Advancing treatment for Duchenne: Give a chance to an unexplored drug to treat children with an incurable disease
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Spread Strategies: Moving forward, what are the main strategies for scaling impact?
Marketplace: Who else is addressing the problem outlined here? How does the proposed project differ from these approaches?
Team
Merck Serono S.A.
14600
Ewen
Sedman
Chief Business Officer, Translational Innovation Platforms R&D
USA
2013
All, once develop it will be made available for all the children affected by this fatal pediatric disease
New products & services: Entries in this category develop and offer essentials product and services that address needs currently unmet.
2.4M and a licence to develop Rimeporide in Duchenne Muscular Dystrophy
Type of partner 1 [business]: pharmaceutical company
-An international mid-size pharmaceutical and life science company, that develops and commercials treatments for neurological and oncological diseases.
- Merck Serono is partnering with EspeRare to enable and accelerate the development of one of its dormant drug (Rimeporide), with the prospect of addressing Duchenne, a paediatric rare incurable disease.
-Merck Serono has granted EspeRare the rights to develop Rimeporide that has previously been developed for heart failure in Duchenne and provide an endowment to the foundation to develop the opportunity.
-Partner 2
-Type of partner [social]: non-profit drug development engine
-A non-profit organisation established in Switzerland in 2013,that is recognised to be operating in the public's interest. Its mission is to drive and accelerated de development of treatment for rare diseases.
-In-line with its philanthropic Product Development Partnership model, through this collaboration EspeRare is enabling the development of unexplored assets for an incurable rare diseases.
-EspeRare is contributing its drug development know-how as well as its patient and expert network. It is driving animal studies, and is developing protocols for clinical trials. It has also raised public and philanthropic funds to co-finance the program activities.
Duchenne Muscular Dystrophy (DMD) is a severe genetic neuromuscular disease that affects 1 in 3,500 boys with an estimated of 50,000 boys affected in the US and Europe. It is caused by a mutation in the DMD gene. Individuals with DMD have progressive loss of muscle function in early childhood. This progressive muscle wasting typically leads to loss of ambulation before 10 years of age. In their 20s, the disease progresses to paralysis, increased difficulty in breathing as well as cardiac muscle dysfunction that leads to heart failure. DMD kills most of the boys it affects by their 20s and currently there is no treatment for this disease. If we are successful, then a major proportion of the 50,000 children will have access to an effective treatment. Additionally, we will have proven a new pathway to affordable treatment development that is set to be replicated for other diseases.
Individuals, Foundations, NGOs, Businesses, National government, Other.
The development of Rimeporide in Duchenne until its first milestone (preclinical validation of its therapeutic opportunity) has been secured by a hybrid funding mechanism that is in line with the non-profit positioning of EspeRare’s drug development model. Concretely EspeRare has received funding from Merck Serono that is complemented by grants from the Swiss government Technology and Innovation bureau and from the French Telethon (AFM). In addition, EspeRare receives in kind support from Genetic Alliance, an international advocacy organization based in the USA. Past the first milestone, Merck Serono has committed to reinvest in the project to finance the early clinical development of the opportunity. Beyond the Duchenne program, EspeRare is building a portfolio of drug development programs in rare diseases, and has initiated 2 other programs.
As a mother of a child affected by a rare disease, Caroline is aware of the power of patient engagement in rare disease drug development. In this context in 2011, she encountered another mother of children with a rare disease, US Ashoka Fellow Sharon Terry, pioneer in citizen health and patient empowerment, with whom she developed a patient-centric model for drug development that became a building block in the EspeRare model. In 2012, Caroline with its two co-founders, successfully received an initial funding of €2.8 million from Merck Serono to launch the foundation and develop its first program in Duchenne. By establishing EspeRare, the founders Caroline Kant, Florence Porte and Beatrice Greco along with Sharon Terry, the foundation’s president, achieved a breakthrough in developing an innovative model to accelerate drug development for underserved patient burden by rare diseases.
For EspeRare to be able to give a chance to this dormant drug to address Duchenne, Merck Serono had to grant this valuable pharmaceutical asset to EspeRare and endow the foundation with a donation. For this to happen, the partnering model needed to merge pharmaceutical commercial interests and the foundation’s philanthropic mission of driving therapeutic development for a small and underserved patient population. This was overcome by building a value proposition that serves both interests. On one-hand EspeRare de-risks and accelerate the development of a Rimeporide in Duchenne and the other hand Merck Serono did commit to an agreement model that secures full development and commercialization of the drug within an ethical, access to health framework.
The partners have signed a licence agreement that grants EspeRare the rights to develop Rimeporide in Duchenne. In this partnership, Merck Serono is contributing its pharmaceutical asset and expertise. EspeRare is bringing its patient/expert network and its operational agility. In the future, Merck Serono has the opportunity, within an appropriate ethical framework, to buy back the program for late development and commercialisation. A Joint Committee drives shared decision-making between on the Duchenne program while leaving EspeRare free to engage with other partners.
This partnership is transformative because it requires that an NGO performs, in a very complex environment and with the discipline of a company, drug development. It also requires that a pharma company risk sharing an asset and supporting its development without blockbuster potential. Partnerships between for profit and non-profit organizations require the creation of a novel structure to allow the best of both cultures to flourish, and new interfaces to be established. For instance, Caroline carefully selects the good contact person within the pharmas companies who can share her vision and be able to bring his organization forward; she also gained the trust of patients’ organisations and fully integrated them in the process. On the other hand, Merck Serono has agreed to explore a new way of doing business while being flexible and collaborative to address a rare disease.
Ashoka team