In December 2020 AMO- Pharma announced the initiation of the Phase 2/3 clinical trial, REACH-CDM. This placebo-controlled study is testing the efficacy and safety of the potential drug AMO-02 (also known as tideglusib) in the treatment of congenital myotonic dystrophy.
AMO-02 inhibits an enzyme called glycogen synthase kinase 3ß (GSK3ß), which is over-active in people with congenital myotonic dystrophy. AMO-02 has been shown to reduce the levels of GSK3β in mouse models and in human cells from people with myotonic dystrophy. Importantly, previous research has shown that AMO-02 can reach the muscles, brain and other tissues.
The REACH-CDM study was designed based on the results of positive phase 2 clinical trial data. The trial aims to recruit 56 children and adolescents with congenital onset myotonic dystrophy. Initial trial sites are in the United States and Canada, with plans to extend to trial sites in additional countries following local approval processes. We will keep you updated as more information is released.
Read AMO-Pharma’s press release for more information.
Read the scientific publication of the Phase 2 clinical trial results here.
If you have any research questions, please contact our Research Line on research@musculardystrophyuk.org