best vitelliform macular dystrophy, is expected in the first quarter of 2027.1
In the OPGx-LCA5 program, the registrational Phase 3 trial is fully enrolled with 8 participants, with dosing expected to start in the fourth quarter of 2026.1 The company also cited Phase 1/2 data shown at Retina in Rio 2026, pointing to durable gains in visual acuity and light sensitivity in adults out to 24 months, with early but positive signals in pediatric participants through six months.1
A third program, OPGx-RDH12, cleared a regulatory hurdle: the FDA cleared the IND for RDH12-associated Leber congenital amaurosis, with a Phase 1/2 trial expected to begin dosing in the fourth quarter of 2026.1
Separately, the FDA approved an expanded use of the company's approved eye drop: the supplemental NDA for phentolamine ophthalmic solution 0.75%, marketed as RYZUMVI, was approved for treating presbyopia in adults, which triggers a milestone payment to Opus from its commercial partner Viatris Inc.1
On the balance sheet, Opus held $89.0 million in cash and equivalents as of September 30, 2026, and said that combined with potential future funding under its note purchase agreement with Oberland Capital Management, it expects cash resources to fund operations into 2029.1
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