Discovery and Preclinical Pharmacology of INE963, a Potent and Fast-Acting Blood-Stage Antimalarial with a High Barrier to Resistance and Potential for Single-Dose Cures in Uncomplicated Malaria.

Benjamin R. Taft, Fumiaki Yokokawa, Tom Kirrane, Anne-Catherine Mata, Richard Huang, Nicole Blaquiere, Grace Waldron, Bin Zou, Oliver Simon, Subramanyam Vankadara, Wai Ling Chan, Mei Ding, Sandra Sim, Judith Straimer, Armand Guiguemde, Suresh B Lakshminarayana, Jay Prakash Jain, Christopher Bodenreider, Christopher Thompson, Christian LanshoeftWei Shu, Eric Fang, Jafri Qumber, Katherine Chan, Luying Pei, Yen-Liang Chen, Hanna Schulz, Jessie Lim, Siti Nurdiana Abas, Xiaoman Ang, Yugang Liu, Iñigo Angulo-Barturen, María Belén Jiménez-Díaz, GlaxoSmithKline Gamo, Benigno Crespo-Fernandez, Philip J. Rosenthal, Roland A. Cooper, Patrick Tumwebaze, Anna Caroline Campos Aguiar, Brice Campo, Simon Campbell, Jürgen Wagner, Thierry T Diagana, Christopher Sarko

Research output: Contribution to journalArticlepeer-review

Abstract

A series of 5-aryl-2-amino-imidazothiadiazole (ITD) derivatives were identified by a phenotype-based high-throughput screening using a blood stage Plasmodium falciparum (Pf) growth inhibition assay. A lead optimization program focused on improving antiplasmodium potency, selectivity against human kinases, and absorption, distribution, metabolism, excretion, and toxicity properties and extended pharmacological profiles culminated in the identification of INE963 (1), which demonstrates potent cellular activity against Pf 3D7 (EC50 = 0.006 μM) and achieves "artemisinin-like" kill kinetics in vitro with a parasite clearance time of/kg is fully curative in the Pf-humanized severe combined immunodeficient mouse model. INE963 (1) also exhibits a high barrier to resistance in drug selection studies and a long half-life (T1/2) across species. These properties suggest the significant potential for INE963 (1) to provide a curative therapy for uncomplicated malaria with short dosing regimens. For these reasons, INE963 (1) was progressed through GLP toxicology studies and is now undergoing Ph1 clinical trials.

Original languageAmerican English
Pages (from-to)3798-3813
Number of pages16
JournalJournal of Medicinal Chemistry
Volume65
Issue number5
StatePublished - Mar 2022

Funding

FundersFunder number
Finance team
NITD
National Institutes of HealthRD/15/0001
National Institute of Allergy and Infectious DiseasesR01AI139179

    ASJC Scopus Subject Areas

    • Molecular Medicine
    • Drug Discovery

    Keywords

    • Animals
    • Antimalarials
    • Folic Acid Antagonists
    • Malaria
    • Falciparum
    • Mice
    • SCID
    • Plasmodium falciparum
    • Folic Acid Antagonists/therapeutic use
    • Mice, SCID
    • Malaria/drug therapy
    • Antimalarials/pharmacology
    • Malaria, Falciparum/drug therapy

    Disciplines

    • Parasitic Diseases

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