Posted by Michael Wonder on 30 May 2017
AMO Pharma announces FDA fast track designation for AMO-02 for treatment of congenital myotonic dystrophy
30 May 2017 - AMO Pharma Limited today announced that the U.S. FDA has granted fast track designation for AMO-02, the company's investigational therapy in development for the treatment of congenital myotonic dystrophy.
AMO-02 (tideglusib) is in clinical stage development for the treatment of the severe form of myotonic dystrophy known as congenital DM1. In cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to be increased. Inhibitors of GSK3ß have been shown to correct the activity of RNA binding proteins, such as CUGBP1, in animal models of DM1.
AMO-02 is an inhibitor of GSK3ß that has demonstrated pre-clinical efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples derived from patients with congenital DM1.
