AstraZeneca has revealed early results from its phase 1 trial of AZD1390, a potential first-in-class ATM kinase inhibitor designed to treat glioma, a type of brain tumor. The drug is intended to work alongside radiation therapy for tumors that have ATM loss, a form of DNA repair deficiency.
According to AstraZeneca, more than 60% of patients involved in the trial experienced tumor shrinkage after treatment with AZD1390 in combination with radiotherapy. These findings were shared in a presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.
The study included patients with high-grade gliomas who were not candidates for surgery. Among a subgroup of 14 patients whose tumors had an ATM loss of function mutation, 10 showed a partial response and one achieved a complete response. The company noted that “these results suggest promising antitumor activity in a molecularly selected population.”
Michael Buckland, Director of the Brain and Mind Centre at the University of Sydney and an investigator on the study, said, “Targeting the ATM pathway may offer a new approach for patients with fewer treatment options, and AZD1390 is the first molecule of its kind to demonstrate this kind of potential.”
AZD1390 was able to cross the blood–brain barrier and appeared well-tolerated among patients. A maximum tolerated dose of 500 mg once daily was determined and will be used in the next part of the study. AstraZeneca noted that further efficacy data is needed, but the early results are encouraging.
The company is now planning a phase 2 expansion of the trial to further explore AZD1390’s benefits in specific patient populations, especially those with the ATM mutation identified through molecular testing.
