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A important progress for patients living with Duchenne: Rimeporide starts its clinical development

23Mar

EspeRare Today achieves a significant milestone by enrolling patients with DMD in Rimeporide phase Ib trial:Our first DMD patient was recruited in France

Rimeporide, EspeRare's first programme reaches today a decisive clinical stage milestone. After encouraging preclinical results obtained in leading institutions and an orphan drug designation granted from the European Medicines Agency, the recruitment of patients with Duchenne Muscular Dystrophy from 6 to 14 years of age in a phase Ib study with Rimeporide started in Paris at the centre of I- Motion. Leading European neuro-paediatricians will be recruiting up to 20 patients with DMD in 2016 at the Armand Trousseau Hospital/ I-motion (Paris, France), the Great Ormond Street Hospital (London, UK), the Santa Creu i Sant Pau (Barcelona, Spain) and the San Raffaele Hospital (Milan, Italy).

This study is investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of a 4-week treatment with rimeporide in these patients. In contrast to many therapeutics under development in DMD, rimeporide is indicated to all patients with DMD regardless of the causative mutations and age. To our knowledge, Rimeporide is one of the few clinical stage therapy intended to reduce inflammation and fibrosis both in skeletal muscles and in the heart.

 

Our hope is that Rimeporide may be a big step forward alone or in combination with other available treatments to address DMD including cardiomyopathy which at present is a progressive disease with very limited therapeutic options.

 

For more information see our website www.esperare.org/en/duchenne-programme and also www.clinicaltrials.gov