2019 R&D portfolio in review: Leishmaniasis

Short-term strategies to develop better treatment regimens using existing drugs progressed, with Phase IIb/III studies in Africa testing new regimens for visceral leishmaniasis (VL) and post-kala-azar dermal leishmaniasis (PKDL) and a Phase II study in South Asia testing a new regimen for PKDL. Results from HIV/VL Phase III studies in Ethiopia and India were presented to national and international authorities to inform review of HIV/VL treatment guidelines. In addition, multiple new chemical entities have progressed in pre-clinical and Phase I studies, marking significant progress toward DNDi’s long-term goal of developing an entirely new generation of all-oral drugs for VL.

For cutaneous leishmaniasis (CL), preliminary results from a Phase II study in Latin America showed the combination of thermotherapy with a shorter course of oral miltefosine to be significantly better than thermotherapy alone for the treatment of uncomplicated CL in the Americas.

2019 R&D portfolio in review: Chagas disease

Preliminary results of a Phase II study evaluating new therapeutic regimens of benznidazole showed that all treatment arms were effective and had good safety profiles. The two-week course of treatment with benznidazole alone was particularly promising, showing 83% efficacy while cutting treatment time by 75% compared to the standard eight-week treatment, with no patients discontinuing treatment due to side effects. Based on these results, DNDi is planning to run an international, multi-site confirmatory Phase III study.

2019 R&D portfolio in review: Filaria – river blindness

With first-in-human studies for emodepside in healthy volunteers successfully completed, preparations are underway to run a Phase II clinical trial at two sites in Ghana. In the Democratic Republic of Congo, training and trial site renovations are advancing ahead of a Phase II proof-of-concept study for TylAMac, which was shown to be safe and well-tolerated in Phase I studies. And new efforts to identify novel treatments against ‘nematode’ worms have kicked off following the launch of a new multidisciplinary consortium: the Helminth Elimination Platform (HELP).

2019 R&D portfolio in review: Mycetoma

In Sudan, the first-ever double-blind, randomized clinical trial for fungal mycetoma is ongoing and had enrolled 101 patients by January 2020. The primary objective of the study is to demonstrate the superiority of the new chemical entity fosravuconazole over the current standard treatment, itraconazole. The study’s targeted enrollment of 165 patients should be reached by the end of 2020.

Chagas Disease. A clinical approach

This book offers a comprehensive overview of Chagas disease, including its vectorial and congenital transmission, and molecular diagnosis, which is essential for screening, and developing and providing timely, effective anti-trypanosomal treatment.

SpringerLink, September 2019