Towards an innovative single-dose oral treatment for Sleeping Sickness

Sleeping sickness (Human African Trypanosomiasis, HAT) is a neglected tropical disease transmitted through the tsetse flies, threatening 65 million people in currently 36 sub-Saharan African countries - mainly the poorest people often living in remote areas with limited access to health facilities. It can lead to death if untreated. Although treatment options exist, the disease is still complex to diagnose and treat and therefore requires specialized staff. Therefore, research and development for innovative treatment methods are crucial with a view to facilitating successful treatment outcomes and utlimately elimination of the disease.

Sleeping sickness is targeted by the World Health Organization for elimination as a public health problem by 2020, with a view to fully eliminate the disease (zero cases) by 2030.

The so-called “Product development partnerships” (PDPs), such as Drugs for Neglected Disease initiative, serve as platforms that bring together public and philantropic funding to address the lack of investment into research and development for neglected tropical diseases (NTDs) like sleeping sickness. Given the risky character of drug development and the lack of commercial incentives in the case of NTDs (due to a lack of purchasing power of the beneficiaries), the pharma industry is typically not ready to invest alone into such undertakings. In this sense, the PDPs typically serve as a risk-spreading mechanism that allows the pharma industry to step in by contributing its know-how and commiting itself to delivery of the developed products at an affordable price, while the PDPs drive forward the overall project and secure the funding through public-private sources.

This one-time financial support to Drugs for Neglected Diseases initiative aims at improving simple treatment options for sleeping sickness, especially for the poorest populations living in rural areas with little access to health facilities.

By developing a one-time oral treatment (called “Acoziborole”) that can be taken at home in one time, this project has the potential to significantly drive sustainable elimination of disease transmission (zero cases) by 2030.

This one-time funding contributes to the development of a patient-centered, single dose oral therapy for sleeping sickness that is affordable, safe and efficacious and easy to administer in rural contexts.

This contribution prevents a 9-month delay in the future development of a promising medicine in the next, critical development stage. SDC’s support is thus decisive for accelerating access to new treatment options for sleeping sickness patients in urgent need.

Expected results:  

The key output of this one-time funding is the continuation of clinical activities in support of the application for approval of Acoziborole to the European Medicines Agency (expected in 2021). These activities include:

• Progressing chemistry manufacturing and control activities
• Executing in-human studies that are part of the standard application package for regulatory approval 

Results from previous phases:   Previouis core funding of SDC has been allocated by DNDi inter alia to progress the preclinial activities for Acoziborole in 2012, 2013, 2014 and 2017 (total amount: EUR 333’792). As a result of the success of these preclinical activities, Acoziborole has now reached a critical development stage that has up to now delivered promising results during in-human studies.

Phase 1 01.03.2019 - 30.09.2020   (Completed)