IRD: Updates Before Entering Quiet Period

Research evidence excerpt

IRD: Updates Before Entering Quiet Period

FLASH NOTE

July 9, 2026

Debjit Chattopadhyay, Ph.D. debjit.chattopadhyay@guggenheimpartners.com IRD: Updates Before Entering Quiet Period

212 823 6584

Moritz Reiterer, Ph.D. Key Message: We caught up with the mgmt before the company enters a quiet period.

moritz.reiterer@guggenheimpartners.com Key updates for OPGx-BEST1: (1) Cohort 1 (low dose, 1.5E9 vg/eye) 3-month data likely

212 372 6368 between 9/7 and 9/11; (2) 20% to 30% reduction in the subretinal fluid in Cohort 1 is

Hannah Wei required to advance to Cohort 2. While up to 30% fluid reduction is clinically meaningful,

hannah.wei@guggenheimpartners.com the internal hurdle to advance the program into a pivotal study is 100% reduction in

212 518 5896 the majority of patients. Given the high internal hurdle, we think dose escalation is the

Ry Forseth, Ph.D. likely outcome. OPGx-LCA5 program achieved regulatory alignment on primary efficacy

ry.forseth@guggenheimpartners.com endpoint (mean improvement of at least 7 dB in retinal sensitivity across the central

212 518 9226 16 test loci). The BLA could be submitted with 6 month efficacy data and preliminary

results support this timeline, in our opinion. Mgmt does not anticipate the requirement

for microperimetry in pivotal trial will restrict label. Note, 7 / 8 participants have already

IRD been enrolled. Figure 1 - Preliminary data support 6-month BLA submission for the OPGx-LCA5 Opus Genetics, Inc.

Sector: Biotechnology program. (IRD)

Improvement post-treatment in study eye of patient 01-06 (C, D), no changes in control eye (A, B). Sensitivity

Company Update gainsevaluated,reachedexceptnormalone limitsadditionalin 3 monthsrow of points(D). Thiswill methodologybe used. is identical to how primary efficacy endpoint will be