Results of Chaperone/ERT Study Expected for Second Half of 2011

Category: Archive
January 10, 2011

In a press release, Amicus Therapeutics announce expected key milestones in 2011, among them results from a phase 2 study of AT2220 co-administered with Enzyme Replacement Therapy (ERT) for Pompe Disease.

“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. Preclinical proof of concept has been established for Fabry disease and Pompe disease… The Company expects to initiate a Phase 2 study with its pharmacological chaperone AT2220 co-administered with ERT for Pompe disease in the first half of 2011 and expects results from this study to be available in the second half of 2011. The Company intends to seek U.S. FDA approval to lift the current partial-hold on the AT2220 program as part of its development plan.”

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