Justis P. Ehlers, MD, on the HELIOS trial for NPDR

Note: Captions are generated with the assistance of AI.

Justis Ehlers, MD, spoke with Ophthalmology Times to share key findings from his recent presentation at the 2025 Retina Soceity meeting, which was held in Chicago, Illinois.

Ehlers shared data from the HELIOS phase 1 trial, which investigated a novel treatment for non-proliferative diabetic retinopathy (NDPR) without center-involving diabetic macular edema. The study compared a sham group with a group treated with a single injection of OTX-TKI (axitinib), a tyrosine kinase inhibitor with a sustained release platform potentially allowing dosing every 6 to 12 months.

The primary goal of the phase 1 trial was to assess safety, which showed no safety signals or signs of intraocular inflammation. The presentation highlighted a post-hoc analysis examining retinal vascular parameters, leakage, and fluid dynamics. Using spectral domain OCT, researchers found that the sham group trended towards increased fluid, while the OTX-TKI group demonstrated sustained fluid reduction throughout the study. Quantitative ultra-wide field angiography revealed progressive leakage in the sham group, contrasting with significant leakage reduction in the OTX-TKI group.

A notable finding was the sustained leakage reduction over the entire 12-month study period, which differed from typical anti-VEGF trials where reduction is often short-lived. The treatment showed a pan-retinal effect, with consistent results across different retinal regions.

The researchers emphasized that non-proliferative diabetic retinopathy currently lacks effective intervention strategies. Most clinicians do not use anti-VEGF therapy due to treatment burden and risk-benefit considerations, despite its potential to impact disease severity. The Helios trial results were promising, with no eyes in the OTX TKI group developing vision-threatening complications, compared to the expected progression in the sham group. The potential for a once-yearly treatment that could "reset the clock" on disease progression represents a significant advancement in managing early-stage diabetic eye disease. The study, while small and preliminary, provides encouraging insights and supports planning for larger-scale clinical trials to further evaluate OTX TKI's potential in treating diabetic eye conditions.