From the Valley of Death to the Bridge of Life: Models for De-Risking Early Translational Projects from Around the World

BIOTECHNOLOGY INNOVATION ORGANIZATION#BIO2016 Mechanistically-driven R&D Need to show MoA, identify (non)responders - discovery and translational partnerships Early industry R&D increasingly acquisition-based In-licensing & co-development commonplace Late stage failures remain an issue Need access to new tools and technologies - early fail paradigm Clear need for collaboration, but… Why partner up?

BIOTECHNOLOGY INNOVATION ORGANIZATION#BIO2016 We don’t always speak the same lingo I hope they understand what we need I hope they understand what this is Щольыат адолэжкэн с вэл эа. थिस् हस् ग्रेअट् पोटेन्टिअल्

BIOTECHNOLOGY INNOVATION ORGANIZATION#BIO2016 Drug, patient, doctor, payer Establishing safety, efficacy Predictive value of pre-clinical models Understand/validate mode of action Maximising impact Selection/validation stratification biomarkers Clinical trial design, endpoints Market understanding, management team Freedom-to-operate Scale-up, manufacturing Clinical workflow, reimbursement environment

BIOTECHNOLOGY INNOVATION ORGANIZATION#BIO2016 Gordon McCauley - Interim CEO Center for Drug Research & Development, Canada Stewart Hay - Chief Executive Officer Therapeutic Innovation Australia Christopher Austin - Director NIH NCATS, USA Carolyn Buser - Vice President, Discovery Partnerships with Academia; GlaxoSmithKline Moderator: Anton Ussi - Operations & Finance Director EATRIS European Infrastructure for Translational Medicine, EU About the speakers

About EATRIS - your gateway to Europe 80 translational research institutes across Europe Accessible via single contact point - Cutting edge tools, technologies, clinical expertise, patient cohorts

Bridging the gap and filling the gap EATRIS performs two daily functions 1.Facilitate (public-private) collaboration & provide research services, access to ancillary expertise 2.Develop and validate tools, technologies and collaboration models to improve pipeline productivity

How does EATRIS de-risk projects - Examples Imaging innovation hub - 1 pharma, 4 academic sites, jointly developing experimental medicines tools for immune- inflammation therapies, e.g. novel PET tracers Portfolio support - Drug development services for biotech client with portfolio of assets Public-public collaboration - supporting charities and public funders in selection, design & development of early stage assets

BIOTECHNOLOGY INNOVATION ORGANIZATION#BIO2016 Outlook: International, cross-sector collaboration Predictivity, patient selection Gene tox profiling / stratification Tissue/organ-on-a-chip 3D cultures for screening Maximising impact Basket trials, adaptive designs, Enhanced regulatory clarity & dialogue Market understanding, management team Novel funding models, venture philanthropy Supporting public/charity funders in translation