Ocular Therapeutix (OCUL) Study update summary

Study design and patient population

• SOL-1 was a phase III, FDA-aligned, superiority trial evaluating AXPAXLI (OTX-TKI) in wet AMD, enrolling 344 subjects with high baseline vision and anti-VEGF dependency.

• Subjects were randomized after two aflibercept loading injections, with most having gained 10+ letters, representing a population expected to lose vision over time.

• The trial included a comparator arm with the same dosing schedule as the investigational drug, ensuring unbiased efficacy and durability assessment.

• The study was conducted under a Special Protocol Assessment (SPA) agreement with the FDA, confirming alignment on design, endpoints, and analysis.

Efficacy results

• AXPAXLI met the primary endpoint: at week 36, 74.1% maintained vision (loss <15 ETDRS letters) vs. 55.8% for aflibercept (p=0.0006).

• At week 52, 65.9% of AXPAXLI subjects maintained vision after a single injection, with statistical significance over aflibercept (p<0.0001).

• Fewer AXPAXLI subjects lost ≥10 letters at both week 36 and 52 compared to aflibercept, with strong secondary endpoint results.

• AXPAXLI showed tighter anatomic control: 55.9% maintained central subfield thickness within 30 microns of baseline at week 36 vs. 37.8% for aflibercept.

• High rescue-free rates: 74.7% at week 36 and 68.8% at week 52, with delayed time to first rescue compared to aflibercept.

Durability and clinical implications

• AXPAXLI demonstrated sustained VEGF suppression and disease control up to 12 months in 66% of treated subjects.

• Durability was supported by the hydrogel platform, enabling predictable drug release and synchronized resorption.

• The therapy offers a potential 9-12 month treatment horizon from a single injection, unprecedented in an FDA-aligned phase III trial.

• Clinicians anticipate broad use in both treatment-naive and maintenance populations, with potential to reduce injection burden and improve adherence.