One-year results from the HELIOS trial reveal that Ocular Therapeutix's investigational intravitreal axitinib implant (OTX-TKI) shows promise in treating non-proliferative diabetic retinopathy (NPDR). The study demonstrated stability or improvement in the Diabetic Retinopathy Severity Scale (DRSS) with a generally well-tolerated safety profile.
Diana V. Do, MD, Professor of Ophthalmology and Vice Chair for Clinical Affairs at the Byers Eye Institute, Stanford University, presented the findings on behalf of the HELIOS investigators at the Hawaiian Eye and Retina 2025 Meeting.
Key Findings from the HELIOS Trial
The HELIOS trial was a multicenter, double-masked, randomized, parallel-group study involving patients with moderately severe to severe NPDR without center-involved diabetic macular edema (CI-DME). OTX-TKI, a sustained-release drug in hydrogel, is administered via a single intravitreal bioresorbable hydrogel injection, designed to release the drug over 6 to 12 months.
Key results from the Phase 1 trial include:
- DRSS Stability or Improvement: OTX-TKI showed DRSS stability or improvement with durability through 48 weeks. 23.1% of patients in the OTX-TKI arm had a 2-step or greater DRSS improvement, and 46.2% of patients had a 1- or 2-step or greater DRSS improvement at 48 weeks. The DRSS did not worsen at 48 weeks in any patients in the OTX-TKI arm.
- Prevention of Disease Progression: No patients in the OTX-TKI arm developed PDR or CI-DME through week 48. In contrast, 37.5% of patients in the sham control arm developed PDR or CI-DME through week 48.
- Fluid Suppression: A single OTX-TKI injection showed durable fluid suppression and more stable fluid control through week 48.
- Safety Profile: OTX-TKI was generally well-tolerated, with no incidence of treatment or injection procedure-related intraocular inflammation, iritis, vitritis, or vasculitis.
The Need for Early Intervention
Dr. Do emphasized the importance of early intervention in diabetic retinopathy, a chronic, progressive disease. While anti-VEGF (vascular endothelial growth factor) therapy has proven effective, and the need for proactive NPDR treatment has been established in the PANORAMA and Protocol W studies, less than 1% of NPDR patients receive anti-VEGF therapy. Furthermore, a majority (62.7%) of retina specialists do not recommend treating NPDR patients without DME.
This has led to what Dr. Do described as an unsustainable treatment burden, characterized by frequent injections and worse outcomes in eyes with interrupted or reduced treatment. "There is an unmet need for early intervention with a longer-lasting treatment option," she stated.
It is important to note that AXPAXLI’s (OTX-TKI) efficacy and safety profiles have not been fully established, and it has not yet been approved by the FDA or any other health agency.
