POM and POC

Designing the Proof of Concept (PoC) and Proof of Mechanism (PoM) studies with the clinical plan and regulatory strategy in mind

Proof of Mechanism (PoM)

  • Usually in Healthy Volunteers, Phase 1 study if Phase1/2 in patients
  • Essential for the selection of appropriate dose for PoC, disease model, and biomarkers
  • Investigate drug concentration at the target site of action
  • Investigate drug engagement with target molecular receptor or enzyme (e.g., Receptor binding)
  • Investigate if the biological response matches the expected effect
  • Analyze PK/PD relationship 

Proof of Mechanism (PoC)

  • Phase 2 study in patients with the selected disease
  • Inform on indicative effectiveness and desired clinical activity
  • PoC lacks statistical power
  • High-quality PoC study yield insight into drug interaction with disease physiology
  • High-quality reads-out enable highly educated Go/noGo decisions 

The PoM and PoC trials’ design is based on the Pillar Theorem:

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