Screening of drug libraries obtained from biotech and pharmaceutical companies, along with active screening of the literature, identified oxfendazole, a veterinary product used for deworming in animals, as a potential macrofilaricidal treatment for river blindness.

Based on highly encouraging pre-clinical data on the efficacy of oxfendazole, DNDi is evaluating the use of oxfendazole for filarial infections. DNDi is moving ahead with pharmaceutical development of the drug, and the HELP Consortium, of which DNDi is a member, will conduct a Phase I clinical trial.

HELP Consortium: an international network to speed up identification of new treatments for worm-related diseases 

Launched in 2019, the Helminth Elimination Platform (HELP) led by the Swiss Tropical and Public Health Institute (Swiss TPH) convenes key players in research and development for filarial diseases. The multidisciplinary consortium works to identify new treatments against nematode worms, including onchocerciasis, lymphatic filariasis, hookworm, and whipworm. The HELP consortium is comprised of the Swiss TPH and DNDi in Switzerland; the Institute for Medical Microbiology, Immunology and Parasitology at the Universitätsklinikum Bonn in Germany; the Muséum National d’Histoire Naturelle in France; the Ifakara Health Institute in Tanzania; the University of Buea in Cameroon; and Bristol-Myers Squibb and Elanco Animal Health in the United States.

Project updates

2020

No serious adverse events occurred in the single ascending dose or multiple ascending dose studies (NCT02234570 and NCT03035760, clinicaltrial.gov) conducted by the Oxfendazole Development Group under the auspices of the US National Institute of Allergy and Infectious Diseases. Based on the data in healthy volunteers, oxfendazole applied orally in a liquid formulation was safe and well tolerated at doses up to 60 mg/kg and showed acceptable safety and tolerability profiles after five repeated daily doses of up to 15 mg/kg, with a few reports of non-serious adverse events in the two studies. There was no increase in adverse events with an increased dose.

Therefore, the available data suggest that oxfendazole is safe for use in humans and a Phase I bioavailability study is planned using a solid, field-adapted tablet. With recently obtained funding from the European Union’s Horizon 2020 research and innovation programme, the HELP Consortium will conduct a bioavailability Phase I trial in Tanzania to evaluate the safety, tolerability, and pharmacokinetics of the tablet.

2019

DNDi developed a field-adapted tablet. 

Scientific article