In addition, Merck Serono will transfer the rights of rimeporide, a compound previously developed for heart failure by Merck. The Foundation will investigate this compound in Duchenne Muscular Dystrophy (DMD), a lethal genetic rare disease affecting 1 in every 3 600 males.
"The EspeRare Foundation is the first initiative of its kind in the framework of the EPP. It is the result of our employees' determination to 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," said François Naef, Chairman of the Board of Directors of Merck Serono S.A. "The Foundation will play a central role by 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 EspeRare Foundation's mission is to uncover the potential of existing drugs to address severe therapeutic unmet needs in rare diseases, through an established network of patient organizations, pharmaceutical companies, biotechs, regulators and academic institutions. The initial endowment from Merck Serono will enable the Foundation to start its activities with the development of rimeporide in DMD, while attracting additional private and public funding for this project and future repositioning programs in rare diseases.
The EspeRare Foundation was established by three Merck Serono employees: Caroline Kant, Florence Porte and Béatrice Greco. Sharon Terry, President and CEO of Genetic Alliance and an Executive Board Member of the International Rare Disease Research Consortium (IRDiRC), will join EspeRare as President of the Foundation.
"As a nonprofit organization, our priorities are not determined by commercial incentives or the size of a market, they are solely driven by the medical needs of patients suffering from rare diseases and the wealth of good science already available. We strive to apply our patient-centric model and our pharma R&D expertise to advance the discovery of new treatments for these underserved patients," commented Caroline Kant, Executive Director and Co-Founder of the EspeRare Foundation. "With our first program in Duchenne, our goal is to improve lives of young boys affected by this debilitating and fatal disease," she added.
Sharon Terry, President of the EspeRare Foundation, added: "The International Rare Disease Research Consortium has publicly and boldly declared that we, as a global community, will have 200 new drugs for rare diseases by 2020. The EspeRare Foundation has a great potential to become an important component in the new system that must be built to achieve this goal. The current pharma model needs to be adapted, new pathways are being forged and the Foundation will be a catalyst and incubator for a new way of addressing rare diseases."
The EspeRare Foundation will be presented to the public and to the international rare diseases community, at the first IRDiRC conference, taking place in Dublin, Ireland, on April 16-17, 2013.
The EspeRare Foundation is the seventh organization to benefit from the support of the Merck Serono EPP. Since 2012, this program has contributed to the successful creation of local biotech start-ups (Prexton Therapeutics, Asceneuron, Calypso Biotech) and service companies (Quartz Bio, TQM Insight, Ondaco). The EspeRare Foundation headquarters will be hosted in Eclosion, a public incubator for life sciences start-ups located in Plan-Les-Ouates, Geneva.