The following information is taken from the Pompe Community Update Newsletter with permission by Amicus:
Earlier in the Pompe Program, the use of AT2220 as a monotherapy for Pompe disease was explored. However, after two participants experienced serious adverse events in a Phase 2 trial in February 2009, AT2220 was put on clinical hold. These events were thoroughly studied by the company and the principal investigator. The events resolved for the patients who subsequently returned to clinical baseline status. Additional questions about the drug and its dosing were investigated, including through an innovative Phase 1 study of muscle tissue pharmacokinetics in healthy volunteers. This study measured how particular muscle tissue absorbed, metabolized and eliminated AT2220. (The results of this distinctive study were presented at the 7th Annual WORLD Symposium 2011.) After careful review of these new data, the company decided to shift its AT2220 research from monotherapy to co-administration. After data submission and discussions with the FDA, AT2220 was released from clinical hold in March 2011, thus enabling Amicus to proceed with the Phase 2 co-administration study of AT220 and ERT.