The Drugs for Neglected Diseases initiative (DNDi) is very pleased to announce two 2021 Projects of the Year, which recognize DNDi R&D teams and key partners for outstanding progress in the areas of drug discovery and clinical research. Nominated by the DNDi Scientific Advisory Committee and selected by the DNDi Executive Board, the 2021 awards recognize two projects from among the more than 50 active projects in DNDi’s R&D portfolio.
S07 series lead optimization for visceral leishmaniasis – 2021 Project of the Year in drug discovery
Existing treatments for visceral leishmaniasis can be painful, toxic, lengthy, costly, and poorly adapted for use in the most affected communities. To provide people of all ages with safer, simpler, and more effective treatment options, DNDi and partners are discovering entirely new compounds that can be developed into all-oral drugs.
The S07 series is the first project to emerge into lead optimization from the NTD Drug Discovery Booster, a global consortium of pharmaceutical companies that have opened their libraries of millions of unique molecules so that researchers can screen them in silico and in vitro to identify new potential treatments for neglected tropical diseases. With support from the Japanese Global Health Innovative Technology (GHIT) Fund, among other donors, more than 18,000 compounds have been screened for antiparasitic activity to date through this international initiative.
Following Discovery Booster efforts conducted in partnership with Takeda Pharmaceutical Company Limited to identify and advance novel compounds against visceral leishmaniasis, the DNDi-Takeda collaboration is now progressing medicinal chemistry optimization for S07 series lead compounds to further enhance their safety and efficacy profiles. With support from the GHIT Fund, the partnership aims to advance an optimized lead to deliver a pre-clinical candidate compound by 2023.
‘The S07 series is a prime example of the advances our discovery partnerships are making to find the missing tools we need to meet patient’s needs and reach global targets for neglected tropical diseases,’ said Dr Laurent Fraisse, Research and Development Director at DNDi. ‘We congratulate Takeda and the DNDi Drug Discovery team for their progress, which demonstrates that innovative collaborations like the NTD Drug Discovery Booster can play a powerful role in accelerating scientific discovery and future medical innovation.’
Acoziborole for sleeping sickness – 2021 Project of the Year in clinical research
DNDi’s goal is to revolutionize the treatment of sleeping sickness – or human African trypanosomiasis (HAT) – by replacing toxic, cumbersome medicines with simple, all-oral cures that can help eliminate the disease altogether. In 2018, DNDi and partners completed the development of the first all-oral treatment for sleeping sickness, fexinidazole.
Together with Sanofi, our industrial partner for fexinidazole, we are now working to complete the development of acoziborole, a single-dose oral treatment for sleeping sickness that can be administered at the point of diagnosis.
‘Acoziborole can provide a dramatic boost for sleeping sickness ‘test-and-treat’ programmes – particularly in remote communities and settings affected by conflict and instability – and help anchor efforts to sustain elimination of the disease,’ said Dr Fraisse. ‘These advances in clinical development would not be possible without the steady commitment of Sanofi, the Institut National de Recherche Biomédicale (INRB) of the Democratic Republic of Congo (DRC), the National HAT Control Programmes of the DRC and Guinea, and the DNDi sleeping sickness team.’
In 2012, acoziborole became the first new chemical entity to enter clinical development from DNDi’s own lead optimization programme. Last year, our teams completed 18-month post treatment follow-up for all patients in DNDi’s pivotal Phase II/III clinical trial for the drug in the Democratic Republic of Congo (DRC) and Guinea. Following its approval, Sanofi will be responsible for the manufacturing, supply, registration, and distribution of acoziborole. The treatment will be provided to patients free of charge through affected countries’ public health systems thanks to a 20-year collaboration between Sanofi and the World Health Organization.