FDA Alignment On LCA5 Ph3 Design Supports On Track Dosing Initiation In 4Q26

Research evidence excerpt

FDA Alignment On LCA5 Ph3 Design Supports On Track Dosing Initiation In 4Q26

Quick Note

July 6, 2026 Opus Genetics, Inc. (IRD)

Rating: FDA Alignment On LCA5 Ph3 Design Supports On TrackOUTPERFORM Dosing Initiation In 4Q26Price:

$4.23

12-Month Price Target: We see the alignment with the FDA on the design of the registrational Ph3 study

$10.00 of OPGx-LCA5 as an important milestone achieved by Opus. Importantly, the

finalization of the study size (only eight patients) and the possibility of a BLA

submission on primary endpoint analysis at six months confirm the FDA's strong

Analysts support for an expedited pathway, which we believe is based on the agency's

Yun Zhong, Ph.D. recognition of an urgent unmet medical need as well as the promising data from(917) 608-0166

yun.zhong@wedbush.com the Ph1/2 study. Opus reported that seven patients have already been enrolled

into the six-month run-in period, which we believe has exceeded management's

prior expectation to complete all patient enrollment before dosing initiation in

4Q26. Overall, we believe that the regulatory progress bodes well for OPTGx-LCA5

program's clinical success, and that positive data from the Ph3 study will provide

important evidence for Opus' overall inherited retinal disease pipeline, which now

includes five programs that management highlighted at the recent R&D Science

Forum to target a combined significant patient population (approximately 23,000

in the U.S.) (note).

Ph3 Patient Number Suggests Highest Efficiency. Recall when Opus announced

positive FDA feedback on the Ph3 plan for OPGx-LCA5 in Nov. '25 (note),

management had planned to leverage an LCA5 patient registry for patient

identification with the aim to enroll "as few as eight patients", so the final Ph3

size suggests the highest efficiency.