AMO Pharma Limited, a UK-based clinical-stage specialty biopharmaceutical company, on Monday announced agreement with regulatory agencies in the US, UK and Canada on design for a registrational clinical study intended to assess the safety and efficacy of the company's investigational therapy AMO-02 (oral tideglusib) in the treatment of congenital myotonic dystrophy type 1 (cDM1).

The design requirements and primary outcome choice for this registrational study are based on advice provided to AMO Pharma by the US Food and Drug Administration (FDA), the UK Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada following meetings over the last six months. AMO-02 is an investigational compound that has not been approved by any regulatory authority. To date, the safety and efficacy of AMO-02 have not been established.

Based on this regulatory advice, the registrational study for AMO-02 is expected to use hospitalisation as the primary efficacy endpoint. Hospitalisation represents a significant burden for people living with cDM1 and their caregivers, as cDM1 is a potentially life-threatening disorder, associated with multiple medical co-morbidities. AMO Pharma also plans to engage the cDM1 community through a survey to better understand the impact of symptoms and hospitalisations associated with this disorder. The clinical study will also include a range of functional assessments as secondary outcome measures to help characterise disease progression and capture key features of cDM1, which can vary widely in its clinical presentation.

The scientific advice received by AMO Pharma from regulators is consistent with the language regarding cDM1 adopted in the FY27 Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriations bill.

AMO Pharma expects to provide an update on the planned initiation of the study during the third quarter of 2026.