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IU researchers find new drug to prevent progression to vision loss safe and effective for people with diabetes

Mark Kelley IU Ventures

More than 37 million adults in the United States have diabetes, and many of them don’t even know they have it, according to the U.S. Centers for Disease Control and Prevention.

Diabetes can be a difficult disease to manage. It happens when the body doesn’t use insulin properly or doesn’t make enough insulin, allowing too much blood sugar to build up in the bloodstream. Over time, people with diabetes can experience other health problems as a result, such as diabetic retinopathy, which can lead to vision loss and blindness. Diabetic retinopathy affects more than 8 million people in the U.S. and 93 million worldwide.

“Currently, there are not many treatment options for diabetic retinopathy, particularly the earlier stages,” said Mark R. Kelley, PhD, Betty and Earl Herr Professor of Pediatric Oncology Research at Indiana University School of Medicine. “The early stages of diabetic retinopathy do not have any real treatment options except watching and waiting for progression to a more advanced stage, which requires eye injections of anti-VEGF as the most commonly used option. Our drug is an oral tablet that could be taken earlier in the disease progression.”

Kelley said a new drug called APX3330 could provide relief for many of these patients. At IU School of Medicine, Kelley had originally licensed the drug to Apexian Pharmaceuticals for oncology development. Following the completion of a safe phase 1 trial in oncology, the drug was then licensed to Ocuphire Pharma for back-of-the-eye diseases.

Researchers recently completed a phase 2b trial on the APX3330. About 100 patients with diabetic retinopathy were randomized to receive either 600 mg of the oral APX3330 drug or a placebo daily over 24 weeks. Kelley said those who received the drug did not experience as much worsening of vision loss as those who received the placebo. There were also few side effects and no serious adverse events.

Kelley said these results show the drug displays a strong safety profile as an oral drug and non-invasive option in protecting vision in both eyes for diabetic retinopathy patients.

“This is the first oral drug to successfully show safety and slow progression of diabetic retinopathy in humans,” Kelley said. “We will have an end-of-phase-2 meeting with the U.S. Food and Drug Administration to get their advice in anticipation of advancing to a phase 3 trial of the drug.”

Learn more about APX3330 and the phase 2 study results.

The views expressed in this content represent the perspective and opinions of the author and may or may not represent the position of Indiana University School of Medicine.
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Christina Griffiths

Christina is the media relations specialist for the IU School of Medicine Dean's Office of Strategic Communications.