IMI 8 th Call Angela Wittelsberger, PhD Scientific Project Manager IMI Open Info Day, Bucharest 10 December 2012

Open collaboration in public-private consortia (data sharing, wide dissemination of results) “Non-competitive” collaborative research for EFPIA companies Competitive calls to select partners of EFPIA companies (IMI beneficiaries) Key Concepts IMI Open Info Day, Bucharest 10 December 2012

Innovative Medicines Initiative: Joining Forces in the Healthcare Sector IMI Open Info Day, Bucharest 10 December 2012

Private Investment in kind (€ 1 billion) EU Public Funding cash (€ 1 billion) EFPIA ACADEMIA HOSPITALS PATIENTS’ ORGANISATIONS SMALL AND MEDIUM-SIZED ENTERPRISES REGULATORS Pharma 1 Pharma 2 Pharma 3 Pharma 4 Pharma 5 Pharma 6 A Typical IMI Consortium IMI Open Info Day, Bucharest 10 December 2012

5 Topics IMI Call 8 IMI Open Info Day, Bucharest 10 December 2012

6 Topics IMI Call 8 TopicIndicative budget EFPIA / IMI JU (in million €) ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections 26.0 / 58.9 ND4BB Topic 1C (Innovative trial design & clinical drug development) 25.4 / 26.4 Developing an aetiology based taxonomy of human disease 18.0 / 18.0 European induced pluripotent stem cell bank up to 30.0 / up to 40.0 IMI Open Info Day, Bucharest 10 December 2012

7 Timelines IMI Call 8 Launch of Call 8: December 2012 Submission of EoIs in SOFIA: from shortly after launch until end of February 2013 Stage 1 evaluation completed: April 2013 Stage 2 evaluation completed: July 2013 Indicative start of projects: October 2013 IMI Open Info Day, Bucharest 10 December 2012

NewDrugsForBadBugs (ND4BB) in IMI Call 8 1)ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections 2)ND4BB Subtopic 1C: Innovative trial design & clinical drug development IMI Open Info Day, Bucharest 10 December 2012

9 The ND4BB programme ND4BB cross-topic collaboration and dissemination (Topic 1 WP1, Topic 2 WP8, Topic 3 WP1, Topic n WPn) Topic 1: Clinical development Steering Committee Project level decision making body Subtopic 1A: Work Packages: 1-4 Subtopic 1B: Work Packages 5A, 5B*, 5C, 5D*, 5E-F Subtopic 1C: Work Packages 6A, 6B*, 6C*, 6D ND4BB Information Centre Topic 2: New drugs into bad bugs Steering Committee Project level decision making body Work Packages: 1-8 Topic 3: Development of new drugs combatting Gram- negative infections Subtopic 3A: Work Packages: 1-3, 5A, 6*, 7*, 8 Subtopic 3B: Work Packages: 4**, 5B Topic n: Work packages 1-n Subject to milestone approval and potentially Call for additional beneficiaries Potentially subject to Call for additional beneficiaries if needed to provide additional Hit-to-Lead efforts Topics launched under Call 6 Topics to be launched under Call 8 Future topics to be launched * **

ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections Focuses on Gram-negative infections only Invites public and private partners with Hits and Leads with a novel mechanism of action Invites experts and professionals in medicinal chemistry, microbiology, biochemistry, pharmacokinetics Aims to combine expertise and knowledge to create a “European Drug Discovery Centre of Excellence for antibiotic resistance”. Goal is the delivery of novel Leads and Development Candidates Successful Development Candidates can move forward up through Phase 1 clinical trial IMI Open Info Day, Bucharest 10 December 2012

ND4BB Topic 3: Invitation summary Invites public and private partners with Hits and Leads: Novel targets Known targets, novel MoA Mechanism of Action Known targets, known MoA Each can be valuable and each has positives and negatives Invites experts and professionals in drug discovery medicinal chemistry, microbiology, biochemistry, pharmacokinetics, etc. Academics, institutions, SMEs IMI Open Info Day, Bucharest 10 December 2012

ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections European Drug Discovery Centre of Excellence for antibiotic resistance Hit-to-Lead programs novel mechanism of action from public and private partners medicinal chemistry, microbiology, biochemistry, pharmacokinetics, etc. from academia, SME and EFPIA Qualified Leads Qualified Candidates Phase-1 ready Phase 1 clinical trial GSK/Sanofi collaboration

ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections European Drug Discovery Centre of Excellence for antibiotic resistance Hit-to-Lead programs novel mechanism of action from public and private partners medicinal chemistry, microbiology, biochemistry, pharmacokinetics, etc. from academia, SME and EFPIA Qualified Leads Qualified Candidates Phase-1 ready Phase 1 clinical trial GSK/Sanofi collaboration Portfolio Management Committee (50:50 EFPIA:Public)

ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections European Drug Discovery Centre of Excellence for antibiotic resistance Hit-to-Lead programs novel mechanism of action from public and private partners medicinal chemistry, microbiology, biochemistry, pharmacokinetics, etc. from academia, SME and EFPIA Qualified Leads (3) Qualified Candidates (2) Phase-1 ready (1-2) Phase 1 clinical trial (1-2) GSK/Sanofi collaboration Portfolio Management Committee (50:50 EFPIA:Public) max. 4 hit-to-lead programs running in parallel, max. 8 total

ND4BB Topic 3: Objectives 1.Provide a unique platform for collaboration and exchange between private and public partners. 2.Establish a vibrant drug discovery hub across Europe with the resource, skills and expertise to generate a pipeline of “Leads” and “Development Candidates” originating from public or private partners. 3.Discover three novel-mechanism antibacterial Leads. 4.Identify two novel-mechanism Clinical Candidate molecules for the treatment of systemic Gram-negative infections. 5.Progress at least one novel-mechanism Clinical Candidate into preclinical and Phase 1 clinical studies. IMI Open Info Day, Bucharest 10 December 2012

ND4BB Topic 3: Work packages & Suptopics WP1: ND4BB Project Management, Collaboration and Dissemination WP2: Portfolio Management Committee WP3: Establishment of the ND4BB Drug Discovery Platform WP4: Delivery of novel “Leads” from public partners WP5: Delivery of Clinical Candidates PART A: GSK/Sanofi Collaboration PART B: Public partners WP6: Delivery of Phase 1-Ready Antibacterials WP7: Clinical Phase 1 WP8 : Partnering Outreach Subtopic 3A Subtopic 3B IMI Open Info Day, Bucharest 10 December 2012

EFPIA PARTICIPANTS: GlaxoSmithKline R&D, Sanofi, AstraZeneca, Basilea INDICATIVE DURATION OF THE PROJECT: 6 years INDICATIVE BUDGET EFPIA in-kind contribution: €26.0M IMI JU contribution: €58.9M ND4BB Topic 3: Discovery and development of new drugs combatting Gram-negative infections IMI Open Info Day, Bucharest 10 December 2012

NewDrugsForBadBugs (ND4BB) in IMI Call 8 ND4BB Subtopic 1C (Innovative trial design & clinical drug development) IMI Open Info Day, Bucharest 10 December 2012

ND4BB Subtopic 1C WP6: Conduct of clinical trials supporting the development of MEDI4893, a monoclonal antibody targeting S. Aureus alpha toxin. WP6A: Epidemiologic Surveillance of Healthcare-associated infections among surgical and intensive care unit patients WP6B: Phase 1b/2a study with MEDI4893 for Staph. aureus ventilator associated pneumonia (VAP) WP6C: Phase 1b/2a study with MEDI4893 for prevention of surgical site infections by Staph. aureus WP6D: Project management, dissemination and collaboration WP7: Conduct of clinical trials supporting the development of AZ9773. IMI Open Info Day, Bucharest 10 December 2012

ND4BB Subtopic 1C: Invitation summary Invites participants with capability for conducting active-surveillance, observational and clinical studies in ICU and surgical patient populations Expertise with data handling and standardization Expertise in clinical project management Expertise in statistics and PK/PD modeling approaches Expertise in bacterial, especially S. aureus virulence factors Proposals for novel diagnostics/biomarkers to be utilized in clinical trial designs. IMI Open Info Day, Bucharest 10 December 2012

ND4BB Subtopic 1C EFPIA PARTICIPANTS: GlaxoSmithKline, AstraZeneca, Janssen R&D, Sanofi INDICATIVE DURATION OF THE PROJECT: Six years INDICATIVE BUDGET EFPIA in-kind contribution: €25.4M IMI JU contribution: €26.4M IMI Open Info Day, Bucharest 10 December 2012

Other topics in IMI Call 8 Developing an aetiology based taxonomy of human disease IMI Open Info Day, Bucharest 10 December 2012

Disease Taxonomy – major objectives Initiate a new taxonomy for selected complex diseases, based on aetiological mechanisms defined by molecular evidence The new classification should provide the basis for patient selection and stratification to facilitate clinical trials and speed up the development of new more effective medicines Provide the rationale for more specific diagnostic tools Provide the basis for the identification of novel targets or pathways for future therapeutic interventions Development of cross therapeutic approaches ̶Clinical classification approaches ̶Molecular classification approaches ̶Imaging classification approaches IMI Open Info Day, Bucharest 10 December 2012

