Efficacy and safety of acoziborole in patients with human African trypanosomiasis caused by Trypanosoma brucei gambiense: a multicentre, open-label, s

https://www.thelancet.com/journals/laninf/article/PIIS1473-3099(22)00660-0/fulltext

Human African trypanosomiasis (HAT; also known as sleeping sickness) is caused by the protozoan parasite Trypanosoma brucei gambiense (gambiense HAT) and is transmitted by the tsetse fly. This neglected tropical disease is mostly fatal when left untreated. HAT remains endemic in sub-Saharan Africa, mainly in the Democratic Republic of the Congo, although surveillance and control programmes have achieved a steady decline in gambiense HAT incidence worldwide with 565 cases reported in 2020 (a decrease from 2110 cases in 2016).
Acoziborole, an oral benzoxaborole-6-carboxamide from the Drugs for Neglected Diseases initiative (DNDi) drug discovery programme, showed high activity in vitro in both stages of gambiense HAT in murine models, and low toxicity and strong efficacy in preclinical studies allowing progression to phase 1. A first-in-human study (unpublished) showed the tolerability and pharmacokinetic profile of acoziborole and determined that a single therapeutic dose of 960 mg in fasted patients provided the desired exposure to the parasite in blood and cerebrospinal fluid (CSF) with a good safety profile. Here, we aim to assess the safety and efficacy of acoziborole in adult and adolescent patients with gambiense HAT.

 

https://www.bloomberg.com/news/arti...-raises-hopes-of-wiping-out-sleeping-sickness

A drug co-developed by Sanofi is highly effective at treating the sometimes lethal disease called sleeping sickness and could help eradicate the ailment by the end of the decade.

A single oral dose of acoziborole was 95% effective at curing patients with a late-stage form of the parasitic disease, according to a study published in The Lancet. The drug was co-developed by the Drugs for Neglected Diseases initiative.

The illness is endemic across much of sub-Saharan Africa. Tsetse flies transmit the parasite, which mostly affects people in rural areas where there’s often little medical infrastructure, according to the World Health Organization.
While the disease is far less common today thanks to control measures, there were still about 1,000 reported cases in 2019 and diagnosing people is difficult. The WHO considers 3 million people to be at moderate or higher risk.

The Gambiense variant of human African trypanosomiasis is the most common and “could become the first disease that is eradicated thanks to a drug rather than a vaccine,” Jacques Pepin, an independent expert, said in a comment published alongside the study.