Preclinical characterization and target validation of the antimalarial pantothenamide MMV693183.


Journal

Nature communications
ISSN: 2041-1723
Titre abrégé: Nat Commun
Pays: England
ID NLM: 101528555

Informations de publication

Date de publication:
20 04 2022
Historique:
received: 17 05 2021
accepted: 09 03 2022
entrez: 21 4 2022
pubmed: 22 4 2022
medline: 23 4 2022
Statut: epublish

Résumé

Drug resistance and a dire lack of transmission-blocking antimalarials hamper malaria elimination. Here, we present the pantothenamide MMV693183 as a first-in-class acetyl-CoA synthetase (AcAS) inhibitor to enter preclinical development. Our studies demonstrate attractive drug-like properties and in vivo efficacy in a humanized mouse model of Plasmodium falciparum infection. The compound shows single digit nanomolar in vitro activity against P. falciparum and P. vivax clinical isolates, and potently blocks P. falciparum transmission to Anopheles mosquitoes. Genetic and biochemical studies identify AcAS as the target of the MMV693183-derived antimetabolite, CoA-MMV693183. Pharmacokinetic-pharmacodynamic modelling predict that a single 30 mg oral dose is sufficient to cure a malaria infection in humans. Toxicology studies in rats indicate a > 30-fold safety margin in relation to the predicted human efficacious exposure. In conclusion, MMV693183 represents a promising candidate for further (pre)clinical development with a novel mode of action for treatment of malaria and blocking transmission.

Identifiants

pubmed: 35444200
doi: 10.1038/s41467-022-29688-5
pii: 10.1038/s41467-022-29688-5
pmc: PMC9021288
doi:

Substances chimiques

Antimalarials 0
Folic Acid Antagonists 0
Pantothenic Acid 19F5HK2737
pantothenamide 2MO0ZY19DW

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Pagination

2158

Subventions

Organisme : NIAID NIH HHS
ID : R01 AI109023
Pays : United States
Organisme : NIAID NIH HHS
ID : R01 AI139179
Pays : United States
Organisme : NIDDK NIH HHS
ID : T32 DK120509
Pays : United States
Organisme : Wellcome Trust
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 100993/Z/13/Z
Pays : United Kingdom

Informations de copyright

© 2022. The Author(s).

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Auteurs

Laura E de Vries (LE)

Department of Medical Microbiology, Radboudumc Center for Infectious Diseases, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.
Department of Immunology and Infectious Diseases, Harvard T.H. Chan School of Public Health, Boston, MA, USA.

Patrick A M Jansen (PAM)

Department of Dermatology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

Catalina Barcelo (C)

Medicines for Malaria Venture, Geneva, Switzerland.

Justin Munro (J)

Department of Chemistry and Huck Center for Malaria Research, The Pennsylvania State University, University Park, PA, USA.

Julie M J Verhoef (JMJ)

Department of Medical Microbiology, Radboudumc Center for Infectious Diseases, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

Charisse Flerida A Pasaje (CFA)

Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.

Kelly Rubiano (K)

Department of Microbiology & Immunology, Columbia University Irving Medical Center, New York, NY, USA.

Josefine Striepen (J)

Department of Microbiology & Immunology, Columbia University Irving Medical Center, New York, NY, USA.

Nada Abla (N)

Medicines for Malaria Venture, Geneva, Switzerland.

Luuk Berning (L)

TropIQ Health Sciences, Nijmegen, The Netherlands.

Judith M Bolscher (JM)

TropIQ Health Sciences, Nijmegen, The Netherlands.

Claudia Demarta-Gatsi (C)

Medicines for Malaria Venture, Geneva, Switzerland.

Rob W M Henderson (RWM)

TropIQ Health Sciences, Nijmegen, The Netherlands.

Tonnie Huijs (T)

TropIQ Health Sciences, Nijmegen, The Netherlands.

Karin M J Koolen (KMJ)

TropIQ Health Sciences, Nijmegen, The Netherlands.

Patrick K Tumwebaze (PK)

Infectious Diseases Research Collaboration, Kampala, Uganda.

