An IU School of Medicine researcher is seeing his work progress to a phase 2 study to help patients with diabetic retinopathy (DR) and diabetic macular edema (DME). The study, called ZETA-1, is evaluating a small molecule oral drug candidate called APX3330, with the goal of enrolling 100 patients across the U.S. Mark Kelley, PhD, Betty and Earl Herr Professor of Pediatric Oncology Research at IU School of Medicine, has developed APX3330 from the bench to clinic, which is a potential first oral therapy for DR and DME.
“It’s been a long path to get to this phase 2 study, but it’s really rewarding,” said Kelley.
APX3330 inhibits the transcription factor regulator Ref-1 (reduction-oxidation effector factor-1). Kelley has been studying the target Ref-1 as an impactful way to block inflammation, as well as the development of new blood vessels, for several decades, both in cancer patients and those with ocular diseases. Kelley received support from the Indiana Clinical and Translational Sciences Institute (CTSI) Project Development Teams (PDT) in 2012 for this research. With his pilot funding, Kelley was able to hire an outside vendor to put together a plan to help him start the clinical trial process. This led to the licensing of the molecule by Apexian Pharmaceuticals, where Kelley is co-founder and Chief Scientific Officer. Ocuphire Pharma acquired APX3330 from Apexian Pharmaceuticals, which initially licensed the molecule from Indiana University.
“It’s hard to identify a drug and develop it from the bench all the way to clinical trials in academia alone,” said Kelley, who is also a member of the Back of the Eye (Retina) Medical Advisory Board for Ocuphire. “It takes a lot of people helping and the Indiana CTSI has really helped researchers move drugs from academia to industry.”
After receiving support from the Indiana CTSI and learning how valuable it can be through the clinical trial process, Kelley joined the Indiana CTSI Think Tank Program for Drug Discovery and Development with the goal of helping other researchers follow in his footsteps.
“Our goal is to make it easier for the next people coming along,” said Kelley. “The theme of all of this is teamwork. You can’t do it alone. We’re here to help the researchers and patients of Indiana and beyond.”
Diabetes is the leading cause of blindness among adults age 20 to 74. Diabetic retinopathy is the most common issue for people with diabetes which affects the eyes. It is caused by high blood sugar levels, which damage blood vessels in the retina. This complication affects more than 7 million people in the U.S.
The ZETA-1 trial is a randomized, placebo-controlled, double-blinded study designed to evaluate the efficacy of APX3330 to improve diabetic retinopathy over 24 weeks. All Ocuphire trials are named after stars or star systems. ZETA-1 is named for Zeta, a wide double star located in the southern constellation, Reticulum. Learn more about the study here.