Clinical Trial Begins for New Drug to Combat Neglected Tropical Diseases

Pavankumar Kamat

January 12, 2022

UK researchers have begun the first clinical trial of a novel drug candidate against the neglected tropical diseases, lymphatic filariasis (LF) and onchocerciasis.

LF and onchocerciasis are severely disabling diseases caused by parasitic filarial nematodes. These are usually transmitted via other insects, such as blackflies for onchocerciasis (aka 'river blindness') or mosquitoes for LF. They affect over 150 million people globally, particularly in developing countries. A bacterial endosymbiont called Wolbachia plays a crucial role in the nematodes' lifecycle and is essential for their survival.

Current drug treatments primarily target the microfilarial stage of the nematodes and confer very little utility against the adult parasites. The molecule codenamed AWZ1066S has been designed to target Wolbachia and not the nematode itself, thereby disrupting the worm's lifecycle and resulting in a macrofilaricidal outcome.

Following successful pre-clinical studies, the phase 1 trial to establish the drug’s safety, tolerability and pharmacokinetics commenced on December 21, 2021. The first patient was administered AWZ1066S at the NIHR Royal Liverpool and Broadgreen Clinical Research Facility, based at Liverpool University Hospitals NHS Foundation Trust.

Potential Game Changer

The molecule was originally developed though the Liverpool School of Tropical Medicine's anti-Wolbachi (A·WOL) consortium, which is funded by The Bill & Melinda Gates Foundation.

The University of Liverpool's Department of Chemistry led the drug screening and optimisation efforts. The University has also collaborated with Eisai Co., to accelerate the drug development process. Professor Mark Taylor, director of the A·WOL Consortium believes "AWZ1066 has real potential to be a game changer".

Professor Paul O’Neill, University of Liverpool, who is leading the research, commented: "Our multi-parameter chemical optimisation approach has delivered a molecule with high potency and specificity against the target pathogen along with the desired oral exposures and preclinical safety profile. We are all excited to see this novel synthetic molecule enter human trials."

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