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.
Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.