IMI Projects : SMEs Contributions and Benefits from Large Public-Private Initiatives on Precompetitive Projects

Transcription

1 IMI Projects : SMEs Contributions and Benefits from Large Public-Private Initiatives on Precompetitive Projects Prof. Hüseyin FIRAT, MD,PhD IMI SAFE-T Applicant Consortium coordinator Partner of the IMI BT-Cure and Pharmacog consortia IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014

2 Overview Biomarker Discovery, Development and Regulatory Qualification TcLand Expression Biomarkers on Inflamatory Diseases Acquired by Firalis in July 2013 IMI BT-Cure Consortium Biomarkers on Alzheimer Created on January 2014 IMI Pharmacog Consortium Amoneta Diagnostic Firalis Biomarkers on Cardiovascular Diseases Founded in 2007 IMI SAFE-T Consortium 2

3 Company Overview Thanks to IMI projects, Firalis gained overall expertise and cumulated know-how, and become an acknowledged key player in the field of biomarker qualification. Firalis develops, manufactures and markets biomarkers (BM) and BM-based tools for drug development and clinical applications. Firalis business model stands on a tripod : I. BIOMARKER R&D : Firalis research activities focus on biomarker qualification in specific cardiovascular disorders. In this parallel, Firalis joined several international programs including the IMI-JU SAFE-T Project, where it has coordinated the consortium during initial phase. II. III. BIOMARKER PRODUCTS : Firalis develops, manufactures and markets BM-based diagnostic solutions; which are first marketed as «Research-Use-Only» (RUO) kits and then transform into «In Vitro Diagnostics» (IVD) tools, upon clinical validation and regulatory qualification of biomarkers used in the corresponding kit. BIOMARKER SERVICES : Firalis is conducting several research projects with and for the biopharmaceutical industry. 3

4 SAFE-T Project Impact at Firalis developing biomarkers for cardiovascular disorders Laboratory facilities : Assay development, & Testing Co-CHIPS Project OSEO funded Firalis SAS University of FREIBURG / IMTEK ISO17025 accreditated and ISO13485 certified labs ISO9001 certified R&D NF compliant Biobanking Co-SAVE Project: biomarkers for drug-induced cardiovascular risks First Products RUO Pre-IDE for IVD candidates Validation of RUO products CardioScan100 VascularScan100 Extension of RUO range IVD clinical studies Extension of RUO and IVD range Regulatory filing for IVD products Marketing Co-development of BM candidates (1) RUO : Research Use Only (2) IVD : In Vitro Diagnostic Qualification of RUO products In clinical samples Cardiovascular IVD Products 4

5 Firalis clinical BM qualification platform The qualification of biomarkers requires close cross-talks in numerous fields of expertise. Firalis created a network of clinical centers of excellences, biobanks, biomarker laboratory, statistic and data mining groups to accelerate the translation of candidate biomarkers toward qualification in key contexts of use. ISO 17025: for testing and calibration laboratories ISO 9001: Quality Management Systems ISO13485: Assay development activities NFS96-900F: Quality of biological resource centres (BRCs)

6 Key servicing activities Biomarker identification Biomarker Assay development Biomarker data analysis Biomarker clinical qualification Sample screening Literature search System biology Ab production Development of assays Validation of assays Testing activities Integrative data analysis from pre & clinical studies x-omic data analysis Pathway analysis Predictive modelling Clinical Study design Biobanking Database creation and maintenance Regulatory consultancy Biomarker solutions Biobanking and analytics 6

7 Analytical testing Platform Multimode reader ELISA: Colorimetric, Fluorometric, Chemoluminescent Bead based Multiplexed immunoassays BioRad Luminex 200 Planar Phase Multiplexed Immunoassays Meso Scale Discovery Sector Imager 6000 Ultra-sensitive immunoassays Singulex Erenna technology Edge System by HTG Molecular Fully Automated, Extraction Free Gene Expression Analysis qpcr ABI PRISM 7900 High throuput PCR and TLDA assays Multianalyte immunoessay Cyplex platform Clinical chemistry SIEMENS Dimension EXL 200 Biochemistry Immunology/serology Enzymology

8 IMI SAFE-T Consortium The first launched IMI-JU project First funded IMI-JU Project Initiated by Firalis as applicant coordinator Start: June 2009, Duration: 6 years, Global budget: ~36 Mio Public consortium coordinator PSTC Within SAFE-T, Firalis particularly focuses on drug induced cardiovascular toxicities 8

9 IMI SAFE-T Consortium The first launched IMI-JU project Three organs needing better clinical monitoring of drug-induced injuries Kidney: current standards such as Urea and creatine increase only once 50-60% of kidney function is lost Liver: current standards are not sufficiently sensitive and specific and do not adequately discriminate normal physiologic adaptation from pathologic phenomenon Vascular System: currently no biomarkers available for drug-induced vascular injury in human Duration: 5 years Global budget: ~36 Mio Kickoff meeting: June 15, 2009 Objectives Evaluate candidate safety BMs for monitoring drug-induced organ toxicities Develop assays and devices for clinical application of safety BMs Compile enough evidence to qualify safety BMs for regulatory decision making in drug development Gain evidence for how safety BMs may also be used in the diagnosis of diseases and in clinical practice 9

10 SAFE-T Structure and Deliverables Evidence-based decision making More reliable causality assessment Better mechanistic understanding Safer translation to clinical development Earlier and more specific signal detection Enhanced clinical monitoring Improved patient safety Reduced attrition rates Accelerated and safer approval of innovative medicines 10

11 Key Achievements at Year 5 Most challenges addressed, few concerns remaining Excellent collaboration within the consortium Biomarker qualification strategy defined Biomarker candidates prioritized, assay development completed Central biobank for sample storage up and running Database and data capture system up and running Collaboration with US based consortia : PSTC/C-Path initiated, VCRC and DILIN New partners included in 2010/2011 to increase capacity for clinical studies Aquisition of 2/3 SMEs by big multinationals, a sign of attractivity and excellence? Regulatory interactions via briefing meetings with EMA/FDA SAFE-T participants >200, 1 1

12 Key Message from SAFE-T Experience Large public private cooperation, such as SAFE-T, provide great opportunities to develop precompetitive tools for drug development All participants need to put their heart and soul, Appropriate time provided to plan and execute such complex project The IMI consortia are in an ideal position to provide their lessons learned 12

13 IMI Program Attractivity for SMEs Pros & Cons Pros International working experience with EFPIA- Academia-other SMEs World-wide visibility, scientific and clinical excellence Possibility to collaborate with other consortia (e.g., PSTC/C-Path) and Health Authorities Acquisition of know-how, increased scientific and operational competence Ability to work in a quality environment with regulatory advise Availability and access to a variety of in-kind contributions from EFPIA members Possibility to exploit the IPs and market products for the Pharma industry Eased access to the Pharma market as potential clients Cons Various perceptions of IP rights across the different stakeholders SMEs invest and lose money without direct benefit (Low indirect cost rate) EFPIA in-kind contribution is as yet significantly below target Collaboration with non-eu projects may lead to a diversion on EU research funding Redundant or un-necessary in-kind contribution from EFPIA, no reporting system established yet Acquisition of SMEs by big structures, introducing an imbalance among the SME partners 13

14 Key Partners & References Clients Partners Other collaborators Web-Imagint-FP7 Web-Mitocare-FP7

15 Located in a very active region of the Biovalley (Switzerland, Germany, France) 620 m² of lab space and biobanking, L2/L3 facilities 15