Drug intelligence / Profile preview

AZD3470

Development stage
Phase 2
Lead developer
AstraZeneca
Modality
Small Molecules
Administration
Oral
01

Overview

AZD3470 is a novel, potent, and selective second-generation small molecule inhibitor of protein arginine methyltransferase 5 (PRMT5), developed for the treatment of cancer. It is specifically designed to target tumors with MTAP (methylthioadenosine phosphorylase) deficiency. By inhibiting PRMT5, which is often overactive in certain cancer cells—especially those lacking MTAP—AZD3470 aims to slow or stop tumor growth while potentially reducing side effects compared to less targeted therapies. The drug is being evaluated as monotherapy and in combination with other anticancer agents in relapsed/refractory hematologic malignancies and advanced/metastatic solid tumors that are MTAP deficient[1][2][3][4][7].

02

Targets

PRMT5 (Protein arginine N-methyltransferase 5)

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