NCI SBIR Program Overview The Montana Bioscience Alliance August 28, 2012 Patricia A Weber, DrPH

1. NCI SBIR Program Overview The Montana Bioscience Alliance August 28, 2012 Patricia A Weber, DrPH Program Director

3. Today’s Presentation • Program Overview & Eligibility • SBIR/STTR Reauthrization • General Funding Information • SBIR Phase II Bridge Award (NCI) • Managing NCI’s Small Business Portfolio • SBIR Development Center • New Funding Opportunities • Investor Forum 3

4. Congressionally Mandated Programs • SBIR:Set-aside program for small business concerns to engage in Federal R&D with the potential for commercialization Federal agencies with an extramural R&D budget > $100M • STTR:Set-aside program to facilitate cooperative R&D between small business concerns and U.S. research institutions with the potential for commercialization Federal agencies with an extramural R&D budget > $1B Set-Aside (FY12) 2.6% 0.35% 4

5. NIH = 27 Institutes & Centers23 Participate in the SBIR/STTR Program The Office of the Director (OD) National Institute on Aging (NIA) National Institute on Alcohol Abuse & Alcoholism (NIAAA) National Institute of Allergy & Infectious Diseases (NIAID) National Institute of Arthritis & Musculoskeletal & Skin Diseases (NIAMS) National Cancer Institute (NCI) National Institute of Child Health & Human Development (NICHD) National Institute on Deafness & Other Communication Disorders (NIDCD) National Institute of Dental and Craniofacial Research (NIDCR) National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK) National Institute on Drug Abuse (NIDA) National Institute of Environmental Health Sciences (NIEHS) National Eye Institute (NEI) National Institute of General Medical Sciences (NIGMS) National Heart, Lung, & Blood Institute (NHLBI) National Human Genome Research Institute (NHGRI) National Institute of Mental Health (NIMH) National Institute of Neurological Disorders & Stroke (NINDS) National Institute of Nursing Research (NINR) National Institute on Minority Health & Health Disparities (NIMHD) National Center for Complementary & Alternative Medicine (NCCAM) Fogarty International Center (FIC) National Center for Advancing Translational Sciences (NCATS) National Library of Medicine (NLM) National Institute of Biomedical Imaging & Bioengineering (NIBIB) NIH Clinical Center (CC) Center for Information Technology (CIT) Center for Scientific Review (CSR) No funding authority 5

6. Reasons to Seek SBIR/STTR Funding • Provides seed funding for innovative technology development • Provides recognition, verification and visibility • Helps provide leverage in attracting additional funding or support (e.g., venture capital, strategic partner) • Not a Loan •  No repayment is required •  Doesn’t impact stock or shares in any way (i.e. non-dilutive) • Intellectual property rights retained by the small business • Bayh-Dole Act (1980) 6

7. SBIR Eligibility • Applicant must be a Small Business Concern (SBC) • Organized for-profit U.S. business • 500 or fewer employees, including affiliates • PD/PI’s primary employment (i.e., >50%) must be with SBC at the time of award and for duration of the project period • At least 51% U.S.- owned by individuals and independently operated • OR At least 51% owned and controlled by another (one) business concern that is at least 51% owned and controlled by one or more individuals 7

8. STTR Eligibility • Applicant is a Small Business Concern • Formal Cooperative R&D Effort • Minimum 40% by small business • Minimum 30% by U.S. research institution • U.S. Research Institution: College or University; Non-profit research organization; Federally-Funded R&D Center (FFRDC) • Intellectual Property Agreement • Allocation SBC of IP rights (to SBC) and rights to carry out follow-on R&D and commercialization • Principal Investigator’sprimary employment may be with either the Small Business Concern or the research institution 8

9. Three-Phase Programs • PHASE I – R41, R43 • Feasibility Study • $150K, 6-months (SBIR) • $100K, 12-months (STTR) Fast-Track Application Combined Phase I & II • PHASE II – R42, R44 • Full Research/R&D • $1M (SBIR) / $750K (STTR), 2-years • Commercialization plan required • PHASE III • Commercialization Stage • Use of non-SBIR/STTR Funds 9 Note: Actual funding levels may differ by topic.

