Amicus announces additional results from ATB200-02 study

Amicus announced additional results from a global Phase 1/2 clinical study, or ATB200-02, to investigate AT-GAA in patients with Pompe disease, an inherited lysosomal storage disorder caused by an enzyme deficiency that leads to accumulation of glycogen in cells. Patients treated with AT-GAA for up to 18 months showed improvements in six-minute walk test, or 6MWT, distance and other measures of motor function and muscle strength, stability or increases in forced vital capacity, or FVC, and durable reductions in biomarkers of muscle damage and disease substrate. These clinical results are being featured at the International Annual Congress of the World Muscle Society. Safety and tolerability data in all 20 patients reflect a maximum of 28+ months of treatment. To date, adverse events have been generally mild and transient. Data on functional outcomes are available for 19 of the 20 patients enrolled. Muscle function improved in 17 of 19 patients at month 12. Muscle function improved in 17 out of 18 patients with available data at month 18. Six-minute walk test distance, a primary measure of motor function in Pompe disease patients, improved in both ERT-naive and ERT-switch patients with continued benefit observed out to month 18. Improvements were generally consistent across both cohorts. three of the four non-ambulatory ERT-switch patients showed improvements in upper extremity strength from baseline to month 18, as measured by quantitative muscle testing and manual muscle testing. Pulmonary function improved in ERT-naive patients and was generally stable in ERT-switch patients. Treatment with AT-GAA resulted in persistent and durable reductions in key biomarkers of muscle damage and disease substrate across all patient cohorts out to month 18 and continue to suggest a positive effect on muscle tissue.

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