A phase I study targeting the APE1/Ref-1 redox signaling protein with APX3330: first clinical agent targeting APE1/Ref-1 in cancer
APX3330 showed preliminary signals of disease control and on-target pharmacology in this first-in-human study. Based on safety and PD, the RP2D is 600 mg/d. Given the small sample size, efficacy conclusions are exploratory (ClinicalTrials.gov Identifier: NCT03375086).
Authors
- Nehal J. Lakhani (ORCID: https://orcid.org/0009-0005-6327-0176)
- Bert H. O’Neil (ORCID: https://orcid.org/0000-0001-7032-7453)
- Richard A. Messmann (ORCID: https://orcid.org/0000-0002-2112-0277)
- Randall Wireman
- Eyram Kpenu (ORCID: https://orcid.org/0000-0001-9939-7340)
- Amber L. Mosley (ORCID: https://orcid.org/0000-0001-5822-2894)
- Mateusz Opyrchal (ORCID: https://orcid.org/0000-0003-2454-7441)
- Jun Wan
- Safi Shahda
- Sheng Liu
- Mark R Kelley
- Hao Liu
Institutions
- Van Andel Institute (US)
- Maryland Oncology Hematology (US)
- Cancer Research And Biostatistics (US)
- Intellia Therapeutics (United States) (US)
- Institute of Molecular Genetics (RU)
- Comprehensive Blood & Cancer Center (US)
- Community Health Network (US)
- ApeX Therapeutics (United States) (US)
- Indiana University – Purdue University Indianapolis (US)
Publication Details
- Journal
- The Oncologist
- Published
- 2025-12-19
- DOI
- https://doi.org/10.1093/oncolo/oyaf423
- Primary Topic
- DNA Repair Mechanisms
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Riley Children's Foundation
- National Cancer Institute