Tomas Yeo (T)

Department of Microbiology & Immunology, Columbia University Irving Medical Center, New York, NY, USA.

Anna C C Aguiar (ACC)

Sao Carlos Institute of Physics, University of São Paulo, São Carlos, São Paulo, Brazil, São Carlos, SP, Brazil.

Iñigo Angulo-Barturen (I)

The Art of Discovery, Derio, Spain.

Alisje Churchyard (A)

Department of Life Sciences, Imperial College London, South Kensington, London, United Kingdom.

Jake Baum (J)

Department of Life Sciences, Imperial College London, South Kensington, London, United Kingdom.

Benigno Crespo Fernández (BC)

Global Health, GlaxoSmithKline, Tres Cantos, Madrid, Spain.

Aline Fuchs (A)

Medicines for Malaria Venture, Geneva, Switzerland.

Francisco-Javier Gamo (FJ)

Global Health, GlaxoSmithKline, Tres Cantos, Madrid, Spain.

Rafael V C Guido (RVC)

Sao Carlos Institute of Physics, University of São Paulo, São Carlos, São Paulo, Brazil, São Carlos, SP, Brazil.

María Belén Jiménez-Diaz (MB)

The Art of Discovery, Derio, Spain.

Dhelio B Pereira (DB)

Research Center for Tropical Medicine of Rondonia, Porto Velho, Brazil.

Rosemary Rochford (R)

Department of Immunology and Microbiology, University of Colorado Anschutz School of Medicine, Aurora, CO, USA.

Camille Roesch (C)

Malaria Molecular Epidemiology Unit, Institut Pasteur du Cambodge, Phnom Penh, Cambodia.
Malaria Translational Research Unit, Institut Pasteur, Paris & Institut Pasteur du Cambodge, Phnom Penh, Cambodia.

Laura M Sanz (LM)

Global Health, GlaxoSmithKline, Tres Cantos, Madrid, Spain.

Graham Trevitt (G)

Sygnature Discovery, Nottingham, United Kingdom.

Benoit Witkowski (B)

Malaria Molecular Epidemiology Unit, Institut Pasteur du Cambodge, Phnom Penh, Cambodia.
Malaria Translational Research Unit, Institut Pasteur, Paris & Institut Pasteur du Cambodge, Phnom Penh, Cambodia.

Sergio Wittlin (S)

Swiss Tropical and Public Health Institute, Basel, Switzerland.
University of Basel, Basel, Switzerland.

Roland A Cooper (RA)

Department of Natural Sciences and Mathematics, Dominican University of California, San Rafael, CA, USA.

Philip J Rosenthal (PJ)

Department of Medicine, University of California, San Francisco, CA, USA.

Robert W Sauerwein (RW)

Department of Medical Microbiology, Radboudumc Center for Infectious Diseases, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.
TropIQ Health Sciences, Nijmegen, The Netherlands.

Joost Schalkwijk (J)

Department of Dermatology, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

Pedro H H Hermkens (PHH)

Hermkens Pharma Consultancy, Oss, The Netherlands.

Roger V Bonnert (RV)

Medicines for Malaria Venture, Geneva, Switzerland.

Brice Campo (B)

Medicines for Malaria Venture, Geneva, Switzerland.

David A Fidock (DA)

Department of Microbiology & Immunology, Columbia University Irving Medical Center, New York, NY, USA.
Center for Malaria Therapeutics and Antimicrobial Resistance, Division of Infectious Diseases, Department of Medicine, Columbia University Irving Medical Center, New York, NY, USA.

Manuel Llinás (M)

Department of Chemistry and Huck Center for Malaria Research, The Pennsylvania State University, University Park, PA, USA.
Department of Biochemistry & Molecular Biology, The Pennsylvania State University, University Park, PA, USA.

Jacquin C Niles (JC)

Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.

Taco W A Kooij (TWA)

Department of Medical Microbiology, Radboudumc Center for Infectious Diseases, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands. taco.kooij@radboudumc.nl.

Koen J Dechering (KJ)

TropIQ Health Sciences, Nijmegen, The Netherlands. k.dechering@tropiq.nl.

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