Advancing treatments for rare diseases together

News

23Mar

EspeRare Today achieves a significant milestone by enrolling patients with DMD in Rimeporide phase Ib trial:Our first DMD patient was recruited in France

Rimeporide, EspeRare's first programme reaches today a decisive clinical stage milestone. After encouraging preclinical results obtained in leading institutions and an orphan drug designation granted from the European Medicines Agency, the recruitment of patients with Duchenne Muscular Dystrophy from 6 to 14 years of age in a phase Ib study with Rimeporide started in Paris at the centre of I- Motion. Leading European neuro-paediatricians will be recruiting up to 20 patients with DMD in 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).

This study is investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of a 4-week treatment with rimeporide in these patients. In contrast to many therapeutics under development in DMD, rimeporide is indicated to all patients with DMD regardless of the causative mutations and age. To our knowledge, Rimeporide is one of the few clinical stage therapy intended to reduce inflammation and fibrosis both in skeletal muscles and in the heart.

 

Our hope is that Rimeporide may be a big step forward alone or in combination with other available treatments to address DMD including cardiomyopathy which at present is a progressive disease with very limited therapeutic options.

 

For more information see our website www.esperare.org/en/duchenne-programme and also www.clinicaltrials.gov

15Mar

EspeRare is invited to present its innovative business model during the next expert meeting of the United Nations Conference on Trade and Development (UNCTAD) on 16th and 17th March 2016 in Geneva.

This multi-year meeting will focus on the impact of investment, innovation and entrepreneurship on productive capacity building and sustainable development. Countries’ representatives and entrepreneurs will show the role played by entrepreneurship and small and medium enterprises in this field.

The foundation’s CEO will explain how EspeRare’s business model addresses the translational gap in rare disease research and development and bridges academic research efforts with pharmaceutical development. The foundation is acting as a development accelerator, minimizing the risks and giving a chance to patients neglected by the medicine all around the world to access treatments. This presentation will be the opportunity to discuss how international policies can support the development of EspeRare’s business model and further enhance its impact on global health.

Delegates from about 70 countries including policy makers, investment experts, academia and private sector development institutions are expected to participate to this interactive debate.

About the UNCTAD
The UNCTAD was established in 1964 to address development issues by promoting international policies to support the equal integration of developing countries in the global economy and help them to ameliorate the conditions of their populations.

For more information please visit the UNCTAD website http://www.unctad.org

12Mar

EspeRare will present a poster on the "Translational Development of Rimeporide, a Sodium-Hydrogen Exchanger (NHE-1) Inhibitor, as a therapy for Patients with Duchenne Muscular Dystrophy" at the 5th Myology International Confenrence in Lyon from 14th to 18th March 2016.

Poster for Myology 2016 jpeg
Click on picture to view

For more information on the Myology Congress click here

26Feb

EspeRare's innovative business model will be presented at the GPHI2 conference. On the 3rd and 4th March 2016, over 200 leaders from the business, humanitarian, health and academic sectors will gather at the IMD Business School in Lausanne to address priority issues in the field of health in Fragile Environments. Two key themes will take central stage of the event:

a) Ensuring and expanding access to health in fragile environments.
b) Enhancing the response to emerging and unmet needs.

For more follow this link 

04Dec

McKinsey supported EspeRare to think through its business model by defining its innovative value proposition and growth priorities. As part of a longstanding partnership with Ashoka, an organization which identifies and support social entrepreneurs, McKinsey has worked with Esperare to enhance the delivery of its highly distinctive approach and to maximize its impact for patients. The consulting company also applied knowledge and experience to support the foundation in the development of a methodology to assess its qualitative and quantitative public health impact.

McKinsey & Company is a management consulting firm that helps leading organizations across the globe to improve their performance.

For more click on the following link

11Oct

EspeRare presented the “Development of Rimeporide, a sodium-hydrogen exchanger inhibitor, as a therapy for Patients with Duchenne Muscular Dystrophy”  at the 20th International Congress of the World Muscle Society in Brighton from 30th September to 4th October 2015.

Development of Rimeporide, a Sodium-Hydrogen Exchanger (NHE-1) Inhibitor, as a therapy for Patients with Duchenne Muscular DystrophyClick on picture to view

For more information on the WMS Congress click here

23Sep

Lausanne, Switzerland September 23th 2015, Caroline Kant's achievements of driving forwards the novel business model of EspeRare was honored in a ceremony held at the EPFL Rolex learning center. She became the CFE Woman Entrepreneur of the Year. This prize promotes entrepreneurial performances of a successful woman in business in Switzerland. “I am very proud that a mission-driven business was chosen for this year’s award, this shows that in today’s economy, social entrepreneurship is starting to gain impact recognition” commented Caroline Kant.

