Advancing treatments for rare diseases Together



The last advances of the Rimeporide clinical study have been presented at the 22nd World Muscle Society congress, in St Malo. Numerous scientists and physicians have shown interest for this project.

Enrollment into this phase IB clinical trial should be complemented by the end of the year. Full results should be available during the first quarter of 2018. EspeRare is initiating discussions with  worldwide clinicians in order to design the phase II study which should start during 2018, depending  on funding.

Poster presented at the World Muscle Society congress



The Food and Drug Administration (FDA), the US Department of Health and Human Services, has granted an Orphan Drug Designation (ODD) for rimeporide for the treatment of Duchenne muscular dystrophy (DMD). See here for more details.

ODD is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. In particular, this ODD grants Rimeporide a 7-year data exclusivity in the US.

Rimeporide already received ODD in DMD from the European Medicines Agency in 2015. Further to this designation in Europe, EspeRare has been able to conduct a phase Ib clinical trial of Rimeporide on young DMD patients. This clinical trial will be completed by the end of 2017.


In parallel of developing Rimeporide for the treatment of Duchenne muscular Dystrophy, EspeRare is now also initiating studying Rimeporide's therapeutic potential in Pulmonary Arterial Hypertension.

Rimeporide is a discontinued heart failure drug, that EspeRare has turned into an innovative first-in-class treatment for patients with Duchenne Muscular Dystrophy (click here to check this clinical development programme). Rimeporide has proven to be well-tolerated in multiple animal models and clinical trials in healthy adults and young boys with Duchenne.

 The mechanism of action of Rimeporide, the inhibition of a protein called NHE-1, holds potential in multiple therapeutic indications and EspeRare is working towards establishing collaborations with world-class partners to explore this potential.

 In particular, EspeRare started a collaboration with Larissa Shimoda, Ph.D. and John Huestch, M.D. at the John Hopkins University School of Medicine in the "Division of Pulmonary and Critical Care Medicine". The aim of this collaboration is to explore the therapeutic potientiel of Rimeporide in Pulmonary Arterial Hypertension, using in vitro and in vivo models. Over the last 10 years, this team has published important research articles on the contribution of NHE-1 in the development of this debilitating disease and the use of NHE-1 inhibitors to prevent it.

Positive results from this study would support the initiation of a Phase II clinical trial to test Rimeporide’s therapeutic potential in patients with Pulmonary Arterial Hypertension.


Prof Muntoni is the Principal Investigator of the multicenter european phase Ib clinical study in Duchenne Muscular Dystrophy boys. He presented Rimeporide in an oral communication at the 12th European Paediatric Neurology Society Congress in Lyon on June 22nd.


On this occasion he gave an update on the preclinical and the clinical development of this innovative treatment that addresses in particular the life-threatening cardiac damage in Duchenne Muscular Dystrophy patients.


EspeRare starts two research collaborations to demonstrate preclinical efficacy of Rimeporide in Duchenne.

Geneva, Switzerland, August 19, 2013 - The foundation announces that it has started two collaborations with the University of Geneva (Switzerland) and the Children National Medical Center in Washington DC (USA) to develop Rimeporide, its lead programme in Duchenne Muscular Dystrophy (DMD). The objective of these collaborations is to assess the therapeutic utility of Rimeporide for Duchenne patients. Rimeporide is a safe and specific Na+/H+ exchanger type 1 (NHE 1) inhibitor previously developed by Merck Serono for cardiac failure.

Rimeporide preclinical efficacy will be tested in DMD mice models. The assessment will be conducted under the leadership of Dr. Kanneboyina Nagaraju specialized in DMD at Prof. Eric Hoffman's research center for genetic medicine, Children National Medical Center, Washington DC.
In parallel, Dr Olivier Dorchies, a recognised DMD preclinical expert at the Pharmaceutical biochemistry lab of Prof. Leonardo Scappozza, University of Geneva, will also investigate the dose / efficacy relationship of Rimeporide in a mice model  and evaluate the efficacy in various cell lines.

Preliminary data from both collaborations are expected by second quarter 2014.

This preclinical work together with the already available data will support further development of this compound in DMD patients. This program has ultimately the potential to address muscle degeneration and cardiomyopathy in a broad segment of DMD children.