10. Congressional Reauthorization • SBIR/STTR programs were re-authorized through FY2017 by the 2012 Defense Authorization Act (P.L.112-81) • Increases SBIR set-aside (incrementally) to 3.2% by 2017 • FY2012 set-aside is 2.6% • Increases STTR set-aside to 0.45% by 2017 • FY2012 set-aside is 0.35% • Establishes hard caps on funding levels for Phase I & II awards • Allows companies majority owned by multiple VC firms, hedge funds, and private equity firms to compete for up to 25% of NIH SBIR funds • Allows ability to switch between the SBIR and STTR mechanisms 10

11. Public Comment Period Open Now SBA published SBIR & STTR policy directives August 6. Public Comment period open until October 5, 2012 Submit comments at http://regulations.gov RIN: 3245-AF84 (SBIR) RIN: 3245-AF45 (STTR) 11

12. Public Webinars Wednesday, August 29, 2012 2 pm EDT To attend, email technology@sba.gov with: • Webinar Date • Name • Title • Organization • Address • Phone • Email Address Send questions & comments to SBIRComments@sba.gov 12

13. SBIR/STTR Reauthorization Key Changes

14. Increase in Set-Aside

15. Hard Limits* on Award Sizes This will impact current NIH practices on awards. Comment on policy directives! *NIH will request waivers on limits for specific topics

16. Venture Capital Participation NIH will be allowed to spend up to 25% of SBIR funds on small businesses majority owned by multiple VCs, hedge funds, or private equity firms. Previously not allowed. In effect after Size Rules are finalized, expected 1/1/13. Cross-Program Awards STTR Phase I awardees can receive SBIR Phase II awards, and vice versa Cross-Agency Awards Phase I awardee may receive a Phase II award from a different agency Changes to Eligibility

17. Open Phase II competition Invitations for Phase II contract proposals will be open to all Phase I contract awardees. Second Phase II Award A sequential Phase II award may be given to continue a Phase II project. Collaborations with Federal Labs Waivers no longer required for partnership with Federal Labs and Centers Direct to Phase II pilot Phase II SBIR awards may be awarded without requiring Phase I award. Not yet clear what NIH implementation will be. Changes to Eligibility

18. Improving the SBIR/STTR Programs • Streamlining the Award Process • Working to shorten timelines from application to award decision. NIH given 1 year from solicitation close date. • Administrative Funding Pilot • 3% of SBIR funds for agencies to provide support to improve: • Outreach • Commercialization • Streamlining & Simplifying the Award Process What can we do to make the program better for you? Comment on Policy Directives & tell your Program Officers

19. Implementation Timeline

20. Sign up for NCI mailing list for updates http://sbir.cancer.gov NIH will soon have more info about NIH-specific implementation at http://sbir.nih.gov Public Comments on Policy Directives open until October 5, 2013 Get your voice heard! More Info

21. How to Find NIH and NCI Funding Opportunities

22. http://grants.nih.gov/grants/funding/sbir.htm 22

23. http://sbir.cancer.gov 23

24. Multiple Funding Solicitations • NIH SBIR/STTR Omnibus Solicitations for Grant Applications • Release: January • Receipt Dates: April 5, August 5, and December 5 • Solicitation of the NIH & CDC for SBIR Contract Proposals • Release: August 15, 2012 • Receipt Date: November 13, 2012 • RFP can be found at: • http://grants.nih.gov/grants/funding/SBIRContract/PHS2013-1.pdf • More info about NCI’s topic areas: • http://sbir.cancer.gov/funding/contracts/ 24

25. http://projectreporter.nih.gov/reporter.cfm 25

26. SBIR Success Rates(1998 – 2010) Source: NIH IMPAC, Success Rate FileContact: Division of Information Services, ORIS/OER 26

27. SBIR Success Rates(1998 – 2010) 27

28. FY 2013 SBIR Contract Solicitation

29. Targeted Funding Opportunities • Goal is to improve commercialization success by focusing on more directed research in targeted areas • Invest in the technology priorities of NCI that also have greatest potential for commercialization • Catalyze technology development and draw private sector investment in specific areas • 20% of NCI’s SBIR budget is now invested in contracts NCI has set aside $10M to support new Phase I contracts in 13 different topic areas in FY2013 29

