Amicus has amended the protocol for the 6-month Phase 2 clinical trial of Plicera patients naive to ERT to include modified doses and dose regimens. Amicus expects the results of this study to be available in 2009. In addition, in the second half of 2008, the Company expects to initiate a longer-term study in individuals switching from ERT to Plicera.
AT2220 (1-deoxynojirimycin HCl) is an investigational, oral therapeutic drug candidate being developed for the treatment of Pompe disease. At the ACMG meeting in March, clinical investigators presented encouraging results from an ex vivo response study in cells from patients with Pompe disease as well as three Phase 1 clinical trials of AT2220 in healthy volunteers. The ex vivo response study was designed to test the effect of AT2220 on various Pompe mutations. Blood and skin samples were collected from 30 Pompe patients (26 adults, 3 juveniles and 1 infant) with a variety of different mutations in acid alpha-glucosidase (GAA), the target enzyme in Pompe disease. Cells from these samples where then treated with AT2220. Of the 26 patients with available data, 24 had cells that showed a dose responsive increase in GAA levels including 22 patients who had at least 1 copy of the common splice site mutation IVS1-13T > G. Data from the Phase 1 trials in a total of 72 healthy volunteers showed that AT2220 was generally safe and well tolerated at all doses.
In the second quarter of 2008, Amicus plans to initiate a Phase 2
clinical trial of AT2220 in patients with Pompe disease. The Company also
plans to consider the initiation of a clinical trial of AT2220-ERT
combination treatment in Pompe patients later in 2008
|SOURCE Amicus Therapeutics|
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