1. December 16, 2011 Technology Transfer Tactics Webinar: How your universitys innovations can become a fixture in big pharmas 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. Drug identification and optimization Manufacturing, Marketing and Sales Clinical Development One company performed all stages of drug development Discovery Research, Target Identification Preclinical Development Then: Fully-Integrated Pharmaceutical Company

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

5. New Pharma Model for Sourcing Ideas Open Innovation

6. What does this mean for academia?

7. Childrens 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. Worlds Largest Pediatric Research Enterprise $225M clinical and basic research funding 800,000 sq. 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 Childrens 1869Children's Hospital Boston opens as a 20-bed facility 1920Dr. William Ladd devises procedures for correcting various congenital defects 1922Dr. James Gamble develops a method for IV feeding that saves the lives of thousands of infants at risk of dehydration 1938Dr. Robert Gross performs the world's first successful surgery to correct a congenital cardiovascular defect 1954Dr. John Enders wins the Nobel Prize for successfully culturing the polio virus 1971Dr. Judah Folkman publishes "Tumor angiogenesis: therapeutic implications" in NEJM 1986Dr. Louis Kunkel identifies the gene for Duchenne muscular dystrophy 1990Dr. Joseph Murray wins the Nobel Prize for his pioneering work in organ transplantation

10. From Innovation… 1983Children's physicians report the first surgical correction of hypoplastic left heart syndrome, a fatal condition. 1986Dr. Louis Kunkel identifies the gene for Duchenne muscular dystrophy 1990Dr. Joseph Murray wins the Nobel Prize for his pioneering work in organ transplantation 1998Dr. Anthony Atala successfully transplants laboratory-grown bladders into dogs, a major advance in the growing field of tissue engineering. 2008Dr. George Daley discovers how to reprogram human somatic cells to pluripotent stem cells with defined transcription factors. 2008Dr. Chris Walsh and his colleagues identify several genetic loci associated with Autism 2008Drs. Vijay Sankaran and Stuart Orkin discover the fetal hemoglobin to adult hemoglobin switch is controlled by BLC11A transcription factor.

11. Invention Types

12. NameCompany dmPGE2 (stem cell stimulation) Von Willebrand Factor Anti-Neuropilin Antibody Tissue Engineered Bladder Pomalidomide Omegaven FcRn-Factor VIII …to Product… NameCompany Revlimid Thalidomide Merge EchoIMS Neumega Namenda Dystrophin Diagnostic INF2 DNA Sequencing Test CardioSeal, StarFlex, BioStar Cannula Needle Set Patient Communication Board Plagio Cradle Quickchange Mutagenesis Kit Products on the MarketProducts in Clinical Trials

13. …and Company

14. Thats the Bayh-Dole story So whats new?

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

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

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

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

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

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

21. Research, Target Lead Manufacturing, Marketing and Sales Clinical Development Preclinical Development The Academic Medical Centers Role in Drug Development Patient Treatment 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 wont 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. Pharmas 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 institutions 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