30. SBIR Contracts vs. Grants: What's the difference? 30

31. FY13 NCI Contract Funding Topics • 313 RNAi Cancer Therapeutics using Nanotechnology • 314 Development of Human Tissue Culture Systems that Mimic the Tumor Microenvironment • 315 Development of Companion Diagnostics: Enabling Precision Medicine in Cancer Therapy • 316 Development of CTC Isolation Technologies Enabling Downstream Single Cell Molecular Analysis • 317 Wound Healing Preparations Incorporating Nitric Oxide-Releasing Materials • 318 Test to Predict Effectiveness of Docetaxel Treatment for Prostate Cancer • 319 Technology to Generate Anti-Peptide Capture Reagents for Affinity-Enriched Proteomic Studies • 320 High Quality Cancer-Related Standards for Metabolomics Research • 321 Chemically Defined Glycan Libraries for Reference Standards and Glycomics Research (Joint NCI-NIGMS Program) • 322 Real-Time Integration of Sensor and Self-Report Data for Clinical and Research Applications • 323 Development of Radiation Modulators for Use During Radiotherapy • 324 Novel Imaging Agents to Expand the Clinical Toolkit for Cancer Diagnosis, Staging, and Treatment • 325 Innovative Radiation Sources for Advanced Radiotherapy Equipment 31

32. FY13 NCI Contract Funding Topics Therapeutics & Diagnostics • 313 RNAi Cancer Therapeutics using Nanotechnology • 315 Development of Companion Diagnostics: Enabling Precision Medicine in Cancer Therapy • 316 Development of CTC Isolation Technologies Enabling Downstream Single Cell Molecular Analysis Advancing Cancer Research • 314 Development of Human Tissue Culture Systems that Mimic the Tumor Microenvironment • 319 Technology to Generate Anti-Peptide Capture Reagents for Affinity-Enriched Proteomic Studies • 320 High Quality Cancer-Related Standards for Metabolomics Research • 321 Chemically Defined Glycan Libraries for Reference Standards and Glycomics Research (Joint NCI-NIGMS Program) 32

33. FY13 NCI Contract Funding Topics (cont’d) Imaging & Radiation Therapy • 323 Development of Radiation Modulators for Use During Radiotherapy • 324 Novel Imaging Agents to Expand the Clinical Toolkit for Cancer Diagnosis, Staging, and Treatment • 325 Innovative Radiation Sources for Advanced Radiotherapy Equipment Health IT • 322 Real-Time Integration of Sensor and Self-Report Data for Clinical and Research Applications NIH Technology Transfer • 317 Wound Healing Preparations Incorporating Nitric Oxide-Releasing Materials • 318 Test to Predict Effectiveness of Docetaxel Treatment for Prostate Cancer 33

34. Topic 314: Development of Human Tissue Culture Systems that Mimic the Tumor Microenvironment • Budget: Phase I $300,000; Phase II $2M • Number of Anticipated Awards: 3 - 5 • Goal: Development of 3D human tissue model culture systems that accurately mimic the tumor microenvironment…validated against known effective anti-cancer agents • Phase I Activities & Deliverables Include: • Develop 3D culture system prototype that incorporates human tumor cells using or easily adapted for use with high content screening platforms • Demonstrate accurate prediction of clinical efficacy in the developed prototype benchmarked against 2D and currently available 3D systems • Phase II Activities & Deliverables Include: • Benchmark performance against known in vivoeffects • Demonstrate ability to scale-up system 34

35. Topic 317: Wound Healing Preparations Incorporating Nitric Oxide-Releasing Materials (NIH Technology Transfer) • Budget: Phase I $200,000; Phase II $1.5M • Number of Anticipated Awards: 1 • Fast-Track proposals not accepted • Goal: Develop a wound-healing dressing using NCI-developed NO-releasing material technology. • *Contractor will be granted royalty-free, non-exclusive license but is encouraged to submit an application for a commercialization license to NIH OTT • Phase I Activities & Deliverables Include: • Prototype development • Material characterization • Proof of concept in vitro studies • In vivo efficacy studies • Phase II Activities & Deliverables Include: • Stability studies • Capacity for commercial production & manufacture 35

36. Topic 322: Real-Time Integration of Sensor and Self-Report Data for Clinical and Research Applications • Budget: Phase I $200,000; Phase II $1M • Number of Anticipated Awards: 2 – 3 • Goal: Secure, privacy-compliant mobile applications and paired analytic systems to control the collection, transfer, integration, analysis, and reporting of objective and self-reported health-related measures. • Phase I Activities & Deliverables Include: • Establish project team with broad expertise • Prototype including front-end mobile application(s), integration with sensors, and back-end user-interface controls for data integration • Phase II Activities & Deliverables Include: • Beta-test and finalize: • Front-end mobile application • File transfer, screening, data importation protocols and systems • Data integration and visualization tools • User-interface systems • Usability testing 36

