Ocuphire falls as oral APX3330 in diabetic retinopathy fails in Phase II trial

Shares of US retinal eye disorder specialist Ocuphire Pharma (Nasdaq: OCUP) slumped more than 29% to $2.66, after it released disappointing top-line efficacy and safety results from its ZETA-1 Phase II trial evaluating oral APX3330 for the treatment of diabetic retinopathy (DR).

Ocuphire Pharma acquired an exclusive, global sublicence from Apexian Pharmaceuticals in 2020 for the firm’s Ref-1 Inhibitor program, including lead candidate APX3330.

The company noted that APX3330 did not meet the primary endpoint (% of patients with a ≥ 2-step improvement in DRSS at week 24 in the study eye). Given the oral systemic delivery of APX3330, however, it is important to evaluate the effect on both eyes. A potential Phase III registration primary endpoint is a ≥ 3-step worsening of DRSS as a composite of both eyes (binocular). This secondary endpoint was pre-specified and evaluated in the ZETA-1 trial.