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Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Posted on: December 22, 2015

CRANBURY, N.J., Dec. 22, 2015 (GLOBE NEWSWIRE)—Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug Administration (FDA), is now effective which allows Amicus to begin site initiation and enrollment of a Phase 1/2 study of ATB200 in patients with Pompe disease. Amicus intends to seek regulatory authorization to evaluate ATB200 in European patients as well. This novel enzyme replacement therapy (ERT) consists of a uniquely engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure, administered in combination with a small molecule pharmacological chaperone (AT2221). In preclinical studies, ATB200 was associated with increased tissue enzyme levels and reduced substrate, which was further improved when AT2221 was co-administered with ATB200.

“The start of our Pompe clinical program is a major step forward for people living with Pompe disease and their families.  This novel ERT has been developed over the past several years by Amicus scientists and represents the potential for a very differentiated biologic treatment option for patients. There is still so much need in the Pompe community and we are dedicated to this mission. We look forward to seeing data from this study in 2016,” stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc.

The key features of this Phase 1/2 study include:

  ~Open-label, dose-escalation to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous ATB200 co-administered with oral AT2221
  ~Subjects in the first cohorts will be adult Pompe patients switched from currently marketed ERT
  ~Primary treatment period will be 18 weeks, with all patients eligible to enroll in an open label extension study
  ~Dose selection for a Phase 3 clinical study will be informed by the findings from this Phase 1/2 study

Amicus plans to present additional study design details in early 2016.  First patient dosing is expected in early 2016.

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