Ocular Therapeutix (OCUL) 2025 EuRetina presentation summary

Study background and rationale

• Diabetic retinopathy can progress to vision-threatening stages, and early anti-VEGF intervention reduces this risk, but is rarely used in early disease due to treatment burden.

• OTX-TKI, an intravitreal hydrogel with axitinib, is designed for durable treatment to reduce injection frequency.

Study design and methodology

• Phase 1 HELIOS trial randomized NPDR patients (DRSS 43 or 57, no CI-DME) 2:1 to OTX-TKI or sham, with monthly follow-up for one year.

• Retinal volumetric changes were measured using masked, machine-learning OCT analysis.

Efficacy and safety outcomes

• At week 48, 75% of OTX-TKI patients showed DRSS improvement, and none developed vision-threatening complications, compared to 37.5% in sham.

• No moderate or severe adverse events, ocular SAEs, or intraocular inflammation were reported in either group.