Fact checked byHeather Biele

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February 09, 2024
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APX3330 ‘promising’ oral treatment option for slowing progression of diabetic retinopathy

Fact checked byHeather Biele
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Key takeaways:

  • APX3330 is an oral, small-molecule Ref-1 inhibitor being developed for treating diabetic retinopathy.
  • It reduced the percentage of patients who developed proliferative diabetic retinopathy over the study period.

Oral APX3330 showed clinically meaningful response compared with placebo in slowing progression of diabetic retinopathy in the phase 2 ZETA-1 trial.

Ocuphire Pharma’s study results were presented at the virtual Angiogenesis, Exudation and Degeneration 2024 conference.

“Treatments like APX3330 offer a hope that we will be able to help preserve and protect patients’ vision and help them maintain the highest quality of life.” Veeral Sheth, MD, MBA, FACS, FASRS

“Diabetic retinopathy affects millions of Americans and is a disease we see every day in our clinics,” Veeral Sheth, MD, MBA, FACS, FASRS, partner and director of clinical trials at University Retina and clinical assistant professor at the University of Illinois at Chicago, told Healio. “We know that without intervention, many of these patients will progress to serious and potentially irreversible vision threatening disease. Treatments like APX3330 offer a hope that we will be able to help preserve and protect patients’ vision and help them maintain the highest quality of life.”

The multicenter, randomized, placebo-controlled ZETA-1 trial enrolled 103 adults with moderately severe to severe nonproliferative diabetic retinopathy (NPDR) or mild proliferative diabetic retinopathy. Researchers used the Diabetic Retinopathy Severity Scale (DRSS) to assess patient eligibility, and participants were assigned to the small-molecule Ref-1 inhibitor APX330 600 mg or placebo twice daily for 24 weeks.

The primary outcome of interest was at least a two-step improvement on the DRSS at week 24, while secondary outcomes included improvement or worsening of disease, progression to vision-threatening complications, best corrected visual acuity, and safety and tolerability.

According to results presented by Sheth, only 5% of patients treated with APX330 had at least three-step worsening on the binocular DRSS person-level scale compared with 13% on placebo.

“We are shifting our view on how to measure success with newer systemic treatments,” Sheth told Healio. “The binocular 17-step, person-level DRSS score is an evolution in how we think about treating this disease.”

In addition, APX3330 reduced the percentage of patients who developed proliferative diabetic retinopathy over the study period, and fewer patients treated with APX3330 lost visual acuity compared with placebo.

Researchers reported limited adverse events, mostly mild in severity and similar to or fewer than those with placebo. Patients were able to continue their routine medications for diabetes comorbidities.

“Given its favorable safety profile, APX3330 may represent a promising oral treatment option for delaying or preventing disease progression in patients with NPDR who otherwise are monitored and untreated until they progress to sight-threatening disease,” George Magrath, MD, MBA, MS, Ocuphire’s CEO, said in related press release. “We look forward to advancing our oral APX3330 program.”

Based on these results, Ocuphire plans to submit a special protocol assessment to initiate a phase 3 trial.

Reference:

Editor’s note: This article was updated Feb. 13, 2024, to clarify certain aspects of the study.