ANNUAL REPORT 2015 Mission & vision MISSION VISION At EspeRare, we address the translational A world in which patient engagement, gap in paediatric rare diseases by uncovering good science, pharmaceutical excellence the potential of existing therapeutic inter- and health authorities come together to ad- ventions to tackle unmet life-threatening dress the medical needs of children affected medical needs. by rare diseases, ultimately alleviating the healthcare burden of their conditions. $VDQRQSURĆWZHDFKLHYHWKLVWKURXJKD collaborative approach centred on patient engagement with the aim of giving people universal access to these therapies. Picture of EspeRare founders : Florence Porte-Thomé, Caroline Kant-Mareda & Béatrice Greco 2 EspeRare | Annual Report 2015 Message from the President 4 2015 highlights 6 Addressing rare diseases 8 Advancing rare disease treatments 10 The translational gap, a major roadblock for new treatments 12 Building our portfolio 14 First programme: Rimeporide in Duchenne Muscular Dystrophy 16 Second programme: Cilengitide in Focal Segmental Glomerulosclerosis 18 Organisation 20 Financial View 26 How you can support EspeRare 34 Established in Switzerland in 2013, EspeRareLVDQHZQRQSURĆWIRXQGDWLRQZLWKJOREDO reach, focused on accelerating the development of treatments for underserved patients affected with rare diseases. EspeRare | Annual Report 2015 3 Message from the President The EspeRare foundation was launched at the International Rare 'LVHDVHV 5HVHDUFK &RQVRUWLXP ĆUVW Congress in Dublin in April 2013. Our goal is to pioneer a collaborative model that accelerates and de-risks the devel- opment of treatments for underserved patients affected by rare diseases. Our Product Development Partnership model for rare diseases is a unique QRQSURĆW EXVLQHVV PRGHO WKDW EULQJV together public, private, academic and philanthropic sectors to develop unex- plored or sleeved treatments for debili- tating rare diseases. 4 EspeRare | Annual Report 2015 “ If opportunity doesn’t knock, build a door.” Milton Berle Dear Friends, It is hard to believe, but EspeRare is three We are very excited to have reached a point As we have validated the relevance of our years old! In this short time, we have only where we have validated the relevance novel model, we are now shifting into an felt a strong increase in interest and need RI RXU XQLTXH PRGHO ZLWK RXU ĆUVW 5 ' expansion phase. We are scaling up with the for the niche in which we have placed programme: the rescue of Rimeporide, development of a portfolio of therapeutic our efforts. While there is a tsunami of a drug that was left abandoned by a programmes in multiple rare diseases. interest in rare diseases, and even in drug pharmaceutical company and for which we As part of a longstanding partnership repurposing, all of the other activities KDYH REWDLQHG 2USKDQ 'UXJ 'HVLJQDWLRQ with Ashoka, McKinsey has worked with DUH LQ WKH IRUSURĆW VSDFH 2XU PLVVLRQ LV this year in Duchenne muscular dystrophy. Caroline and her team to enhance the comprehensive: not only do we want to Thanks to our highly collaborative and delivery of our unique approach and repurpose existing drugs for rare conditions, philanthropic model, not only have we given maximise our impact for the patients we but we are also concerned that access a chance to this drug to reach those children serve. To this end, McKinsey supported us to these drugs is fair and equitable. We in need by combine public, philanthropic in thinking through our business model and are concerned that current trends in rare and private funding but we are proud to GHĆQLQJRXUJURZWKSULRULWLHV diseases show astronomical prices being set report that we are now investigating this and accepted by some markets around the drug in boys affected by Duchenne in four Personally, as I work in the rare disease world. The EspeRare model was developed European clinical reference centres. world, particularly in a leadership role in as an alternative to this costly approach that organisations such as the International is starting to burden health economics and EspeRare has received important Rare Disease Research Consortium and the creating access to medicine challenges. recognition in this past year. Caroline Accelerating Medicines Partnership, I know .DQW RXU &(2 ZDV QDPHG 6ZLVV :RPDQ there is a need for EspeRare because it can We are convinced that accelerating the Entrepreneur of the Year. This recognition, implement the model that great strategists process of moving molecules that have from the Club of Female Entrepreneur, are formulating. We look forward to undergone initial development into another recognizes her tremendous vision in stronger and more energetic collaborations indication is a critical and less risky path to JLYLQJ ELUWK WR D