In a press release, Amicus Therapeutics announce upcoming activities in the chaperones study:
“Amicus has commenced activities for a Phase 2 study of AT2220 co-administered with ERT for Pompe disease. The Company expects to dose the first patient during the third quarter of 2011, and anticipates results in the first half of 2012. The broader use of pharmacological chaperones co-administered with ERT represents an important expansion of the Company’s chaperone technology platform. Preclinical studies have shown that pharmacological chaperones bind to and stabilize the infused ERT, potentially improving its pharmaceutical and pharmacological properties. Preclinical proof-of-concept studies in Fabry and Pompe diseases have demonstrated that a pharmacological chaperone co-administered with ERT can prevent the loss of activity of ERT in the circulation, increase tissue uptake, and increase substrate reduction.”
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