Opus Genetics (IRD) investor relations material

Pipeline overview and program updates

• Advancing seven AAV gene therapy assets for inherited retinal diseases, targeting bestrophinopathies, Leber's congenital amaurosis (LCA), and retinitis pigmentosa, with a platform approach enabling parallel development and capital efficiency.

• Two lead programs (BEST1 and LCA5) are in clinical trials, with five more expected in the clinic by 2027.

• LCA5 phase III enrollment ongoing; BEST1 phase I-II cohort 1 completed; RDH12, MERTK, and RHO programs entering clinic over the next 12-18 months, with up to four clinical trial readouts expected in 2027.

• Multi-asset pipeline and first-mover advantage in several indications, with meaningful ex-U.S. market opportunities.

• Cash runway extends into 2029, supporting five clinical programs through multiple inflection points.

Clinical trial data and development milestones

• LCA5 gene therapy shows significant improvements in visual acuity and functional endpoints in both adult and pediatric cohorts, with meaningful patient-reported outcomes.

• LCA5 Phase 1/2 trial 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.

• BEST1 program completed first cohort enrollment; microperimetry and imaging endpoints are being used to correlate structural and functional improvements.

• Early BEST1 results show favorable safety (no ocular inflammation or adverse events) and initial efficacy (12-letter gain in visual acuity, 23% reduction in central subfield thickness).

• Preclinical data for RDH12, MERTK, and RHO demonstrate restoration of enzyme/protein function and photoreceptor preservation in relevant animal models.

R&D strategy and innovation priorities

• Focus on diseases with preserved retinal structure, rapid proof-of-concept potential, and availability of animal/cell models.

• Use of mutation-independent, single AAV constructs for silence-and-replace strategies (notably for RHO).

• Data-driven dose exploration and adaptive clinical trial designs, leveraging independent data monitoring committees.

• Streamlined, cost-effective development leverages rare disease regulatory advantages and a validated delivery approach.