Positive topline results reported in clinical trial of novel myotonic dystrophy drug

Key takeaways:

• The REACH-CMD study included 56 children and adolescents with congenital myotonic dystrophy.
• Treatment with AM0-02 resulted in significant improvement in motor skills, muscle strength and daily functionality.

A global clinical-stage specialty biopharmaceutical company has announced positive topline results from its study of its investigational therapeutic AM0-02 to treat children and adolescents with congenital myotonic dystrophy.

According to a release from AMO Pharma Limited, the REACH-CMD clinical trial, a double-blind, placebo-controlled randomized study of 56 young persons at sites in the United States, Canada, New Zealand and Australia, did not meet its primary endpoint of a statistically significant benefit compared with placebo.

However, the company said, further statistical analysis showed significant benefits in motor skills, muscle strength and daily functionality with AM0-02 treatment. These included improvements on the Peabody Picture Vocabulary Test, reduction of skeletal and heart muscle biomarker creatine phosphokinase, improvement in the 10 meter walk/run test and pharmacokinetic benefits seen in increased plasma levels of the drug correlating to greater clinical improvement.

All participants were given the option to continue treatment in a year-long, open-label extension study. The company reported a 98% holdover rate, and 85% of those in the OLE opted to continue treatment after its completion.

“We are very encouraged by the consistent benefit shown across multiple clinically confirmed measures of efficacy,” AMO Pharma Chief Medical Officer Joe Horrigan, MD, said in the release. “These data reflect a broad profile of benefit in cognitive, motor, muscle, real world adaptive behavior and biochemical measures associated with treatment with no reported treatment-related serious adverse events.”