At its inception, DNDi’s short-term strategy was to make better use of existing treatments by combining drugs already in use. In September 2009, DNDi and partners launched the first new treatment option for sleeping sickness in 25 years: nifurtimox and eflornithine combination therapy (NECT). NECT is included on the WHO Essential Medicines Lists (EML) for adults (since 2009) and children (since 2013), and virtually all T. b. gambiense endemic countries are now using NECT as first-line treatment for stage 2 g-HAT.

As a medium-term strategy, DNDi initiated a compound mining effort to identify existing chemical compounds with potential against kinetoplastid diseases, resulting in the rediscovery of fexinidazole.

After a complete Phase I programme, DNDi engaged in g-HAT patient studies. Inclusion into a pivotal Phase II/III study in stage 2 g-HAT is complete and patient follow-up is ongoing. Two complementary studies will examine efficacy and safety in adults with stage 1 and early stage 2 g-HAT, in children aged 6-14 years, and a third one is being planned for r-HAT patients. Additional information will be obtained from a study in special population groups and to provide preliminary evidence on treatment compliance and effectiveness in ambulatory patients. Sanofi is the industrial partner.

In order to build a strong pipeline for long-term drug discovery, DNDi established a HAT Lead Optimization Consortium resulting in identification of the oxaborole SCYX-7158, which successfully progressed through pre-clinical development. Phase I clinical development was completed in 2015 and preparations are underway for a prospective Phase II/III efficacy study in patients, to be initiated in 2016. Other backup compounds were evaluated by the consortium and remain available for further development if necessary. In addition, DNDi supports the HAT Platform that was launched in Kinshasa, Democratic Republic of the Congo (DRC) in 2005. The HAT Platform is a clinical research and access-supporting network for HAT endemic countries that brings together key players in the research on sleeping sickness in endemic countries and those involved in HAT from the international research arena, with partners having a role in developing HAT health policy participating in the Platform. Ideally a new treatment for adults and children would be effective against both stages of the disease and both parasite sub-species, non-toxic, have at least 95% efficacy at 18 months post end of dosing follow-up examination, be safe for pregnant and breastfeeding women, easy to use (short-course or once a day), oral, require no monitoring, affordable, and adapted to tropical climates.


DNDi aims to deliver:

  • A safe, effective, and orally administered drug to replace current first-line HAT treatments, and to improve and simplify current case management.
  • The ideal goal is to develop two drugs that are effective against both stage 1 and 2 of HAT and both subspecies of the parasite Trypanosoma brucei gambiense (g-HAT) and Trypanosoma brucei rhodesiense (r-HAT).
  • If successful, this would represent a fundamental shift in disease management, as it would remove the need both for a risky and painful lumbar puncture test to confirm the disease stage, and for hospitalization as treatment would no longer rely on administering a drug intravenously.