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

Key milestones and pipeline updates

• Anticipates pivotal year with BEST1 mid-year data readout and ongoing pivotal LCA5 trial; multiple additional assets expected to enter the clinic in 2024.

• Commercial asset Ryzumvi, partnered with Viatris, is marketed for dilation reversal; sNDA for presbyopia submitted, with potential approval expected this year.

• BEST1 phase I/II trial ongoing, targeting 9,000 US patients; mid-year data to focus on safety, target engagement, and functional improvement.

• LCA5 pivotal trial enrolling, with all six treated patients showing clinically meaningful vision improvements; pediatric cohort expected to amplify results.

• At least two additional gene therapy programs planned to enter the clinic in 2024.

Scientific approach and differentiation

• Focuses on gene augmentation for diseases with normal retinal structure but impaired function, enabling efficient, quick trials and high probability of success.

• Utilizes well-characterized AAV vectors and known promoters, leveraging expertise from Dr. Jean Bennett for translational accuracy.

• Subretinal delivery allows precise, localized dosing with negligible systemic exposure and well-characterized ocular safety profile.

• Programs selected for clear structure-function association, enabling rapid efficacy assessment and biologic impact.

• Differentiation includes de-risked clinical approach, efficient manufacturing, and focus on development over discovery.

Clinical data and safety

• BEST1 program aims to demonstrate improvement in electrophysiology, OCT imaging, and visual function; logical endpoints allow for clear assessment.

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

• Both lead programs have shown no serious adverse effects to date, supported by extensive non-clinical safety data and low vector doses.

• Microperimetry and other functional tests confirm robust biological activity and safety across endpoints.