For more on the award follow this link

04Aug

Following AFM’s positive review of EspeRare’s plans to further develop Rimeporide in children affected with Duchenne muscular dystrophy (DMD), the french leading patient organization has renewed its financial support to EspeRare’s program. This grant will co-finance a biomarker study and partly the phase Ib clinical trial activities that are currently being initiated in several clinical centers in Europe.
In the context of this additional commitment the French organization, pioneer in funding and supporting rare disease research, has also invited the Rimeporide program into its strategic portfolio. This will provide EspeRare a facilitated access to AFM’s network of biomedical experts and strategic guidance to best advance Rimeporide for boys burdened by such debilitating disease.

02Jul

The foundation's CEO will serve as a core member of a strategic effort to advance progress in the field of Data Mining and Repurposing. The purpose of this Task Force is to gather global experts and identify opportunities for collaborations to speed up the exploitation of these new discovery tools.
The International Rare Diseases Research Consortium teams up researchers and organisations investing in rare diseases research in order to achieve two main objectives by the year 2020, namely to deliver 200 new therapies for rare diseases and means to diagnose most rare diseases.
The Executive Committee of IRDiRC selected several topics for action which could be instrumental in accelerating R&D, among which, data mining and repurposing. As a key player in this field, EspeRare’s CEO, Caroline Kant has been invited to contribute to this “Data mining and repositioning Task Force”.

About IRDiRC
The International Rare Diseases Research Consortium (IRDiRC) was initiated by the European Commission and the US National Institutes for Health Research and launched in April 2011 to foster international collaboration in the rare diseases field. IRDiRC will team up researchers and organizations investing in rare diseases research in order to achieve two main objectives, namely to deliver 200 new therapies for rare diseases and means to diagnose most rare diseases by the year 2020.

For more click on the following link

04May

Geneva, Switzerland, May 4th 2015 – The European Medicines Agency (EMA) has granted an Orphan Drug Designation (ODD) for rimeporide, its lead compound for the treatment of Duchenne muscular dystrophy (DMD). DMD is a rare, life-threatening disease affecting boys early in childhood that causes muscle weakness and muscle loss. It is the most common and serious form of paediatric muscular dystrophies.

Rimeporide is a selective sodium/proton exchanger type-1 inhibitor, originally developed by Merck Serono in the intended indication of congestive heart failure. In clinical studies to date, rimeporide was shown to have a clinically acceptable safety profile in adults. Rights to rimeporide were transferred to EspeRare in 2013. Since then, EspeRare successfully conducted two non-clinical studies at the Children Medical Centre in Washington (USA) and the University of Geneva (Switzerland). The results were presented at the ICNMD XIII Congress on Neuromuscular Diseases in Nice (France) last year. “Rimeporide’s potential to address skeletal muscle inflammation, fibrosis and cardiomyopathy in a broad population of patients, regardless of their mutational status, could make it an ideal complement to treatments designed to augment or replace dystrophin. Obtaining this ODD is an important milestone for rimeporide and supports its clinical development” commented Florence Porte-Thomé, EspeRare’s R&D Director. The decision, which was made consequent to a positive recommendation from EMA's Committee for Orphan Medicinal Products, represents the first time a sodium/proton exchanger inhibitor has received orphan status for DMD in Europe, positioning this mode of action as an innovative therapeutic approach for DMD.

Leveraging on robust safety and nonclinical efficacy evidence to support clinical development, EspeRare will launch the first clinical study in boys with DMD during the second half of 2015 to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of this drug candidate in these patients. “The nonclinical package indicates rimeporide’s potential to transform Duchenne muscular dystrophy from a life-threatening to a chronic disease” explains Professor Denis Duboc, Cardiologist at the Hospital Cochin in Paris.

Obtaining this orphan drug designation for rimeporide is a demonstration that dormant therapeutic assets can show potential to treat patients affected by orphan diseases. This success encourages EspeRare to further build on its model by expanding its partnerships with biopharmaceutical companies and working towards building a robust portfolio of programs for patients affected by these underserved diseases.
 
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare genetic paediatric disease that affects approximately 1 in 3,500 male babies worldwide. It is a rapidly progressive form of muscular dystrophy caused by a mutation in a gene which encodes the dystrophin protein. Its absence causes progressive skeletal muscle degeneration leading to a loss of ambulation around the age of 10. Then progressive respiratory muscle weakness and cardiac failure both represent major life-threatening complications. Today there is no cure for DMD.
 
