RWC 2025: Updates on the HELIOS trial for diabetic retinopathy

At the Retina World Congress 2025 in Lauderdale, Florida, Dilsher S. Dhoot, MD, from Retina Consultants of America in Santa Barbara, California shared research on the pharmaceutical candidate, OTX-TKI which is being evaluated as a potential treatment for diabetic retinopathy.

The presentation focused on the HELIOS trial, which demonstrated promising results for a potential new treatment approach. The study showed that OTX-TKI was well-tolerated and safe, with no serious adverse events reported. Notably, all improvements in diabetic retinopathy severity score (DRSS) occurred in the OTX-TKI treatment arm, while all worsening was observed in the sham arm. This suggested a significant positive impact of the treatment.

A particularly compelling finding was the complete absence of vision-threatening complications in the OTX-TKI group, with all such complications occurring in the sham arm. The researchers conducted a post-hoc analysis using innovative technology, which examined ultra widefield fluorescine angiography. The analysis measured leakage across different retinal areas, including the total retina, peripheral retina, and posterior pole. This detailed examination provided additional evidence of the treatment's biological activity and potential effectiveness.

Dhoot also expressed his optimism about tyrosine kinase inhibitors (TKIs) as a treatment class, highlighting their potential for sustained biological activity. The research suggested that TKIs could potentially maintain effective levels in the eye for 6 months to a year, positioning them as a potentially foundational therapy for diabetic retinopathy. The long-term vision for this treatment approach was to reduce activity fluctuations and potentially improve patient vision stability. While acknowledging that some patients might require supplemental therapy, the researcher believed that the majority could achieve stable results with this innovative treatment. Dhoot concluded with a call for further studies to explore the full potential of OTX-TKI in treating diabetic retinopathy, marking an exciting advancement in ophthalmological research.