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Go/No Go Decision after Ph1

Driving a Critical Go/No Go Decision After Ph1
Situation: 

A promising drug candidate achieved its Ph1 PK endpoints in healthy volunteers. Since PD could only be assessed in patients, we were asked to provide a critical analysis of Ph1 results.

Solution: 
  1. Developed a mechanistic PK/PD model
  2. Set model parameters to match in vitro and in vivo values from literature and in-house data
  3. Calibrated unkown model parameters to Ph1 PK data
  4. Identified key sensitive parameters and missing data
  5. Influenced team to collect additional data that increased the prediction confidence
  6. Identified a key liability and predicted a path forward
Result: 
  • Influenced management to discontinue lead, saving $15M milestone payment
  • Canceled Ph2, saving an estimated $100M
  • Supported development of improved molecule with model-defined criteria
  • Accelerated Ph1 for the improved molecule
  • Accurately predicted Ph1 PK of improved molecule currently in Ph2