Steinert’s disease: mixed results for AMO-02

AMO-02 (or tidegluzib) is a drug candidate developed by AMO Pharma for the treatment of Steinert disease (or myotonic dystrophy type 1). In 2020, results from the first clinical evaluation of 16 people with T1D treated for 3 months showed improvements in cognitive function and fatigue, and, in some participants, behavioral disturbances.

To confirm these results, a phase II-III clinical trial was conducted against placebo in 56 children and adolescents suffering from congenital Steinert disease (REACH CDM study). They received either AMO-02 or placebo for 6 months.
The main evaluation criterion was the score obtained according to “ Congenital T1DM rating scale completed by a physician ”, a clinical questionnaire completed by a physician to assess the overall impact of the congenital form of the disease. He found no difference between patients receiving AMO-02 and those receiving placebo.

However, AMO Pharma reports that there were significant improvements in physical (10-minute walk test) and cognitive (picture vocabulary test), as well as reductions in the enzyme creatine phosphokinase (CPK), a measure of muscle and heart damage.

Source
AMO Pharma Announces Confirmation of Data from the REACH-CDM Clinical Study for AMO-02 in the Treatment of Myotonic Dystrophy AMO Pharma, Press Release September 6, 2023