Opus Genetics (IRD) Chardan's 10th Annual Healthcare Conference summary

Presbyopia approval and economics

• FDA approved phentolamine for presbyopia; approval brings a $10M milestone expected this quarter, up to $130M in total milestones, and royalties starting in the lower teens and scaling higher.

• The drop is the first approved presbyopia treatment without a retinal-detachment warning; it avoids ciliary-body engagement and is positioned to reduce night glare and dimming concerns linked to strong miotics.

LCA5 pivotal trial

• The eight-patient pivotal trial uses each patient’s six-month pretreatment period as control; primary endpoint is the proportion achieving >7 dB improvement on central-16 microperimetry.

• Prior study average improvement was 10.5 dB; dosing is underway and expected to conclude soon, with top-line data by end of 2027.

• Addressable population is estimated at 150–200 patients, most seen at the University of Pennsylvania; the program could qualify for a priority review voucher, and management expects high penetration based on safety and efficacy.

BEST1 program

• BEST1 mutations cause retinal degenerative diseases including BVMD and ARB; central photoreceptors may remain viable for decades, providing a broad therapeutic window. BIRD-1 is an adaptive, open-label, dose-escalation Phase 1/2 study of subretinal OPGX-BEST1.

• Low-dose Cohort 1 treated five participants at 1.5×10⁹ vg/eye; treatment was well tolerated, with no serious adverse events, dose-limiting toxicities, intraocular inflammation or treatment-related systemic adverse events.

• All five Cohort 1 participants improved on at least one visual-function measure, four of five had structural improvement, and microperimetry improved in three of four participants.

• Cohort 2 enrolled 11 patients versus five planned; data and a potential pivotal-trial transition update are expected in Q2 2027. FDA-aligned pivotal endpoint is microperimetry, including ≥3 dB improvement across ≥5 prespecified loci anchored to a patient-reported outcome.

• U.S. symptomatic BEST1 population estimated at 23,600 versus 8,400 identified through genetic testing; a 150-provider survey supported the larger treatable-patient estimate. Phase III and commercial drug supply are expected early next year, with CMC readiness driving timing.