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AMO Pharma Reports Long-Term Safety Data from REACHCDM-X Study of AMO-02

AMO Pharma Limited, a clinical-stage biopharmaceutical company focused on addressing rare genetic disorders, has released new findings from its ongoing REACHCDM-X study. This study is crucial for understanding the safety and efficacy of AMO-02, a treatment targeting congenital myotonic dystrophy type 1 (DM1). The company shared insights that have emerged from almost four years of research, highlighting the drug's promising safety profile.

Key Findings

In the preliminary analysis, AMO Pharma noted that AMO-02 has demonstrated a generally good safety and tolerability profile over the study period. As of August 2025, a total of 45 participants are still on treatment. Notably, 20 of these individuals have been receiving AMO-02 for over three years, with only a single adverse event leading to treatment discontinuation—an instance of elevated liver enzymes.

The adverse events reported during the study were predominantly mild or moderate, with respiratory infections and gastrointestinal issues being the most frequently observed—a pattern consistent with the natural course of congenital DM1. Across more than 151 patient-years of treatment exposure, AMO-02 presented a remarkably low hospitalization rate of just 0.14 events per patient per year, with no fatalities or cardiovascular complications reported.

Additionally, participants aged 10 years and older showed little to no decline in performance on a 10-meter walk/run test, a secondary endpoint in the trial, further underscoring the treatment's potential benefits.

Next Steps Toward Approval

AMO Pharma acknowledges the positive implications of these findings and plans to present this data to the U.S. Food and Drug Administration (FDA) in a meeting scheduled for Q4 2025. During this meeting, they aim to discuss the trajectory for a New Drug Application (NDA) for AMO-02. Furthermore, submissions have also been made to Health Canada and the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), emphasizing the company's commitment to gaining regulatory validation across multiple global health authorities.

Given the potential life-threatening implications associated with congenital myotonic dystrophy, Dr. Mike Snape, the CEO of AMO Pharma, expressed enthusiasm about the data, which advances our understanding of the clinical benefits AMO-02 could deliver. He emphasized the importance of real-world evidence and the relevance of these outcomes for families impacted by DM1.

The REACHCDM-X Study

The REACHCDM-X study, which started in August 2021, is recognized as the largest and longest-running interventional study targeting congenital and childhood-onset myotonic dystrophy. This open-label extension comes after the double-blind REACHCDM trial, and it has expanded to include a broader age range of participants—up to 45 years old, reflecting AMO Pharma’s dedication to comprehensively understanding DM1.

By allowing patients to continue treatment over the long term, the study aims to uncover consistent safety data that will inform future regulatory submissions and therapeutic approaches.

AMO Pharma's Broader Commitment

In addition to AMO-02 for DM1, AMO Pharma is actively developing therapies for other rare genetic conditions, including AMO-01, a candidate for Phelan-McDermid syndrome, and AMO-04, which shows promise for treating Rett syndrome. This portfolio reflects the company's resolve to address unmet needs in pediatric populations plagued by severe neurogenetic disorders, many of which lack effective treatments.

Overall, the insights garnered from the REACHCDM-X study signify a hopeful advancement in stemming the tide of congenital myotonic dystrophy, representing the potential for substantial clinical benefits for patients and families affected by this debilitating condition.

For further information, visit AMO Pharma.