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Collaboration for innovation: Establishment of a pan-nematode drug development platform

Periodic Reporting for period 2 - HELP (Collaboration for innovation: Establishment of a pan-nematode drug development platform)

Reporting period: 2021-03-01 to 2022-08-31

Infections with helminths (worms) constitute the largest proportion of Neglected Tropical Diseases (NTDs), with an estimated 1.8 billion infected individuals worldwide and over 5 billion people living in endemic countries at risk of further infections. Worm-infections strongly affect mental, physical and economic human development.
To target the elimination of parasitic worms, a drug research and development (R&D) pipeline is urgently needed to provide new chemotherapeutics and eventually help reaching the 2030 sustainable development goals (SDGs) on Good Health and Well-being.
The Helminth Elimination Platform (HELP) project aims to establish such a pipeline for drug development against parasitic worm infections, with a focus on onchocerciasis (filarial infections) and soil-transmitted helminth (STH) infections. To attain this ambitious goal and to deliver new highly efficacious drugs as soon as possible, HELP intervenes at different stages of the drug development process. In particularly, it aims to:
• Identify new drug candidates (early stage of drug development / discovery phase).
• Progress the candidates with the best profile through standard pre-clinical studies.
• Advance Corallopyronin A (CorA), a novel compound with proven efficacy against filariae, towards phase 1 clinical trials.
• Conduct two phase 1 clinical trials with oxfendazole and oxantel pamoate under current regulatory guidelines to eventually allow for filing with a regulatory authority.
With this strategy, the consortium will establish a much-needed drug R&D pipeline and expects to have a strong positive impact on the society by reducing the disease burden and its social and economic consequences in the endemic countries in the future.
The HELP project has so far performed the following work:
Industrial partners Celgene and Bayer Animal Health / Elanco have provided pre-selected compounds to the academic partners (Swiss TPH, UKB, MNHN and UOB). The compounds were tested for their activity on different worm species in the laboratories of the academic partners. A total of 23 promising candidates were identified and subsequently tested for their activity against infection with filariae and STH in animal models (mice, hamsters, jirds). Of all the compounds tested, at least five would be suitable for further development in standard pre-clinical development packages.

Swiss TPH together with the partners DNDi and IHI have prepared a Phase 1 clinical trial for Oxfendazole (testing in healthy, adult volunteers). After obtaining ethical approvals from the Tanzanian and Swiss authorities the trial recruitment successfully has been finalized.
After completing a data review for oxantel pamoate and obtaining regulatory advice by Swissmedic in the first period of the project, a clinical trial with oxantel pamoate is currently being prepared by Swiss TPH, DNDi and IHI. Additionally, the oxantel pamoate tablets to be used in the clinical trial were developed by DNDi, since the product is not available as a tablet on the market. We expect the final product to be synthesized in early 2023.
In addition, many activities for the dissemination of HELP-related activities have been performed under the lead of DNDi by all partners. Of note, the content of the HELP-website has been expanded and continuous social media posting on current issues was very active.
At least five compounds that are active against different worm species of the laboratories have been tested to the level that they could enter the next phase of non-clinical development.
Oxantel pamoate, which is not on the market in a tablet formulation that is approved for human use, has been developed in an optimized and child friendly formulation, which can be used in clinical trials (and potentially beyond).