Sleeping Sickness Investigational Therapy Demonstrates up to 95% Efficacy

A single-dose of acoziborole could simplify the treatment of this fatal infection.

Photo is courtesy of the CDC


Acoziborole, an investigational single-dose oral treatment developed for the treatment of sleeping sickness, demonstrated up to a 95% efficacy in a recent trial against the parasitic infection.

Between 2016 and 2019, DNDi and its partners led an open-label, Phase II/III study to assess the safety and efficacy of acoziborole (manufactured by Sanofi) in patients with early- and late-stage of the gambiense strain of human African trypanosomiasis (g-HAT).

The clinical trial was led by The Drugs for Neglected Diseases initiative (DNDi) and its partners in the Democratic Republic of the Congo (DRC) and Guinea.

For the study, 208 patients were recruited at 10 hospitals in the DRC and Guinea.

The 18-month treatment success rate for acoziborole was 95% in late-stage g-HAT patients, corresponding to the best results from studies with existing treatments (94%). In addition, 100% of the 41 patients with early-stage g-HAT were considered as treatment successes at all timepoints. The study shows that acoziborole has a favorable safety profile, with no significant drug-related safety signals reported.

The results were published in The Lancet Infectious Diseases.

The existing treatment requires hospitalization and infusion treatment, and in sub-Saharan Africa where sleeping sickness is prevalent, this can be difficult for people living in remote areas.

“Sleeping sickness is a nightmare disease that affects patients in some of the most remote settings in West and Central Africa, where distance from hospital can be measured in days. We are now on the cusp of a potential treatment that can be given in one day, in a single dose of three pills; this would be a revolution for doctors and communities,” Dr Victor Kande, Former neglected tropical diseases expert advisor at the Ministry of Health of the DRC, principal investigator of the trial, and lead author of The Lancet article, said in a statement.

“By simplifying the treatment paradigm, acoziborole would be an innovation that enables a sustainable response to sleeping sickness for health systems. With these new data, we have hope that we may be able to finally eliminate the disease, once and for all, by opening the door to a ‘screen-and-treat' approach at the village level,” Dr Antoine Tarral, head of the sleeping sickness program at DNDi and co-author of the paper said in a statement.

Sleeping sickness is transmitted by the bite of a tsetse fly infected with parasites, and the infection is fatal without treatment. In the early stages of the disease, people have headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing neuropsychiatric symptoms such as sleep disruption, confusion, lethargy, and convulsions and death. The tsetse fly is found in sub-Saharan Africa and the infection is endemic.

“Based on these findings, the benefit–risk profile of acoziborole for adults and adolescents with gambiense HAT, regardless of disease stage, is considered positive,” The Lancet authors wrote. “Given the high efficacy and good safety at all stages of disease, acoziborole eliminates the need for routine lumbar puncture at diagnosis and during follow-up, which requires trained staff, is associated with complications, and is a source of anxiety for patients.”

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