Ocuphire Pharma announces presentation on APX3330 at ARVO 2024

Ocuphire Pharma announced that clinical data from its ZETA-1 trial evaluating APX3330 in diabetic retinopathy on a validated binocular person-level scale was presented yesterday at the Association for Research in Vision and Ophthalmology Annual Meeting, taking place May 5-9, 2024 in Seattle, Washington. Presentation Highlights: ZETA-1 was a Phase 2, randomized, double-masked trial evaluating the efficacy and safety of oral APX3330 compared to placebo in 103 participants with DR completed in January 2023. A subset analysis was conducted to evaluate the efficacy of APX3330 in slowing DR progression using the target population of the planned Phase 2/3 study and the Food and Drug Administration’s-agreed upon registration endpoint of a 3-step change on a binocular diabetic Retinopathy Severity Scale. This 17-step person-level scale accounts for the DRSS scores of the two eyes and then anchors the step to the worse eye. The subset comprised 68 participants from the ZETA-1 trial who had a baseline DRSS score of 47 or 53 in at least one eye and 43, 47 or 53 in the other eye. Analysis of the ZETA-1 Phase 2 subset using the binocular person-level scale showed that no participants in the APX3330 group had a greater than or equal to 4-step worsening at week 24 compared to 15.2% in the placebo group, representing a 100% reduction between groups. Similarly, only 5.7% of APX3330-treated subjects had a greater than or equal to 3-step worsening at week 24 compared to 15.2% of placebo subjects, representing a 62.5% reduction between groups. Fewer participants in the APX3330 group developed proliferative diabetic retinopathy by week 24 compared to the placebo group. APX3330 showed favorable safety and tolerability, with similar ocular adverse events between APX3330 and placebo groups.