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1Photo credits: Ceridwen Hughes, http://www.samebutdifferentcic.org.uk/
February 2018
Fast forwarding treatments for children with rare diseases
Bringing treatments to children affected by rare diseasesin a non-profit, socially responsible wayEspeRare
• 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 self-sustainable model and our life-saving treatments
EspeRare is using philanthropic funding as a social investment
EspeRare’s non-profit model multiplies the impact of philanthropic investments to alleviate the suffering of millions of children & their families
By establishing EspeRare, we fulfil our dream of dedicating our pharma expertise to children battling rare diseases
“ “
Caroline Kant Florence Porte Beatrice Greco
Who?• Three former pharmaceutical executives• Collective experience of 40 years in pharma• Initial financial endowment from Merck Serono.
When and Where?• Founded in 2013, recognized by Swiss authorities as a
non-profit with full tax exemption and member of theTranslational Giving Europe network
• EspeRare is part of the Campus Biotech hub, theGeneva life-science pole of excellence hosting researchinstitutes 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
EspeRare, a unique Swiss non-profit venture fast-forwarding the treatments for children with rare diseases
Foundationorigin
EspeRare bridges the translational “valley of death” between academia research and pharmaceutical drug development
EspeRaregoals
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
A novel approach to enable the development of repositioning opportunities for rare diseases
EspeRaregoals
Mission → developing accessible treatments for children suffering from life-threatening rare diseases
Proprietaryinformatics platform for opportunity
identification
Patient engagement
Hybrid funding (public, philanthropic, private and
profit from R&D)
Access to medicine objectives
Repositioningexisting drugs
Collaboration with Key Opinion Leaders
Drug repositioning: a de-risked & faster approach to therapeutic development with sound business case in orphan diseases
EspeRareFocus
Drug repositioning:
Rescuing untapped opportunities for children with rare diseases with 40% less investment, 20-45% less cycle timeand 30% higher chance to reach patients
Rimeporide
EspoiR-005
FloWatch device (commercial registration)
Duchenne muscular dystrophy
Focal seg. glomerulosclerosis
Congenital heart defects
EspoiR-003
RimeporidePulmonary hypertension
Phase 1/2PreclinicalRare diseases addressed
Outlicensing 2018
Preclinical PoC 2018
Preclinical PoC 2018
Diagnostic dev
Market re-launch/fundraising
Pediatic cancers
Our portfolio: 5 drug development programs addressing diseases affecting 2 million childrenOur Portfolio
X-linked ectodermal dysplasia EspoiR-004 Late stage clinicaldev set-up
A de-risked clinical asset in Duchenne Muscular dystrophy addressing skeletal & cardiac muscular degeneration
Project example: Rimeporide
Addressing Duchenne muscular dystrophy• Genetic disease affecting 50,000 boys worldwide• Low life expectancy : 2nd 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 PROCESS• 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
“ “
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Impacting rare diseases: uncovering new opportunities with our proprietary IT repositioning platform
Securing the Future
Our drug repositioning platform: a collaborative, multi-layer approach to identify drug repositioning candidates for selected rare diseases
1. Prioritize Rare Indications
Select cpds with de-risked properties for drug development &
business potential2. Select IND ready
assets
3. In silico discovery of repositioning opportunities
Apply a range of computational approach for opportunity
identification
Prioritizing according to diseases drug development criteria's
Today EspeRare has achieved proof of concept: maximizing impact for orphan diseases & handing over business model to others
Growth & Impact plan
~5 millionpatients
~2 million
patients ü Platform and capacity building
ü Team expansionü 6-7 programs at
steady stateü Financial return on 2
programs
5 programs
6-7 programs
ü5 programsü3 of which at
advanced stage
ü Self-financed with profit reallocation
ü Expansion to underserved patients & very rare diseases
ü Replication capability (toolbox, governance)
ü Financial return on programs
ü Seed funding for about 10 EspeRare’s sister organizations (S1, S2,..)
ü Model replication in other niche markets ( personalized medicine, neglected diseases)
ü Maximised and globalized impact
6-7 programs
~10 millionpatients
S3
S2
S4S
5
S1
~50 millionpatients
S6
S7
S8
6-7 programs per organization
Reachsustainability
EspeRare model replication
Impact Expansion
Achieved !
Proof of concept
2018-2019
2025
2030
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Contact:EspeRare Foundation
Campus Biotech Innovation park I Avenue Secheron 15 I CH-1202 Geneva
Thank you !
