IMI2: linking academia with industry in pharmaceutical research. Hugh Laverty 21 June 2018, UKRO Conference, Southampton

Transcription

1 IMI2: linking academia with industry in pharmaceutical research Hugh Laverty 21 June 2018, UKRO Conference, Southampton

2 IMI Europe s partnership for health IMI1: bn budget 59 projects > 5 bn IMI2: bn budget More ambitious More open Greater scope 2.5 bn Partnership bn

3 The way in which pharmaceutical companies develop new medicines is changing Regulatory environment Rising R&D costs EU Pricing RWE New medicines development HC Reform Patients Generics Declining R&D productivity

4 IMI2 building on successes of IMI1 European Commission From H M EFPIA & Partners in Research 1425 M Associated Partners 213 M IMI M Focused: stratified medicines and healthcare priorities Healthcare solutions: prevention and treatment End-to-end: R&D, regulatory and access move integration a step further Multi-sector: within and beyond life sciences 4

5 IMI2 overall objectives improve the current drug development process through development of tools, standards & approaches to assess efficacy, safety & quality of health products develop diagnostic & treatment biomarkers for diseases clearly linked to clinical relevance & approved by regulators reduce time to clinical proof of concept (e.g. for cancer, immunological, respiratory, neurological/neurodegen. diseases) increase success rate in clinical trials of priority meds (WHO) develop new therapies for diseases with high unmet need, (e.g. Alzheimer s) & limited market incentives (e.g. AMR) reduce failure rate of vaccine candidates in phase III trials through new biomarkers for efficacy & safety checks - IMI2 legislation -

6 IMI 2 Strategic Research Agenda Antimicrobial resistance Osteoarthritis Cardiovascular diseases Diabetes Neurodegenerative diseases Psychiatric diseases Respiratory diseases Immune-mediated diseases Ageing-associated diseases Cancer Rare/Orphan Diseases Vaccines

7 IMI 2 Strategic Research Agenda Priority Themes 1. Neuro-degeneration 2. Immuno-inflammation 3. Metabolic disorders 4. Infection control 5. Translational Safety 6. Data and knowledge management 7. Oncology Support Technologies 1. Imaging 2. ICT 3. Medical devices. Enablers Patient access to innovative solutions (MAPPs) - Target validation - Stratified medicine, precision medicine - Innovative trials - Data generation & interpretation - Prevention, disease interception - Patient adherence - Health disease management - Regulatory framework - Reimbursement/patient access

8 The Vision for IMI2 Science is driving the manner in which we view disease Population Individual Molecular diagnosis based on biological knowledge We treat a population. Some respond and some don t We treat a targeted population They all respond

9 IMI Ecosystem for innovative collaborations IMI is a neutral platform where all involved in drug development can engage in open collaboration on shared challenges. Allow engagment in a cross-sector and multi-disciplinary consortium at the forefront of cutting-edge research; Provide the necessary scale by combination of funding, expertise, knowledge, skills and resources; Build a trustful collaboration upon a creative spirit, innovative and critical thinking; Gain from each other (new knowledge, skills, ways of working) Be transformative to make a difference in drug development and ultimately patients life.

10 HOW DOES IMI WORK?

11 IMI 2 life cycle Call topics definition Strategic Research Agenda Annual Work Plan Strategic Governing Groups Consultation Member-Associated States/Scientific Committee Call Launch / Evaluation / Grant award Consortium agreement, Grant agreement, Implementation and Reporting

12 Strategic Governing Groups - SGG (major actors in Topic Development Process) Driven and Chaired by Industry, contribution from European Commission and IMI office Translational Safety Immunology Infections Control Neurodegeneration Metabolic Disorders Knowledge and Data Management Oncology

13 How does IMI work? Two stage procedure Topic definition phase Stage 1 Stage 2 Granting phase Industry consortium Academic research teams Mid-size enterprises SMEs Hospitals Regulators Patients organisations Applicant consortium Industry consortium Definition of topics by industry consortium SP Submission & Evaluation FP Submission & Evaluation Signature of Consortium and Grant Agreements Call launch Selected stage 1 team merge with industry team Start of the Granting phase Project launch!

