Opus Genetics (IRD) Corporate presentation summary

Strategic approach and pipeline overview

• Employs a portfolio strategy targeting seven IRD AAV gene therapy assets, enabling multiple data readouts and milestones across indications.

• Focuses on cost-effective development and streamlined timelines, leveraging rare disease regulatory advantages for potentially faster approvals.

• Two lead programs, OPGX-BEST1 and OPGX-LCA5, are in clinical trials, with five gene therapy programs expected in the clinic by 2027.

• Holds worldwide rights to all gene therapy programs and has a partnered commercial program for presbyopia.

Lead clinical programs: OPGX-BEST1 and OPGX-LCA5

• OPGX-BEST1 targets BEST1-related retinal degenerative diseases, showing favorable safety and early efficacy in Phase 1/2 with no ocular inflammation or dose-limiting toxicities.

• Enrollment for OPGX-BEST1 Phase 1/2 is complete, with full Cohort 1 three-month data expected in September 2026.

• OPGX-LCA5 addresses early-onset, severe hereditary retinal degeneration, demonstrating visual acuity and functional improvements in both adult and pediatric participants.

• OPGX-LCA5 has received multiple regulatory designations and is positioned for rapid advancement, with Phase 3 dosing planned for Q4 2026.

Clinical and preclinical data highlights

• OPGX-BEST1 demonstrated a 12-letter gain in BCVA and a 23% decrease in central subfield thickness in the sentinel participant over three months.

• Canine models of ARB treated with OPGX-BEST1 showed reversal of lesions and microdetachments, supporting clinical translation.

• OPGX-LCA5 showed improvement in visual acuity and full-field stimulus test in 5 out of 6 participants, with durable effects up to 18 months in adults.

• Treated eyes in OPGX-LCA5 trials identified more objects in virtual reality mobility tests, indicating functional vision gains.