QRQSURĆW RUJDQLVDWLRQ WR with donors, partners, and patients in the alleviating the burden of these diseases. repurpose interventions, decreasing the coming year. They are rare, and affect only small enormous amount of time and money it populations, but taken collectively they generally takes to alleviate the suffering Sincerely, represent a major healthcare burden as they of individuals affected by rare diseases. affect more than 1 in 10 people worldwide. The Swiss economic journal Bilan also Sharon Terry recognised EspeRare as a top 8 Swiss start President up for 2015. EspeRare | Annual Report 2015 5 2015 highlights JAN-FEB MAY 2015 2015 EspeRare selected as a Top Swiss start-up The Rimeporide drug receives the Orphan with an innovative business model Drug Designation in Duchenne Muscular Dystrophy (DMD) Featured by the economic journal Bilan in January for its innovative business model, EspeRare is also The European Medicines Agency has granted the presented in the magazine edition of February in the 2USKDQ 'UXJ 'HVLJQDWLRQ WR 5LPHSRULGH IRU '0' top 8 Swiss start-ups, among 50 promising start-ups a life-threatening muscle disease affecting boys early in from all industries in which one should invest. Q childhood. This designation is a key recognition of the therapeutic potential of EspeRare’s leading drug under development, which is currently being tested in boy’s affected by this devastating disease. Q JULY AUGUST 2015 2015 EspeRare part of a strategic task force to AFM-Téléthon renews its support to address rare diseases EspeRare’s therapeutic development efforts for Duchenne To reach its objective to diagnose most rare diseases DQG WR ĆQG QHZ WUHDWPHQWV IRU UDUH GLVHDVHV The French leading patient organisation has renewed by 2020, the International Rare Diseases Research LWV ĆQDQFLDO VXSSRUW WR (VSH5DUHèV SURJUDPPH LQ Consortium (IRDiRC) created a data mining and 'XFKHQQH 7KLV JUDQW LV FRĆQDQFLQJ FOLQLFDO WULDO repurposing task force to enhance collaborations activities currently being initiated in several hospitals and exploit tools to accelerate the development of in Europe. The AFM has also invited the Rimeporide QHZ WUHDWPHQWV 7KH IRXQGDWLRQèV &(2 ZDV LQYLWHG programme into its strategic portfolio, which gives to become a member of this group and provide her access to AFM’s network of biomedical experts to H[SHUWLVHDQGH[SHULHQFHLQWKLVĆHOGQ best advance Rimeporide for boys burdened by such debilitating disease. Q 6 EspeRare | Annual Report 2015 “ The voyage of discovery is not in seeking new landscapes but in having new eyes.” Marcel Proust SEPTEMBER OCTOBER 2015 2015 CFE Swiss Woman Entrepreneur Award Rimeporide in Duchenne presented at granted to EspeRare’s CEO the 20th WMS Congress This award highlights the exceptional entrepreneurial EspeRare actively participated in the 20th work of a business woman in Switzerland. By winning International World Muscle Society Congress this price, Caroline Kant gains a strong recognition for in Brighton (UK) with a poster presenting the her efforts in driving forwards the novel business model rimeporide compound and its possible applications in of EspeRare. “I am very proud that a mission-driven Duchenne Muscular Dystrophy. Q business was chosen for this year’s award, this shows that in today’s economy, social entrepreneurship is starting to JDLQLPSDFWUHFRJQLWLRQëFRPPHQWHG(VSH5DUHèV&(2Q DECEMBER DECEMBER 2015 2015 McKinsey supports the foundation (VSH5DUHEHFRPHVD6,%DIĆOLDWH enhancing its impact for patients 7KH 6ZLVV ,QVWLWXWH RI %LRLQIRUPDWLFV LV D QRQSURĆW McKinsey supported EspeRare to think through coalition of over 60 bioinformatics research and service LWV EXVLQHVV PRGHO E\ GHĆQLQJ LWV LQQRYDWLYH YDOXH groups in Switzerland. EspeRare has been invited to proposition and growth priorities. As part of a MRLQ WKLV QHWZRUN DV DQ DIĆOLDWHG LQVWLWXWLRQ :LWK WKLV longstanding partnership with Ashoka, McKinsey partnership, the foundation will be able to collaborate has worked with Esperare to enhance the delivery and leverage world-class bioinformatics collaborations of its highly distinctive approach and to maximize its and infrastructure that will support the development of impact for patients. Q its drug repositioning platform for rare diseases. Q EspeRare | Annual Report 2015 7 Addressing rare diseases WHAT IS A RARE DISEASE? EMPOWERED ADVOCACY ORGANISATIONS IN RARE DISEASES In Europe, any disease affecting less than Particularly in rare conditions, disease 1 person in 2000 is considered rare. In the advocacy organisations are key part- 86DGLVHDVHRUGLVRUGHULVGHĆQHGDVUDUH ners at each stage of drug development: when it affects less than 200,000 people in the US.1 ÆGiven the scarcity and fragmentation of medical expertise, patients are highly Rare diseases are chronic,