Academic Public Private Partnership Program (AP4)

1. Academic Public Private Partnership Program (AP4) Dr. Edward Sausville Associate Director Developmental Therapeutics Program Division of Cancer Treatment & Diagnosis National Cancer Institute AACR Annual Meeting July 11, 2003 Washington, DC http://dtp.nci.nih.gov Exhibit Booth #342-344

2. Purpose • To stimulate cancer intervention discovery and development at academic/non-profit centers in partnership with other academics, industry, non-profits and government. • To incorporate the latest technologies to find novel, mechanistically targeted drugs and other intervention strategies for underserved diseases • To combine the necessary expertise to reduce the time required to translate novel interventions to the clinic.

3. Impetus • The need for a new intervention discovery and development assistance mechanism was promoted by several Progress Review Groups. • New discovery paradigm (combichem, HTS, genomics,) has to date not lead to an increase in rate of NDAs when practiced by pharma/biotech. • Hypothesis: academic/non-profit centers add value to this effort if appropriately partnered.

4. AP4 Concept Development • Modeled after Industrial/University Cooperative Research Centers (I/UCRC) at National Science Foundation (NSF) which stimulates industrial-academic partnerships • I/UCRC created 25+ years ago: FY ’00 - NSF funds of $5.2 M generated $68M budget supporting 1,750 faculty and students at 50 centers.

5. NCI Study of I/UCRC • Could the I/UCRC concept be adapted for NCI use? Input from: • NCI Office of Scientific Planning and Assessment • NIH Office of the General Counsel • NCI Technology Transfer Branch • NCI-CTEP • Members of DTP Grants and Contracts Operations Branch • Members of PRGs

6. AP4 Primary Features • Academic or Non-profit Director • Each Center can have many contributing “partners” - academics, pharma, biotechs, non-profits, gov’ts • All research is carried out at the Center • All partners agree to a membership agreement • Steering Committee (SC) - AP4 Director, partners and NCI; SC determines $$ to Center projects • Focus is preclinical discovery & early development of interventions with access to NCI development and clinical resources

7. AP4 – Proposed Grain Size ~15 Planning Grants for $ 50K direct costs each ~6 Partnerships of $450K to $600K direct costs each based on: $300K total partner fees = $450K/yr (direct) NCI $450K total partner fees = $600K/yr (direct) NCI

8. AP4 – Connections AP4 Director (College/Non-profit) Funding Funding Partner 1 Voting Member & Coordinator Evaluations Voting Members Advice (Non- Voting) NCI Program Director Steering Committee Partner 2 Requests For Support/ Progress Reports Review & Approvals Partner 3 Project 1 Project 2 Project 3

9. NCI Center $$ Support Structure • Year 1: $ 450,000 (100%) • Year 2: $ 450,000 (100%) • Year 3: $ 450,000 (100%) • Year 4: $ 337,500 (75% funding) • Year 5: $ 225,000 (50% funding) (To encourage increased membership)

10. AP4 Director • AP4 Director will: • Use a one year planning grant to identify potential partners; hold a meeting and formulate a partnership which benefits all parties • Administer all aspects of subsequently approved 5-year funded partnerships. • Organize partnership meetings and projects. • Provide yearly partnership evaluations to NCI through an appointed evaluator.

11. Membership Agreement • Agreement establishes a framework for the relationship between all partners • All partners must agree to terms of the agreement which spells out: • IP issues • Membership fees: contribution of each member • Creation of the Steering Committee • Publication rights • Evaluation metrics

12. Possible Center Evaluation Metrics • # of Cancer-relevant target(s) characterized • Establishment of high throughput, target-based screens • Method of analysis of data from HTS successfully utilized to identify true hits. • Communication between and contribution of all partners to the Center • Number of patents, INDs filed, clinical trials supported • Number of licenses for new IP • Number of NDAs.

13. Intellectual Property • IP issues should be resolved during planning grant period • How IP is handled may vary from group to group • Bayh-Dole may be modified by consent of Center members • NCI should advise, but not mandate how IP will be handled by each Center

14. Grant Mechanism • Cooperative Agreement: • Substantial NCI scientific and/or programmatic involvement with the awardee. • NCI's role is to support and/or stimulate the recipient's activity by working with the award recipient. . . , but it is not to assume direction, prime responsibility, or a dominant role in the activity.

15. AP4 Unique Features • Dynamic management: SC will make go/no go decisions on projects; direct authority to add new projects and shift funds – a fundamental difference from traditional grant arrangements. • Priority access to DTP development resources for SC approved projects (must meet DCTD Drug Development Group criteria). • AP4 is a test case for NCI – a partnership between academics, industry, non-profits and the gov’t. • Mingling of funds from gov’t. and partners will lessen gov’t. financial responsibility.

16. Attraction for Partners • NCI $ support for high risk research of interest to the cancer research community • Establishes long-term relationship between academia, industry, and non-profits • Each partnership determines its own ground rules – defines beneficial interdisciplinary membership • Can develop students knowledgeable in drug discovery and development-relevant research

17. AP4 Initiation Sequence • NCI - Announcement of program • Proposed AP4 Director – may submit LOI • Proposed AP4 Director - submits application for planning grant • NCI - peer-review of planning grant • Proposed AP4 Director – uses planning grant to find potential partners and form partnership • (Evidence of planning grant success is a submitted Center proposal) • NCI – peer review of Center proposals

18. AP4 – Anticipated Time Line • 2003 • February/March - Presentation to EC and BSA • July – Present program to the AACR • July - Appears in NIH guide • November - Receipt of planning grant applications • 2004 • February - Review of planning grant applications • June - Planning grant awards • 2005 • June - Receipt of Center applications • September - Review of Center applications • November - NCAB Funding approval • January ‘06 – Awards made

19. AP4 Conclusion • What is proposed – AP4 Centers will be Cooperative Agreements based at academic or non-profit institutions; membership will be comprised of academics, industry, government and non-profits. • Why - to discover new interventions translatable into therapies, recent NDA filings tell us technology alone is not enough. It is essential that multi-disciplinary and multi-institutional talents be partnered in this effort. • Uniqueness – focus on partnering, new technologies, and underserved diseases, with Gov’t and members sharing costs; SC vested with the power to make immediate go/no go decisions.