AMO Pharma Reports Long-Term Safety Data for AMO-02 in Congenital and Childhood-Onset DM1

AMO Pharma Reports Long-Term Safety Data for AMO-02 in Congenital and Childhood-Onset DM1

By, Admin 16 September 2025

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.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!