Opus Genetics (IRD) 44th Annual J.P. Morgan Healthcare conference summary

Key milestones and pipeline updates

• Anticipates pivotal data readout for BEST1 gene therapy in mid-year, with initial data at a medical conference in Q1 and multiple additional gene therapy assets entering the clinic this year.

• LCA5 pivotal trial is ongoing, with all six treated patients showing clinically meaningful vision improvements; pediatric enrollment is underway, and data readout is expected in 2027.

• Commercial asset Ryzumvi (phentolamine ophthalmic solution) is partnered with Viatris, with a supplemental NDA for presbyopia submitted and potential approval expected this year.

• Pipeline includes seven gene therapy programs, focusing on efficient, quick trials for inherited retinal diseases with high probability of success.

• Company leverages grant funding and patient group partnerships to advance preclinical assets.

Scientific approach and differentiation

• Focuses on diseases with normal retinal structure but impaired function, enabling rapid efficacy assessment through gene augmentation.

• Uses well-characterized AAV vectors and established delivery methods, reducing clinical risk and manufacturing complexity.

• Subretinal delivery targets a small number of retinal cells, minimizing systemic exposure and side effects.

• Relies on structure-function association, selecting indications where restoring a missing gene can quickly reverse pathology.

• Differentiation includes leveraging expertise from Dr. Jean Bennett and focusing on translational efficiency from animal models to humans.

Clinical data and safety profile

• BEST1 phase I/II trial will assess safety, target engagement (via electrophysiology and OCT), and functional vision improvements.

• LCA5 program demonstrated rapid and durable improvements in visual acuity, FST, and mobility tests, with effects seen as early as one month and lasting at least 18 months.

• No serious adverse events reported in BEST1 or LCA5 trials; low vector doses and robust non-clinical safety data support a favorable safety profile.

• Pediatric patients in LCA5 trials show even greater vision gains, supporting early intervention.

• Regulatory designations include rare pediatric, orphan, regenerative, and RMAT status.