The ATM Kinase Inhibitor AZD0156 Is a Potent Inhibitor of <i>Plasmodium</i> Phosphatidylinositol 4‐Kinase (PI4Kβ) and Is an Attractive Candidate for Medicinal Chemistry Optimization Against Malaria

J John G. Woodland (Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa) D Dina Coertzen (Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa) K Kathryn J. Wicht (South African Medical Research Council Drug Discovery and Development Research Unit, Department of Chemistry and Institute of Infectious Disease and Molecular Medicine University of Cape Town Rondebosch, Cape Town 7701 South Africa) V Virginia Franco Hidalgo (GlaxoSmithKline Tres Cantos Medicines Development Campus Madrid Spain) C Charisse Flerida A. Pasaje L Luiz C. Godoy (Department of Biological Engineering Massachusetts Institute of Technology Cambridge MA 02139 USA) T Tarrick Qahash M Mmakwena M. Mmonwa (Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa) G Godwin A. Dziwornu (Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa) L Lynn Wambua (South African Medical Research Council Drug Discovery and Development Research Unit Institute of Infectious Disease and Molecular Medicine University of Cape Town Cape Town 7925 South Africa) S Sarah Harries (Department of Chemistry University of Cape Town Rondebosch Cape Town 7701 South Africa) C Constance M. Korkor (Department of Chemistry University of Cape Town Rondebosch Cape Town 7701 South Africa) M Mathew Njoroge L Liezl Krugmann (Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa) D Dale Taylor (Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa) M Meta Leshabane (Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa) H Henrico Langeveld (Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa) T Tayla Rabie (Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa) J Janette Reader (Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa) M Mariëtte van der Watt (Institute for Sustainable Malaria Control University of Pretoria Hatfield Pretoria 0028 South Africa) N Nelius Venter (Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa) E Erica Erlank (Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa) A Ayesha S. Aswat (Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa) L Lizette L. Koekemoer (Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa) T Tomas Yeo J Jin H. Jeon (Department of Microbiology and Immunology, Columbia University Medical Center) D David A. Fidock (Department of Microbiology and Immunology, Columbia University Medical Center) F Francisco Javier Gamo (GlaxoSmithKline Tres Cantos Medicines Development Campus Madrid Spain) S Sergio Wittlin (Swiss Tropical and Public Health Institute) J Jacquin C. Niles M Manuel Llinás L Lauren B. Coulson (South African Medical Research Council Drug Discovery and Development Research Unit, Department of Chemistry and Institute of Infectious Disease and Molecular Medicine University of Cape Town Rondebosch, Cape Town 7701 South Africa) L Lyn‐Marié Birkholtz (Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa) K Kelly Chibale

Abstract

Abstract New compounds targeting human malaria parasites are critical for effective malaria control and elimination. Here, we pursued the imidazoquinolinone AZD0156 (MMV1580483), a human ataxia‐telangiectasia mutated (ATM) kinase inhibitor that completed Phase I clinical trials as an anticancer agent. We validated its in vitro activity against the two main forms of the Plasmodium falciparum parasite in the human host, viz. the asexual blood (symptomatic) stage and sexual gametocyte (transmission) stage. Resistance selection, cross‐resistance, biochemical, and conditional knockdown studies revealed that AZD0156 inhibits P. falciparum phosphatidylinositol 4‐kinase type III beta ( Pf PI4Kβ), a clinically‐validated target for the treatment of malaria. Metabolic perturbations, fixed‐ratio isobolograms, killing kinetics and morphological evaluation correlated AZD0156 inhibition with other known PI4Kβ inhibitors. The compound showed favorable in vivo pharmacokinetic properties and 81% antimalarial efficacy (4 × 50 mg kg −1 ) in a P. berghei mouse malaria infection model. Importantly, a cleaner biochemical profile was measured against human kinases (MAP4K4, MINK1) implicated in embryofoetal developmental toxicity associated with the Pf PI4Kβ inhibitor MMV390048. This improved kinase selectivity profile and structural differentiation from other PI4Kβ inhibitors, together with its multistage antiplasmodial activity and favorable pharmacokinetic properties, makes AZD0156 an attractive candidate for target‐based drug repositioning against malaria via a medicinal chemistry optimization approach.

Article Details

Volume / Issue Vol. 64, Issue 28
Published July 07, 2025
ISSN 1433-7851
Publisher Wiley

Journal Info

Angewandte Chemie International Edition

Wiley

ISSN: 1433-7851 Physical Sciences

Authors (34)

J

John G. Woodland

Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa

D

Dina Coertzen

Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa

K

Kathryn J. Wicht

South African Medical Research Council Drug Discovery and Development Research Unit, Department of Chemistry and Institute of Infectious Disease and Molecular Medicine University of Cape Town Rondebosch, Cape Town 7701 South Africa

V

Virginia Franco Hidalgo

GlaxoSmithKline Tres Cantos Medicines Development Campus Madrid Spain

C

Charisse Flerida A. Pasaje

L

Luiz C. Godoy

Department of Biological Engineering Massachusetts Institute of Technology Cambridge MA 02139 USA

T

Tarrick Qahash

M

Mmakwena M. Mmonwa

Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa

G

Godwin A. Dziwornu

Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa

L

Lynn Wambua

South African Medical Research Council Drug Discovery and Development Research Unit Institute of Infectious Disease and Molecular Medicine University of Cape Town Cape Town 7925 South Africa

S

Sarah Harries

Department of Chemistry University of Cape Town Rondebosch Cape Town 7701 South Africa

C

Constance M. Korkor

Department of Chemistry University of Cape Town Rondebosch Cape Town 7701 South Africa

M

Mathew Njoroge

L

Liezl Krugmann

Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa

D

Dale Taylor

Holistic Drug Discovery and Development (H3D) Centre University of Cape Town Rondebosch Cape Town 7701 South Africa

M

Meta Leshabane

Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa

H

Henrico Langeveld

Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa

T

Tayla Rabie

Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa

J

Janette Reader

Department of Biochemistry, Genetics and Microbiology Institute for Sustainable Malaria Control University of Pretoria Hatfield 0028 South Africa

M

Mariëtte van der Watt

Institute for Sustainable Malaria Control University of Pretoria Hatfield Pretoria 0028 South Africa

N

Nelius Venter

Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa

E

Erica Erlank

Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa

A

Ayesha S. Aswat

Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa

L

Lizette L. Koekemoer

Wits Research Institute for Malaria Faculty of Health Sciences University of the Witwatersrand Johannesburg 2193 South Africa

T

Tomas Yeo

J

Jin H. Jeon

Department of Microbiology and Immunology, Columbia University Medical Center

D

David A. Fidock

Department of Microbiology and Immunology, Columbia University Medical Center

F

Francisco Javier Gamo

GlaxoSmithKline Tres Cantos Medicines Development Campus Madrid Spain

S

Sergio Wittlin

Swiss Tropical and Public Health Institute

J

Jacquin C. Niles

M

Manuel Llinás

L

Lauren B. Coulson

South African Medical Research Council Drug Discovery and Development Research Unit, Department of Chemistry and Institute of Infectious Disease and Molecular Medicine University of Cape Town Rondebosch, Cape Town 7701 South Africa

L

Lyn‐Marié Birkholtz

Department of Biochemistry, Genetics and Microbiology Hatfield Pretoria 0028 South Africa

K

Kelly Chibale