Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy

Phase 2 Completed 103 enrolled Ocuphire Pharma, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The objective of this study is to evaluate the safety and efficacy of APX3330 to treat diabetic retinopathy (DR) and diabetic macular edema (DME).

Timeline

Start: 2021-04-08
Primary completion: 2023-01-25
Completion: 2023-01-25

Outcome

Sources disagree · no verdict is set; each source's reading is shown below

release “The study met its primary safety endpoint, demonstrating a favorable safety and tolerability profile with no adverse events reported.” reads as met primary endpoint opusgtx.com ↗

release “In the ZETA-1 Phase 2 trial, APX3330 did not meet the primary endpoint” reads as missed primary endpoint opusgtx.com ↗

release “One of its assets, APX3330, failed to meet its primary endpoint in a Phase 2 clinical trial” reads as missed primary endpoint opusgtx.com ↗

Publications

Lalunio H, Stupka N, Goodman CA, Hayes A. The Potential of Targeting APE1/Ref-1 as a Therapeutic Intervention for Duchenne Muscular Dystrophy. Antioxid Redox Signal. 2025 May;42(13-15):641-654. doi: 10.1089/ars.2024.0620. Epub 2024 Dec 27.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	APX3330	Small molecule	600 mg	Oral