OTX-TKI Shows Durable Reduction of Retinal Leakage in NPDR
Overview
A post hoc imaging analysis from the phase 1 HELIOS trial indicates that a single intravitreal injection of OTX-TKI may reduce retinal leakage for up to 12 months in patients with moderately severe to severe nonproliferative diabetic retinopathy (NPDR). The treatment demonstrated a decrease in leakage area and index compared to sham, suggesting a potential durable therapeutic effect.
Background
Nonproliferative diabetic retinopathy (NPDR) is characterized by retinal vascular leakage and progressive disease activity, which can be quantified by fluorescein angiography. OTX-TKI is a bioresorbable intravitreal implant releasing axitinib, a tyrosine kinase inhibitor targeting VEGFR2, designed to reduce retinal vascular leakage. The HELIOS phase 1 trial evaluated safety and exploratory efficacy endpoints in patients with moderately severe to severe NPDR over 12 months.
Data Highlights
| Parameter | OTX-TKI Group | Sham Group |
|---|---|---|
| Leakage Area | Decreased over 12 months | Increased over 12 months |
| Leakage Index | Decreased over 12 months | Increased over 12 months |
| Retinal Regions | Reduction in peripheral and posterior pole leakage | Increase in peripheral and posterior pole leakage |
Key Findings
- Single intravitreal injection of OTX-TKI reduced retinal leakage area and leakage index for up to 12 months.
- Sham-treated patients showed progressive increase in retinal leakage consistent with disease progression.
- Leakage reduction was observed across total retinal area, peripheral retina, and posterior pole.
- Machine learning–augmented ultrawidefield fluorescein angiography enabled quantitative assessment of leakage changes.
- No vision-threatening complications were reported at week 48 in the OTX-TKI group.
- Findings support potential long-acting treatment effect of OTX-TKI in moderately severe to severe NPDR.
Clinical Implications
OTX-TKI may offer a novel, long-acting intravitreal treatment option to reduce retinal vascular leakage and potentially slow progression in patients with moderately severe to severe NPDR. Quantitative leakage assessment via ultrawidefield fluorescein angiography can serve as a useful biomarker to monitor treatment response. Further larger studies are warranted to confirm these preliminary efficacy signals.
Conclusion
The HELIOS trial post hoc analysis suggests that OTX-TKI provides a sustained reduction in retinal leakage in NPDR patients for up to 12 months, supporting its potential as a durable therapeutic option. These findings warrant further investigation in larger, controlled trials.
References
- Talcott KE et al. 2025 -- OTX-TKI May Decrease Retinal Leakage in NPDR, 2025 ASRS Annual Meeting
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