London-headquartered AMO Pharma has announced the launch of a ‘pivotal’ phase III study of its congenital myotonic dystrophy (CDM1) candidate AMO-02 (tideglusib).

AMO-02 is a dual action action mRNA modulating/GSK3ß kinase inhibitor in development as a potential treatment for CDM1.

The launch of the REACH-CDM study follows the completion of discussion between AMO Pharma and the US Food and Drug Administration (FDA).

This included making amendments to the trial protocol to address potential issues associated with execution of the study due to the COVID-19 pandemic.

The phase III study was designed based on results of positive phase II data. In prior research, AMO-02 has been shown to access brain, muscle and other tissues and reduce the effect of expansion repeat in mRNA – the pathological basis for CDM.

The REACH-CDM trial will aim to enrol a total of 56 patients initially in sites in the US and Canada, with additional sites in Australia, New Zealand and other countries to be added depending on approval.

"Congenital myotonic dystrophy is a devastating disease that has a profound impact on the health and quality of life of patients and families. We are very encouraged by the levels of interest in the REACH-CDM trial from investigators and researchers and from families who are so anxious for a treatment," said Ibs Mahmood, chief executive officer of AMO Pharma.

"We are dedicated to advancing the development program for AMO-02 as rapidly as possible and are grateful to the regulators, investigators and leaders in patient advocacy for their support," he added.