Fast forwarding treatments for children with rare diseases

Transcription

1 Fast forwarding treatments for children with rare diseases February Photo credits: Ceridwen Hughes,

2 EspeRare Bringing treatments to children affected by rare diseases in a non-profit, socially responsible way EspeRare is using philanthropic funding as a social investment EspeRare is combining pharmaceutical know-how and philanthropic investments to revive dormant IP and save children who are otherwise not addressed by the pharma industry Over the last 5 years, EspeRare has develop 5 treatments that together have the potential to transform the lives of 2 million children in 4 different life-threatening diseases EspeRare is looking for socially-minded donors and investors to fund our ground-breaking selfsustainable model and our life-saving treatments EspeRare s non-profit model multiplies the impact of philanthropic investments to alleviate the suffering of millions of children & their families

3 Foundation origin EspeRare, a unique wiss non-profit venture fast-forwarding the treatments for children with rare diseases Who? Three former pharmaceutical executives Collective experience of 40 years in pharma Initial financial endowment from Merck erono. When and Where? Founded in 2013, recognized by wiss authorities as a non-profit with full tax exemption and member of the Translational Giving Europe network EspeRare is part of the Campus Biotech hub, the Geneva life-science pole of excellence hosting research institutes and biotech companies Why? To propose a more effective, fast-forwarding approch With an inclusive non-profit business model Resulting in accessible treatments for children suffering from rare and life-threatening diseases Florence Porte Caroline Kant Beatrice Greco By establishing EspeRare, we fulfil our dream of dedicating our pharma expertise to children battling rare diseases

4 EspeRare goals EspeRare bridges the translational valley of death between academia research and pharmaceutical drug development Using its collaborative approach and solid industrial drug development expertise, EspeRare coordinates all necessary R&D activities to address the therapeutic death valley in rare diseases

5 EspeRare goals A novel approach to enable the development of repositioning opportunities for rare diseases Proprietary informatics platform for opportunity identification Hybrid funding (public, philanthropic, private and profit from R&D) Repositioning existing drugs Patient engagement Access to medicine objectives Collaboration with Key Opinion Leaders Mission developing accessible treatments for children suffering from life-threatening rare diseases

6 EspeRare Focus Drug repositioning: a de-risked & faster approach to therapeutic development with sound business case in orphan diseases Drug repositioning: Rescuing untapped opportunities for children with rare diseases with 40% less investment, 20-45% less cycle time and 30% higher chance to reach patients

7 Our Portfolio Our portfolio: 5 drug development programs addressing diseases affecting 2 million children Rare diseases addressed Preclinical Phase 1/2 Duchenne muscular dystrophy Pulmonary hypertension Pediatic cancers Focal seg. glomerulosclerosis Rimeporide Rimeporide EspoiR-005 EspoiR-003 Outlicensing 2018 Preclinical PoC 2018 Preclinical PoC 2018 Diagnostic dev X-linked ectodermal dysplasia EspoiR-004 Late stage clinical dev set-up Congenital heart defects FloWatch device (commercial registration) Market relaunch/fundraising

8 Project example: Rimeporide A de-risked clinical asset in Duchenne Muscular dystrophy addressing skeletal & cardiac muscular degeneration Addressing Duchenne muscular dystrophy Genetic disease affecting 50,000 boys worldwide Low life expectancy : 2 nd to 3rd decade of life High unmet medical need What is EspeRare doing with Rimeporide? Transforming a dormant asset into a novel and commercially viable treatment, improving vital and functional prognosis. What is the action plan? ü COMPLETED Validation of Rimeporide's therapeutic potential with a successful phase Ib in children with DMD and robust preclinical package and Orphan Drug designation Critical baking and input from the patient community (funding, access to experts, input on dev. design) Ø IN PROCE Partnering discussions to transition the program in late stage drug development Preclinical Proof of Concept in Hypoxic Pulmonary Hypertension Rimeporide has the potential to transform Duchenne from a life-threatening disease to a chronic one Main clinical Key opinion Leader in Cardiology in DMD, France

9 ecuring the Future Impacting rare diseases: uncovering new opportunities with our proprietary IT repositioning platform Our drug repositioning platform: a collaborative, multi-layer approach to identify drug repositioning candidates for selected rare diseases 1. Prioritize Rare Indications Prioritizing according to diseases drug development criteria's 2. elect IND ready assets elect cpds with de-risked properties for drug development & business potential 3. In silico discovery of repositioning opportunities Apply a range of computational approach for opportunity 9 identification

10 Growth & Impact plan Today EspeRare has achieved proof of concept: maximizing impact for orphan diseases & handing over business model to others 2030 Achieved! ~2 million patients 5 programs ü5 programs ü3 of which at advanced stage ~5 million patients 6-7 programs ü Platform and capacity building ü Team expansion ü 6-7 programs at steady state ü Financial return on 2 programs 2025 ~10 million patients 6-7 programs ü elf-financed with profit reallocation ü Expansion to underserved patients & very rare diseases ü Replication capability (toolbox, governance) ü Financial return on programs ~50 million patients 5 ü eed funding for about 10 EspeRare s sister organizations (1, 2,..) ü Model replication in other niche markets ( personalized medicine, neglected diseases) ü Maximised and globalized impact programs per organization Proof of concept Reach sustainability Impact Expansion EspeRare model replication

11 Thank you! Contact: EspeRare Foundation Campus Biotech Innovation park I Avenue echeron 15 I CH-1202 Geneva kant.caroline@esperare.org 11