Clinical Report: New Data From ARVO Support Efficacy of Axitinib in NPDR

Overview

The phase 1/2 HELIOS trial presented at the 2025 ARVO meeting demonstrated that intravitreal axitinib hydrogel (OTX-TKI) significantly reduces retinal-RPE volume and central subfield thickness in patients with nonproliferative diabetic retinopathy (NPDR) without center-involved diabetic macular edema (CI-DME). These findings suggest OTX-TKI may serve as a long-lasting therapeutic option for NPDR.

Background

Nonproliferative diabetic retinopathy (NPDR) is a common complication of diabetes that can lead to vision loss. Current treatment options are limited, particularly for NPDR without CI-DME, where intravitreal therapies are not routinely recommended. The introduction of novel therapies like axitinib could provide new avenues for managing this condition and preventing progression to more severe forms of diabetic retinopathy.

Data Highlights

Key Findings

• OTX-TKI treatment resulted in a significant reduction in mean retinal-RPE volume compared to sham treatment.
• Central subfield thickness decreased in the OTX-TKI group, while it increased in the sham group.
• Intraretinal fluid volume decreased in the OTX-TKI group, indicating improved retinal health.
• No rescue anti-VEGF therapy was administered during the study, highlighting the efficacy of OTX-TKI alone.
• The findings support further investigation of OTX-TKI as a long-acting treatment for NPDR.

Clinical Implications

The results suggest that OTX-TKI may provide a durable treatment option for patients with NPDR, potentially reducing the need for frequent interventions. Clinicians should consider the implications of these findings in the context of existing treatment guidelines and the need for further research.

Conclusion

The HELIOS trial findings indicate that OTX-TKI may be a promising therapy for managing NPDR, warranting further investigation to confirm its long-term benefits and safety profile.

References

1. The ASCO Post, 2011 -- Axitinib Improves Progression-free Survival over Sorafenib in Advanced Renal Cell Carcinoma
2. The ASCO Post, 2026 -- Addition of Axitinib to Long-Acting Octreotide in Advanced Extrapancreatic Neuroendocrine Tumors
3. Diabetic Retinopathy Preferred Practice Pattern® - PubMed, 2023
4. The ASCO Post — Addition of Axitinib to Long-Acting Octreotide in Advanced Extrapancreatic Neuroendocrine Tumors
5. The ASCO Post — Addition of Axitinib to Long-Acting Octreotide in Advanced Extrapancreatic Neuroendocrine Tumors
6. Diabetic Retinopathy Preferred Practice Pattern® - PubMed
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