The Innovative Medicines Initiative. Pierre Meulien CNIO, Madrid - Spain

Transcription

1 The Innovative Medicines Initiative Pierre Meulien CNIO, Madrid - Spain

2 IMI Europe s partnership for health > 5 bn 2.5 bn Partnership Pharma industry 2.5 bn EU contribution from FP7 / H2020 Pharma contribution in kind

3 Why do we need IMI? Because drug development is very complex risky inefficient lengthy expensive Because Biological mechanisms underlying disease are complex Clinical trial designs need to be adapted to scientific knowledge Regulatory pathways should be adapted due to scientific drivers

4 What does IMI do? IMI facilitates bringing new knowledge closer to the patient through an integrated platform involving stakeholders from the public and private sectors IMI responds to both industrial needs and public health challenges Through IMI funded projects the innovation process is streamlined through end - to- end integration

5 An international, cross-sector community 108 other teams 970 academic teams 202 SME teams Over researchers working for: open collaboration improved R&D productivity innovative approaches to unmet medical needs 31 patient orgs 552 EFPIA teams Figures from June 2016

6 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, Article 2b

7 IMI budget breakdown Drug Discovery Metabolic disorders Drug safety Stem cells Data Management Brain disorders Cancer Infectious diseases Other Inflamatory disorders Biologicals Geriatrics Vaccines Education and Training Lung diseases Sustainable chemistry Drug delivery Drug kinetics Figures from May 2016

8 Spanish Participation in IMI 131 participations involved 75 from Academic and other research organisations 13 SMEs 18 from EFPIA companies 1 patient organisation 24 other Spanish researchers have received over 42 million in funding Ranked 8 th in IMI-1

9 % of Spanish Participants by Legal type 1% 10% SME's 57% 18% 14% Other EFPIA Academia and research organisations Patient organisations

10 IMI 2 budget ( ) EU funding goes to: Universities SMEs Mid-sized companies Patient groups etc bn IMI 2 total budget billion bn Other 213 m EFPIA companies receive no funding contribute to projects in kind Associated Partners e.g. charities, non-efpia companies

11 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!

12 What does a typical IMI project look like? Industrial partners align themselves around a real challenge for industry and agree to work together and commit resources. New ideas from public sector, universities, SMEs etc. are needed to address the challenge Scale is a key to success and is provided through IMI funding. Outcomes should be transformative for the industry as well as having a clear public value

13 Some Specific Examples

14 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

15 NATURE REVIEWS DRUG DISCOVERY COMMENT Towards the taxonomy of human disease Martin Hofmann-Apitius, Marta E. Alarcón-Riquelme, Chris Chamberlain & Duncan McHale Nature Reviews Drug Discovery 14, (2015) Published online 30 January 2015 Consortia have begun to establish 'mechanism-based taxonomies' for inflammatory and neurodegenerative diseases that could aid drug development and personalized therapy. IMI projects PRECISEADS and AETIONOMY

16 IMI s cancer projects CANCER-ID Establishment of standard protocols for, and clinical validation of, blood-based biomarkers PREDECT Developing advanced, transferable in vitro models for breast, prostate and lung cancers Budget 86 million ONCOTRACK Developing & assessing novel approaches for identification of new markers for colon cancer QUIC-CONCEPT Tools & imaging biomarkers that show earlier and more accurately how tumours respond to drugs in clinical trials

17 deconstruction PREDECT characterise and compare = Tissue Slices 3D Co-culture 3D Bioreactor 3D Spheroids 2D on Plastic reconstruction PREDECT starts at both ends of complexity, with stepwise increases / decreases in complexity. Every step is compared to each other and primary tumors

18 ONCOTRACK a systems biology approach to model the tumour cell The premise: most attempts to identify novel biomarkers probably fail because the source data do not sufficiently represent the complexity of the tumour OncoTrack - An in silico model (ModCellTM) is being populated with genomic, transcriptome, genetic, biochemical and pathologic information derived from tumour samples (CTC, free circulating DNA, CSC, xenografts) collected prospectively from patients diagnosed with colon cancer This approach allows detailed analysis of signalling pathways in the tumour Major deliverable: Biological validation of the computational prediction of treatment response

19 New Projects in Cancer Haematological Malignancies Preclinical platform for paediatric oncology.new opportunity in Prostate Cancer (Big Data) in Call 10 New Oncology Strategy for IMI (Stefan Scherer)

20 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)

