Opus Genetics (IRD) investor relations material

Strategic approach and pipeline overview

• Employs a portfolio strategy targeting multiple inherited retinal diseases (IRDs) with AAV gene therapies, enabling multiple data readouts and milestones across seven assets, two of which are in clinical trials.

• Focuses on rare diseases with regulatory advantages, streamlined timelines, and cost-effective development, leveraging validated science and delivery methods.

• Pipeline includes gene therapies for LCA5, BEST1, RHO, RDH12, MERTK, CNGB1, and NMNAT1, with worldwide rights retained.

• Commercial partnership for Phentolamine Ophthalmic Solution 0.75% provides non-dilutive funding and future royalty streams.

Lead clinical programs: OPGX-BEST1 and OPGX-LCA5

• OPGX-BEST1 targets BEST1-related retinal degenerative diseases, showing positive safety, tolerability, and biological activity in early Phase 1/2 results; three-month data from Cohort 1 expected mid-2026.

• OPGX-LCA5 addresses early-onset, severe hereditary retinal degeneration, with positive Phase 1/2 safety and efficacy in both adults and children; Phase 3 trial enrollment ongoing.

• Both programs have received multiple regulatory designations, including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy, with potential for Priority Review Voucher upon approval.

Clinical data and efficacy highlights

• OPGX-BEST1 demonstrated no ocular inflammation or treatment-related adverse events in the sentinel participant, with early signals of functional and structural improvement (12-letter BCVA gain, 23% CST decrease).

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

• Microperimetry and mobility tests indicated increased retinal sensitivity and improved navigation ability in treated eyes.