14 An international, cross-sector community Figures from Jan 2018 Over researchers working for: open collaboration improved R&D productivity innovative approaches to unmet medical needs

15 IMI1 budget distribution per disease area Budget distribution per disease area Updated January 2017 Brain disorders % Drug safety % Metabolic disorders % Stem cells % Cancer % Data Management % Inflammatory disorders % Drug Discovery % Infectious diseases % IMI 1 Total contributions EFPIA contributions IMI contributions Biologicals % Vaccines % Geriatrics % Education and Training % Lung diseases % Sustainable chemistry % Drug Delivery % Drug kinetics % Relative effectiveness %

16 IMI2 budget distribution per disease area Distribution of funding per scientific area Updated February 2017 Diabetes / metabolic desorders % Neurodegeneration % Oncology % Other % Infections control % IMI 2 Total contributions Data and knowledge management % Translational safety % EFPIA contributions IMI contributions Associated partners contributions

17 UK participation in IMI IMI1 Participations: 372 (1741) 21% Budget: 238,575,125 25% IMI2 Participations: 240 (1175) 20% Budget: 189,858,122 32% Figures as of June 2018

18 Some Specific Examples

19 The problem Antibiotics resistance 'as big a risk as terrorism' UK medical chief Three million Europeans catch infections in hospital annually Antibiotic resistance: we must act now, says WHO 17 Janvier 2008: La résistance des bactéries aux antibiotiques a atteint une dimension planétaire

20 IMI - New Drugs for Bad Bugs How to get new drugs into bad bugs Pan-European network of clinical/laboratory sites Collaboration on clinical trials Platform for antibiotic development New business models Part of EU AMR programme 7 projects 700 million budget

21 New Drugs for Bad Bugs (ND4BB) Cross-project communication & collaboration TRANS- LOCATION Research on penetration & efflux in Gramnegatives Data hub & learning from R&D experience ENABLE Discovery & development of new drugs combatting Gramnegative infections COMBACTE Enabling clinical collaboration & refining clinical trial design Clinical development of compounds for Gram positives COMBACTE- CARE Clinical development of antibacterial agents for Gramnegative, antibiotic resistant pathogens COMBACTE- MAGNET Systemic molecules against healthcareassociated infections iabc Inhaled antibacterials in bronchiectasis and cystic fibrosis DRIVE-AB Driving reinvestment in R&D & responsible use of antibiotics ND4BB Information Centre All data generated is submitted and made accessible to all partners Drug discovery Drug development (Grampositives) Drug development (Gramnegatives) Economics & stewardship

22 ND4BB programme Clinical development of new compounds against AMR Clinical Development Phase I Phase II Phase III Regulatory Review Commercialisation Phase IV Patients COMBACTE COMBACTE-CARE COMBACTE-MAGNET iabc MEDI4893 for prevention S. Aureus infections incl. MRSA; Minocycline in A. baumannii infections Aztreonam-Avibactam for treatment Gram negative infections incl. MDR MEDI 3902 for prevention P. aeruginosa infections AIC499 for treatment Gram negative infections inhaled formulation of Murepavadin (POL7080) in CF patients tobramycin inhalation powder in BE patients + Observational studies to inform the development of new compounds Sustainable clinical trial investigator (CLIN-NET), laboratory (LAB- NET) networks + methodological (STAT-NET) & epidomiological (EPI- NET) networks

23 IMI2 Ebola+ programme Projects on Ad26.ZEBOV / MVA-BN-Filo prime-boost candidate vaccine regimen EBOVAC1, EBOVAC2: Clinical studies in Africa & Europe EBOMAN: Vaccine manufacture EBODAC: Vaccine deployment & compliance VSV-EBOVAC Goal: characterise the immune response triggered by the rvsv- ZEBOV candidate vaccine FILODIAG, MOFINA, EbolaMoDRAD Goal: all aim to develop rapid diagnostic tests capable of delivering reliable results at the point of care in as little as 15 minutes

24 Mofina Successfully miniaturised Altona Realstar Filovirus assay to fit Alere Q automated PCR system. Allows accurate diagnosis in the field with 75 minute turnaround CE-IVD approved for use in Europe FDA registration in progress

25 Alzheimer s disease a major unmet need Alzheimer s disease in numbers 46.8 million affected globally 10.5 million in Europe Global cost USD 818 billion (EUR 732 billion)

26 IMI action on Alzheimer s disease PHARMA-COG Matrix of biomarkers Test efficacy of new treatments EMIF Linking & analysing data Identify those at risk AETIONOMY New classification of AD/PD Personalised treatments Total budget 169 million EPAD Adaptive clinical trials Faster drug development & patient access

27 Sharing data to improve clinical trials for schizophrenia patients 67 studies 25 countries 1 database By redesigning clinical trials, you could: make them shorter (6 weeks 4 weeks) require fewer people (79 46 patients per arm) cut costs (savings of 2.8 million) gain insights into effects of treatment on negative symptoms (e.g. lack of emotion)

28 Collaborative Drug Discovery: ELF LIBRARY DESIGNS EFPIA contribution (>300,000 cpds) Joint European Compound Library Public contribution (200,000 cpds by 2017) DRUG TARGETS European Screening Centre - uhts - Compound logistics - Hit triage - Medicinal Chemistry

