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Juvenile Diabetes Research Foundation (JDRF) www.jdrf.org

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JDRF: Mission and General Strategy To find a cure for diabetes and its complications Proactively accelerate the discovery , developmen t, and delivery of disease-modifying drugs and devices to better treat and cure diabetes and its complications

3.
Diabetes Market Landscape <ul><li>Cost of diabetes in US (2008) = $218 billon (direct and indirect costs: medical expenditures & lost productivity) </li></ul><ul><li>Diabetes Market (drugs & devices - worldwide sales, 2008): </li></ul><ul><ul><li>Insulin Products $9 billon </li></ul></ul><ul><ul><li>Drugs $8 billon </li></ul></ul><ul><ul><li>Blood Glucose Monitoring $7 billon </li></ul></ul><ul><ul><li>Insulin Pumps $1.5 billon </li></ul></ul><ul><li>Unmet medical need: </li></ul><ul><ul><li>Help patients control glucose, stay healthy, and prevent complications </li></ul></ul><ul><ul><li>Develop new therapies and improve efficacy of existing therapies </li></ul></ul><ul><ul><li>Reduce overall cost of diabetes care and impact on the healthcare system </li></ul></ul>

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JDRF: Support <ul><li>Patient-centric organization: Cure, Treat, Prevent </li></ul><ul><li>Over past 39 yrs, JDRF funded >$1B of research; FY10 research budget of $115M </li></ul><ul><li>Funding potentially transformative early stage research, translational research, and clinical research in academia and industry worldwide </li></ul><ul><li>Support also includes advocacy and addressing regulatory, reimbursement, adoption issues, etc. </li></ul><ul><li>Partnering is critical for its success </li></ul>

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JDRF: Partners JDRF Venture Capital Industry People with Type 1 Diabetes Providers, Educators Academia Funders: NIH, Government, Foundations FDA Payers JDRF

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JDRF: Support <ul><li>Type 1 diabetes with type 2 diabetes impact </li></ul><ul><li>All stages: At-risk, Recent onset, Established diabetes </li></ul>

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JDRF: Diabetes Stage-Specific Focus Treat Prevent Prevent Treat Cure Established Diabetes Recent Onset Diabetes At-Risk

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JDRF: Support <ul><li>Type 1 diabetes with type 2 diabetes impact </li></ul><ul><li>All stages: At-risk, Recent onset, Established diabetes </li></ul><ul><li>Highest Funding Priorities </li></ul><ul><ul><li>Beta Cell Therapies: Beta Cell Regeneration and Reprogramming </li></ul></ul><ul><ul><li>Immune Therapies: Antigen-Specific Immunotherapy and Vaccines </li></ul></ul><ul><ul><li>Glucose Control: Closed Loop Artificial Pancreas Device, Glucose-responsive Insulin </li></ul></ul><ul><ul><li>Complication Therapies: Complication protective therapies </li></ul></ul><ul><li>Proactively managed pipeline </li></ul>

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JDRF: R&D Strategy JDRF’s Role: To advance IP and product development by “de-risking”, increasing value, and filling gaps Patients <ul><li>JDRF acts as a catalyst, driver, funder, advocate </li></ul><ul><li>Partnering and handing off with “Line of Sight” to the Patient </li></ul>Clinical Development Translational Research Basic Research

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JDRF: Industry Partners- 1 EU FP Funding (through Phase I ) Immune Therapies Beta Cell Therapies

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JDRF: Industry Partners- 2 Glucose Control Complication Therapies

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GNF-JDRF Partnership: Filling a Critical Gap Deliver novel IND candidates for out-licensing or co-development with Novartis or other pharma  cell survival -Housey -GNF <ul><li>. </li></ul><ul><li>GNF </li></ul> cell survival -Kafman -Nadler -Others <ul><li>-Stoffel </li></ul><ul><li>Kim </li></ul><ul><li>Newgard </li></ul><ul><li>-Schreiber </li></ul><ul><li>-Dor </li></ul>Reprogram. -Hecksher- Sorensen -Schreiber XOMA-052 -Survival Reprogram -Herrera -Ferber -Kay/Grompe -Bouwens -RFA pending Progenitor cells -Sander/ Cirulli -Ferrer -Bonner-Weir - Bouwens Cell Target Path- way Target ID Lead ID Lead Develop Lead Optim EIH Enable Clinical Proof of concept <ul><li>Kim/Crabtree </li></ul><ul><li>Dor </li></ul><ul><li>Bhushan </li></ul><ul><li>Vale </li></ul><ul><li>Habener </li></ul><ul><li>Others </li></ul> cell survival -Johnson -Zick-Others PPI/DPPIVi -Rabinovitch Gastrin therapy -Transition Leading Pharma Partners Leading Academic Partners

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JDRF: Organizational Leadership <ul><li>Alan Lewis, PhD: President, CEO (alewis@jdrf.org) </li></ul><ul><li>Richard Insel, MD: EVP Research, CSO (rinsel@jdrf.org) </li></ul><ul><li>Robert Goldstein, MD, PhD: SVP, Scientific Affairs (rgoldstein@jdrf.org) </li></ul><ul><li>Karin Hehenberger, MD, PhD: SVP, Strategic Alliances (khehenberger@jdrf.org) </li></ul>

14.
JDRF: Summary/Key Points <ul><li>Focus on: </li></ul><ul><ul><li>Patients: Cure, Treat, Prevent </li></ul></ul><ul><ul><li>Science: Beta Cells, Immune Therapies, Glucose Control, Complications </li></ul></ul><ul><ul><li>IP: Create IP and advance its development </li></ul></ul><ul><li>Execute through </li></ul><ul><ul><li>Funding: Academia, Industry </li></ul></ul><ul><ul><li>Outreach/Advocay: Patients, NIH, FDA, Insurance Industry, Providers </li></ul></ul><ul><ul><li>Partnering: Industry, Foundations, VC </li></ul></ul>

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Q&A