21 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

22 IMI dementia portfolio highlights Challenge 1: What are the underlying causes of dementia? Challenge 2: Who is at greatest risk of developing dementia? AETIONOMY: Identifying subgroups of dementia, based on underlying genetic / molecular causes, to allow tailored treatments Patients involved EMIF: Linking & analysing data from many studies to identify markers of risk Challenge 3: How can we improve clinical trial design? EPAD: Setting up innovative clinical trials that allow multiple treatments to be tested at same time Challenge 4: Can brain scans aid research & treatment? AMYPAD: Studying how brain scans of amyloid plaques could aid diagnosis, treatment & research

23 Despite the vast amount of research on schizophrenia and depression in the past two decades, there have been few innovative drugs to treat these disorders. Precompetitive research collaborations between companies and academic groups can help tackle this innovation deficit, as illustrated by the achievements of the IMI- NEWMEDS consortium.

24 NEWMEDS: Tackling the challenges of schizophrenia and depression research 9 (11*) EFPIA companies; 7 Public organizations; 3 SME Total Budget: 23.2 M Animal models that truly translate a patient relevant phenotype WP 7, 9 Discovery phase Experimental Human tools that enable early decision making in clinical trials Phase 1 Phase IIa Sub Phase 2b Phase 3 mission Platforms that will enable target identification of biologically validated targets Find ways to improve the way we run clinical trials * 2 EFPIA companies joined the consortium during the life time of the project 24

25 Headline achievements of the NEWMEDS program Gained novel key insights into the biology of schizophrenia and depression Standardised the application of cognitive models across several industrial partners Developed translational tools/platforms for drug discovery [animal models, human imaging] Developed new web tools for analysis [image analysis, biomarker significance] Provided information for the design of more efficient clinical trials

26 Topics for the near future? Paediatric Clinical Trial Network Oncology Advanced Therapies Patient Engagement Digital Health Biopreparedness Microbiome

27 IMI and Advanced Therapies Held 2 workshops Developed Concept paper Key issues identified: In vitro and in vivo model systems for preclinical testing Vector systems for gene therapy Application of gene editing technology Regulatory environment for preclinical testing Demonstrations of clinical safety and efficacy- (control groups?) Communication to the general public on the benefits of ATMPs Manufacturing challenges related to specific products (cells, vectors etc) Batch to batch consistency, regulatory frameworks, controlling raw materials etc Need for common technology platforms? Common Standards? Pricing, reimbursement, access etc. New business models Hospital Exemption issue

28 Participation of new players We need to be more inclusive as regards who partners with IMI We need other sectors to get involved (Dx, Imaging, ICT etc) Evolution of Generics space (repurposing of existing drugs, combinations etc) Smaller EU Countries Involvement of more SMEs Associated Partners of IMI and Partners in Research (through EFPIA) Partnerships with European infrastructures and or other initiatives like EIT-Health for example Partnerships with other funders

29 IMI 2 Calls for proposals

30 IMI 2 Call 8 Open Call on Ebola & related diseases Call launched December 2015, will remain open for two years. Opportunity to capture emerging scientific advances and progress those rapidly into healthcare interventions. Deliverables of projects funded must increase our preparedness to react to future outbreaks of Ebola and other filoviral haemorrhagic fevers. Projects can address vaccines, diagnostics, treatments, etc. Indicative IMI budget: 70 million 55 million (remaining) Cut-off dates: 16 March 2016, 15 September 2016, 16 March 2017, 14 September 2017, 15 March More information: bit.ly/imi2call8

31 IMI 2 Call 10 December 2016 (deadline for applications- March 28 th 2017) Understanding hypoglycaemia: underlying mechanisms and clinical determinants plus consequences for people with diabetes How big data could support better diagnosis and treatment outcomes for prostate cancer (part of IMI big data programme) Improving the care of patients suffering from acute / chronic pain Creation of a pan-european paediatric clinical trials network Biomanufacturing 2020: Development of innovative high throughput analytical tools and methods to characterize cell culture fluid during development and commercial cell culture processes Unlocking the solute carrier gene-family for effective new therapies Enhanced patient voice in medicines lifecycle Precision medicines approaches in autism spectrum disorders More information:

32 How is IMI addressing the challenges in drug development? Through IMI s projects we are trying to put patients at the centre share risk (among public & private players) increase efficiency (by developing common tools) reduce duplication of effort (esp. at early stages) reduce timelines (by using a personalised medicine approach) integrate the latest science into drug development use data and knowledge management to work more effectively We do this by creating a neutral platform where all involved in drug development academics, industry, SMEs, patients, regulators, others can engage in open collaboration on shared challenges.

33 Thank you