#USA #London #Myotonic Dystrophy #AMO Pharma #AMO-02

AMO Pharma Unveils Long-Term Safety Data for AMO-02

AMO Pharma Limited, a clinical-stage biopharmaceutical company specializing in rare genetic disorders, recently shared compelling long-term safety data from its ongoing REACHCDM-X study. This pivotal research focuses on AMO-02, a treatment aimed at addressing congenital myotonic dystrophy type 1 (DM1), a serious condition with limited treatment options.

The findings reflect nearly four years of patient experience and indicate that AMO-02 has maintained a favorable safety profile throughout the duration of the study. Mike Snape, CEO of AMO Pharma, expressed satisfaction with the results, particularly highlighting the low rates of hospitalizations observed among participants.

Study Highlights

The REACHCDM-X study involves an open-label extension of a previous double-blind trial and has garnered attention for being the largest and longest-running interventional study of its kind focused on congenital and childhood-onset DM1. Key highlights from the study include:

• - Widespread Tolerance: AMO-02 was reported as generally safe and well-tolerated by the participants. As of August 2025, 45 patients continued to receive treatment, including 20 individuals who have been on AMO-02 for over three years.
• - Adverse Events: Of the participant cohort, only one withdrew due to an adverse event, specifically elevated liver enzymes. Most adverse effects recorded were mild or moderate in severity, predominantly respiratory infections and gastrointestinal issues, which align with the natural symptoms of the disorder.
• - Hospitalization Rates: The hospitalization rate for participants receiving AMO-02 was an impressive 0.14 per patient per year across over 151 patient-years of treatment exposure—no deaths or cardiovascular events occurred during the study.
• - Functional Outcomes: In a 10-meter walk/run test, older participants exhibited little to no decline in functional ability throughout the treatment year, showcasing the treatment's potential efficacy.

Regulatory Discussions Ahead

AMO Pharma has submitted the recent safety data to the U.S. Food and Drug Administration (FDA), with a meeting scheduled for Q4 2025 to discuss the pathway toward New Drug Application (NDA) submission for AMO-02. The company also plans to engage with regulatory bodies in Canada and the UK regarding the next steps for AMO-02's approval.
Dr. Snape underscored the significance of these findings—strong safety data provide crucial insights into the potential clinical benefits for patients with congenital DM1, a condition often associated with serious complications. The company aims to utilize real-world evidence to further understand AMO-02's effectiveness, a strategy expected to influence future investigations significantly.

AMO Pharma's Vision

Founded with the goal of developing therapies for unmet medical needs, AMO Pharma focuses on serious childhood conditions with few or no effective treatments. Alongside AMO-02, the company is also progressing other investigational products targeting disorders like Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) and Phelan-McDermid syndrome.
As the pharmaceutical community watches closely, AMO Pharma's developments signal hope for many families dealing with the challenging impacts of congenital myotonic dystrophy. Stay informed for updates on this promising treatment's journey through the regulatory landscape and its implications for future therapeutic advancements.

For more information, visit AMO Pharma's website.