Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study

Category: Archive
May 14, 2017

CRANBURY, N.J., May 15, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD) today announced positive functional data from initial patients in a global Phase 1/2 study (ATB200-02) to investigate ATB200/AT2221 in patients with Pompe disease. Patients who completed six months of treatment with ATB200/AT2221 showed improvements in the six-minute walk test (6MWT) distance and other measures of motor function, in addition to stability or improvements in forced vital capacity (FVC). Consistent with previous results presented at the 2017 WORLDSymposium™, patients treated with ATB200/AT2221 continue to show improvements in biomarkers of muscle damage and disease substrate.

“We are very pleased to see improvements in six minute walk distance and other measures of motor function in both naïve and ERT-switch patients, as well as stability or improvements in forced vital capacity. The consistency and magnitude of improvements exceeded our expectations and follow the initial improvements seen on key biomarkers of muscle damage and disease substrate,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “These preliminary functional results are very encouraging and suggest a clinically meaningful improvement for patients.  We look forward to additional data from all patients in the third quarter as we continue in our mission to develop an improved treatment option for people living with Pompe disease.”…

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