Opus Genetics (IRD) Investor update summary

Clinical trial results and product updates

• Positive top-line results from VEGA-3 and LYNX-2 phase III trials for phentolamine ophthalmic solution 0.75% met primary endpoints, showing statistically significant and sustained improvements in near vision for presbyopia and dim light disturbances.

• VEGA-3 demonstrated a 27.2% responder rate for ≥15-letter improvement in binocular DCNVA at 12 hours post-dose on Day 8, compared to 11.5% for placebo, with efficacy maintained over 6 weeks and no evidence of tachyphylaxis.

• LYNX-2 achieved its primary endpoint for improved mesopic low contrast visual acuity in post-keratorefractive patients, with significant patient-reported outcomes and no new safety signals.

• Significant patient-reported functional benefits were observed at Days 3, 8, and Week 6, including satisfaction with near vision upon awakening.

• FDA granted Fast Track designation for phentolamine in treating chronic night driving impairment in keratorefractive patients.

Safety and tolerability

• Safety profile was consistent with previous trials, with no treatment-related serious adverse events.

• Most common adverse events were mild, including conjunctival hyperemia, instillation site irritation, dysgeusia, and headache in 2.6% of patients.

• Patients will continue to be monitored for long-term safety over 48 weeks.

Pipeline and regulatory milestones

• Plans to submit an FDA application for phentolamine in presbyopia in the second half of 2025, with LYNX-3 trial initiation for dim light disturbances also expected this year.

• Eligible for up to $130 million in milestone payments and tiered royalties from Viatris partnership.

• Gene therapy programs target rare inherited retinal diseases using a patented AAV vector approach, aiming for multiple product approvals over the next five years.

• OPGX-LCA5 program received RMAT designation from the FDA, with initial pediatric data expected in Q3 2025.

• OPGX-BEST1 IND filing and phase I/II trial planned for this year; additional preclinical work ongoing for other IRD targets.