Developing an aetiology-based taxonomy of human disease Topic A: Systemic Lupus Erythematosus (SLE) & related connective tissue disorders & Rheumatoid Arthritis (RA) Topic B: Neurodegenerative disorders with a focus on Alzheimer’s disease and Parkinson’s disease Chronic Obstructive Pulmonary Disorders (COPD) Topic postponed Taxonomy platform Coordinated by UCB EFPIA in-kind commitment: €18 million IMI JU budget: up to €18 million New Calls for next disease areas IMI Open Info Day, Bucharest 10 December 2012

Expertise in disease classification, taxonomy, molecular aetiologies Expertise in omics technologies, study of genetic mutations and polymorphisms, gene expression data, protein modifications and expression patterns, protein interactions, informatics & modeling Expertise in clinical research, preclinical research, imaging, epidemiology Expertise in data basing, curation, harmonisation, standardisation and sharing (incl. legal and ethical aspects) Disease Taxonomy – invitation summary IMI Open Info Day, Bucharest 10 December 2012

Developing an aetiology-based taxonomy of human disease EFPIA PARTICIPANTS: UCB, Lundbeck, MerckSerono, Pfizer, Eli Lilly, Bayer INDICATIVE DURATION OF THE PROJECT: Five years INDICATIVE BUDGET EFPIA in-kind contribution: €18M (add details) IMI JU contribution: €18M Topic A (SLE + RA) Topic B (neurodegenerative disorders) EFPIA in-kind contribution€10M€8M IMI JU contribution€10M€8M IMI Open Info Day, Bucharest 10 December 2012

Other topics in IMI Call 8 European induced pluripotent stem cell bank IMI Open Info Day, Bucharest 10 December 2012

Scientific Generation of a full complement of geno- & phenotypic data for key patient cohorts Research and implementation of current standard practices for the generation and differentiation of iPS cells Development of automatable processes for iPS cell culture and banking Application of best practise in cryopreservation & biobanking to develop a ‘commercial standard’ state of the art iPS facility Provision of quality protocols and training in iPS cell growth & development Operational Set-up of a sustainable, not-for-profit, specialist production, storage and distribution centre for iPS cells across Europe hosted in appropriate facility Provide patient derived iPS cells to a defined quality and within a defined time from placing an order Supply an iPS differentiation service during the latter half of the call Provide searchable anonymized geno-, phenotypic & clinical data associated with each iPSC line European induced pluripotent stem cell bank IMI Open Info Day, Bucharest 10 December 2012

Applicants that have identified a broad and useful cohort of patient derived iPS cell lines Expertise in tissue/somatic cell collection and the generation and differentiation of iPS cells Expertise in cell culture, cryopreservation and quantitative analysis in cell biology Expertise related to data, security, standards (incl. ethical and confidentiality policies) Appropriate infrastructure and laboratory space to support the bank European induced pluripotent stem cell bank – invitation summary IMI Open Info Day, Bucharest 10 December 2012

European induced pluripotent stem cell bank EFPIA PARTICIPANTS: Pfizer, Sanofi, NovoNordisk, Servier, Lundbeck, AstraZeneca, Novartis, Lilly, UCB INDICATIVE DURATION OF THE PROJECT: Six years INDICATIVE BUDGET EFPIA in-kind contribution: up to €30M IMI JU contribution: up to €40M IMI Open Info Day, Bucharest 10 December 2012

IMI Call 8 – upcoming webinars European induced pluripotent stem cell bank – Thursday 6 December, 13:00 CET Developing an aetiology-based taxonomy of human disease – Wednesday 12 December, 10:30 CET ND4BB Subtopic 1C – Tuesday 11 December, 15:00 CET ND4BB Topic 3: Discovery and development of new drugs combating Gram – negative infections – Monday 17 December, 15:00 CET Webinar on IMI’s rules and procedures on Thursday 13 December at 14:30 CET IMI Open Info Day, Bucharest 10 December 2012

IMI – future topics IMI Open Info Day, Bucharest 10 December 2012

Future topics Development of drug-drug combinations Leveraging emerging technology for pharmacovigilance Further topics derived from the Scientific Priorities IMI Open Info Day, Bucharest 10 December 2012

THANK YOU ! IMI Open Info Day, Bucharest 10 December 2012