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Define Best-In-Class Profile

Leveraging Competitor Data to Define Best-In-Class Inhibitor With an Improved Safety Window
Situation: 

A decision was made to halt development on a high value target due to mechanism related safety challenges. Subsequent to that decision, two competitor molecules showed a modest therapeutic window in the clinic, despite not showing any selectivity in vitro and in cellular assays. It was unclear which properties to optimize to produce a differentiated best-in-class molecule with an improved therapeutic window.

Solution: 
  1. Built a systems pharmacology model of competitor compounds
  2. Identified potential contributors to therapeutic window
  3. Worked with the team to identify compounds and experiments to validate the mechanism
  4. Determined the best-in-class compound profile
Result: 
  • A molecule matching the best-in-class profile has been advanced to preclinical development
  • Convinced management to support a program despite being years behind the frontrunners
  • Accelerated timelines to deliver a truly differentiated best-in-class compound