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Strong Proof-of-Concept Data for Chaperone's Ability to Stabilize and Enhance Activity and Uptake of Currently Marketed ERT Products for Pompe Disease
Results to be Presented at LDN WORLD Symposium in February 2013Initiation of Repeat-Dose Pompe Study Anticipated in 3Q13
CRANBURY, N.J., Jan. 4, 2013 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced positive preliminary results from all 4 dose cohorts in a Phase 2 study (
Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (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 data
1 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.
John F. Crowley, Chairman and Chief Executive Officer stated, "Study 010 has established human proof-of-concept that AT2220-ERT co-administration increases GAA enzyme activity in muscle. We look forward to initiating our repeat-dose clinical study to investigate the effect of AT2220-ERT co-administration on ERT stability and activity, ERT-related immunogenicity, and other clinical measures. We believe that co-administration may deliver significant benefits compared to ERT alone and become an important therapy for people with Pompe disease."