Phase IIb Design

Goal: Definition of doses and regimen to test in Phase IIb

Available data

• Plasma concentration measurements in healthy volunteers (Phase I) and patients (Phase IIa)
• Efficacy readout in patients (Phase IIa)
• IV administration only in patients. SC in healthy volunteers.

Questions

• Are the pharmacokinetics different in patients and healthy volunteers?
• What is the SC bioavailability and the rate of absorption?
• Which SC doses and regimen would be feasible in order to reach a minimum efficacious threshold at Cmin?
• What is a reasonable concentration threshold for efficacy?

Approach

• Integration of all available data
• Dose/concentration/efficacy response characterization
• Simulation based assessment of candidate dose scenarios is human

Benefit for Client’s Project

• Better understanding of patient PK
• More informed dose selection
• Optimization of information generated in Phase IIb study (informing dose/concentration/response relationship)