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news.Amicus Therapeutics has infused the first patient with AT2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) to treat Pompe disease.
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The multi-center, open-label Phase 2 Study 010 is designed to assess the safety and pharmacokinetic (PK) effects of four increasing oral doses of AT2220 co-administered with ERT versus ERT alone in individuals with Pompe disease.
All subjects will be given a regularly scheduled ERT infusion.
Patients will receive a single oral dose of AT2220, one hour prior to the initiation of the next ERT infusion.
Plasma enzyme activity and protein levels will be demonstrated during each infusion.
Muscle biopsies will be taken seven days after each infusion to measure tissue ERT activity with and without the chaperone, as well as the level of AT2220.
Amicus Therapeutics chairman and CEO John Crowley said they believe that chaperone-ERT co-administration has the potential to improve treatment outcomes for Pompe patients.
Amicus CMO Pol Boudes said results from Study 010, if positive, may form the basis for later stage studies that would allow them to assess the effect of AT2220 co-administered with ERT on glycogen reduction and ERT-related toxicity in patients with Pompe disease.