Chaperone Monotherapy
As monotherapy agents, pharmacological chaperones are designed to bind to the endogenous protein (lysosomal enzyme), stabilizing the enzyme in its properly folded active form and facilitating cellular trafficking to lysosomes. This allows for increased activity, improved cellular function and potentially reduced cell stress. Individuals with genetic mutations that result in some residual biological activity are potentially eligible for pharmacological chaperone monotherapy.
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Chaperone ERT Combination Therapy
In combination with ERT, pharmacological chaperones are designed to bind to infused exogenous enzyme, stabilizing the enzyme in its properly folded and active form in patients receiving ERT. This allows for enhanced tissue uptake, greater lysosomal activity and more reduction of substrate.
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Chaperone ERT Combination Therapy
In combination with ERT, pharmacological chaperones are designed to bind to infused exogenous enzyme, stabilizing the enzyme in its properly folded and active form in patients receiving ERT. This allows for enhanced tissue uptake, greater lysosomal activity and more reduction of substrate.
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Fabry Disease
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called α-galactosidase A (α-GAL A).
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Parkinson’s Disease
Individuals with Gaucher disease have an estimated 20-fold increased risk of developing Parkinson's disease, and an estimated 5% to 10% of those diagnosed with Parkinson's are carriers of the Gaucher disease gene.
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Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called acid α-glucosidase (GAA).
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Gaucher Disease
Gaucher disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called β-glucocerebrosidase (GCase).
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Migalastat HCI
In collaboration with GlaxoSmithKline (GSK), Amicus is developing the pharmacological chaperone migalastat HCl (AT1001) for Fabry disease as an oral monotherapy, and in combination with enzyme replacement therapy (ERT).
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Migalastat HCI
In collaboration with GlaxoSmithKline (GSK), Amicus is developing the pharmacological chaperone migalastat HCl (AT1001) for Fabry disease as an oral monotherapy, and in combination with enzyme replacement therapy (ERT).
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Migalastat HCI
In collaboration with GlaxoSmithKline (GSK), Amicus is developing the pharmacological chaperone migalastat HCl (AT1001) for Fabry disease as an oral monotherapy, and in combination with enzyme replacement therapy (ERT).
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Duvoglostat HCl
AT2220 (duvoglostat HCl) is a novel, orally-administered pharmacological chaperone in clinical development for co-administration with ERT for Pompe disease.
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Study 010
Open-label Phase 2 study to evaluate the safety and pharmacokinetic effects of four increasing oral doses of duvoglustat HCl co-administered with ERT versus ERT alone for Pompe disease. Each patient receives their current dose and regimen of ERT alone at one infusion and oral AT2220 administered prior to ERT at their next infusion.
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Study 011
Placebo-controlled, double-blind Phase 3 study of migalastat HCl in males and females with Fabry disease who have α-Gal mutations that are considered amenable to chaperone monotherapy based on a proprietary cell-based assay.
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Study 012
Randomized, open-label, 18-month Phase 3 study investigating the safety and efficacy of migalastat HCI compared to ERT in males and females with Fabry disease who have α-Gal mutations that are considered amenable to chaperone monotherapy based on a proprietary cell-based assay.
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Study 013
Open-label Phase 2 study to compare a single administration of oral migalastat HCl co-administered with infused ERT versus ERT alone in males with Fabry disease. Each patient receives their current dose and regimen of ERT alone at one infusion and oral migalastat HCl administered prior to ERT at their next infusion.
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AT2101, AT3375
Amicus is investigating the pharmacological chaperones AT3375 and AT2101 in combination with ERT in preclinical studies for Gaucher disease.
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AT3375
AT3375 is a next-generation pharmacological chaperone in preclinical development for Parkinson’s disease in Gaucher gene carrier’s and potentially the broader Parkinson’s population.
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