New data from the phase 1/2 HELIOS trial of intravitreal axitinib hydrogel (OTX-TKI; Ocular Therapeutics) were presented at the 2025 Association for Research in Vision and Ophthalmology meeting. The post hoc analysis investigated the impact of the tyrosine kinase inhibitor OTX-TKI on macular fluid metrics in patients with nonproliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CI-DME).

Using masked, machine learning-assisted spectral-domain OCT analysis, the study evaluated volumetric retinal changes in 21 eyes (13 OTX-TKI, 8 sham). Over 48 weeks, OTX-TKI–treated eyes demonstrated consistently greater reductions in mean retinal-RPE volume and central subfield thickness compared to sham, with no rescue anti-VEGF therapy administered. At week 48, the mean retinal-RPE volume decreased by 0.92 mm³ in the OTX-TKI group vs 0.16 mm³ in sham; central subfield thickness declined by 15.4 µm vs a 4.7 µm increase in the sham group. Additionally, intraretinal fluid volume decreased in the OTX-TKI group but increased in sham.

“The results of the HELIOS analysis support the sustained therapeutic signal from treatment with a single OTX-TKI injection,” said trial investigator Justis Ehlers, MD, of Cole Eye Institute at the Cleveland Clinic, who presented the data. “Advanced imaging analysis demonstrates both durable volumetric reduction of intraretinal fluid and improvements in quantitative panretinal leakage. These findings provide evidence that OTX-TKI could be a potential long-lasting therapeutic for addressing nonproliferative diabetic retinopathy. Additional data will be needed to further confirm these findings.”

The findings further suggest that OTX-TKI may reduce subclinical retinal fluid and potentially slow NPDR progression. The results support further investigation of OTX-TKI as a long-acting treatment to stabilize retinal structure and mitigate vision-threatening complications in NPDR. RP