Case Study: Developing a Complex Dosing Drug Schedule for a DART Pompe Disease Study

Amics Therapeutics, based in Cranbury, NJ (USA), focuses on finding treatments for a DART Pompe disease studyrange of devastating and rare and orphan diseases. Its lead biologics program is ATB200/AT2221, a uniquely engineered Pompe disease enzyme replacement therapy that will be administered in combination with a pharmacological chaperone and tested in multiple human genetic diseases.

A Short-Half Life Investigational Drug Suggests an Every-Other-Day Complex Dosing Regime

The Amicus and Covance dialogue began at the 2017 Society of Toxicology conference and resulted in a large series of DART studies being awarded to Covance at its center for biotech excellence in Greenfield, IN. Early in the engagement, Covance’s most experienced US-based DART team member provided consultation on study design to make it more consistent with FDA guidelines and expectations. Continue reading