Positive Results for Phase 2 Proof-of-Concept Study for Congenital and Childhood Onset Myotonic Dystrophy Type 1


The results of a phase 2 proof-of concept study of AMO-02, an investigational therapy for childhood-onset myotonic dystrophy type 1 were presented at the Muscular Dystrophy Association Meeting this week. In the study, patients treated with 1000 mg/day AMO-02 for 14 weeks showed improvements in functional daily living, levels of fatigue, and cognitive function compared to those treated with 400 mg/day. Improvements were measured most effectively with clinician- and caregiver-completed rating scales and showed statistically significant treatment-associated effect sizes across the period of treatment with AMO-02. Co-occurring autism spectrum symptoms also improved for several patients. There were no early discontinuations or dose adjustments required for adverse effects.

"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," said Michael Snape, MD, chief executive officer of AMO Pharma.

"As the first clinical trial devoted to this patient population, these results are especially encouraging," said Dr. Horrigan who presented the results. "We look forward to continuing to expand our research and further evaluate the efficacy of AMO-02 in larger multi-site clinical trials in the US, Canada and the UK."



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