ATB200 co-administered with AT2221 is in development for the treatment of Pompe disease. Pompe disease is an inherited, genetic disorder which results in the deficiency of the enzyme ‘acid alpha-glucosidase’. This deficiency leads to progressive accumulation of glycogen, a type of sugar, usually stored in muscle tissues particularly around the heart, skeletal muscle and diaphragm. Enzyme replacement therapy is a recommended treatment approach.
ATB200 (cipaglucosidase alfa) is a recombinant human acid alfa glucosidase (rhGAA) enzyme that shows significantly improved uptake into muscle cells compared with algucosidase alfa at similar doses and translates into optimised glycogen reduction in disease-relevant muscles in non-clinical models. AT2221 (miglustat) is the pharmacological chaperone that stabilises and enhances the plasma exposure of ATB200, resulting in improved delivery of active enzyme to key disease-relevant tissues. If licensed, ATB200/AT2221 will offer an additional treatment option for patients with Pompe disease with potentially improved efficacy.
Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.