AZD1390 is an investigational ATM inhibitor aimed at increasing the efficacy of radiation. As of Feb 2024, 75 recurrent GBM patients and 36 newly diagnosed GBM patients with unmethylated MGMT had received AZD1390 in the multicenter Phase 1 trial. The treatment was generally well tolerated, with the majority of adverse events being low-grade. For rGBM patients who received doses sufficient for target engagement (as determined by a Phase 0 study), mOS was 12.7 months. Survival data for the ndGBM cohort is still maturing.
The Ivy Brain Tumor Center also presented data at AACR from their Phase 0/1 study on AZD1390, with results showing the drug was well tolerated, achieved high concentrations in tumor tissue, and successfully modulated its biomarker target.