December 16, 2011 Technology Transfer Tactics Webinar:

1. December 16, 2011 Technology Transfer Tactics Webinar: How your university’s innovations can become a fixture in big pharma’s pipeline Nurjana Bachman PhD - Business Development Manager

2. December 16, 2011 Technology Transfer Tactics Webinar: How big pharma can help you develop innovative therapeutics for patients Nurjana Bachman PhD - Business Development Manager

3. Then: Fully-Integrated Pharmaceutical Company One company performed all stages of drug development Discovery Research, Target Identification Drug identification and optimization Preclinical Development Clinical Development Manufacturing, Marketing and Sales

4. Now: Fully-Integrated Pharmaceutical Network One company partners to perform all stages of drug development Discovery Research, Target Identification Drug identification and optimization Preclinical Development Clinical Development Manufacturing, Marketing and Sales Academia, other companies, CROs, manufacturing partners

5. New Pharma Model for Sourcing Ideas Open Innovation

6. What does this mean for academia?

7. Children’s Ranked #1 in US • #1 ranked in: • Heart and Heart Surgery • Neurology and Neurosurgery • Urology • Orthopedics • Kidney • Cancer • #2 ranked in: • Neonatology • Diabetes and Endocrine Disorders • Gastroenterology • #3 ranked in: • Pulmonary • * US News & World Report, May 2011

8. World’s Largest Pediatric Research Enterprise $225M clinical and basic research funding 800,000sq. feet of research space 40 clinical departments 225 specialized clinical programs ~600,000 inpatient/outpatient visits 8,720 total employees 2 Nobel Prize winners 11 members, Howard Hughes Medical Institute 7 members, National Academy of Sciences 11 members, Institute of Medicine

9. History of Innovation at Children’s

10. From Innovation…

11. Invention Types

12. …to Product… Products on the Market Products in Clinical Trials

13. …and Company

14. That’s the Bayh-Dole story So what’s new?

15. Our Assets Investigators Animal models Patient samples George Daley MD PhD (HHMI) Clifford Woolf, MD, PhD Translational research infrastructure Patents Assays Expertise Marsha Moses PhD Christopher Walsh MD PhD (HHMI) Access to alternative funding sources Thought leadership

16. The Academic Medical Center’s Role in Drug Development Discovery Research, Target Identification Drug identification and optimization Preclinical Development Clinical Development Manufacturing, Marketing and Sales Academia Industry

17. Then: Academia a Source Foundation Other academic Govt Pharma Academic Medical Center Information, IP VC CRO Start-up Biotech

18. Now: Academia a Development Partner Other academic Foundation Govt Pharma Academic Medical Center VC CRO Start-up Biotech

19. New Integrated Functions in the Licensing Office Business Development Patents & Licensing Technology Development Clinical Trials

20. Children’s Technology Development Fund Each project mentored by Board member $50k or $150k awarded for 1 year product development project with CRO Technologies selected with Advisory Board

21. The Academic Medical Center’s Role in Drug Development Research, Target Patient Treatment Lead Preclinical Development Clinical Development Manufacturing, Marketing and Sales NIH: National Center for Advancing Translational Sciences Pharma programs: Pfizer, GSK, Eli Lilly, Roche, Novo Nordisk, Sanofi, UCB, Bayer, etc. Foundations: CF Foundation, LLS, JDRF, Epilepsy Foundation, etc. Academic programs: e.g. Internal Technology Development Funds

22. The Academic Medical Center: the new FIPCO? No– Mission-driven, not profit driven- different incentive structure Multiple funding sources- primary funding from government Without partners, treatments won’t get to patient With more ability, more leverage, and ability to create a better partnering opportunities Pharma “open innovation” lets us participate in new ways But– How do we structure it so that it benefits both parties?

23. Collaboration Partnership Structures Old way: • Sponsored research/licensing: negotiated on a per project basis New way- on a continuum: • From…Company-defined grant programs: application based… • Company defines access, terms and level of commitment • …To “Strategic Alliances” • Joint Steering Committee decision-making structure • Request for proposals across the institution • Joint project plan development • Long term commitment

24. Key Aspects to Consider • Ownership • Control (Joint decision-making) • Commitment • Time • Dollars • Really integrating projects into company pipelines • Commitment of internal resources • Ability to get treatment to the patient • Bi-directional knowledge-sharing Best case scenario: • Specific resources offered • Commitment jointly defined and shared • Information and expertise shared • Value recognized through shared ownership of assets developed • Helps inform our future commercialization efforts

25. Pharma’s Open Innovation: Advantages to Academia* • Access to complementary expertise and new resources • Forms basis for ongoing communication and relationship • Streamlines negotiations (expectations set up front) • Informs ongoing commercialization efforts • Ability to influence drug development and facilitate getting treatment to patient • * Assumes fair deal structure

26. How to Become a Fixture in Pharma Pipelines • a.k.a. How pharma can help you develop innovative therapeutics • Understand the breadth of your assets • Understand partners’ needs, goals, resources and limitations • Understand your institution’s needs and limitations • Articulate these to the industry partner • Build long-term relationships • Communication and transparency!

27. This webinar is sponsored by Merrill DataSite – The Secure Virtual Data Room Solution for the Life Sciences Industry