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AMO Pharma reveals data from myotonic dystrophy study

Clinically significant results have emerged from the trial evaluating the company’s AMO-02 candidate.

AMO Pharma Limited, a company concentrating on rare childhood-onset neurogenetic disorders with limited treatment options, has announced results from the company’s REACH-CDM trial.

The study concerns the investigational candidate AMO-02 for the treatment of children and adolescents with congenital myotonic dystrophy.

Topline results from the research, based on a clinician-administered rating instrument, demonstrated a positive benefit across both the treatment and placebo groups. This may, however, be linked in part to difficulties in patient monitoring and compliance with reporting protocols caused by the COVID-19 pandemic.

While the study did not meet the primary endpoint of a statistically significant benefit over a placebo, a clinically significant benefit was established during functional and objective assessments among the treatment group when compared to the placebo.

Meanwhile, treatment with AMO-02 was associated with improvements in cognitive performance, a reduction in a widely used biomarker of skeletal and cardiac muscle integrity and progress in the 10m walk/run test.

Furthermore, a combined statistical analysis of outcomes assessing muscle strength, motor skills, cognitive ability and biomarker data delivered a statistically significant benefit following treatment with AMO-02 compared to the placebo.

Benefits witnessed with the AMO-02 candidate were related to pharmacokinetic parameters, showing that increased plasma levels from the drug resulted in increased clinical improvement.

Dr Ibraheem Mahmood, chief executive officer at AMO Pharma, commented: “We are immensely grateful to all the families who took part in this study, patient advocates in the myotonic dystrophy community, investigators who worked with us to face the challenges presented by the COVID pandemic, and our investors for supporting this work.”

He added: “These results provide strong further validation of the potential benefits of treatment with AMO-02 in multiple key areas that represent the most severe symptoms and disabilities associated with DM1. We are now working to discuss the next steps with regulators in order to advance this programme.”

Dr Joe Horrigan, chief medical officer at AMO Pharma, concluded: “We are very encouraged by the consistent benefit shown across multiple clinically confirmed measures of efficacy. These data reflect a broad profile of benefit in cognitive, motor, muscle, real-world adaptive behaviour and biochemical measures associated with treatment with no reported treatment-related serious adverse events.”

Participants in the trial were invited to continue treatment in an open-label extension study and 98% of patients opted to continue with the treatment.