Advancing treatments for rare diseases together

News

27Feb

EspeRare's CEO in the spotlight in Biotech Finance

Esperare, Foundation, Interview, Caroline Kant, Journal, Finance

For its issue "Special rare diseases", the journal Biotech Finances has chosen to feature EspeRare's CEO, Caroline Kant.The interview summarises Caroline Kant's career path as well as EspeRare's aims, realizations and business plan, thereby informing finance and biotechs specialists of EspeRare's unique business model.

http://www.eei-biotechfinances.com/

03Feb

EspeRare Foundation receives financial support from AltroDomani Onlus and Parent Project Onlus

With this financial support, AltroDomani Onlus and Parent Project Onlus associations contribute to EspeRare’s first sponsored clinical study conducted with Rimeporide in patients with DMD in Europe (RIM4DMD). This support will provide financial back up for the follow-up of patients who participate in this clinical trial in Italy and facilitate the organisation of the travel and accommodation of these patients and their families during their study visits at the hospital.

The RIM4DMD clinical study was initiated in June 2016 at the San Raffaele Hospital in Milano where Dr Stefano Previtali is the principal investigator. Recruitment at this site is ongoing.
 
The study is also open for recruitment in 3 other clinical centres in Europe: the Santa Creu i Sant Pau Hospital (Barcelona, Spain), the Armand Trousseau Hospital/ I-motion (Paris, France) and the Great Ormond Street Hospital (London, UK).

To find out more about EspeRare's RIM4DMD clinical project, please click here.

To find out more about AltroDomani Onlus, please click here.

To find out more about Parent Project Onlus, please click here. And to read this news on Parent Project Onlus' website, please click here.

01Dec

EspeRare on screen!

EspeRare is on screen in UBS branches to promote social innovators.

To find out more about this UBS programme, click here.

16Nov

EspeRare Launches a New Program for Newborn Babies with Congenital Heart Defects

EspeRare is delighted to add a new program to its portfolio and to announce the relaunch of a previously marketed active implantable medical device, FloWatch.  EspeRare recently obtained the licensing rights to this clinically-proven device that has medical benefits in protecting the heart and lungs of babies born with severe congenital heart defects. This is EspeRare’s first “medical devices” program, adding diversification to our present portfolio of 4 therapeutic drug programs. This is also EspeRare’s first, market-ready therapeutic intervention. EspeRare’s medical devices team is working hard to have the first FloWatch units manufactured and marketed before the end of 2017.

FloWatch is a technology that could be offered to up to 1000 babies per year in Europe and to over 30,000 in Africa and Asia. In these latter regions, and in line with EspeRare’s ethical goals of universal access to medicine, we are evaluating a distribution model for FloWatch that is socially responsible, maximizes medical impact, and whereby profits made from the FloWatch sales in the developed countries support philanthropic distribution in the poorer countries.

Find out more about the FloWatch programme

Find out more about Congenital Heart Defects

Find out more about the FloWatch device

 

05Oct

Update on clinical development of Rimeporide in Duchenne presented at the 21st International Congress of the World Muscle Society

EspeRare is presenting its latest update on the clinical development of Rimeporide in Duchenne Muscular Dystrophy, "Translational Development of Rimeporide, a Sodium-Hydrogen Exchanger (NHE-1) Inhibitor, for Patients with Duchenne Muscular Dystrophy", at the 21st International Congress of the World Muscle Society in Grenada (Spain), October 4th to 8th.

rimeporide_esperare_WMS2016Click on picture to view

For more information on the WMS 2016 congress click here

05Aug

EspeRare initiates the development of a new therapeutic approach for Duchenne

EspeRare is pleased to announce a collaboration between Duchenne UK, a Duchenne patient organisation, and Professor Dominic Wells at the Royal Veterinary College, to test a potential new anti-inflammatory and anti-fibrotic therapy on the mdx mouse model for Duchenne Muscular Dystrophy (DMD).

Duchenne UK is providing £67,980 to fund an eight-month program that will assess the potential benefits of EspeRare’s ER03 compound in DMD. This preclinical studies will look at the scientific rationale to “re-position” the compound as a treatment for DMD.

This compound has already been tested in several animal models of inflammation and fibrosis. It has also been tested for safety in healthy volunteers, in several Phase I studies as well as in a Phase II study.

Those living with Duchenne lack the muscle protein dystrophin which acts as a ‘shock absorber’ for muscle. Without a shock absorber, everyday use of muscles cause them to become damaged. This damage promotes inflammatory processes which in turn cause hardening and scar formation. Muscles damaged in this way are termed fibrotic. It is hoped that this potential new therapy will be effective in treating both inflammation and fibrosis in the damaged muscles of those living with Duchenne. This would be a treatment for all those living with Duchenne, regardless of their age or DMD gene mutation.

