Researchers at the Retina World Congress 2025 in Lauderdale, Florida, presented promising results from the HELIOS trial evaluating OTX-TKI as a potential treatment for diabetic retinopathy, demonstrating significant improvements in disease markers and an absence of vision-threatening complications in treated patients.
Dr. Dilsher S. Dhoot from California Retina Consultants and Retina Consultants of America shared the findings, highlighting that OTX-TKI was well-tolerated with no serious adverse events reported during the study period.
"We saw that all DRSS [diabetic retinopathy severity score] improvements occurred in the OTX-TKI arm and all worsening occurred in the sham arm," Dr. Dhoot explained during his presentation. "We also looked at vision-threatening complications, and we saw that all patients with vision-threatening complications were in the sham arm, with no patients in the OTX-TKI arm having vision-threatening complications."
Advanced Imaging Analysis Confirms Treatment Benefits
A post-hoc analysis utilizing innovative ultrawidefield fluorescein angiography technology developed with Justin Sayers and his team from Cole Eye Institute at Cleveland Clinic provided additional evidence of OTX-TKI's biological activity.
The analysis measured retinal leakage across multiple areas, including the total retina, peripheral retina, and posterior pole. Results showed that patients receiving OTX-TKI experienced improvements in leakage parameters, while those in the sham group demonstrated worsening leakage patterns.
"Patients with OTX-TKI improved in terms of leakage, compared to those patients in the sham group, who worsened in terms of their leakage," noted Dr. Dhoot. "This is more compelling data of the action and the biologic activity of OTX-TKI."
Potential Long-Term Benefits of Tyrosine Kinase Inhibitors
The HELIOS trial results suggest that tyrosine kinase inhibitors (TKIs) as a therapeutic class may offer significant advantages for diabetic retinopathy patients. Researchers believe TKIs could potentially maintain effective therapeutic levels in the eye for extended periods ranging from six months to a year.
This sustained biological activity positions TKIs as a potentially foundational therapy for diabetic retinopathy, with the possibility of reducing treatment frequency while maintaining disease control. The approach could help stabilize vision for patients by minimizing the fluctuations in disease activity that often occur with current treatment options.
While acknowledging that some patients might require supplemental therapy, researchers expressed optimism that the majority could achieve stable results with this innovative treatment approach.
Diabetic Retinopathy Treatment Landscape
Diabetic retinopathy remains a leading cause of vision loss globally, affecting approximately one-third of people living with diabetes. Current treatment options often require frequent interventions, creating a significant burden for patients and healthcare systems.
The development of longer-acting treatments like OTX-TKI could represent an important advancement in addressing the unmet needs of this patient population. By potentially reducing treatment frequency while maintaining or improving efficacy, such therapies could significantly improve patient quality of life and treatment adherence.
Dr. Dhoot concluded his presentation by emphasizing the need for further studies to explore the full potential of OTX-TKI in treating diabetic retinopathy. The research team looks forward to additional clinical investigations that will help determine the optimal use of this promising therapy in clinical practice.
The HELIOS trial results mark an exciting advancement in ophthalmological research and offer hope for improved treatment options for patients with diabetic retinopathy.
