Ocular Therapeutix (OCUL) 2025 Retina World Congress presentation summary

Study design and methodology

• Multi-center, double-masked, randomized, parallel group study evaluated OTX-TKI in moderately-severe to severe NPDR without CI-DME, with 2:1 randomization (OTX-TKI:Sham) and monthly visits over 48 weeks.

• Primary outcome was safety and tolerability; secondary outcomes included DRSS changes, rescue therapy, BCVA, and CSFT changes.

• Advanced OCT feature analysis used machine learning for segmentation and feature extraction, validated by certified readers.

Efficacy and clinical outcomes

• OTX-TKI patients showed greater reductions in retinal volume from baseline at week 48 (-0.92 mm³) compared to sham (-0.16 mm³).

• Sustained control of intraretinal fluid was observed in OTX-TKI patients, with a mean change of -0.017 mm³ at week 48 versus +0.042 mm³ for sham.

• No OTX-TKI patients developed PDR or CI-DME through 48 weeks, while 37.5% of sham patients did.

• OTX-TKI patients demonstrated stabilization of EZ integrity, with less attenuation compared to sham.

Case examples and quantitative findings

• Sham patient developed CI-DME by week 48, with CST increase of +48 μm and macular volume increase of +0.89 mm³.

• OTX-TKI patient showed a 1-step DRSS improvement, CST decrease of -31 μm, and macular volume decrease of -2.03 mm³ by week 48.

• Quantitative analysis confirmed consistent improvement in fluid metrics and EZ integrity in OTX-TKI group.