Proof of Principle studies are an early stage of clinical drug development when a compound
has shown potential in animal models and early safety testing. This step of proof-of-principle
(PoP) or proof-of-concept (PoC) often links between Phase-I and dose ranging Phase-II
studies. These small-scale studies are designed to detect a signal that the drug is active on
a pathophysiologically relevant mechanism, as well as preliminary evidence of efficacy in a
clinically relevant endpoint. Sponsors use these studies to estimate whether their compound …