pistoia alliance european conference 2015 - ann martin / imi

Ann Martin 14 April 2015 • Pistoia Alliance • Zurich, CH

Innovative Medicines Initiative Update and Opportunities

!  What is the Innovative Medicines Initiative? !  IMI achievements !  How does IMI work?

Outline

‘We must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.’

Rethinking drug discovery – Turning the Titanic? Elias A. Zerhouni, President Global R&D, Sanofi Editorial in Science Translational Medicine, January 2014

IMI – Europe’s partnership for health

> €5 bn

Partnership 2008 - 2024

€2.5 bn

€2.5 bn

IMI – key concepts

!  Focus on unmet needs !  Non-competitive collaborative research !  Competitive Calls for proposals !  Open collaboration in public-private consortia

!  Data sharing, dissemination of results…

!  Industry contribution is in kind

IMI 1 – € 2 bn budget breakdown

Infectious diseases

Drug discovery

Brain disorders

Metabolic disorders

Drug safety Stem cells

Cancer Data management

Inflamatory disorders

Biologicals Geriatrics

Lung diseases Education &

training Sustainable chemistry

Drug delivery Drug kinetics Relative

effectiveness

An international, cross-sector community 650

acad-emic

teams

120 SMEs

409 EFPIA teams

25 patient orgs

17 regul-ators

Over 6 000 researchers working for: !  collective intelligence

networks !  improved R&D

productivity !  innovative approaches

to unmet medical needs

IMI achievements

Antimicrobial resistance – a growing threat

25 000 killed

€1.5 billion

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

!  €229 million !  4 projects launched

Action on Alzheimer’s disease

Matrix of biomarkers "  Test efficacy

of new treatments

Linking & analysing data "  Identify those

at risk

New classification of AD/PD " Personalised

treatments

‘Adaptive’ clinical trials "  Faster drug

development & patient access

Autism – a common disorder

!  Affects 1 in 110 !  Lifelong condition !  Difficulties in social

interaction & communication, unusual repetitive behaviours

!  Major impact on families & carers 0

!  New insights into underlying causes

!  Could the brain changes be reversed?

!  Gender & autism !  2 major clinical trials for early

detection and monitoring of ASD in children

!  Working with regulators on treatment guidelines

IMI – delivering results on autism

Sharing data to improve clinical trials for schizophrenia

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)

23 000 patients 67 studies

25 countries 1 database

IMI contributes to drug safety

SAFE-T project

!  153 potential biomarker candidates for drug-induced injury of kidney, liver & vascular system evaluated

!  17 exploratory clinical studies

!  Dialogue with regulatory agencies

!  MoU with PSTC (C-Path)

IMI projects deliver excellent science

0 0.5 1 1.5 2 2.5 3

IMI

FNIH

Wellcome Trust

TI Pharma

World average

Citation index

IMI – a partner for SMEs 15% of funding recipients 18.4% of budget

135

SMEs

in 4

6 pr

ojec

ts

Funding

Visibility

Knowledge

Access

Networking

IMI – a partner for SMEs in diabetes research !  IMIDIA generated the first human pancreatic beta cell line !  A French SME was at the heart of the research

‘Thanks to this collaboration, the robustness of our beta cells has been validated by large pharma companies – a major advantage for a biotechnology company like Endocells.’ – Anne-Fabienne Weitsch, CEO of Endocells

IMI – putting patients at the heart of drug development U-BIOPRED – towards personalised medicine for severe asthma !  The challenge – recruit 1 025 people !  Patients involved in project as partners &

through ethics board, safety monitoring board, and patient input platform

!  Offered patients’ perspective on recruitment, study design, project communication

!  Project now starting to deliver results!

How does IMI work?

Goals of IMI 2 programme

$  Increase the success rate of clinical trials of new medicines & vaccines

$  Speed up the earlier stages of drug development

$ Develop new treatments for areas of unmet need $ Develop new biological markers to diagnose diseases and

assess treatments

$  Improve the drug development process by creating tools to assess the efficacy, safety and quality of medicines

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

Topic definition phase

Typical IMI project life cycle Negotiation phase Stage 1

Identification of topics and willingness to collaborate by EFPIA companies and associated partners

Signature of Project Agreement and Grant Agreement

Submission of short proposals by applicant consortia & evaluation by independent experts

Patients’ organisations

Academic research

teams

Regulators

Hospitals

SMEs

Mid-size enterprises

Industry consortium

Stage 2

Preparation of full proposal & evaluation by independent experts/ethical panel

Industry consortium

Applicant consortium

Call launch

Invitation to selected team to merge with industry team

Start of the negotiation

phase Project launch!

Why take part in IMI projects?

!  Scientific excellence !  Impact on drug development, regulatory procedures,

patients’ lives !  Access to expertise of scientists from universities,

industry, biotechs, regulators, patient groups… !  New business opportunities !  Under IMI 2 – improved funding rates !  Flexible intellectual property policy protects partners

while promoting use of knowledge

IMI’s flexible intellectual property policy

Support to industry

Freedom of access

Compensation for IP

Dissemination of information

Incentive to participate flexibility

+ trusted party

IMI’s IP policy allows unprecedented levels of sharing

Companies pooling legacy

toxicity data

European platform for antibiotic

development

Companies pooling &

sharing old trial data European

Lead Factory compound collection

Project partners

validate each other’s findings

Apply for funding !  Look out for new IMI Calls

!  www.imi.europa.eu !  IMI newsletter !  Twitter, LinkedIn

!  Link up with other experts !  Read and understand the

Call documents !  Info sessions /

webinars !  Contact the IMI

Programme Office !  Submit your proposal

How to take part in IMI projects Contribute to IMI as an Associated Partner Your contribution is matched by the EU !  Read the IMI Strategic

