IMI Projects : SMEs Contributions and Benefits from Large Public … · 2014. 9. 16. · Firalis...
Transcript of IMI Projects : SMEs Contributions and Benefits from Large Public … · 2014. 9. 16. · Firalis...
IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014
IMI Projects : SMEs Contributions and Benefits from Large Public-Private Initiatives on Precompetitive Projects
Prof. Hüseyin FIRAT, MD,PhD
IMI SAFE-T Applicant Consortium coordinatorPartner of the IMI BT-Cure and Pharmacog consortia
OverviewBiomarker Discovery, Development and Regulatory Qualification
Firalis
Amoneta
Diagnostic
TcLandExpression
Biomarkers on Cardiovascular DiseasesFounded in 2007
Biomarkers on Inflamatory DiseasesAcquired by Firalis in July 2013
Biomarkers on AlzheimerCreated on January 2014
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014 2
IMI SAFE-T Consortium
IMI Pharmacog Consortium
IMI BT-Cure Consortium
Company Overview
Thanks to IMI projects, Firalis gained overall expertise and cumulated know-how, andbecome an acknowledged key player in the field of biomarker qualification.
Firalis develops, manufactures and markets biomarkers (BM) and BM-based tools for drugdevelopment and clinical applications.
Firalis business model stands on a tripod :
I. BIOMARKER R&D : Firalis research activities focus on biomarker qualification in specificcardiovascular disorders. In this parallel, Firalis joined several international programs includingthe IMI-JU SAFE-T Project, where it has coordinated the consortium during initial phase.
II. BIOMARKER PRODUCTS : Firalis develops, manufactures and markets BM-based diagnosticsolutions; which are first marketed as «Research-Use-Only» (RUO) kits and then transforminto «In Vitro Diagnostics» (IVD) tools, upon clinical validation and regulatory qualification ofbiomarkers used in the corresponding kit.
III. BIOMARKER SERVICES : Firalis is conducting several research projects with and for the bio-pharmaceutical industry.
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014 3
Cardiovascular IVD 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
SAFE-T Project Impact at Firalis
developing biomarkers for cardiovascular disorders
Co-CHIPS ProjectOSEO funded Firalis SAS University of FREIBURG / IMTEK
Laboratory facilities :
Assay development, & Testing
• First Products RUO• Pre-IDE for IVD candidates
ISO17025 accreditated and ISO13485 certified labsISO9001 certified R&D NF96 900 compliant Biobanking
(1) RUO : Research Use Only(2) IVD : In Vitro Diagnostic
Validation of RUO products
Qualification of RUO products In clinical samples
Co-SAVE Project: biomarkers for drug-induced cardiovascular risks
4
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)
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014
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 solutionsBiobanking and analytics
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014 6
Analytical testing Platform
Bead based MultiplexedimmunoassaysBioRad Luminex 200
Multimode readerELISA: Colorimetric, Fluorometric, Chemoluminescent
Ultra-sensitive immunoassaysSingulex Erenna® technology
qPCRABI PRISM 7900High throuput PCRand TLDA assays
Clinical chemistrySIEMENS Dimension EXL 200BiochemistryImmunology/serologyEnzymology
Planar Phase MultiplexedImmunoassaysMeso Scale DiscoverySector Imager 6000
Edge System by HTG MolecularFully Automated, Extraction Free Gene Expression Analysis
Multianalyte immunoessayCyplex platform
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014
IMI SAFE-T ConsortiumThe 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
Within SAFE-T, Firalis particularly focuses on drug induced cardiovascular toxicities
PSTC
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-20148
IMI SAFE-T ConsortiumThe 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
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
• Duration: 5 years• Global budget: ~36 Mio €• Kickoff meeting: June 15, 2009
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-20149
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
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-201410
11
Key Achievements at Year 5Most 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 andrunning
Collaboration with US based consortia : PSTC/C-Path initiated, VCRC and DILIN
New partners included in 2010/2011 toincrease capacity for clinical studies
Aquisition of 2/3 SMEs by bigmultinationals, a sign of attractivity andexcellence ?
Regulatory interactions via briefing meetings with EMA/FDA
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014
SAFE-T participants >200, www.safe-t.eu
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
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-201412
IMI Program Attractivity for SMEsPros & 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
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-201413
Key Partners & References
Web-Imagint-FP7
Partners
Web-Mitocare-FP7 http://www.imi-safe-t.eu/
Clients
Othercollaborators
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014
• Located in a very active region of the Biovalley(Switzerland, Germany, France)
• 620 m² of lab space and biobanking, L2/L3 facilities
H. Firat IMI2, Hôpital Saint-Antoine, Paris/ 09-Sept-2014 15