AMO Pharma has announced results from its ongoing phase II study investigating two doses of tideglusib (AMO-02) in adults with congenital and juvenile onset myotonic dystrophy.
The subjects had an average age of 22 years – six of whom had congenital-onset myotonic dystrophy – and they underwent once daily oral treatment with 1000mg AMO-02. The results showed that treatment was generally safe and well tolerated, and no subjects discontinued treatment due to adverse events or other issues. The data show multiple endpoints with favourable changes that were statistically significant.
Some improvements in central nervous system symptoms, autistic features and activities in daily living were also noted.
In a press release, AMO Pharma’s CEO, Michael Snape, said:
"AMO Pharma would like to thank the families taking part in this study, as well as the staff at Newcastle and all our partners in this work. Without their contribution we would not have been able to gather these data and we truly appreciate all the efforts that they have made. I am very proud that we can report encouraging preliminary data for families affected by congenital myotonic dystrophy, for whom no sponsor led studies have previously been conducted. We are now working to complete the second cohort of this study and finalize planning of our next study in children and adolescents with this condition. We look forward to making further announcements regarding our next steps,"
Prof Hanns Lochmuller highlighted that it has been a neglected area, and added, “It is important to expand our knowledge of clinical study methodologies in this patient population. The participants, their families and caregivers came from across the UK and were recruited through the joint efforts of Newcastle University and Hospital Trust, the UK patient registry and the patient organizations MDUK and MDSG, showing that efficient recruitment can be achieved and trials conducted in this rare disease.”