News

The ella fund will support the development of EspoiR-005 (ER-005) in cancers affecting children.
This initial endowment will fund a series of proof-of-concept screening studies to better characterize ER-005 effects in childhood cancers. To this end EspeRare is collaborating with the Centre Léon Bérard in Lyon, France, a cancer referral and treatment centre and research institute specialised in rare tumours, comprising a paediatric hemato-oncology unit.

Our new therapeutic candidate is a first-in-class candidate for the treatment of rare paediatric cancers. EspeRare obtained the rights to that molecule from a pharmaceutical partner and gained access to the results of previous studies on that compound, including safety and efficacy in humans.

Vast progress has been done in cancer treatments over the last years, however cancers affecting children have not received the same attention from the industry. In view of the severe unmet needs for these underserved young patients, should this initial research phase supported by the ella fund produce promising results, EspeRare intends to rapidly progress the development of this therapy into patients.

About The ella fund

The ella fund is a philanthropy fund sheltered by the Swiss Philanthropy Foundation that seeks to empower girls and women through education, healthcare and entrepreneurship. With this goal in mind they have recognised EspeRare as a promising venture philanthropic drug development organisation led by women.

The Fondation Pictet financially supports EspeRare’s new programme in a disabling and rare skin disease called X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). EspeRare is honoured that such a prestigious foundation acknowledges this new project, designed to cure the most threatening symptoms of this disease.

XLHED is a serious rare disease that is life-threatening, particularly in the first years of life. Infants affected by this genetic disorder cannot sweat and are at risk of sudden death due to hyperthermia and severe respiratory issues. See here for more information on the disease.

This new programme, called EspoiR-004 (ER-004) is the first and only therapy for this disease. It is also one of the first treatments designed to be delivered in-utero, paving the way for other such treatments.

On the occasion of the 10th anniversary of its TV show Toutes Taxes Comprises (TTC), the Swiss TV channel RTS carried out a report on the Health Valley Switzerland.
EspeRare illustrates, alongside Sophia Genetics and Mindmaze, the diversity, complementarity and dynamics of this ecosystem.

The Health Valley Switzerland, whose name is inspired by Silicon Valley in the USA, is a grouping of biomedical companies from French-speaking Switzerland. The network involves public and private research centres, companies and start-ups working in biomedical technologies, as well as hospitals and clinics. It includes more than 1,000 member companies and 25,000 employees. The Health Valley fosters interaction among its members, and gives international visibility to the biomedical expertise of French-speaking Switzerland.

The Food and Drug Administration (FDA), the US Department of Health and Human Services, has granted an Orphan Drug Designation (ODD) for Rimeporide for the treatment of Duchenne muscular dystrophy (DMD). See here for more details.

ODD is designed to promote the development of drugs that may provide significant benefit to patients suffering from rare, life-threatening diseases. In particular, this ODD grants Rimeporide a 7-year data exclusivity in the US.

Rimeporide already received ODD in DMD from the European Medicines Agency in 2015. Further to this designation in Europe, EspeRare has been able to conduct a phase Ib clinical trial of Rimeporide on young DMD patients. This clinical trial will be completed by the end of 2017.

EspeRare is currently looking to re-launch the development of ER004 (formerly known as EDI200) as a treatment for babies affected by X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).

XLHED is a serious rare disease that is life-threatening, particularly in the first years of life. Infants affected by this genetic disorder cannot sweat and are at risk of sudden death due to hyperthermia and severe respiratory issues. See here for more information on the disease.

ER004 is the first and only therapy for this disease. ER004 was developed until 2015 by Edimer Pharmaceuticals (US). Unfortunately its development was stalled following a clinical setback as the treatment, when administered to newborn babies, did not seem to provide therapeutic benefits.

In the meantime, Prof. Holm Schneider, a German medical expert, renewed hope for XLHED patients. When he administered ER004 in-utero to 3 babies (including Maarten & Linus, the babies in the picture), he could show that these patients were subsequently able to sweat normally. These results highlight the strong potential for this therapy, when administered during pregnancy, to address the most debilitating aspects of XLHED. See here for more information on Maarten and Linus.

Using Prof. Holm Schneider’s promising new in-utero approach, EspeRare is now restarting the development of this promesing therapy. Currently, the main hurdle to overcome is to find the right regulatory and funding path forward. Once those challenges are addressed, clinical development in Europe is set to restart as early 2018.

Importantly and in-line with our patient-centered approach, EspeRare is partnering with XLHED patient associations, such as the American organisation NFED, to better understand the patients needs and their expectations from a therapy. See here for news from NFED.

Together with our partners, we are working very hard to ensure a way forward for this ground-breaking treatment. We hope to be able to share more exciting news on this program soon!

EspeRare President’s TedMed talk in now online.

In this inspiring speech, Sharon Terry shares her personal story about how, since science didn’t understand her kids’ rare disease, she and her husband decided to study it themselves. Thus they paved the way for patient empowerment in research and drug development.

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.

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