Judith Caron, Programs Director, CQDM May CQDM/CIHR C OLLABORATIVE F UNDING P ROGRAM IN P ERSONALIZED M EDICINE TO A CCELERATE D RUG D ISCOVERY

Our mission To fund support breakthrough technologies that will 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 THE ONLY PRE-COMPETITIVE PHARMA CONSORTIUM IN CANADA

Pfizer AstraZeneca Merck Lilly Novartis Boehringer Canada Québec GSK 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 Fully paid-up Worldwide Perpetual This model allows High financial leverage ( fold) To derisk R&D To improve R&D productivity

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

Completed 18 competitions within 6 funding programs 450 projects reviewed Established an international mentorship program (over 80 mentors) Raised $65 M of public and private funding Built a strong R&D ecosystem including pharmas, SEMs and academia HIGHLIGHTS

OUR PORTFOLIO A network of 400 researchers A clear focus on  Scientific excellence and innovation  Strong impact on biopharmaceutical research  Translational research  Multidisciplinary approach  Public/private partnerships 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

SEVERAL THERAPEUTIC INDICATIONS Across many 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

 Each project is assigned a mentor from each pharma sponsor  The mentors are international experts from the global Pharma organizations  The mentors meet with the investigators twice a year, on site, and provide guidance on how to deliver results with highest impact  They can provide some additional resources (clinical samples, database, specific expertise)  The mentors become the champions of the funded projects in their respective corporate organizations THE MENTORSHIP PROGRAM: A WIN/WIN COMBINATION A network of 80 mentors

RIGHTS FOR CQDM PHARMA MEMBERS Results Deliverables New IP Know how Data Copyrights No new IP Owned by Researchers and Research Entities Background IP CQDM project R&D use Fully paid-up, perpetual, worldwide licence option to use deliverables for R&D Results – data – process - technology - platform Service agreement with the PI (when relevant)

OUR ACHIEVEMENTS 32 outstanding research projects engaging $37.3M 10 projects completed with a success rate of 90% 80% of the technology already used by the pharma members 3 major investments by pharma members to use the results at the end of the project Success stories of great value for biopharmaceutical research HTS discovery platform for VLP vaccine antigens Biomarkers for monitoring beta cells mass and function in diabetes patients Biosensors for GPCR signaling pathways Early diagnosis of pulmonary hypertension ERG for non invasive stratification of psychiatric disorders Flash walk mapping for GPRCs Synthetic lethality in oncology Several tangibles economic benefits for Quebec and Canada Support of 256 jobs Technology maturation for 12 SMEs 25 partnerships with industry Creation of 5 starts-up Additional $14.5 M from industry

CQDM/CIHR C OLLABORATIVE F UNDING P ROGRAM IN P ERSONALIZED M EDICINE TO A CCELERATE D RUG D ISCOVERY

SCOPE OF THE CQDM/CIHR PROGRAM This pan-Canadian program offer public and private partners the opportunity to work together to develop novel technologies and tools that address current and critical challenges faced by the pharmaceutical industry in the area of personalized medicine. To develop cutting-edge next-generation technologies, computational tools and devices with an immediate and strong impact on the discovery/development of new drugs  Not intended to fund the development of specific molecules  Not intended to fund the discovery of biomarkers with ‘’omics’’ approaches

RESEARCH AGENDA  Research projects focused on the invention and development of next-generation of technologies, computational tools and devices in the following research areas: cancer infection and immunity neurodegeneration affecting cognition  No technologies/tools/devices funded through other initiatives in personalized medicine  Broad range of research fields/disciplines: biological and health sciences, chemistry, engineering, nanotechnologies, computational sciences, medical devices, imaging, biosensors, in silico and in vitro screening systems, and improved disease-relevant animal models, etc.  Well-defined near-term application with potential to significantly impact biopharmaceutical R&D

Research performed in Canada Academic sector: laboratories, affiliated research institutes, hospitals Private sector: SMEs, biotechs, CROs Public/private partnerships mandatory: minimum 1 academic researcher and 1 industrial researcher working collaboratively Address most important challenges of the biopharmaceutical industry Highly innovative projects with strong and wide potential impact on the pharmaceutical drug R&D process and the Canadian healthcare system Multinational pharmaceutical companies not eligible to receive funds ELIGIBILITY

