Winning the endgame against sleeping sickness: Efficacy profiling of acoziborole against African trypanosomes with the focus on Trypanosoma brucei rhodesiense

by Cal M, Keller S, Rocchetti R, Braillard S, Ioset JR, Mäser P, Kaiser M. PLoS Neglected Tropical Diseases 2026. doi: 10.1371/journal.pntd.0014762

Summary: Acoziborole has obtained a positive opinion from the European Medicines Agency as a single-dose treatment for human African trypanosomiasis (HAT) caused by Trypanosoma brucei gambiense infection. However, to win the endgame against HAT, acoziborole also needs to be extended to infections caused by T. b. rhodesiense. To strengthen its preclinical dossier, the authors of this manuscript conducted an in-depth evaluation of acoziborole against the bloodstream forms of T. brucei subspecies with a focus on T. b. rhodesiense. Acoziborole had high activity against different T. b. rhodesiense strains, including one which was multidrug-resistant. Although T. b. gambiense appeared more sensitive than T. b. rhodesiense at very short exposure times, since acoziborole has an extremely long elimination half-life, it is expected to be have similar efficacyin patients with both forms of HAT.

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