About Orphan Drug Designation (ODD)
EMA's Orphan Medicinal Product Designation is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. In addition to granting 10 years of market exclusivity, the designation also provides special incentives for sponsors including eligibility for protocol assistance, possible exemptions or reductions in certain regulatory fees during development or at the time of application for marketing approval.
 For more information, please visit the EMA website  http://www.ema.europa.eu/ema/

Press relations: Gwen Laporte- laporte.gwen@esperare.org

02Apr

The annual report highlights the foundation's achievements and the financial statements.

Please click here to read our new annual report.

02Mar

We are very proud to announce this new opportunity for EspeRare. The pharmaceutical company Boehringer Ingelheim has invited the foundation to join its programm "Making more Health". In this context EspeRare explores the possibility to create a business partnership with this big pharmaceutical company and to benefit from the pro-bono support of experts within the company to foster the foundation future development. This is the recognition for EspeRare of its pioneering model and a true encouragement to continue its commitments and growth.

About Boehringer Ingelheim
Boehringer Ingelheim is a german family-owned company created in 1885. It belongs to the  world’s 20 leading pharmaceutical companies. They develop new medications of high therapeutic value for human and veterinary medicine,  from development phase to marketing.
Involvment in social projects is part of Boheringer Ingelheim corporate culture, like the initiative “Making more Health” in partnership with Ashoka, an organization which identifies and support social entrepreneurs with innovative changing solutions for the world’s most urgent social problems.

About Making More Health
Boehringer Ingelheim’s initiative “Making More Health” aims to select and support most promising solutions to challenging health problems with the help of Ashoka's know-how. The pharmaceutical group brings its professional expertise in the healthcare and business sector, its experience and its management skills. The group also developed a system to foster employees commitment in these social actions.
The three axes of "Making more Health" action are the regular selection of "Fellows, a programme for young people engagement to develop new solutions in health care and a programme with company employees active on-site support.
All these concrete actions aim to bring individuals and communities innovative solutions to undeserved health problems.

To learn more about this, see following links:
http://www.makingmorehealth.org/MMH_Activities/caroline-kant.html
http://www.makingmorehealth.org/

09Feb

Less than 2 years after its launch, EspeRare has made the cut in the top 8 Swiss start-ups. Bilan magazine presents in its February edition the 2015 selection of 50 Swiss start-ups in which one should invest. The selection gives an overview of promising start-ups from all industries. EspeRare has received special recommendations from members of the jury.

For more see link

06Jan

"Social entrepreneurship…which business model to change the world?"
In this article from the economical journal Bilan, Aurore Bui selects six social entrepreneurs with innovative business models, including Caroline Kant for EspeRare.
Please click here to read the article.

22Sep

EspeRare is exploring the therapeutic potential of cilengitide in Focal Segmental Glomerulosclerosis with the University of Bristol, UK. Cilengitide is an investigational compound developed by Merck Serono, the biopharmaceutical division of Merck.

Focal Segmental Glomerulosclerosis (FSGS) is a rare disease that causes impair renal function due to scaring of glomeruli (structures within the kidneys composed of capillary blood vessels actively involved in filtering harmful substances out of the blood). The underlying disease mechanism involves the alteration of cells called podocytes within those glomeruli. Published scientific data suggests that the inhibition of αvβ3 integrin could potentially modulate the pathologic processes characterized by inappropriate motility of podocytes in this rare renal disease.

EspeRare has initiated a collaboration with Professor Moin Saleem at University of Bristol to test the ability of cilengitide, a αvβ3 integrin inhibitor, to activate the motility of podocytes in several FSGS experimental models. If demonstrated, such effect could potentially translate into a reduced or halted decline of renal function and progression to end stage kidney disease in humans. In that context, cilengitide represents an attractive investigational candidate for a potential future therapy for FSGS patients.

This first assessment of the therapeutic potential of cilengitide in non clinical models of Focal Segmental Glomerulosclerosis is set to be completed by the end of 2014, and is co-funded by Merck Serono.

About Cilengitide
Cilengitide is an investigational compound developed by Merck Serono. The small molecule is a potential first in a class of αvβ3 integrin inhibitor that was in clinical development in oncology until Phase III. The development of this drug candidate was halted in 2013.
Merck Serono has also previously provided support to EspeRare in the development of another of its investigational products, Rimeporide, in the deadly pediatric disease Duchenne Muscular Dystrophy, through initial fund donation, and transfer of compound rights to EspeRare. This joint project is still ongoing.

