We are leveraging our biologics and Chaperone-Advanced Replacement Therapy (CHART®) to develop ATB200/AT2221, a novel treatment paradigm that consists of a uniquely engineered recombinant human acid alpha-glucosidase (rhGAA) enzyme with an optimized carbohydrate structure (designated ATB200), administered with a small molecule pharmacological chaperone (designated AT2221).
Amicus has initiated a clinical study to investigate the safety and pharmacokinetics of ATB200/AT2221 as a fixed-dose combination therapy in patients with Pompe disease.
In 2013, Amicus completed a Phase 2 safety and pharmacokinetics study, Study 010, that investigated single ascending oral doses of a pharmacological chaperone co-administered with alglucosidase alfa in patients with Pompe disease. Each patient received one infusion of ERT alone then a single oral dose of the pharmacological chaperone just prior to the next ERT infusion.
ATB200/AT2221: Phase 1/2 Safety Study (ATB200-02 Study)
Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous ATB200 Co-administered with Oral AT2221 in Patients with Pompe Disease
Active, not recruiting
Open-label, dose-escalation study of patients 18 to 65 years with a diagnosis of Pompe disease Pompe disease patients to assess if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.