APX3330 oral treatment for diabetic retinopathy shows encouraging phase 2 data
Click Here to Manage Email Alerts
PARK CITY, Utah — APX3330, an investigational systemic treatment for diabetic retinopathy, showed encouraging outcomes in the phase 2 ZETA-1 trial, paving the way to phase 3.
“It is a promising oral candidate for slowing the progression of diabetic retinopathy ... a noninvasive approach to our diabetic patients,” Veeral Sheth, MD, MBA, FACS, FASRS, said at Clinical Trials at the Summit.
Image: Adobe Stock
APX3330 (Ocuphire Pharma) is a selective inhibitor of Ref-1, which is responsible for three distinct pathways: angiogenesis, oxidative stress and inflammation, he said. By simultaneously addressing all three pathways, this compound has the potential to restore neurovascular homeostasis.
The primary endpoint of ZETA-1 was a two-step or greater improvement in Diabetic Retinopathy Severity Scale (DRSS) score in the study eye. However, because APX3330 is a systemic therapy, phase 2/3 will look at binocular disease progression using a binocular 17-step DRSS score.
“A three-step worsening is considered clinically meaningful with this scale,” Sheth said.
Phase 3 will focus on patients with nonproliferative diabetic retinopathy (NPDR) with a severity score of 8 to 11. These patients are at the highest risk for progressing to proliferative diabetic retinopathy (PDR).
In ZETA-1 study, fewer treated patients (3%) worsened by three or more steps on binocular DRSS score as compared with patients who received placebo (20%). This represents an 85% reduction between treatment and placebo. A clear trend was seen toward fewer patients worsening with treatment as compared with placebo.
Fewer treated patients (7%) developed PDR as compared with those in the placebo group (27%), accounting for a 74% reduction.
APX3330 showed a favorable safety and tolerability profile. Ocular adverse events were similar between treatment and placebo, and participants with DR continued routine medications to manage comorbidities.
Ocuphire is now set to commence the ZETA-2 phase 2/3 trial.
“In the phase 3 trial, we are really going to be focusing on patients with more severe NPDR that are more likely to progress to PDR,” Sheth said.
Collapse
Sheth V. Clinical update on oral APX3330 for diabetic retinopathy. Presented at: Clinical Trials at the Summit; June 8, 2024; Park City, Utah.
Read more about
Click Here to Manage Email Alerts