1. An Academic Model to Bridge the Valley of Death April 17, 2009 Scott Weir, PharmD, PhD Institute for Advancing Medical Innovation University of Kansas

2. Reducing Cancer Burden in Kansas Ewing Kauffman Foundation Drug Discovery, Delivery and Development State Economic Impact Academic, Industry and Disease Philanthropy Partnerships Pharmaceutical industry expertise and best practices Aligning The Stars in Kansas Key Success Factors

3. Reducing Cancer Burden in Kansas *American Cancer Society. Statistics for 2008. 25,700 citizens in Kansas and western Missouri diagnosed in 2008* 11,028 deaths in 2008* Cancer mortality rate reductions in KS (-0.5%) and MO (-0.7%) lags national trend (-2.1%) $4.3 Billion annually in medical expenses and lost productivity *Cancer patients must travel 200 to 700 miles to reach an NCI- designated Comprehensive Cancer Center Regional Problem: Cancer

4. More advanced cancer prevention, early detection, treatment, and survivorship Drug Discovery, Delivery & Development Community-based Approach Institute for Advancing Medical Innovation Results Clinical Trials Office NCI Designation Differentiators Prevention & Control Research Prevention & Control Research Breast Cancer Prevention & Survivorship Centers Rural Primary Care Outreach Reducing Cancer Burden in Kansas

5. Largest foundation devoted to innovation and entrepreneurship $16.2M over five years Proof of Concept funding Industry adjunct faculty Project managers Fellowships Ewing Kauffman Foundation Entrepreneurship

6. Advance Medical Innovations To Commercialization Novel Drugs & Drug Targets Drug Products & Delivery Platforms Drug Discovery Research Training the Next Generation Of Medical Innovators Drug Delivery Research Institute for Advancing Medical Innovation Drug-Device Combination Products Drug-Device Combination Products Integrated Bio-Engineering & Drug Research Bio- Engineering Research Biomaterials & Medical Devices Biomaterials & Medical Devices Ewing Kauffman Foundation

7. Drug Discovery, Delivery and Development Capitalizing on Pharmacy Strengths NIH Funding History Medicinal Chemistry »Chemical Methodologies and Library Development Center »Specialized Chemistry Center »Faculty Pharmaceutical Chemistry »Dr. Higuchi, The Father of Physical Pharmacy »Alza Corporation founded at KU »NCI formulation contract (Taxol®, Velcade®) »Dr. Val Stella

8. Reengineered Discovery Process 1 Target Selection & Validation Target Production 2 3 High Throughput Screening 4 Chemical Hit Identification Target -> Chemical Hit Chemical Hit -> Lead 5 Define Lead Selection Criteria 6 Prediction of Physio- Chemical Properties 7 In vitro Potency & Selectivity In vivo Proof of Concept 8 Early ADMET Lead -> 9 Pre – Formulation Screening 10 In vivo and In vitro ADMET Profiling 11 Prepare for IND Enabling Activities Development Candidate Novel Drug Target 10 – 18 months~ 18 months Drug Discovery, Delivery and Development Development Candidate

9. Our Drug Pipeline 62 Projects 36 Cancer Projects

10. Investors in Our Program State Economic Impact Drug Delivery Center of Excellence Entrepreneurship training Faculty recruitment Facilities Facilitating collaborative research R&D Infrastructure Innovation centers Federal grant matching Heartland BioVentures

11. Kansas Centric Big Picture Target Selection & Validation HTS Identified Chemical Hits Optimized Chemical Lead Candidates Development Candidate Selected Drug Discovery Early Stage Drug Development GLP Drug Safety GMP Drug Substance & Product Mfg IND Prep Submission Filing Late Stage Drug Development Phase I Clinical Trial Phase II Clinical Trials Phase III Clinical Trials 3 Years1 Year6-8 Years Product Registration NDA Preparation Submission And Approval 1.5 Yr Within Universities Kansas Start-Up National Drug Development Accelerator Commercialization Entity State Economic Impact

12. Partnerships Assembling the Bridge Advancing Innovations to Clinical Proof of Concept Pharma Partners Pharma Partners Academic Partners Academic Partners Disease Philanthropy Partners Disease Philanthropy Partners KU Researchers, Cores and Centers KU Researchers, Cores and Centers NIH NCI NIH NCI Investors

13. “LLS, University of Kansas Cancer Center Announce Innovative Partnership to Accelerate Drug Development” January 7, 2009 Press Release Currently $1.5M in funding supporting two projects Counts toward cancer-related research funding April 22 Health Canada Pre-CTA Meeting Partnerships

14. “Institute for Pediatric Innovation Awarded Funding by University of Kansas to Analyze the Use of Extemporaneously Prepared Medicines in Children’s Hospitals” June 30, 2008 Press Release KU is the drug development partner with Beckloff Associates Inc. First pediatric product developed and ready for GMP manufacturing Kauffman Foundation grant funding development of two products annually Partnerships

15. Pharma “profiling” Go/no go decisions Pre-determined criteria Industry experienced leadership and project management High performance project teams Cores and centers Partnerships Portfolio review and prioritization Drug targets Continuous pipeline review Leveraging industry partners (e.g., GLP, GMP) Integration of intellectual property management activities Pharmaceutical Industry Best Practices

17. Offering Our Platform to Collaborators Within the University Industry

18. Measurable Outcomes In the Clinic in 2009! Advancing Innovations to Clinical Proof of Concept Nanotax® Industry/Academia Collaboration Nanotax® Industry/Academia Collaboration Reformulated Chemotherapy Agent Industry/Academia Collaboration Reformulated Chemotherapy Agent Industry/Academia Collaboration Repurposed Agent for Blood Cancers Academia/Philanthropy/ Industry Collaboration Repurposed Agent for Blood Cancers Academia/Philanthropy/ Industry Collaboration SR-13668 Phase 0 Academia/Academia/NCI Collaboration SR-13668 Phase 0 Academia/Academia/NCI Collaboration