Research Agenda !  Identify points that match

your priorities !  Contact the IMI Programme

Office

Ann Martin dd.mm.yyyy • Event name • City, Country

Data & Knowledge Management Portfolio

Clinical  Trial  Simulator  -­‐  Prototype  v.2  Adap9ve  Op9mal  Designs  –  prototype  V.1  

Model Based  Drug  Development  

Compound  Target  Pathway  Disease  data    

$  Created the Open PHACTS Discovery Platform linking information and facts from multiple public databases

$  Created an API for drug discovery with an ecosystem of apps

$  Methodology for delivery business-relevant solutions

$  Use by bench and computational scientists

$  Established itself as a– a unique forum for partners

$  the Open PHACTS Foundation – support the existing infrastructure and widen scientific scope

Efforts to Automate clinical research

1DESIGN  Feasibility  Study  

XML   XML  

Regulators  

Clinical Trial Management

System (CTMS)

Document Management

System

2EXECUTION  Pa?ent  

Recruitment  

3EXECUTION  

Data  Collec?on  

FDA-­‐EMA  SUBMISSION  

XML  

Case  Report  Form  

(CDISC  ODM)  

Trial Registry

Clinical Data Management

System (CDMS)

XML  

Protocol  (CDISC  PRM  –  SDM)  

EMIF European Medical Information Framework

Platform Make data available for browsing and analysis in multiple ways Alzheimers Disease Identification and validation of markers that predict Alzheimer’s progression Metabolics Discovery of predictors of the metabolic complications of adult and paediatric obesity    

Research topics provide focus

"  Supports collaborative projects

"  Analysis needs of translational data

"  Deploys open-source tranSMART

"  Service with public data

"  Ethics / data protection

"  Standards research

"  transMART Foundation o  35 transMART implementations o  multiple development teams o  commercial services

studies  including  omics  images  clinical  assessment  data

Abirisk  Oncotrack  UBIOPRED  Predict-­‐TB  RA-­‐MAP  ....  

J&J    eTRIKS    TraIT        TheHyve    IDBS    Sanofi  Pfizer  Takeda      RecombinantbyDeloiPe    Thomson  Reuters        Michigan  Harvard    

22  transla?onal  ,  1400  gene  expression  studies  

European Translational and Knowledge Management Services

Multiple data types supported

Recognising Adverse Drug Reactions

•  A mobile phone app for reporting of suspected ADRs to EU regulators.

•  Assess the use of the app to provide info on medicines.

•  Explore identification of potential safety issues from user comments in social media.

•  Develop recommendations for use mobile technologies and social media in pharmacovigilance and monitoring of the safety of medicines.

Under Evaluation Call 3, 4

Patient input into drug development Today

Pharma 1 Patients Advocacy Groups Regulators

Pharma 2

Pharma 3

Future

Pharma Patients Advocacy Groups Regulators

Pharma

Pharma

Knowledge Repository

Knowledge repository to enable patient-focus medicine development

Challenges in Managing Chronic Disease Today

•  Physician visits are time-limited evaluations based on subjective observations of both the patient and the physician or psychiatrist

•  Changes in disease state for each of these diseases can occur on timescales much shorter than the interval between physician visits

•  Through technological advances over the last decade it is now possible to objectively, remotely, and continuously measure aspects of patient physiology, behavior and symptoms

39

40

Multi-Platform Biomarker Data from Controlled Studies

Physician Notes

Caregiver Reports

Literature Patents Epidemiology

Patient Hospital Records Data

Vision of Tomorrow: Next Generation Patient Centric

Data MOVE FROM DIAGNOSE AND TREAT TO PREDICT

AND PREEMPT 40

Continuous Real-Time Patient Data Home Monitoring. Remote Sensing. (Actigraphy, Physiological)

Predict and Preempt: "  relapse in depression

"  exacerbation in MS "  epileptic fit

" Onset of mania

" Psychotic break etc.

Remote Assessment of Disease and Relapse (RADAR)

Current Inidicative Topics

Current indicative topics - 1

!  Patient perspective elicitation on benefits and risks of medicinal products, supplementing benefit risk assessments by regulators and HTAs (health technology assessments) from development through the entire life cycle

!  Development of a quantitative system toxicology (QST) platform !  Diabetic kidney disease (DKD) biomarker idea

Current indicative topics - 2

!  Inflammation and Alzheimer’s disease (AD): modulating microglia function – focussing on TREM2 and CD33

!  Enabling magnetic encephalography (MEG) as a biomarker for diagnosis and disease progression in mild cognitive impairment (MCI) and Alzheimer’s disease (AD)

!  Understanding the role of amyloid biomarkers in diagnosis, clinical management and treatment of patients with cognitive impairment

!  Evolving models of patient engagement and access for earlier identification of Alzheimer’s disease

!  Bringing apolipoprotein E (APOE) biology to validated Alzheimer’s disease targets

Future IMI Calls - timelines & info

!  Find out more: http://bit.ly/futuretopics !  Call launch: Summer 2015

Disclaimer - All information regarding future IMI Call topics is indicative and subject to change. Final information about future IMI Calls will be communicated after approval by the IMI Governing Board.

www.imi.europa.eu @IMI_JU

Thank you

Ann Martin • Principal Scientific Manager ann.martin@imi.europa.eu

IMI general presentation 21-10-2014

DKM Project Master

Change History

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