If this research program shows promise, the compound will be able to go straight to a Phase II Proof of Concept study in DMD patients. In line with our commitment to repositioning existing drug and bring medicines to market in an accelerated way, Duchenne UK and EspeRare are committed to further develop this treatment in Phase II, if the compound shows efficacy.

Emily Crossley and Alex Johnson, Co-Founders of Duchenne UK, said; “We are delighted to be able to join forces with Esperare and the Royal Veterinary College, to confirm the efficacy in DMD and advance our knowledge about this compound. By leveraging on the available data package and capitalizing on its safe use in humans in previous studies, we hope that if it shows promise, we could go into patients with DMD as soon as early 2018.”

Florence Porte, CSO of EspeRare said: “Thanks to the generosity of Duchenne UK, it has been possible to transform this shelved asset into a potential novel therapeutic option for patients with DMD. This innovative repositioning approach may offer to patients with DMD a new and safe disease modifier addressing inflammation and fibrosis and which may be combined with dystrophin replacing therapies.”

For more about Duchenne UK -> link

Duchenne UK news release -> link

For more about Royal Veterinary College -> link

28Jul

EspeRare CEO becomes an ambassador of the UBS social innovators program

EspeRare is proud to collaborate with UBS on this international search for groundbreaking social innovators. In collaboration with ASHOKA, the financial institution will select high-potential social enterprises that address some of society’s most pressing challenges and, over the course of a year, support them to scale up their operations and impact. Caroline Kant, EspeRare CEO has become an ambassador to this program, as part of her mentoring role she will help select the wining social innovators and share her experience in pioneering social impact for orphan diseases.

"To address the unprecedented magnitude of today’s social challenges, financial and social innovation needs to come together. It is my hope that this program will demonstrate that social and financial success can merge to flu greater good” commented Caroline Kant

For more on the UBS program -> Link to the webpage
To view Caroline Kant's profile as a UBS ambassador -> Link 

27Jul

EspeRare receives key support from Duchenne Parent Project Spain

Duchenne Parent Project Spain has been instrumental for the set-up of the Spanish clinical activities that focuses on testing Rimeporide in patients affected by Duchenne Muscular Dystrophy. Over the last few months, Dr Marisol Montolio Del Olmo helped EspeRare make the link with Dr. Jordi Díaz Manera from the Santa Creu i Sant Pau Hospital in Barcelona who will lead the first safety study of Rimeporide in 6 to 14 years old boys affected by Duchenne in Spain. In addition, DPP Spain is also providing administrative and funding supports for the travel and accommodation of patients and families who will be participating in this clinical trial.

Florence Porte Thomé, R&D Director of EspeRare said “Without the intervention of DPP Spain, it may not have been possible for EspeRare to open a clinical site in Spain. This partnership is a prime example of the power and importance of collaborating with patients organization to develop treatments for Duchenne Muscular Dystrophy.”

The study is now opened to recruitment in four clinical centers in Europe, the Santa Creu i Sant Pau Hospital (Barcelona, Spain), Armand Trousseau Hospital/ I-motion (Paris, France) and the San Raffaele Hospital (Milan, Italy) and soon in the Great Ormond Street Hospital (London, UK). “

For more information please visit: 

-> www.clinicaltrials.gov 

-> DPP Spain news release

 

29Jun

EspeRare at the G21 Swisstainability Forum

The foundation's co-founders participated to the 6th edition of the Swisstainability Forum to debate on new business models to foster economic and social sustainability of Switzerland.

The G21 was held the 28-29 June 2016 at the SwissTech Convention Center, EPFL, Lausanne.

This annual event is meant to be the platform for the economic transition in Switzerland. Its aim is to encourage meetings and collaboration between scientific, economic, and political actors and NGOs, to support the companies in their innovating and sustainable projects, and to showcase the best initiatives and innovations which will be the leading projects of tomorrow, always in a forward-looking spirit.

For more -> link

23May

EspeRare 2015 annual report is available

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

Please click here to read our new annual report.

23Mar

A important progress for patients living with Duchenne: Rimeporide starts its clinical development

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's model presented at the United Nations

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

Rimeporide in Duchenne presented at the 5th Myology Congress

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

ICRC Global Partnerships for Humanitarian Impact and Innovation event

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 supports EspeRare enhancing its impact for patients

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

Rimeporide in Duchenne presented at the 20th WMS Congress

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

EspeRare's CEO wins the 2015 Swiss Woman Entrepreneur Award

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

AFM-Téléthon renews its support to EspeRare's drug development efforts in Duchenne

Duchenne Rimeporide

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

EspeRare on IRDiRC's data mining & repositioning taskforce

IRDiRC, Big Data, Repositioning

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

EspeRare’s Rimeporide receives Orphan Drug Designation in Duchenne Muscular Dystrophy

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

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