29 European Lead Factory - results Motor neurone disease Based on the hits generated by the European Lead Factory, Dr Richard Mead, University of Sheffield, started the spin-out virtual biotech Keapstone Therapeutics, a world-first type of partnership between Parkinson's UK and the University of Sheffield. Keapstone Therapeutics aims to develop a new therapy for motor neurone disease (MND/ALS) and/or Parkinson s disease. Diabetes Scandicure, in collaboration with the IMI-funded European Lead Factory, has developed inhibitors of a novel biological target for nonalcoholic steatohepatitis (NASH), type-2 diabetes and potentially other metabolic diseases. Antimicrobial resistance ENABLE takes on Oxford antibiotic resistance programme The University of Oxford takes the next step in targeting antimicrobial resistance, with help of the results and hit compounds delivered by the European Lead Factory. The Oxford team, led by Professor Chris Schofield, has got the opportunity to collaborate with the European Gram- Negative Antibacterial Engine (ENABLE) project to further progress this programme towards clinical development. Oncology At the end of 2017, Prof Reuven Stein received an extraordinary Qualified Hit List from the European Lead Factory. His research group studies the tumour microenvironment (TME) and has discovered a protein that plays an essential role in tumour progression. With the promising ELF compounds, they hope to develop new treatments for cancers such as glioma and melanoma. Depression and pain, African sleeping sickness, Chagas disease, Leishmania parasite & Dengue virus

30 Drug repurposing: a strategy to develop new treatments - IMI2 Call13 Large amount of data arising from terminated programmes are available for these compounds This information is useful to test hypotheses in alternative therapeutic indications may accelerate the transition of these compounds from bench to market in a cost effective way Objective: To investigate the therapeutic potential of nine previously deprioritised clinical compounds in new clinical indications in areas of high unmet need: Cardiovascular diseases and diabetes Respiratory diseases Neurodegenerative diseases Rare/orphan diseases

31 Measures of Success New models developed & published Setting new standards Use by industry Impact on regulatory framework Better drugs and impact on medical practice Better evidence-based decision making

32 Future IMI Calls for proposals The following topics are currently under consideration for inclusion in future IMI Calls for proposals: Integrated research platforms enabling patient-centric drug development Blockchain enabled healthcare Emerging translational safety technologies and tools for interrogating human immuno-biology Digital transformation of clinical trials endpoints Microenvironment imposed signatures in tissue and liquid biopsies in immune mediated disease Development and validation of translational platforms in support of synaptopathy drug discovery Antimicrobial resistance (AMR) accelerator programme: The AMR Accelerator programme consists of three pillars under which multiple projects are expected: Pillar A: Capability building network (CBN) Pillar B: Tuberculosis drug development network (TBDDN) Pillar C: Company-specific portfolio building networks (PBNs)

33 Priorities and first batch of ideas for future IMI2 calls Immunology Antimicrobial resistance Digital Health/Big Data Modernisation of clinical trials and regulatory pathways Treatment of non-response and remission Non-invasive molecular imaging of immune cells Clinical trials networks Accelerator of AMR R&D Remote clinical trials Biosensors/digital endpoints in clinical development Addressing the challenge of platform trials (Integrated Research Platforms)

34 Commission Communication 11 Jan "Horizon 2020 interim evaluation: maximising the impact of EU R&I" The Commission intends to explore in the Impact Assessment of the future programme: rationalising partnerships (including KICs and Future and Emerging Technologies Flagships), improve their openness and transparency, and Link them with future EU R&I missions and strategic priorities. It will consider, as part of a strategic process, for example, the additionality of capabilities, relevance for political priorities agreed between the EU, Member States, industry and other stakeholders, flexibility, exit strategies integrated from the start, and delivery of impacts that cannot be achieved through the Framework Programme alone.

35 Expected Timelines FP9 and Partnerships June 2018 Commission adopted proposals for EU's long term 2018 budget and the proposals for the EU programmes Discussion/ negotiation with Council and European Parliament has started FP9 presents criteria for the selection, but does not identify specific, partnerships nor missions. End of 2018 Identification of potential partnerships For partnerships established under Art 187 (public private partnerships established as Joint Undertakings) or Art 185 (public public partnerships) During 2019 Impact Assessments and preparation of the legal basis of proposed partnerships for adoption by the new Commission During 2020 Negotiation with Council and EP for proposals 2021 Implementation through the Work Programmes of FP9

36 New approaches are needed Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem. Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.

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38 Thank you Hugh Laverty Head of Scientific Operations