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Duchenne Muscular Dystrophy: EspeRare Foundation And Drug Repositioning

December 30, 2013

This is the fifth Blog Post in a series examining Duchenne Muscular Dystrophy (DMD) in the rare disease and orphan drug space. This Blog Post discusses the EspeRare Foundation and how the organization is using repositioning or repurposing of an old drug to benefit DMD.

Drug repositioning or drug repurposing (DR) is applying existing drugs to new indications or diseases. DR is increasing in importance to many drug development and pharmaceutical companies. Using DR provides several advantages to companies:

•   Repositioned drug has already passed toxicity and other tests

•   Repositioned drug’s safety profile is already known

•   Repositioned drug decreases the development time and cost of developing a drug.

Merck Serono, a division of Merck, announces in April 2013, the launch of the EspeRare Foundation. The EspeRare Foundation is presented to the public and international rare disease community at the IRDiRC (International Rare Diseases Research Consortium) Conference, in April 2013, in Dublin, Ireland.

The EspeRare Foundation is a nonprofit private organization established in Geneva, Switzerland, to advance treatments for rare diseases. The mission of the foundation is to reposition or repurpose existing or abandoned drugs for the potential of providing new treatments for rare diseases. Merck Serono donates an initial €2.8 million (approximately US $3.8 million) in funding, and the rights of Rimeporide, a compound previously developed for heart failure by Merck. Per the foundation’s website, from 2010-12, half of the 46 FDA approvals in orphan indications are repositioned drugs.

According to Francois Naef, Chairman of the Board of Directors at Merck Serono, the EspeRare Foundation will:

 “ .. develop a unique operating model for the benefit of patients suffering from rare diseases, a field with high unmet needs and hardly no treatments available …. pioneering a collaborative system that brings together the right stakeholders to explore the therapeutic value of existing compounds, ultimately accelerating the R&D process in rare diseases.”

The foundation’s first program is the repositioning of Rimeporide, with the potential to slow muscle degeneration in children with DMD. Rimeporide is discontinued during Phase I for congestive heart failure. The drug is a selective Sodium-Proton Exchanger (NHE-1) inhibitor with known pharmacokinetics, formulation , dosing, safety, and other known profile features. The use of an NHE-1 inhibitor is thought to decrease sodium and calcium overload in muscles of DMD patients, and thus slow muscle degeneration.

Other recent EspeRare Foundation developments for DMD:

•   In August 2013, EspeRare starts two research collaborations to show preclinical efficacy of Rimeporide in DMD mice models (preliminary data from both are expected by 2nd quarter 2014):

1.   University of Geneva (Switzerland)

2.   Children National Medical Center in Washington DC

•  In November 2013AFMTelethon, the French Muscular Dystrophy Association, confirms intent to co-finance the evaluation of the potential of Rimeporide for DMD.

Please Note: “Technician Transfer Liquid To Test Tube” courtesy of US National Institute of Mental Health.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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