AMO Pharma reports Phase II data for myotonic dystrophy candidate

AMO Pharma Ltd. (Surrey, U.K.) reported data from a Phase II trial in 16 patients ages 13-34 with congenital or childhood-onset myotonic dystrophy type 1 (DM1) showing that tideglusib (AMO-02) was generally well tolerated with nasopharyngitis reported as the most common treatment-emergent adverse event. The trial included a 2-week, placebo run-in period followed by 12 weeks of once-daily 400 or 1,000 mg oral tideglusib.

The company said most patients treated with tideglusib showed improvements in cognitive function, fatigue and the ability to perform daily tasks, with a greater response in patients who received high-dose tideglusib vs. low-dose tideglusib. Data were presented at the Muscular Dystrophy Association meeting in Arlington...