Ocuphire Pharma, Inc. announced the successful outcome of an End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA), supporting the advancement of oral APX3330 for the treatment of diabetic retinopathy (DR) into Phase 3 studies based on the recently completed Phase 2 ZETA-1 trial. Results from Phase 2 ZETA-1 results demonstrate that oral APX3330 has the potential to slow or prevent clinically meaningful progression of diabetic retinopathy, as measured by the percentage of subjects with = 3-step worsening on a binocular diabetic retinopathy severity scale (DRSS), which will be the Phase 3 primary endpoint. As recommended by the FDA, Ocuphire plans to submit a Special Protocol Assessment to agree on the clinical trial protocol and statistical analysis plan for the Phase 3 trials and will share specifics on the study design parameters and anticipated timing once agreed with the FDA.

The EOP2 meeting was supported by results from the previously completed Phase 2 ZETA- 1 trial. With a novel dual mechanism of action, APX3330 blocks the downstream pathways regulated by Ref-1 -- which involve angiogenesis (VEGF) and inflammation (NFkB) to decrease abnormal activation of both angiogenesis and inflammatory pathways that are implicated across several ocular diseases, including DR, DME, and age- related macular degeneration (AMD). APX3330 has shown a favorable safety and tolerability profile in 12 clinical trials conducted in healthy, hepatitis, cancer, and diabetic subjects.