Duration: Up to 30 months Funds available: $750K maximum Private matching funds: 25% of total project cost (in cash, in kind, combination) Eligible costs: Salary of research personnel; Material and supplies; Travel expenses; Intellectual property costs; Administrative costs (up to 15% for the CQDM portion). CIHR’s policies and general guidelines, including those specific to overhead costs, will apply to its contribution Non-eligible costs: Equipment or facilities; Salary of the principal investigator and co-investigators FUNDING SPECIFICS

REVIEW AND DECISION PROCESS Scientific evaluation External review committee CQDM/CIHR joint committee Board Final selection Research agreement Projects start LOI Full applications Fit program + matching funds requirements Scientific evaluation External review committee Fund in order of scientific merit  3-step process: CQDM/CIHR joint committee to decide before Scientific evaluation 16 CQDM/CIHR joint committee IMPACT

Scientific excellence and novelty Potential of applications in drug research o immediate impact on drug discovery or drug development o capacity to address important unmet needs Clear well-defined deliverables and milestones Global competitiveness Team expertise/experience Quality of collaboration Alignment with corporate objectives of the private partner(s) EVALUATION CRITERIA OF THE FULL PROPOSALS

IMPACT ON THE DRUG R&D PROCESS Identify most important challenges currently faced by the pharmaceutical industry How will you project address them? How will the expected results and the deliverables of the project impact the drug R&D process? Please specifically discuss the following possible impacts (if applicable):  Opening new therapeutic approaches and research avenues  Bringing more effective medicines to the clinic and to the market  Increasing the efficacy of existing drugs  Reducing the R&D costs, time to market or the risks of development  Other impacts on the drug discovery and development process

POSITIONING/GLOBAL COMPETITIVENESS Not about the team or facilities It’s all about the technology, product, tool, device Identify:  Current state of the art  Competition: o Direct and indirect o Local and international o Existing or in development Compare your technology, product, tool, device Bring out:  Advantages  Differentiating factors  Originality and novelty aspects

MANAGEMENT OF FUNDED PROJECTS Projects are deliverables-based and milestones driven Research agreement included:  Semi-annual milestones  Deliverables  Detailed budget  Gantt chart: major steps, milestones, deliverables, budget Ensure success of project

... mutual benefits for researchers and CQDM pharma members Minimally one pharma mentor Opportunity for researchers to create strong links and collaborate with pharma industry Two meetings/year (co-ordinated by CQDM) Informal interactions throughout year Mentors role? Ensure technology developed fully aligned with industry needs Eager to aid with project success in mind (not to evaluate) May provide additional resources (clinical samples, database, specific expertise) MENTORSHIP PROGRAM: ONE OF A KIND

TIMELINES CQDM/CIHR 2014 COMPETITION Important dates Program launchMarch 18 th, 2014 Deadline for submission of the letters of intentJune 27 th, 2014 Announcement of the selected letters of intentSeptember 11 th, 2014 Deadline to submit full proposalsNovember 27 th, 2014 Final selection announcementMarch 31 st, 2015 Signature of a research agreement Three months window Funding release and beginning of the projectsJuly 1 st, 2015

EXAMPLES OF DELIVERABLES  Software for data analysis or data measurement  Diagnostic tools  Imaging tools  Medical devices or instruments  Prototypes  Delivery systems  Predictive algorithms  Etc......

EXAMPLES OF PROJECTS Cancer Richard Kremer Platform for the enrichment of CTCs to follow disease progression/treatment outcome Infection & Immunity Michel Bergeron Profiling the gut microbiome to predict the emergence of antibiotic resistance Neurodegeneration affection cognition Rick Hoge Profiling patients with AD using a new MRI technique

CONTACT INFORMATION Thank you for your participation! Merci de votre attention! Olivier Jacob-Gravel Program Delivery Coordinator CIHR Etienne Richer Assistant Director CIHR Institute of Genetics More information: competitions/cqdm-cihr- competition.php Judith Caron Director of Programs CQDM