Drug intelligence / Profile preview

acoziborole

Development stage
Phase 3
Lead developer
Drugs for Neglected Diseases initiative
Modality
Small Molecules
Administration
Oral
01

Overview

Acoziborole is an orally bioavailable, structurally novel small molecule antiprotozoal drug of the benzoxaborole class. It was originally invented by Anacor Pharmaceuticals and is being developed by the Drugs for Neglected Diseases Initiative (DNDi) for the treatment of human African trypanosomiasis (HAT), also known as sleeping sickness, caused by *Trypanosoma brucei gambiense*. Acoziborole acts as an inhibitor of the trypanosome cleavage and polyadenylation specificity factor 3 (CPSF3), blocking mRNA processing in the parasite. The drug is notable for its single-dose oral regimen, which has demonstrated high efficacy (>95%) and a favorable safety profile in both early and late-stage HAT patients, including children. Its long half-life allows prolonged exposure from just one dose, making it especially suitable for use in remote settings where access to healthcare may be limited[1][2][4][5][6][7].

Other names
benzoxaborole derivative
02

Targets

CPSF3 (Cleavage and polyadenylation specificity factor 3)

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