Ocular Therapeutix (OCUL) Study result summary

Study overview and design

• SOL-1 was a large, randomized, double-masked, well-controlled phase 3 superiority trial evaluating a single injection of AXPAXLI (0.45 mg) versus aflibercept (2 mg) in 344 newly diagnosed wet AMD patients after an 8-week loading phase, with randomization after peak response to aflibercept.

• The primary endpoint was the proportion of subjects maintaining vision (less than 15 ETDRS letter loss) at week 36, with a key secondary endpoint at week 52, and a superiority design agreed upon with the FDA.

• The trial population was specifically selected to lose vision, making the bar for success high and the results clinically meaningful.

• Secondary and exploratory endpoints included anatomic control (CSFT within 30 microns of baseline), rescue-free rates, and application of SOL-R rescue criteria.

• The study was conducted under a Special Protocol Assessment (SPA) with the FDA, with masking through week 104, re-dosing at weeks 52 and 76, and designed to support a potential superiority label and regulatory submission.

Key efficacy results

• At week 36, 74.1% of AXPAXLI subjects maintained vision versus 55.8% for aflibercept, a statistically significant risk difference of 17.5% (p=0.0006).

• At week 52, 65.9% of AXPAXLI subjects maintained vision versus 44.2% for aflibercept, a 21.1% risk difference (p<0.0001).

• 55.9% of AXPAXLI subjects maintained CSFT within 30 microns at week 36, versus 37.8% for aflibercept (risk difference 17.1%, p=0.0013).

• Rescue-free rates for AXPAXLI were 80.6% at week 24, 74.7% at week 36, and 68.8% at week 52, consistently higher than aflibercept.

• Applying SOL-R rescue criteria, 77.1% of AXPAXLI subjects would have been rescue-free at week 24.

Safety and tolerability

• AXPAXLI was generally well-tolerated, with no observed treatment-related ocular or systemic serious adverse events through week 52.

• No cases of endophthalmitis, retinal vasculitis, retinal detachment, implant migration, or intraocular inflammation were reported in the AXPAXLI arm.

• Ocular adverse events included vitreous floaters (12.4%), cataract (7.1%), conjunctival hemorrhage (6.5%), and mild to moderate iritis and uveitis (1.8% each), all resolving with or without treatment.

• Non-ocular adverse events were similar between arms, with no new safety signals identified.

• More than 95% of patients reached the week 52 durability assessment, with no meaningful discontinuations after repeat dosing.