Amicus Therapeutics (Nasdaq: FOLD) today announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold for the AT2220 (1-deoxynojirimycin HCI) Investigational New Drug Application (IND). AT2220 is a pharmacological chaperone in development as a treatment for Pompe disease. Based on data provided by Amicus, the FDA agreed with the Company's proposal to resume clinical development of AT2220, starting with a new Phase 2 study designed to evaluate the use of AT2220 when co-administered with enzyme replacement therapy (ERT) in subjects with Pompe disease. The Company expects to commence this study in the first half of 2011 and to report preliminary results in the second half of 2011.
Co-administration of Pharmacological Chaperones with ERT
Amicus previously reported promising preclinical data demonstrating that the co-administration of a pharmacological chaperone with ERT has the potential to address key limitations of ERT. The addition of a pharmacological chaperone has been shown to prevent the loss of activity of ERT in the circulation, increase tissue uptake, and increase substrate reduction in multiple disease-relevant tissues. Preclinical proof of concept has been established for Fabry disease and Pompe disease.