About Focal Segmental Glomerulosclerosis (FSGS)
FSGS is a rare form of a nephrotic syndrome that affects both children and adults with peaks at 6-8 and 20-30 years of age, respectively. The life expectancy of a 10 year-old child on hemodialysis due to end stage kidney disease is dramatically reduced.
The cause of FSGS is unknown. The disease effects a specific part of the kidney called glomeruli. The role of the glomeruli is to filter soluble waste such as by-products of cellular metabolism from the blood, the first step to form urine. When barriers in this filter are damaged, proteins begin to leak into the urine and over time cause kidney failure. The diagnosis of FSGS is based on the presence of proteinuria, possibly signs and symptoms secondary to proteinuria (oedema), and histologic examination of kidney biopsy.The disease burden is tremendous to patients with this lifelong chronic disease; particularly it requires management of dialysis and in some cases transplantation. Currently, there is unfortunately no cure for this debilitating disease.

About Professor Moin Saleem, University of Bristol
Professor Moin Saleem, Professor of Paediatric Renal Medicine, at University of Bristol, School of Clinical Sciences, is a world renowned expert in glomerular diseases and particularly the nephrotic syndromes. He is a pioneer in the field of podocyte biology and originated the gold standard of a conditionally immortalised human podocyte cell line, now in use worldwide in academia and industry. In 2007 he was awarded a Medical Research Council grant to set up the UK renal rare disease registry (www.renalradar.org), which has grown into a national strategy adopted by the UK Renal Association.

03Sep

"These pioneers who are changing the world" is the title of this special issue dedicated to sustainable economy.
This folder dedicated to innovation, provides an overview of entrepreneurs at the heart of social innovation and portrays Caroline Kant, co -founder and executive director of EspeRare. 'Hardships in life' can trigger social engagement, the article describes the journey of a woman who decided herself to the advancing treatments for rare diseases.

See-Express Reussir n#27 –September-October 2014 p42-43

11Aug

EspeRare’s president & executive director are authors of the chapter on the role of Disease Advocacy Organizations in orphan drug development within the book entitled Orphan Drugs and Rare Diseases newly published by the Royal Society of Chemistry. Click here to view chapter abstract

The book is an up-to-date monograph written by a combination of academic and industry experts working in the field of orphan diseases drug development, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat.

To view book content click here

Royal Society of Chemistry

05Jul

EspeRare presented the “Development of Rimeporide, a selective sodium-hydrogen exchanger inhibitor, for Patient with Duchenne Muscular Dystrophy”  at the 13th International Congress on Neuromuscular Diseases in Nice from the 5-10th of July 2014.

Development of Rimeporide, a selective sodium-hydrogen exchanger inhibitor (NHE-1), for Patient for Duchenne Muscular Dystrophy
Click on picture to view

For more information on the ICNMD congress click here

 

28Jun

EspeRare & Merck Serono are one of the 3 prize winners of the “Social & Business Co-Creation: collaboration for impact” competition.

The prize was awarded for its project “Advancing treatment for Duchenne” at the 5th Zermatt Summit “The Courage to Dare” on June 27th. The jury was constituted by Ashoka, the Zermatt Summit Foundation, Fondation Guilé, DPD and Boehringer Ingelheim, they reviewed 338 projects, selected 15 finalists and named 3 winning Social & Business Co-Creation projects.

Please follow this link to see all winners

About Merck Serono
Merck Serono is the biopharmaceutical division of Merck. With headquarters in Darmstadt, Germany, Merck Serono offers leading brands in 150 countries to help patients with cancer, multiple sclerosis, infertility, endocrine and metabolic disorders as well as cardiovascular diseases. In the United States and Canada, EMD Serono operates as a separately incorporated subsidiary of Merck Serono.
Merck Serono discovers, develops, manufactures and markets prescription medicines of both chemical and biological origin in specialist indications. We have an enduring commitment to deliver novel therapies in our core focus areas of neurology, oncology, immuno-oncology and immunology.

About the “Social & Business Co-Creation: collaboration for impact” competition:
The competition Social & Business Co-Creation: collaboration for impact, has been developed by Ashoka, the Zermatt Summit Foundation, Fondation Guilé, DPD and Boehringer Ingelheim to recognize innovative Co-Creation projects in Europe led by social-mission organisations, traditional businesses and public institutions.

The 3 winners receives coaching and a first prize of € 20,000, two runner-up prizes of € 10,000. Two early entry prizes of €1,000, a last mile and a special jury prize are also awarded.

About the Zermatt Summit
Created in 2010 as a primary international platform, the Zermatt Summit promotes a constructive, action oriented dialogue between key stakeholders from different universes who share the same purpose. Its role is to inspire, connect and empower high profile entrepreneurs, economists, political and spiritual leaders and NGO representatives who strive to be catalysts of a more inclusive and human globalization. Through a uniquely designed program, the Zermatt Summit team takes participants on an intense three-day journey to share a vision for a better society, develop models for a more human and sustainable world and change their hearts and minds in the process. 

20May

The annual report highlights the foundation's achievements and the financial statement.

Please click here to read our first annual report.

 

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