Rimeporide in patients with Duchenne Muscular Dystrophy

      Objective: To reposition rimeporide as an oral disease modifying treatment for patients with Duchenne Muscular Dystrophy (DMD). Methods: EspeRare is developing rimeporide, a safe, potent, oral and selective NHE-1 inhibitor for patients with DMD. Today, translational studies are ongoing to generate value for rimeporide and ensure its subsequent transition into a pivotal Phase II/III study in patients with DMD. These translational studies are conducted in Golden Retriever Muscular Dystrophic Dogs (GRMD), mdx mice and patients with DMD. Results: In cardiomyopathic hamster, rimeporide was shown to prevent hypertrophy and necrosis in the heart and to improve overall survival. In mdx mice, rimeporide improved specific force, prevented inflammation and fibrosis in skeletal muscles, in the diaphragm and in the heart in a clinically and statistically significant manner. After showing a compelling safety and tolerability in healthy adults, rimeporide is now tested in an open-label, Phase 1b study in young DMD boys to assess its safety, pharmacokinetics and to explore biomarkers (serum and NMR-I and NMR-S) after a 4-week treatment with ascending oral dose. The recruitment in the study has been initiated in March 2017. To date, on the 15 patients who were enrolled, tolerability has been good with no significant new risks identified. There have been no serious adverse events (SAE), no pattern of AE and all AE were considered unrelated to rimeporide. Conclusion: Rimeporide in DMD received Orphan Drug Designation from EMA. Rimeporide has the potential to be a muscle-sparing agent that may alleviate muscle and cardiac damage and inflammation in patients with DMD. Its unique ability to prevent cardiomyopathy as well as the decline in muscle function, with no restriction on age and on genetic subtypes, makes it an important complement to other treatments restoring dystrophin.
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