A phase 1 clinical trial underway at Roswell Park Comprehensive Cancer Center offers a promising treatment option for patients with relapsed or refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Based on preclinical research demonstrating significant efficacy and improved survival, the FDA has granted fast-track designation to the study drug, AUTX-703. This first-of-its-kind KAT2A/B inhibitor, which induces cell differentiation and suppresses the proliferation of cancer cells, has also shown activity in both small cell lung cancer and neuroendocrine prostate cancer.
“AUTX-703 is a highly promising inhibitor of KAT2A, a key driver of leukemia growth in laboratory studies,” says Eunice Wang, MD, Chief of Leukemia at Roswell Park and Site Principal Investigator of the study (NCT06846606). “Treatment with AUTX-703 suggests this novel agent could have potent effects on limiting leukemia progression, and we look forward to being one of the first sites in the world to offer this new therapy to our patients.”
In general, patients with relapsed/refractory AML or MDS face a poor prognosis, with a median overall survival of around five months. While hematopoietic stem cell transplant is the best — and the only potentially curative — treatment option, not all patients are eligible, leaving many with no alternatives.
The non-randomized, open-label clinical trial will focus primarily on the safety and tolerability of AUTX-703 and secondarily the establishment of the recommended phase 2 dose. Sponsored by Auron Therapeutics, Inc., it is offered at only nine locations in the U.S.
BROADCASTMED