Opus Genetics (IRD) Investor presentation summary

Platform and opportunity

• Targeted, one-time AAV gene therapies focus on inherited retinal diseases with preserved retinal structure and measurable functional outcomes.

• Pipeline includes LCA5, BEST1, RDH12, MERTK and RHO, with CNGB1, NMNAT1 and an undisclosed IRD program also listed.

• Programs use subretinal delivery and have potential first-mover positioning; all gene therapy programs may qualify for Priority Review Vouchers.

• Estimated prevalence across select global markets includes ~30,900 RDH12, ~30,200 RHO, ~21,960 MERTK and ~3,240 LCA5 patients.

Clinical catalysts and funding

• Cash balance of $89 million; runway extends into 2029 and is expected to fund five clinical programs through critical inflection points.

• Four clinical data readouts are targeted for 2027: BEST1, LCA5, RDH12 and MERTK; timelines are estimates and may change.

• RDH12 study initiation is targeted for Q4 2026 and MERTK for Q1 2027; BEST1 Cohort 2 results and pivotal study start are targeted for 2027.

• Phentolamine ophthalmic solution 0.75% is a partnered commercial program; an SNDA for presbyopia had a September 2026 PDUFA date.

OPGX-BEST1

• Low-dose Cohort 1 was well tolerated in 5/5 participants, with no serious adverse events, dose-limiting toxicities or intraocular inflammation.

• Structural improvements occurred in 4/5 participants; all 5/5 improved in at least one functional measure, including 3/4 evaluable participants on microperimetry.

• Improvements co-localized with structural changes; earlier-stage participants showed the greatest structural and functional gains.

• Cohort 2 enrollment reached 8 participants; dosing completion was targeted for Q4 2026, with Cohort 1 six-month and Cohort 2 three-month data expected in Q2 2027.

• FDA aligned on a potential pivotal endpoint of ≥3 dB microperimetry improvement in ≥5 prespecified loci, with a randomized controlled trial and patient-reported outcome.