Public-Private Partnerships for Bridging the Innovation Gap in Personalized Medicine

Transcription

1 Public-Private Partnerships for Bridging the Innovation Gap in Personalized Medicine Martin LeBlanc, President and CEO, Caprion The 2014 ISPIM Americas Innovation Forum October 6,

2 CHALLENGES FOR BUILDING VIABLE BUSINESS MODELS IN THE PERSONALIZED MEDICINE AREA Strong unmet need and Rx/Dx industry demand for innovative and validated companion Dx candidates Biomarker innovation gap unlikely to be resolved by private sector alone due to prevailing Rx, Dx and VC industry dynamics Significant business model challenges for biomarker discovery technology platform companies Sustainability and risk-reward challenges of pure service model Venture capital availability challenges for pure biomarker product development models Potential economically viable strategies from hybrid business models that leverage strategic funding from both public and private sector sources Discuss Caprion s approach to biomarker development and innovative private-public partnership models for funding early-stage R&D in Canada/Quebec as strategies for addressing these challenges CONFIDENTIAL 2

3 DRIVERS OF PERSONALIZED MEDICINE- Pharma R&D Productivity Crisis Global Pharmaceutical R&D Spend For the Years Ended December 31, ($ in billions) $60 $50 $40 $30 $20 $ CAGR = 28.2% P CAGR = 34% NME, BLA Approvals For the Years Ended December 31, $ Pharmaceutical R&D Spend NME, BLA Approvals Estimated $1.0 billion investment required for successfully bringing a new drug to market. Approximately 70% to 90% of new drug candidates fail during clinical trials. Submission rate of new drug applications for New Molecular Entities ( NME ) and Biologics License Applications ( BLA ) has decreased over 50% in the past decade Rising percentage of late-stage clinical failures, now estimated at 50% of compounds tested in phase III trials CONFIDENTIAL 3 3

4 DRIVERS OF PERSONALIZED MEDICINE- One Size Does Not Fit all CONFIDENTIAL 4

5 PERSONALIZED MEDICINE- The New Paradigm for Drug Development Patient selection Patient Profiling Targeted Therapy Over 65 personalized medicine drugs & diagnostics currently comercially available of which 75% are in the cancer field Several new PM drugs approved by FDA since 2011 with over 80% response rates and that were developed in less than 6 years ans with significantly lower R&D costs CONFIDENTIAL 5

6 BRIDGING THE BIOMARKER INNOVATION GAP THROUGH PUBLIC-PRIVATE PARTNERSHIPS Clinical education / Adoption Commercial Result consistency Discovery Validation Distribution to laboratories Regulatory approval Clinical validation IVD Assay development Assay prototype testing Biomarker identification Medical needs and biomarker hypothesis LARGE DX COMPANIES Value Inflexion INNOVATION GAP CAPRION EXPERTISE CONFIDENTIAL 6

7 PRE-COMPETITIVE/CONSORTIUM APPROACHES FAVORED AMONG BIOMARKER INITIATIVES Pre-competitive research : middle ground of focused cutting-edge research that lies between fundamental basic research in academia and proprietary research performed in corporate laboratories Concept: work collaboratively to expedite the generation of resources (tools, data and specimens) that each stakeholder can use to drive commercial products. Key biomarker stakeholders: pharma, diagnostic and biotech industries, academia, non-profit foundations and associations, professional societies, advocacy groups, payers, healthcare providers. For the pharmaceutical industry, the competitive space includes: development, regulatory approval and marketing of proprietary therapeutics Precompetitive space includes all preceding stages of the development pipeline, such as understanding disease biology, revealing molecular pathway(s), identifying novel therapeutic targets and biomarkers of efficacy/toxicity that could serve as surrogate endpoints for use in clinical trials process. For drug development industry, precompetitive opportunity centered on ability to share cost and risk of upstream R&D Intellectual property considerations associated with the precompetitive effort must be carefully managed so as to not create a barrier for downstream commercialization CONFIDENTIAL 7

8 CONSORTIUM APPROACH FAVORED AMONG BIOMARKER INITIATIVES FDA Critical Path Institute (C-Path) Biomarker Consortium (BC) Predictive Safety Testing Consortium (PSTC) Innovative Medicines Initiative (IMI), European Personalized Medicine Association (EPEMED) Wellcome Trust Case Control Consortium International Cancer Biomarker Consortium Quebec Personalized Healthcare Network (QPHN) Personalized Medicine Partnership for Cancer (PMPC) Quebec Consortium for Drug Discovery (CQDM) Fonds de Partenariat pour un Quebec Innovant et en Santé (FPQIS) CONFIDENTIAL 8

9 CAPRION CORPORATE OVERVIEW Founded in 2000 Located in - Montreal, Canada and Menlo Park, California 85 employees (65 scientists) Focused on personalized medicine and biologics Leading service provider to pharma, biotech and gov t institutions - Mass spec-based proteomics for target & biomarker discovery and validation - Multiparametric flow cytometry for GLP immune monitoring services Developing 3 in-vitro diagnostic products Venture backed from and Caprion Pharma spun-out service business in 2007 Private equity ownership since Great Point Partners, LLC ( ) - Chicago Growth Partners (since July 2012) CONFIDENTIAL 9

