Mark R. Kelley, the Betty and Earl Herr professor of pediatric oncology research at the Indiana University School of Medicine and associate director of basic science at the IU Melvin and Bren Simon Comprehensive Cancer Center, is celebrating the positive Phase 2 trial results of a first-of-its-kind drug he developed to slow the progression of diabetic retinopathy for patients in earlier stages of the disease. The oral therapeutic is called APX3330.
Ocuphire Pharma, an ophthalmic biopharmaceutical company, licensed APX3330 and is now preparing for the next phase following the positive Phase 2 results of testing the efficacy and safety of the drug in patients.
Diabetic retinopathy is the leading cause of blindness among people with diabetes and of working-age adults between 45-50 years old. By 2050, the number of individuals affected by this disease will skyrocket to more than 14 million people.
“This was the first time APX3330 was utilized for the treatment for eye diseases, specifically diabetic retinopathy,” Kelley said. “Diabetic retinopathy currently affects approximately 10 million people with diabetes and if left untreated, can lead to sight-threatening conditions. The drug is a first of its kind as there are no non-invasive oral treatments on the market to treat diabetic retinopathy. We have learned a lot with these Phase 2 results, and we are excited to keep the momentum going to advance this drug.”
The Phase 2 results showcased that patients treated with the placebo drug had a ≥ 3 step worsening on the binocular Diabetic Retinopathy Severity Scale (DRSS) than patients treated with APX3330. Therefore, APX3330 has the potential to be an early, non-invasive preventative treatment. As an oral, systemic drug, there is also the potential to treat other organs affected by diabetes (e.g., kidney disease, peripheral neuropathy) compared with other treatments on the market.
The primary endpoints for the Phase 3 trials will be to look for the reduction in worsening of diabetic retinopathy in correlation to binocular DRSS, which is a disease severity classification for diabetic retinopathy. Ocuphire will submit a special protocol assessment (SPA) to the FDA that will detail clinical trial protocol and a statistical analysis plan for the upcoming Phase 3 trials.
Kelley originally submitted this technology to the IU Innovation and Commercialization Office to help bring the drug to the market and advance his research. The mission of the office is to transfer IU innovations from lab to market for public benefit and global impact.
Kelley is the chief scientific officer and founder of Apexian Pharmaceuticals, a biotechnology company headquartered in Indianapolis. Apexian is focused on developing novel compounds to treat inflammatory disorders and tumorigenesis, including cancers of the colon, pancreas, skin, blood, diabetic macular edema, inflammatory bowel disorders and others. In 2020, the company entered into an agreement with Ocuphire Pharma to license APX3330. The drug is now Ocuphire’s lead retinal product candidate.
In recent years, IU Ventures, IU’s early-stage venture and angel investment arm, has made investments in Apexian totaling $400,000, though the organization’s relationship with Kelley dates back more than a decade. From 2008 to 2010, he served as a member of the board of directors and the executive committee for the IU Research and Technology Corporation, which now operates as IU Ventures. Kelley has also had an active role in networking and panelist events hosted by IU Ventures to speak about his experiences and provide mentorship to other IU inventors.
Kelley recently was one of two researchers at the IU Simon Comprehensive Cancer Center to receive a five-year, $2.4 million grant from the National Cancer Institute, which will allow him to further his research into therapies that target the critical pathways that pancreatic tumors use to survive.
Read an earlier blog post about Kelley and his research.
Bri Heron, technology marketing manager at Indiana University’s Innovation and Commercialization Office, contributed to this story.
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