Developing treatments for infectious and neglected tropical diseases

Through NITD, we continue to research and develop a promising portfolio of novel drug candidates for the treatment of neglected tropical diseases that affect around 1.6 billion people worldwide.

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Invested in R&D to discover new and better treatments for infectious and neglected diseases (USD)

We made progress on a potential first-in-class compound to treat dengue fever. Although this is the most common vector-borne viral disease in the world, there is currently no specific medicine to treat it. The WHO had listed dengue as a potential threat among 10 diseases, and recent outbreaks – with nearly twice as many dengue infections as in the past 20 years – confirm this. The compound identified by NITD has demonstrated activity against all four dengue serotypes. We completed initial preclinical safety studies and expect to begin clinical trials in 2021.

Cryptosporidium infection is the leading cause of parasitic diarrhea, a major cause of death among young children in developing countries. We reported in 2019 the discovery of the apicomplexan lipid kinase PI4K as a potential molecular target, and NITD has advanced a promising drug candidate, EDI408, through preclinical studies. EDI048 is active in the intestinal enterocytes, where the parasite resides, and is quickly metabolized by the body – a desirable feature in addition to the compound’s safety profile. Although diarrhea is one of the top three killers of children globally, there are only a few novel medicines currently being developed to treat it. In 2020, NITD was awarded a three-year grant from the Wellcome Trust for drug discovery science in cryptosporidiosis and Chagas disease.

With an estimated 50 000 to 90 000 new cases per year, visceral leishmaniasis is the most serious form of leishmaniasis, causing fever, weight loss, spleen and liver enlargement, and death if left untreated. In February, we announced a collaboration with the Drugs for Neglected Diseases Initiative (DNDi) to jointly develop LXE408, a first-in-class inhibitor of the kinetoplastid proteasome, for the treatment of visceral leishmaniasis. Within the scope of the agreement, Novartis is responsible for completing Phase I clinical trials. Upon approval, we have committed to distributing the drug on an affordable basis worldwide to maximize access in endemic countries. DNDi will lead Phase II and III clinical development, with the first Phase II study scheduled to start in early 2021 in India. Additional trials are planned in East Africa, which has the highest burden of visceral leishmaniasis.