A unique Collaborative Model for the Discovery of New Therapeutic Approaches

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1 A unique Collaborative Model for the Discovery of New Therapeutic Approaches

2 THE INNOVATION GAP

3 OPEN INNOVATION TO ADDRESS THE LACK OF PRODUCTIVITY OF BIOPHARMACEUTICAL RESEARCH

4 A PRE-COMPETITIVE PHARMA CONSORTIUM Our mission To fund support breakthrough technologies that significantly enhance biopharmaceutical R&D productivity and accelerate the development of safer and more efficacious drugs To create a collaborative ground between the academic and private sectors based on trust, creativity and mutual benefit

5 A COLLABORATIVE APPROACH TO SHARE THE R&D COSTS AND THE RESULTS Each pharma member has a non-exclusive end-user licence option for R&D purpose Canada This model allows: High financial leverage (10-20 fold) To fund early stage research To derisk R&D To develop new tools that will improve R&D productivity

6 HIGHLIGHTS Raised $65 M of public and private funding Established an international mentorship program Completed 10 competitions within 4 funding programs Built a strong R&D ecosystem including pharmas, SEMs and academia

7 OUR FUNDING PROGRAMS Focused on technologies that accelerate the drug discovery/development process Focus Large scale Multidisciplinary Immediate impact on drug discovery $2M Québec/Ontario Large scale Collaboration QC/ON Immediate impact $1M Québec/Massasuchetts Large scale Collaboration QC/Mass Late stage R&D $2M Explore Highly innovative Disruptive technologies Early concept validation $300k Québec/France Large scale Collaboration QC/Fr Immediate impact $1.4M Pan Canadian Large scale Canadian network Immediate impact $1.5M

8 A clear focus on Scientific excellence and innovation Strong impact on biopharmaceutical research Translational research Multidisciplinary approach Public/private partnerships Interest to work with the pharma industry OUR PORTFOLIO 32 research projects for a total of $37,3M 46 research entities: 25 academic institutions 21 private companies Several different sectors of activity Engineering imaging photonics nanotechnologies instrumentations biomarker development discovery platforms drug delivery A network of 400 researchers

9 A MULTIDISCIPLINARY APPROACH Across several sectors of activities Genomics System biology Informatics Chemistry Engineering Biomaterials Imaging Diagnostic Physics Medical devices Pharmacology Biochemistry Cell Biology Clinical sciences Proteomics Nanotechnology Biophysics Physiology

10 A STRONG IMPACT ON THE WHOLE DRUG DEVELOPMENT PROCESS 5 YEARS OF OPERATIONS $37,3 M COMMITTED Shore Synthetic lethality oncology Chelsky/Gasman Drug targets neuroendocrine tumours Blais-Ouellette/Bischoff Protein-protein interactions CNS target discovery Richards Somatic mutations of reacting T cells inflammatory diseases Bouvier Biosensors GPCR signalling Vezina Discovery platform VLP vaccine Ag Yudin/Marsault Macrocycles library Protein-protein interactions Pineyro Biosensors for ion channels Götte Screening methods antivirals Bouvier/Kieffer New engineered mice for GPCR studies Hébert Dynamic response to GPCR drugs Najmanovich Molecular interactions field platform Purisima In silico predictivity of mab affinity Mes-Masson Microfluidic platform for cancer therapy Escher Real-time in vivo analyte measurement ADME studies Bergeron Resistance to antibiotics Mishra/DiPaolo Parkinson s disease Early diagnosis and treatment Bedell Animal models Alzheimer s disease Maziade ERG biomarkers psychiatric disorders Paramithiotis Early diagnostics diabetes Hoge Patient stratification Alzheimer s disease Martel Magnetic guided drug delivery platform Dupuis Imaging biomarker pulmonary hypertension Park Stromal biomarkers breast cancer Kremer Method to isolate circulating cancer cells

11 A UNIQUE MENTORSHIP PROGRAM Assigned mentors from each pharma member Assist investigators in understanding needs of the biopharma industry Support successful and timely achievement of project May provide additional resources clinical samples databases specific expertise A network of over 60 mentors International experts from global pharma Two meetings / year co-ordinated by CQDM Informal interactions throughout the year Mutual benefits for researchers and CQDM pharma members Early and privileged access to results for CQDM pharma members Opportunity for researchers to collaborate with pharma industry

12 SEVERAL SUCCESSES AFTER 5 YEARS M. Bouvier (University of Montreal) Biosensors for GPCRs siganilling Over 38 biosensors covering the GPCRs landscape HTS tools to better predict drug efficacy and undesired effects Already used by CQDM pharma sponsors Major investment from Pfizer to develop signalling signature with molecules of specific interest J. Dupuis (Montreal Heart Institute) Non-invasive biomarker for early diagnosis of pulmonary hypertension Facilitate patient recruitment Treat early with the expectation to increase drug efficacy Replace the 6 min walk test as the end-point in clinical trials Significant in kind contribution from Merck to develop PET imaging biomarkers L. Vézina (Medicago) - High-throughput discovery platform for VLP vaccines Cost and time reductions (10x) in the discovery of new VLP antigens Capacity to discover candidates for challenging viruses and others antigens Discovery of new candidates for Ebola and Rabies ( under development within the company) Proof of concept with RSV E. Paramithiotis (Caprion Proteomics) - Biomarkers for Beta cell mass/function in diabetes Panel of 30 -cells biomarkers to monitor diabetes with an accuracy of 94% Rapid identification of pre-diabetics subjects for clinical trials Follow the decline of -cell function in subjects leading to the development of novel early diabetic treatments Outstanding collaboration between Caprion and 2 KOLs in diabetes

13 FUTURE CONSIDERATIONS To create Canadian and International networks dedicated to the advancement of next-generation technologies with the ultimate goal of bringing better cures to patients Expand activities across Canada with more specific research agenda to address the most important bottlenecks in biomarphaceutical research Neurosciences Oncology Ab resistance Personalized medicine Initiate an international expansion with a special focus in France, Germany, UK and the US Work in collaboration with international Foundations to bring solutions to unmet medical needs Add new pharma members

14 CO-CREATION OF VALUE FOR ALL STAKEHOLDERS Pharma Access to innovation Very high financial leverage (up to 20x) for R&D SMEs and academic institutions Additional source of non dilutive funding Technology maturation Capacity to fund high risk R&D Access to enabling technologies for drug discovery Close interactions with KOLs in several sectors of activity Funding for translational research Partnerships with big pharmas Technology showcase Access to Pharma mentors

15 A UNIQUE PRE-COMPETIVIE CONSORTIUM TO ACCELERATE THE DISCOVERY OF MORE EFFICIENT AND SAFER DRUGS