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Rimeporide in Duchenne programme

 

Main Rimeporide programme milestones

2013 

EspeRare obtains the rights to develop rimeporide in neuromuscular diseases.

2014

Two non-clinical studies demonstrated Rimeporide’s ability to significantly decrease levels of inflammation and fibrosis in a large panel of skeletal muscles and in the heart in a dystrophic mice model.

2015

Orphan Drug designation granted by the European Medicines Agency (EMA) for Rimeporide in DMD.

Filing of the clinical trial application in four European countries.

2016

Patient are being enrolled in our phase Ib clinical trial. The initiation of the development of Rimeporide in children with DMD has been made possible thanks to several years of research and collaboration with researchers at leading institutions including the Children National Medical Center (Washington DC, USA) and the Sherbrooke University (Canada). In this phase Ib trial, leading European neuro-paediatricians will enroll up to 20 patients with DMD during 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). For more information please visit www.clinicaltrials.gov. 

Beyond 2017

Late stage development of Rimeporide will be conducted by other partners that have the adequate infrastructure to conduct late stage clinical trials, manufacturing and commercialisation. Along the development path, EspeRare will strive to ensure that patients that are expected to benefit from the medication will have access to it. According to current development plans, Rimeporide aims to be marketed by 2020-2021.

Our hope is that Rimeporide becomes a major treatment option for patients suffering with DMD, alone or in combination with other available treatments. In particular, Rimeporide has the potential to address the cardiomyopathy in Duchenne, which is at present a progressive condition with very limited therapeutic options. To our knowledge, Rimeporide is one of the few clinical stage therapies intended to reduce inflammation and fibrosis both in skeletal muscles and in the heart.

The Rimeporide programme background

Rimeporide was a proprietary compound of Merck Serono, previously developed up to clinical stage for congestive heart failure but was discontinued during phase I for strategic reasons. Rimeporide is known as a selective Sodium-Proton Exchanger (NHE-1) inhibitor on which detailed data on pharmacology, formulation, dosing, pharmacokinetic and toxicology profile exists.

In Duchenne Muscular Dystrophy (DMD), the overload of calcium is recognised to be damaging to muscle integrity. Calcium homeostasis can occur through a number of channels or pumps, and NHE-1 is known to impact Calcium load through its intra-cellular regulation of sodium. In Duchenne models, NHE-1 is disregulated and has been shown to be partly involved in sodium and calcium cellular retention.

Rimeporide identity card

  • Duchenne is a heartbreaking disease. Children like Laurent, my cousin, affected with this disease are bright and engaged but as they grow up, they inexorably go weaker and experience the loss of the few abilities they had acquired. I have in my gene the eagerness to find treatments for this disease and I am committed to give them the strength to fight their disease.
    Florence Porte-Thomé
    R&D program Director and Founder of the EspeRare Foundation

About Duchenne Muscular Dystrophy

  • Duchenne muscular dystrophy (DMD) is a severe genetic neuromuscular disease that affects 1 in 3,500 boys with an estimated patient population of 50,000 boys in the developed world.  
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