Phase II success for AT 2220 in Pompe Disease
Amicus Therapeutics has announced positive preliminary results from all 4 dose cohorts in a Phase II study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT 2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease (Myozyme and Lumizyme). Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are the first and only approved treatments for Pompe disease. Based on the Study 010 results, Amicus expects to initiate a repeat-dose clinical study in the third quarter of 2013.
For people with Pompe disease, deficient GAA enzyme leads to the accumulation of glycogen in tissues affected by disease (primarily muscle). Preclinical data1 demonstrated that AT2220 in combination with ERT enhances rhGAA enzyme activity, reduces glycogen accumulation, and potentially mitigates ERT-related immunogenicity in a mouse model of Pompe disease. In Study 010, co-administration of AT2220 to Pompe patients increased rhGAA enzyme activity and enhanced rhGAA enzyme uptake into muscle tissue compared to ERT alone.Learning Zones
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