Phase 2 Study of AT2220 for Pompe Disease

Amicus Therapeutics announced commencement of its Phase 2 study of AT2220 (1-deoxynojirimycin HCI) coadministered with enzyme replacement therapy (ERT) for the treatment of Pompe disease. The FDA has removed the clinical hold for the AT2220 Investigational New Drug Application (IND), which was put in place after adverse events were reported from a Phase 2 trial evaluating AT2220 as monotherapy in adults with Pompe disease. After completing a thorough investigation of the events, including the completion of additional preclinical and Phase 1 studies, Amicus decided to continue development of AT2220 as a pharmacological chaperone when coadministered with ERT.

Pharmacological chaperone technology involves the use of small molecules that selectively bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. AT2220 binds to and destabilizes the acid α-glucosidase (GAA) enzyme, which is deficient in Pompe disease, and restores its biological function of degrading glycogen substrate in lysosomes.

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