New Study Confirms Efficacy of Emodepside Against Parasitic Worm Infections

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Emodepside trial lab on Pemba Island, Tanzania. Photo credit: Lyndsay Taylor.
Emodepside trial lab on Pemba Island, Tanzania. Photo credit: Lyndsay Taylor.
Researchers at Swiss TPH have demonstrated that the novel drug candidate, emodepside, is highly effective in treating parasitic worm infections in humans, particularly hookworms. The results of a Phase 2b trial, published today in The Lancet, confirmed the drug’s strong efficacy and safety profile, building on the promising outcomes of an earlier Phase 2a study. Swiss TPH is collaborating with Bayer to further develop emodepside.

Soil-transmitted helminth infections are caused by different species of parasitic worms, including whipworms, hookworms and roundworms. Worldwide, more than 1.5 billion people are infected with at least one soil-transmitted helminth, with most of the infected population living in lowand middle-income countries. Hookworms alone are responsible for an estimated 460 million cases worldwide.

Infected people can experience symptoms such as stomach pain, diarrhea and anemia, while heavy infections can lead to malnutrition, and impaired physical development.

Safe drugs for the treatment of these infections are available but the efficacy varies widely. The current treatments recommended by the World Health Organization (WHO) are albendazole and mebendazole. With the looming threat of drug resistance, the need for new drugs is all the more urgent.

A promising new drug candidate

Emodepside, a promising anthelmintic candidate originally developed for veterinary use, has shown great potential in treating parasitic worm infections in humans. The results of a new Phase 2b trial, published today in The Lancet, confirmed the drug’s strong efficacy and safety profile, particularly in treating hookworms, building on the promising outcomes of a previous 2a clinical trial. Emodepside has the potential to become a critical treatment option, for individual cases or as part of existing programs for parasitic worm infections.

The study included 293 participants aged 12 to 60 years from Pemba Island, Tanzania, who were infected with hookworms. Researchers at Swiss TPH in collaboration with the Public Health Laboratory Ivo de Carneri (PHL-IdC) tested a single 30 mg dose of emodepside against the standard 400 mg dose of albendazole, focusing on hookworms. Emodepside cured 96.6% of patients, compared to an 81.2% cure rate with albendazole.

A previous study by Swiss TPH demonstrated that a 15 mg dose of emodepside cured 100% of individuals infected with whipworm, a level of efficacy that had not been previously achieved with current treatments.

After analyzing the data from both the Phase 2a and 2b trials, researchers identified the 15 mg dose as the best option for the upcoming Phase 3 trial, with a focus on treating whipworm. "A 100% cure rate of whipworm was already achieved at the 15 mg dose and caused fewer side effects than the 30 mg dose," said Lyndsay Taylor, PhD candidate at Swiss TPH and first author of the study. "What we found is that emodepside is highly effective and safe for treating hookworms, whipworms and roundworms."

As development continues, more information will be gathered about emodepside’s safety, long-term effectiveness, and optimal use. Jennifer Keiser, Head of the Helminth Drug Development unit at Swiss TPH added: "There is a very good chance that emodepside could be a game changer in the fight against helminthiasis and could play a major role in global deworming efforts."

Swiss TPH is collaborating with Bayer to further develop emodepside and fill the empty anthelmintic pipeline. The Phase 3 trial is expected to start next year on Pemba Island and the Philippines.

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