The phase 2 study enrolled 16 participants aged between 13 to 34 years old. Every day for 14 weeks, the participants took an oral dose of either: 400mg tidegusib, 1000mg tideglusib, or a placebo. Their condition was monitored by both doctors and caregivers over the 14 weeks.
Most participants who received the drug had improved cognitive (brain) function and felt less fatigued (tired). They were also better at performing day-to-day tasks and activities. In addition, several participants showed improvements in autistic symptoms.
Both doses of tideglusib were safe and well tolerated. However the higher dose (1000mg/day) appeared to more beneficial than the lower dose (400mg/day).
Dr Michael Snape, Chief Executive Officer of AMO Pharma, said:
These significant data are an important step in the development of AMO-02 as a potentially safe and effective treatment option for many patients living with congenital and childhood onset myotonic dystrophy type 1. We look forward to advancing the clinical development program for AMO-02 and are grateful to the clinicians, caregivers and patients who participated in this landmark trial.
AMO Pharma is planning to further evaluate the efficacy of tideglusib in larger multi-site clinical trials in the US, Canada and UK. We will let you know as soon as the company releases any more information.
Read AMO Pharma's press release
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