Ocular Therapeutix saw its stock slide steeply even after announcing that its AXPAXLI therapy achieved the primary endpoint in the SOL-1 Phase 3 superiority trial for wet age-related macular degeneration (wet AMD).
The company reported that AXPAXLI was superior to aflibercept in preserving vision at Week 36. In the SOL-1 cohort, 74.1% of patients treated with AXPAXLI maintained vision at that timepoint, corresponding to a 17.5% risk difference versus the aflibercept arm, with a p-value of 0.0006. The firm also disclosed that 65.9% of AXPAXLI-treated subjects maintained vision at Week 52, and 68.8% of patients remained rescue-free at Week 52.
Ocular Therapeutix characterized AXPAXLI as generally well-tolerated in the trial and noted there were no treatment-related ocular serious adverse events reported. Based on the SOL-1 dataset, the company intends to pursue discussions with the U.S. Food and Drug Administration that would inform a potential New Drug Application submission. If approved, AXPAXLI could represent the first tyrosine kinase inhibitor to reach the market for wet AMD therapy.
Despite the trial outcome and the safety statement, investors reacted negatively, sending Ocular Therapeutix shares down 35% on Tuesday. The company noted that market participants may be focusing on elements of the data or potential regulatory hurdles, which it said appeared to weigh on the share price.
Ocular Therapeutix is also running a complementary SOL-R Phase 3 non-inferiority trial; topline results from that study are expected in the first quarter of 2027. The company plans to present detailed SOL-1 findings at the 49th Macula Society Annual Meeting later this month.
Context and next steps
The SOL-1 readout provides statistically significant evidence of AXPAXLI's superiority on the primary endpoint at Week 36 and shows additional measures of maintained vision and rescue-free status at Week 52. The company has framed the safety profile as acceptable based on the absence of treatment-related ocular serious adverse events in the trial.
Management intends to engage with regulators prior to any NDA filing, and the outcome of those discussions, together with the pending SOL-R topline data, will shape the regulatory and commercial pathway for AXPAXLI.