Amicus Therapeutics Initiates Trial Of AT2220 In Pompe Disease

Armen Hareyan's picture
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Amicus Therapeutics has initiated a Phase 2 clinical trial of AT2220 (1-deoxynojirimycin HCl), for the treatment of Pompe disease. Amicus will conduct the study in adult Pompe patients in clinical centers throughout North America and Europe. AT2220 is the third compound based on Amicus' pharmacological chaperone technology platform to enter Phase 2 clinical development.

"We look forward to evaluating AT2220 as a potential new oral therapeutic option for patients living with Pompe disease," said Barry J. Byrne, M.D., Ph.D., professor of pediatrics, molecular genetics and microbiology at the University of Florida in Gainesville and an investigator in the Phase 2 trial.

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AT2220 is designed to selectively bind to, stabilize and elevate the cellular activity of acid alpha-glucosidase (GAA), the enzyme deficient in Pompe disease. This deficiency leads to lysosomal accumulation of glycogen inside cells, which is believed to cause the various symptoms of Pompe disease.

Amicus is initiating the multi-national, open-label Phase 2 clinical trial designed to enroll 18 adult patients diagnosed with Pompe disease. The primary objective of the study is to evaluate the safety and tolerability of different dosing regimens of AT2220 over a 12-week period. The study will also explore certain pharmacodynamic and pharmacokinetic measures including the effect of treatment with AT2220 on GAA activity and on glycogen levels in various cells and tissues.

Additional objectives include preliminary assessments of pulmonary and skeletal muscle function. Participants who complete the study may be eligible to participate in a voluntary extension study that will further evaluate the effect of AT2220 on these functional parameters.

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