Clinical Scorecard: OTX-TKI May Decrease Retinal Leakage in NPDR

At a Glance

Key Highlights

• Single intravitreal injection of OTX-TKI implant may reduce retinal leakage for up to 12 months
• Post hoc analysis from phase 1 HELIOS trial showed decreased leakage area and index on ultrawidefield fluorescein angiography
• OTX-TKI implant gradually dissolves over 6 to 12 months, releasing axitinib to target VEGFR2-mediated disease activity

Guideline-Based Recommendations

Diagnosis

• Use ultrawidefield fluorescein angiography to quantify retinal leakage and assess disease activity in NPDR

Management

• Consider single intravitreal OTX-TKI implant injection as a potential long-acting treatment option for moderately severe to severe NPDR without diabetic macular edema

Monitoring & Follow-up

• Monitor retinal leakage area and leakage index via imaging over 12 months to evaluate treatment effect
• Assess Diabetic Retinopathy Severity Scale (DRSS) changes and vision-threatening complications during follow-up

Risks

• Phase 1 HELIOS trial reported absence of vision-threatening complications at week 48

Patient & Prescribing Data

Patients with moderately severe to severe NPDR without diabetic macular edema

A single OTX-TKI implant injection demonstrated sustained reduction in retinal leakage and DRSS improvement over 12 months in a small phase 1 trial

Clinical Best Practices

• Utilize machine learning–augmented imaging platforms combined with expert validation to quantify retinal leakage accurately
• Evaluate both peripheral and posterior pole retinal leakage areas and indices for comprehensive disease assessment
• Consider OTX-TKI implant as a long-acting therapeutic option pending further phase 3 trial results

References

• 2025 American Society of Retina Specialists Annual Meeting