Roadmap for development of therapeutics for helminths

What are we trying to achieve and why? What is the problem we are trying to solve?

To develop a minimum acceptable target product profile (TPP) for a novel active pharmaceutical/phytochemical ingredient (API) with anthelmintic activity

What are the scientific and technological challenges (knowledge gaps needing to be addressed)?

• A TPP is a strategic planning tool widely used across therapeutic areas, as a guide in research and development during the search for a new API. The TPP is useful to identify critical attributes of a potential novel API before its development begins, to ensure that the final product is adapted and responds to the needs of the end-users. The most important challenge here is to elaborate a minimum viable TPP useful to guide the development of a novel therapeutic tool in parasite control. The TPP should include the following desirable/ideal promotional claims for a new API with anthelmintic properties:
• Novel chemical class. New mode of action
• Broad spectrum of anthelmintic activity (nematodes, trematodes, cestodes)
• Activity against nematodes (adult and larval stages) R to existing anthelmintic molecules
• Well tolerated in all animal species. Low tissues residues. Minimum milk and meat withdrawal times required
• Easy to be formulated for administration by different routes
• Compatible with other drug medications. Potential to be used in combination with other ATH compounds
• Adequate quality at an affordable cost in different worldwide livestock production systems
• User and environmental-friendly

What approaches could/should be taken to address the research question?

The elaboration of a TPP to follow the efficacy, quality and safety assessments for a novel API with anthelmintic activity is a main initial challenge to be achieved. The “Attributes” (as listed in attached page below) may be achieved to guide the assessment of the clinical efficacy and safety in the process of development of any potentially new compound with improved anthelmintic activity

What else needs to be done before we can solve this need?

Further epidemiological, genetic and pharmaco-parasitological based research to understand the mechanisms of resistance in different helminth parasites of economic relevance in livestock animals. This is particularly relevant for cattle nematodes, where we still have some efficacious tools within the existing anthelmintic drugs and/or their combined use to expand the spectrum/efficacy

Existing knowledge including successes and failures

The TPP needs to define the properties of a new molecule (synthetic or natural phytochemical) to make it economically viable (worth developing). A reasonable body of information and knowledge on different strategies to improve parasite control in livestock animals under situations of anthelmintic resistance is available and it may contribute with any new development process. The accumulated knowledge for existing/traditional anthelmintic molecules should be a relevant guide in the process of development of a new API to be used in anthelmintic therapy in livestock animals.