Early Development of Erenumab for Migraine Prophylaxis

Modern neuroscience drug development is challenging, success rates are low, and costs are high. A rigorous early development program, which utilizes biomarkers to answer fundamental questions before advancement to later development, will allow us to progress and develop neuroscience medications with higher success rates. Fundamental questions to be answered in early development include the following: does the drug get to the target, does it bind, and does it have an effect? This chapter illustrates some of these concepts by highlighting the development of erenumab, a monoclonal antibody targeting the calcitonin-gene-related protein (CGRP) receptor for migraine prophylaxis, as a case study of how a peripheral target engagement biomarker was utilized to determine dose selection for clinical phase 2 studies.