Sanford-Burnham Update Orlando’s Developing Life Sciences Initiative

1. Sanford-Burnham UpdateOrlando’s Developing Life Sciences Initiative Daniel P. Kelly, M.D. Scientific Director July 10, 2012

2. Sanford-Burnham Medical Research Institute La Jolla Est. 1976 Orlando Est. 2007 • Not-for-profit research • Cancer, neurologic, infectious and childhood disease, diabetes and obesity • # 3among research institutes in NIH funding • # 1 worldwide ranking: citation of Sanford-Burnham studies in biology and biochemistry journals

3. First Medical City facility to open (April 2009) • 175,000 sq ft facility • Largest GOLD LEED independent scientific facility in Florida

4. Medical City at Lake Nona Nemours Children’s Hospital VA Medical Center UCF College of Medicine Sanford-Burnham University of Florida Research Center

5. Infectious & Inflammatory Disease Center Cancer Center Neuroscience, Aging and Stem Cell Center Diabetes & Obesity Center Sanford-Burnham Medical Research Institute Metabolism CV Pathobiology Translational Research Sanford Child Health Center

6. Florida Campus Focus: High-Impact,Disease-Oriented Research Early drug discovery • Pharmaceutical experience • Biotech business development expertise • Diagnostic biomarker discovery • Genomics • Metabolomics Metabolic origins of: Diabetes Obesity Heart and vascular diseases Cancer

7. Why Obesity and Diabetes? • Obesity contributes to a variety of chronic diseases • Obesity is driving a dramatic expansion of Type 2 diabetes – major cause of death in U.S. • Spreading to pediatric age groups: Super-sized kids may have shorter life expectancy than parents

8. Obesity Trends Among U.S. Adults 1990 1999 2009 No Data <10% 10%–14% 15%–19% 20%–24% 25%–29% ≥30%

9. Diabetes Cancer CV Disease Stroke National Center for Health Statistics, 1998 Diabetes is a Lethal Disease • Cardiovascular disease is the leading cause of death • The prognosis is grim for diabetics following heart attack • Cardiovascular disease in diabetics is unique, and refractory to current Rx

10. Diabetic CV Disease Our Research Pursuits: Interdisciplinary, Collaborative, Disease-oriented Cardiovascular Disease Obesity Diabetes

11. Accomplishments to Date • 2/3 of the way towards reaching our faculty recruitment goal of 29 • Over half way towards meeting our 2016 employment goal of 300 positions • Created high-value jobs, paying 130 percent average private sector wage in Orange County

12. Sanford-Burnham at Lake Nona Faculty: Recruitment by Site of Origin Pratley U Vermont F. Rastinejad S. Khorasanizadeh Univ. of Virginia Kelly Han Wash. Univ. Roth Abbott Ayala Vanderbilt Jiang U Mass. Wood Univ. of Alabama Gulick MGH Osborne UC Irvine Gardell Wyeth Collins Hamner Altarejos Salk Perera Savannah Sikder UTSW S. Smith Pennington L. Smith Vanderbilt; Scripps

13. Received over $52 million in research grants Published over 130 scientific papers Written 155 contracts (MTAs, CDAs, RCAs, etc.) Completed 13 patents and 21 invention disclosures (potential patents) Brought multiple pharmaceutical companies to Orlando to consider partnerships Accomplishments to Date

14. We are Diversifying Our Revenue Streams • The percentage of total research dollars from outside of Florida has risen from 40% to 50% in one year ( 2010 – 2011) • Launched commercial research partnership with Takeda Co. and Florida Hospital, bringing revenue and attracting international industry attention to region • Sanford-Burnham has successfully secured commercial research funding with Thermo Fisher, Eli Lilly and Novo Nordisk…others in negotiation

15. Florida Translational Research Fund State of Florida approved recurring $3MM to fund collaborative research with universities and non-profit institutes throughout Florida The program provides scientists statewide with access to Sanford-Burnham’s state-of-the-art technologies for drug discovery Leverages the government’s (state and local) prior investment  

16. State-of-the-Art Technologies Enable Research and Drug Discovery • Robots screen a “library” of compounds to discover small chemical structures that are the leads for the drugs of tomorrow • 1 of 4 NIH Comprehensive Screening Centers in U.S. • Leverage technology to attract additional grants In 2011, scientists successfully synthesized more than 500 new chemical compounds and identified 12 novel probes (early drug candidates).

17. Contributing to Local Growth and Job Creation Sanford-Burnham’s presence in Lake Nona and desire to collaborate helps area institutions attract top doctors and medical researchers “Our location in Lake Nona’s Medical City and proximity to Sanford-Burnham has been enormously helpful as Nemours recruits world-class physician-researchers.” Dr. Lane Donnelly, Chief Medical Officer, Nemours Children’s Hospital “Sanford-Burnham was a catalyst in the formation of the Translational Research Institute at Florida Hospital.” Lars Houmann, President and CEO, Florida Hospital

18. Growth of State of Florida Biotech Industry • Florida’s biotechnology industry has grown by 42 percent over the past five years • Nearly 200 biotechnology companies operate in Florida • 65 percent increase in life science venture dollars in Q1 2012 over Q4 2011 Florida researchers were awarded 2.3% of NIH money in 2012, up from 1.5% in 2006

19. Training Tomorrow’s Scientists in Orange County Inspiring a passion for science: Rigorous postdoctoral training United Negro College Fund – Sanford-Burnham four year scholarship Contribute to science curriculum in local schools Lab clinics for teachers Career days for college and high school students Internships and summer camps

20. Bridging the Translational Divide Discovery Improving Patient Care

21. Towards Personalized Medicine

22. Clinical Partnerships Test Therapeutic Potential of Discoveries Steven R. Smith, M.D. Bench to Bedside Clinical Research Basic Research Bedside to Bench

23. Translational Research Institute for Metabolism and Diabetes (TRI) Florida Hospital/Sanford-Burnham Research Discovery and Validation in Humans First-in-human trials

24. The BRAIN-FAT Connection! Orexin Dr. Dev Sikder The HEART-FAT Connection! ANP Dr. Sheila Collins

25. Emerging Translational Research Partnerships Pharma Academic Centers Regional Health Systems UCF VAMC Nemours Duke UF Wash U Translational Research Institute (Florida Hospital) Sanford-Burnham Sanford-Burnham Sanford-Burnham

26. Collaborative Regional Partnerships Advance Science Joint Research Sharing Technologies  Teaching and Training

27. Sanford-Burnham wishes to express special thanks to Orange County for your support and partnership

28. Sources of Sanford-Burnham Grant Revenue

29. Grant Revenue by Fiscal Year

30. Proposals/Awards

31. Recruitment Progress and Timeline for SBMRI - Fla Sanford gift: renamed to Sanford-Burnham Medical Research Institute at Lake Nona (1/10) Burnham Lake Nona Opens temporary labs at FBC, Orlando Burnham announces plans to expand to Orlando, FL New facility opens 4/09 1 2 3 4 5 6 8 9 11 13 15 17 18 19 2006 2007 2008 2009 2011 2010 Cum. Faculty numbers: Goal 28-30 (2016) Dan Kelly assumes Scientific Director Position; DOC established (7/08) One contract not renewed