London, UK — AMO Pharma Limited, a clinical-stage specialty biopharmaceutical company focused on rare genetic disorders, announced new safety findings from its ongoing REACHCDM-X open-label extension (OLE) study of AMO-02 for congenital and childhood-onset myotonic dystrophy type 1 (DM1).
The preliminary analysis includes nearly four years of continuous treatment data, marking the largest and longest interventional study conducted in congenital and childhood-onset DM1.
CEO Statement
“We are pleased to share new findings that reflect the experience of patients treated with AMO-02 for almost four full years. The results highlight AMO-02’s favorable safety profile, especially the low hospitalization rate observed. We look forward to upcoming FDA discussions to advance AMO-02 as quickly as possible for patients with congenital myotonic dystrophy.”
- Mike Snape, CEO of AMO Pharma.
Key Findings from the REACHCDM-X Study
- Safety profile: AMO-02 has been generally safe and well-tolerated for four years.
- Patient retention: As of August 2025, 45 participants remain on treatment, including 20 for more than three years.
- Withdrawals: 14 participants discontinued treatment; only one due to an adverse event (elevated liver enzymes).
- Adverse events: Mostly mild or moderate, with respiratory and gastrointestinal events consistent with DM1’s natural course.
- Hospitalizations: Across 151 patient-years of exposure, hospitalization rate was 0.14 per patient per year.
- Mortality/cardiovascular events: None reported.
- Secondary endpoint: In the 10-meter walk/run test, patients aged ≥10 years showed little or no decline over one year.
Regulatory Updates
- Data submitted to the U.S. FDA, with submissions also planned for Health Canada and the UK MHRA.
- AMO Pharma will meet with the FDA in Q4 2025 to discuss:
- Development plan for AMO-02 in congenital DM1.
- Use of real-world evidence in regulatory submissions. - A Phase 3 study protocol for adult-onset DM1 has also been submitted to the FDA.
Broader Pipeline
- AMO-02: In development for congenital DM1 and Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC).
- AMO-01: Investigated for Phelan-McDermid syndrome.
- AMO-04: Clinic-ready potential treatment for Rett syndrome and related disorders.
About AMO Pharma
AMO Pharma is a privately held clinical-stage biopharma focused on rare and severe childhood-onset neurogenetic disorders with limited or no treatment options. Its pipeline includes multiple investigational medicines aimed at providing first-in-class therapies for underserved patient populations.
