Ocular Therapeutix (OCUL) American Society of Retina Specialists presentation summary

Study design and methodology

• Phase 1, multi-center, double-masked, randomized study evaluated OTX-TKI in moderately-severe to severe NPDR without center-involved DME.

• Patients were randomized 2:1 to receive a single OTX-TKI hydrogel injection or sham, with monthly follow-up for one year.

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

• Quantitative leakage was assessed using ultra-widefield fluorescein angiography and machine learning-based analysis.

OTX-TKI technology and mechanism

• OTX-TKI is a bioresorbable hydrogel delivering axitinib, a highly potent VEGFR2 inhibitor, via sustained release.

• Axitinib is ~100 times more potent for VEGFR2 than sunitinib or vorolanib, with high selectivity for VEGF receptors.

• Single intravitreal injection is designed for 6–12 months of drug release.

Efficacy results: retinal leakage reduction

• OTX-TKI group showed durable reduction in total retinal vascular leakage area and leakage index over 12 months, compared to sham.

• Peripheral leakage parameters improved in OTX-TKI eyes, while sham eyes worsened.

• Sustained reduction in leakage was also observed in the posterior pole for OTX-TKI eyes.