Proof of concept data in animal models are preclinical in vivo results generated in disease-relevant models to test whether a candidate intervention can produce the intended biological and therapeutic effects. These studies go beyond in vitro target engagement or mechanism-of-action studies. Proof of concept data from animal models provides insight into whether the treatment with the experimental medication is likely to translate into clinical benefit, thereby supporting the progression of a project and informing early clinical trial design.
In anti-infective and other therapeutic areas, proof-of-concept data typically come from validated animal disease models that reproduce key aspects of human pathology and pharmacology and include quantitative measures of efficacy (for example, reductions in pathogen burden, improvements in organ function, or survival). These studies help identify effective dose–exposure–response relationships, refine dosing regimens, and support regulatory and portfolio decisions, while recognising the inherent limitations and uncertainties of extrapolating from animal to human studies.