The Drugs for Neglected Diseases initiative (DNDi) has been awarded $29.2 million to accelerate the development of innovative new drugs for patients in sub-Saharan Africa with sleeping sickness (human African trypanosomiasis – $15 million) and river blindness (onchocerciasis – $14.2 million) by the Bill & Melinda Gates Foundation These two parasitic diseases could be eliminated if new patient care tools are brought to bear.
Announced today, these grants will run from 2020 until 2023 and will help deliver a single-dose cure for sleeping sickness, a terrifying and fatal disease, and greatly aid efforts to develop a short-course treatment for the millions of people at risk of river blindness in Central and West Africa.
‘Both river blindness and sleeping sickness require innovative drugs to improve patient care or achieve elimination,’ said Dr Bernard Pécoul, Executive Director of DNDi. ‘There has been great progress for these two diseases in recent years, with the delivery of fexinidazole, the first all-oral treatment for sleeping sickness developed by DNDi and Sanofi, and the approval in the US of moxidectin for river blindness. Nevertheless, the needs remain acute, and reaching the goal of elimination requires innovation into better, field-adapted tools.’
The financial support will help develop:
New tools to address debilitating filarial diseases and that will shorten the time to onchocerciasis elimination and enable test-and-treat strategies:
- River blindness is transmitted by the bite of an infected blackfly and causes severe itching, skin lesions, and eventually blindness. An estimated 20.9 million people are infected by the disease worldwide. The current approach to eliminating river blindness is based on the mass distribution of ivermectin, which has been tremendously successful in reducing disease prevalence. But these programs need to be repeated for 10-12 years as current treatments only kill the juvenile worms that cause river blindness, but not the adult worms, which can live more than 10 years in the human body.
- DNDi aims to develop a safe, effective, affordable, and field-adapted ‘macrofilaricidal’ drug that can kill adult worms that cause river blindness. This could be used for individual patient treatment – current approaches are largely preventative targeting the larval stages only – with the potential for use as preventive chemotherapy to accelerate the elimination of river blindness in Africa in areas that are difficult to treat.
- In 2018-9, DNDi and partners successfully progressed two new chemical compounds to first-in-human Phase I clinical studies: emodepside with Bayer Pharmaceuticals, and ABBV-4083 with AbbVie and the Liverpool School of Tropical Medicine. Thanks to this renewed financial support, DNDi aims to demonstrate a proof-of-concept for efficacy, safety, and tolerability for the two compounds.
The first single-dose oral treatment for sleeping sickness to support sustainable elimination of the disease:
- Sleeping sickness is transmitted by the tsetse fly. The parasite attacks the central nervous system, causing severe neurological disorders, convulsions, and coma, and the disease is nearly always fatal without treatment. While cases are currently declining, 8.5 million people live in areas at moderate to very high risk in sub-Saharan Africa, particularly in the Democratic Republic of Congo (DRC), which is home to two-thirds of all cases.
- In 2018-9, DNDi and partners, in particular Sanofi, delivered the first all-oral treatment for sleeping sickness, fexinidazole, that is currently being implemented in endemic countries. Fexinidazole eliminates the need for systematic hospitalization of patients and will improve the management of sleeping sickness patients, and facilitate the integration of sleeping sickness treatment into health systems. But a second all-oral, and importantly single-dose treatment, acoziborole, is now being tested in Phase II/III clinical studies in DRC and Guinea. Acoziborole is the first new chemical entity from DNDi’s own lead optimization program to have entered clinical development.
- If successful, acoziborole would bring a simple, safe, and effective treatment that – together with a rapid diagnostic test – could be administered at point of diagnosis. This important feature would be a game-changer for sustainable elimination of the disease.
Both these investments build on previous financial support to DNDi’s earlier-stage R&D activities in filarial diseases and sleeping sickness projects from the Bill & Melinda Gates Foundation.
‘We are extremely grateful to the Bill & Melinda Gates Foundation for its long-standing trust over the past 12 years, our close partnership, and its renewed support to our mission to improve the quality of life and the health of millions of people living with these diseases,’ said Dr Pécoul.
The Drugs for Neglected Diseases initiative (DNDi) is a collaborative, patient needs-driven, not-for-profit research and development (R&D) organization that develops safe, effective, and affordable treatments for the millions of people across the world affected by neglected diseases, notably human African trypanosomiasis (sleeping sickness), leishmaniasis, Chagas disease, filarial infections, mycetoma, paediatric HIV, and hepatitis C. Since its inception in 2003, DNDi has delivered eight new treatments: two fixed-dose antimalarials (ASAQ and ASMQ); nifurtimox-eflornithine combination therapy (NECT) for late-stage sleeping sickness; sodium stibogluconate and paromomycin (SSG&PM) combination therapy for visceral leishmaniasis in Africa; a set of combination therapies for visceral leishmaniasis in Asia; paediatric dosage forms of benznidazole for Chagas disease; a ‘super-booster’ therapy for children co-infected with HIV and TB; and fexinidazole, the first all-oral drug for sleeping sickness.
Ilan Moss, DNDi Communications Manager, DNDi North America
DNDi Press Manager
Photo credit: Xavier Vahed-DNDi