Clinical Read2026-06-30

Phentolamine ophthalmic solution - (LYNX-3)

Opus Genetics Inc.IRD
Indication
Decreased visual acuity under dim (mesopic) light conditions
Stage
phase3
Event
Topline Data
Details

May 7, 2026: Opus Genetics, Inc. (NASDAQ: IRD) presented new clinical and preclinical gene therapy data at ARVO 2026 (Denver).

OPGx-LCA5 (Leber congenital amaurosis type 5): In an ongoing Phase 1/2/3 pediatric study, 6‑month results after a single subretinal injection showed the therapy was well tolerated with no dose-limiting toxicities; adverse events were mild/expected/resolved. Reported outcomes included >30‑fold improvements in cone sensitivity in all treated patients, visual acuity improvement in treated vs baseline and untreated control eyes, supportive dark-adapted pupillary light response changes (amplitude/latency thresholds), improved orientation and mobility test performance, and patient-reported functional vision improvements.

OPGx-BEST1 (ARB and BVMD due to BEST1 mutations): Preliminary 3‑month data from an adult in a Phase 1b/2a study showed no ocular inflammation or treatment-related AEs, up to 12-letter visual acuity improvement, and ~23% reduction in central subfield thickness. The company completed enrollment in Cohort 1 of its Phase 1/2 study and expects to announce 3‑month topline Cohort 1 data in September 2026. It also reported development of potency/expression assays to support manufacturing consistency and regulatory readiness.

OPGx-RHO / mutation-independent RHO approach (adRP models): Nonclinical canine and swine studies reported NOAEL establishment, retinal structure/function preservation with dose-dependent rescue, minimal effective dose and durability signals, and potential interocular vector transfer.