Amicus Therapeutics (FOLD) Announces Positive Pompe Program Updates
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Amicus Therapeutics (Nasdaq: FOLD) today announced additional positive preliminary results from an ongoing Phase 2 open-label drug-drug interaction study (Study 010) to evaluate the safety and plasma pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease (Myozyme® and Lumizyme®).
These Phase 2 results were presented in a poster1 at the 17th International World Muscle Society Congress in Perth, Australia. The Company also announced initial findings from ex vivo studies to characterize the immunogenicity of Myozyme and Lumizyme with and without AT2220.
For people with Pompe disease, deficient GAA enzyme leads to the accumulation of glycogen in tissues affected by disease (primarily muscle). Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are the first and only approved treatments for Pompe disease. The clinical benefit of Myozyme and Lumizyme may be limited by low stability of the recombinant enzyme at neutral pH and body temperature, modest tissue uptake, and immune responses that affect tolerability and efficacy. Published preclinical data2 suggest that AT2220 in combination with this ERT may improve rhGAA enzyme activity, reduce glycogen accumulation, and potentially mitigate ERT-related immunogenicity in patients with Pompe disease.
Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics said, "We continue to be encouraged by preliminary results from Study 010. Following co-administration, we have seen consistent increases in the amount of active enzyme in plasma in all patients, with the greatest increase in Cohort 3. We look forward to completing Study 010 in the fourth and highest dose cohort, and expect to report results by the end of the year."
These Phase 2 results were presented in a poster1 at the 17th International World Muscle Society Congress in Perth, Australia. The Company also announced initial findings from ex vivo studies to characterize the immunogenicity of Myozyme and Lumizyme with and without AT2220.
For people with Pompe disease, deficient GAA enzyme leads to the accumulation of glycogen in tissues affected by disease (primarily muscle). Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are the first and only approved treatments for Pompe disease. The clinical benefit of Myozyme and Lumizyme may be limited by low stability of the recombinant enzyme at neutral pH and body temperature, modest tissue uptake, and immune responses that affect tolerability and efficacy. Published preclinical data2 suggest that AT2220 in combination with this ERT may improve rhGAA enzyme activity, reduce glycogen accumulation, and potentially mitigate ERT-related immunogenicity in patients with Pompe disease.
Pol F. Boudes, MD, Chief Medical Officer of Amicus Therapeutics said, "We continue to be encouraged by preliminary results from Study 010. Following co-administration, we have seen consistent increases in the amount of active enzyme in plasma in all patients, with the greatest increase in Cohort 3. We look forward to completing Study 010 in the fourth and highest dose cohort, and expect to report results by the end of the year."
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