10 DUAL-TRACK BIOMARKER BUSINESS MODEL Proteomics / Immunology Services Fee-for-service business model in partnership with pharma, biotechs and not-for-profit organizations ProteoCarta: Proteomics Services for Biomarkers/Personalized Medicine Biomarker and drug target discovery Multiplexed MRM-MS assays for biomarker verification & validation ImmuneCarta: Immune Monitoring Services for Biologics Development Multi-parametric flow cytometry Functional analyses of complex innate and adaptive immune responses Over 50 leading pharma & biotech clients EBITDA positive since 2006 In-Vitro Diagnostics Leverage non-dilutive funding for development of novel in-vitro diagnostic products: Oncology Infectious disease Diabetes Discover, validate and develop biomarker candidates & retain rights until clinical validation stage Pursue commercial development of LDT tests from CLIA lab in Montreal Out-license hospital-based tests to major Dx companies Over $30 million of non-dilutive funding secured since 2009 CONFIDENTIAL 10

11 CAPRION: STRONG INDUSTRY PARTNERSHIPS Fee-for-service model with pharmas, biotechs and not-forprofit organizations Over 50 industry and government partners All therapeutic areas - Cancer, inflammation, metabolic disease, infectious disease, CNS All sample types - Plasma, serum, CSF, cells, tissue Perform complete studies: - Study design - Sample preparation - Sample analysis - Statistical analysis - Biological interpretation - Comprehensive reporting Dedicated program management CONFIDENTIAL 11

12 CAPRION IN VITRO DIAGNOSTIC PRODUCT DEVELOPMENT PIPELINE Programs Candidate Discovery Candidate Verification Candidate Validation Commercial Deployment Cancer Lung nodules Neuroendocrine tumors Tuberculosis Infectious diseases Brucellosis Lyme disease Pediatric fever Diabetes Disease progression Drug response Over $30M of non-dilutive funding secured from gov t and industry partners for Dx discovery and validation in 3 disease areas since 2009 CONFIDENTIAL 12

13 XPRESYS LUNG NODULE ASSESSMENT TEST COLLABORATION WITH INDI My CT scan shows a lung nodule Is my nodule benign or malignant? Do I need surgery? High unmet need > 3 M lung nodules/yr in US Majority are benign after lengthy diagnostic odyssey US healthcare costs of $12B/yr on these work-ups High unmet need for noninvasive diagnosis of lung nodules Xpresys Lung Test 11-protein blood test to assess with high probability if a lung nodule is benign Discovered and developed by Indi with Caprion Used proteomics approach and multiplexed MRM format in centralized CLIA Lab model Launched by Indi in US in November 2013 Caprion receives royalties on worldwide sales Test co-commercialized by Caprion in Canada with funding support from PMPC (MFEQ) CONFIDENTIAL 13

14 About the PMPC: Public-private partnership between academic, clinical, biotech and global pharmaceutical groups Led by Caprion Proteome Four year project funded by Quebec Gov t and private partners ($21.1M) PMPC Goals: Develop, validate, deploy solutions to enable the practice of personalized medicine for cancer Improve the efficiency and costs of the healthcare system in Quebec Position Québec as global leader of personalized medicine Attract additionnal pharma partners to conduct personalized medicine clinical trials in Quebec

15 PMPC: Public-private Partnership Quebec Clinical Research Organization in Cancer (Q- CROC) Multidisciplinary network of clinicians, basic scientists, and members of the medical community committed to translational cancer research.

16 PMPC Program Thematics Deploy and integrate biomarker discovery and validation platforms with clinical practice for the implementation of personalized medicine Discover, develop, validate and commercialize biomarkers for lung, colon and breast cancer Implement electronic solutions to facilitate personalized medicine decision-making through efficient data management and analysis Provide a framework to address pharmacoeconomic, ethical, legal, reimbursement and approval issues to accelerate the adoption of personalized medicine solutions through the Quebec Healthcare System

17 Integrated Strategy for Enabling Personalized Medicine Broad and unique network of clinicians and hospitals to facilitate clinical development and deployment of personalized medicine solutions Retrospective and prospective access to high quality research grade clinical biospecimens for supporting biomarker discovery and validation Leading edge integrated technology platforms to support biomarker discovery and validation: proteomics, immune monitoring, genomics, bioinformatics Novel focused diagnostic, prognostic and theranostic biomarker applications for full spectrum of cancer applications Integrated electronic solutions to support clinical, scientific and Healthcare System decision-making Conditional reimbursement of innovative cancer drugs and diagnostics based on economic assessment of health and cost benefits to Quebec Healthcare System Approval and integration of novel personalized medicine solutions in the Quebec Healthcare System