DNDI-6148

DNDI-6148 is an investigational oral benzoxaborole small molecule developed by the Drugs for Neglected Diseases initiative (DNDi) in collaboration with Pfizer (formerly Anacor Pharmaceuticals). It was primarily designed for the treatment of leishmaniasis, including visceral leishmaniasis (VL) and cutaneous leishmaniasis (CL), and has also shown potential efficacy against Chagas disease. The compound acts as a potent inhibitor of the Leishmania cleavage and polyadenylation specificity factor 3 (CPSF3) endonuclease, an essential enzyme involved in parasite mRNA processing. Although a Phase I single ascending dose study in healthy volunteers completed in early 2022 demonstrated that the drug was safe and well-tolerated, its clinical development has been deprioritized and paused due to preclinical signals of reproductive toxicity.