Apexian Pharmaceuticals is a clinical stage biotechnology company founded on the scientific discoveries of Dr. Mark R. Kelley, whose work on the APE1/Ref-1 protein has provided unique insight into this important biological target. APE1/REF-1 plays a key role in a variety of inflammatory disorders and tumorigenesis, including cancers of the colon, pancreas, skin, blood, diabetic macular edema, inflammatory bowel disorders and others.
One Drug, One Molecular Target, Multiple Indications: APX3330, is an orally-administered agent targeting the APE1 protein. APE1 maintains NFkB, STAT3, AP-1 and HIF-1⍺ in a reduced form, acting as a regulator of transcription factors. APX3330 selectively binds to APE1, inhibiting its redox signaling activity. Apexian’s extensive data package demonstrating specific targeting the APE1/Ref-1 functions by APX3330 presents strong rationale for exploring this orally administered drug for the treatment of cancers, retinal and choroidal vascular diseases, prevention of chemotherapy-induced peripheral neuropathy (CIPN), and gastrointestinal inflammatory diseases. In addition, the findings from Apexian’s Phase 1 study in patients (NCT03375086) with advanced tumors further support and inform the development of APX3330 for the indications cited above.
Additionally, we have a robust pipeline of second generation compounds demonstrating similar target specificity with increased efficacy.