Amicus reports function outcomes data from Phase I/II of Pompe's disease candidate
Amicus Therapeutics Inc. (NASDAQ:FOLD) reported functional outcomes data from the Phase I/II ATB200-02 Study showing that IV ATB200/AT2221 improved muscle and pulmonary function in patients with Pompe's disease. Amicus CEO John Crowley told BioCentury the company expects to begin a Phase III trial sometime next year, but that Amicus is also working with regulators to "find the fastest path available to bring this medicine to patients."
The open-label, international trial enrolled 20 patients across 3 cohorts: ambulatory enzyme replacement therapy (ERT)-switch patients; non-ambulatory ERT-switch patients; and ERT-naïve patients...
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