Opus Genetics (IRD) Investor presentation summary

Strategic approach and pipeline overview

• Employs a portfolio strategy targeting seven IRD AAV gene therapy assets, with two already in clinical trials and five more expected by 2027.

• Focuses on rare inherited retinal diseases (IRDs) with validated science, streamlined timelines, and cost-effective development.

• Leverages rare disease regulatory advantages for potentially faster approvals and non-dilutive funding.

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

• Significant market opportunity exists across the U.S., EU, Middle East/North Africa, and China.

Lead programs: OPGX-BEST1 and OPGX-LCA5

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

• BEST1 mutations account for ~22,000 patients in select global markets and ~3.5% of all IRDs; the therapy aims to restore retinal ion homeostasis.

• OPGX-LCA5 addresses early-onset, severe hereditary retinal degeneration, with positive safety and efficacy results in both adult and pediatric Phase 1/2 participants.

• LCA5 patients retain central photoreceptors into adulthood, providing a broad therapeutic window for gene replacement.

• Both programs have received multiple regulatory designations, including Rare Pediatric Disease, Orphan Drug, and RMAT, and are eligible for Priority Review Voucher upon BLA approval.

Clinical data and outcomes

• OPGX-BEST1 Phase 1/2 sentinel participant showed a 12-letter gain in visual acuity and a 23% decrease in central subfield thickness over three months, with reduced intraretinal fluid.

• OPGX-LCA5 demonstrated improvement in visual acuity and full-field stimulus test in 5 of 6 participants, with effects lasting up to 18 months in adults and 6 months in pediatric patients.

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

• Microperimetry data showed increased sensitivity and fixation movement toward the fovea in treated eyes.

• Both programs were well-tolerated, with no serious adverse events or dose-limiting toxicities reported.