Opus Genetics (IRD) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Pipeline and upcoming milestones

• LCA5 pivotal-trial readout expected next year; potential BLA filing next year if data hold.

• BEST1 cohort two adds earlier-stage patients at ~3x cohort-one dose; additional cohort expected in Q1.

• RDH12 patient dosing imminent; FST movement within months will serve as a target-engagement guidepost. MERTK treatment is also expected to start imminently.

• BEST1, RDH12 and MERTK data could read out next year or early 2028, alongside the pivotal LCA5 readout next year.

BEST1 clinical findings and trial design

• In cohort one, at least two BVMD patients showed vitelliform-material removal and marked sensitivity gains in the same retinal areas.

• Microperimetry improvement at vitelliform loci was ~4.5 dB; cohort-one subretinal fluid did not change, consistent with advanced disease and limited viable photoreceptors.

• Cohort two results consistent with cohort one across 11 additional patients, including 10 with BVMD, could de-risk phase III; consistency and low variability are the priority.

• Response appears likelier with active vitelliform material in treatable regions and without confounding pathology; registration enrollment is expected to enrich for such patients.

• The primary endpoint is a responder analysis requiring improvement in microperimetry and PGIS; microperimetry loci must be prespecified before surgery, with at least five selected.

Market opportunity and launch planning

• A third-party, 150-specialist U.S. assessment estimated 23,600 Best disease patients, versus an earlier estimate of 8,400.

• A targeted launch approach is in planning, leveraging digital technologies and an estimated 20–30 total FTEs.

• LCA5 affects just under 200 patients; a potential priority review voucher auction is expected to be pursued in 2028.

• RDH12 and MERTK each have an estimated ~15,000–20,000 patients across the MENA region; MERTK is sponsored in Abu Dhabi by the Department of Health.