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.
Ozanimod, administered orally, is a new sphingosine 1-phosphate (S1P) receptor modulator. Treatment with S1P modulators is believed to work by interfering with signalling pathways that contribute to tissue inflammation. If licensed, ozanimod will offer an additional treatment option for adult patients with moderate to severe UC who have had an inadequate response, lost response to, or were intolerant to conventional therapy or a tumour necrosis factor-alpha (TNF) antagonist. In one phase II clinical trial, ozanimod at a daily dose of 1mg resulted in a slightly higher rate of clinical remission of ulcerative colitis than placebo.