Opus Genetics (IRD) Corporate presentation summary

Cohort 1 safety and early efficacy

• OPGX-BEST1 was well tolerated in all 5 participants, with no serious adverse events, dose-limiting toxicities, intraocular inflammation, or treatment-related systemic AEs; ocular treatment-related AEs were mild or moderate.

• Structural improvements were reported in 4/5 participants: vitelliform material decreased in 2/3 BVMD participants and intraretinal fluid decreased in 2/2 ARB participants.

• All 5 participants improved on at least one functional measure; microperimetry improved in 3/4 evaluable participants, BCVA in 3/5, LLVA in 2/5, and contrast sensitivity in 2/5.

• Functional gains co-localized with structural improvements; the earlier-stage participant showed the largest functional gains, while advanced atrophy and scarring limited improvement in one BVMD participant.

Trial design and regulatory path

• BIRD-1 is an adaptive, open-label Phase 1/2 dose-exploration study in adults with BVMD or ARB; Cohort 1 received 1.5×10⁹ vg/eye and Cohort 2 is evaluating 4.5×10⁹ vg/eye.

• Cohort 1 included 5 participants: 2 ARB participants with 6-month follow-up and 3 BVMD participants with 3-month follow-up; Cohort 2 enrollment reached 8 participants, above the planned 5.

• The August 2026 FDA Type C meeting aligned on a potential pivotal endpoint: ≥3 dB improvement in ≥5 prespecified loci in the treated retinal transition zone, alongside a randomized controlled trial and patient-reported outcome.

• 75% of evaluable Cohort 1 participants met the microperimetry threshold; 6-month low-dose and 3-month high-dose Cohort 2 data are expected in Q2 2027, with pivotal dosing planned for 2027.

Market opportunity and program outlook

• BEST1-related disease is estimated to affect 23,600 symptomatic patients in the U.S. and 45,400 globally; BVMD accounts for an estimated 23,200 U.S. patients and ARB for 400.

• Cash runway was projected into 2029, supporting five clinical programs through multiple inflection points; four clinical data readouts are expected in 2027 across BEST1, LCA5, RDH12, and MERTK.

• Other gene therapy programs span LCA5, RDH12, MERTK, RHO, NMNAT1, and CNGB1; all programs have potential to qualify for a Priority Review Voucher.