37. Questions About Contracts? Ms. Anita Hughes Anita.Hughes@nih.gov 301.435.3805 http://sbir.cancer.gov/funding/contracts/ 37

38. More Information on NCI SBIR & STTR Website Application Deadline: November 13, 2012 http://sbir.cancer.gov

39. Innovative Health IT for Broad Adoption by Healthcare Systems & Consumers (PA-12-196) Goal: Accelerate development & commercialization of evidence-based consumer health IT to: • Prevent or reduce the risk of cancer • Facilitate patient-provider communication • Improve disease outcomes in consumer & clinical settings • Phase II or Fast-Track applications only • Strong applicants will have a partnership with large business (e.g. commercial IT firm, EMR vendor, healthcare systems, etc.) Next receipt date December 5, 2012 http://sbir.cancer.gov/resource/hit/ Contact Dr. Patricia Weber, weberpa@mail.nih.gov

40. Innovative Health IT for Broad Adoption by Healthcare Systems & Consumers (PA-12-196) • NCI is responding to the goals of the IOM and ONC by publishing a funding opportunity that encourages small businesses to partner with larger businesses or health organizations to develop user-centered health IT products. • Does not promote the development of healthcare delivery systems that would compete with the over arching infrastructure for EMRs being developed by the ONC. • Instead the focus is on patient-centered, evidence-based health IT tools that are interoperable with existing EMRs • Overall goal is to engage health providers and consumers in the prevention and management of chronic diseases

41. NCI SBIR Phase II Bridge Award 41

42. Three-Phase Programs Phase II Bridge Award • PHASE I – R41, R43 • Feasibility Study • $150K, 6-months (SBIR) • $100K, 12-months (STTR) • PHASE II – R42, R44 • Full Research/R&D • $1M (SBIR) / $750K (STTR), 2-years • Commercialization plan required • PHASE III • Commercialization Stage • Use of non-SBIR/STTR Funds 42 Note: Actual funding levels may differ by topic.

43. Phase II Bridge Award • Follow-on to the SBIR Phase II Award • Goal is to extend SBIR Phase II awards to help cross the “Valley of Death” • Provides up to $3M in additional SBIR funding over 3 years • Strategy is to help early-stage projects by: • Incentivizing partnerships between SBIR grantees and third-party investors • Giving competitive preference and funding priority to applicants that can raise substantial third-party funds (i.e., minimum 1:1 match) • Benefits • Affords NCI the opportunity to leverage millions in external resources • Provides NCI added confidence based on due diligence by outside investors • Provides the small business with commercialization guidance during the award • Ideally, the small business will establish a strong relationship with an investor or strategic partner that will provide additional financing beyond the Bridge Award 43

44. EXAMPLE: Drug Development The “Valley of Death” is the problem Preclinical Development (Lead Development, Animal Studies, File IND) Target Identification & Validation Safety Review (IND) Clinical Trials NDA Review Commercialization Phase I & Phase II SBIR Private Investment / Strategic Partner SBIR Bridge Award SBIR Bridge Award addresses the problem by bridging the “Valley of Death” 44

45. EXAMPLE: Drug Development Preclinical Development (Lead Development, Animal Studies, File IND) Target Identification & Validation Safety Review (IND) Clinical Trials NDA Review Commercialization Phase I & Phase II SBIR Private Investment / Strategic Partner SBIR Bridge Award SBIR Bridge Awardallows NIH to share investment risk by incentivizing Private Investors to evaluate projects and commit funds much earlier 45

46. EXAMPLE: Drug Development Phase I & Phase II SBIR SBIR Bridge Award STOP STOP 2nd Year 1/3 of funds 1st Year 1/3 of funds 3rd Year 1/3 of funds YES YES • Milestones reached? • Matching Funds? • Milestones reached? • Matching Funds? • Milestones reached? • Matching Funds? NO NO Preclinical Development (Lead Development, Animal Studies, File IND) Target Identification & Validation Safety Review Clinical Trials NDA Review Commercialization SBIR Bridge Award Private Investment 46

47. 12 Bridge Awards (to date) 3 therapeutics 6 imaging technologies 3 molecular diagnostics http://projectreporter.nih.gov/reporter.cfm 47 47

48. 12 Bridge Awards (to date) Venture Capital: 1/3 Strategic Partners: 1/3 Individuals & Other: 1/3 48

49. New Approach for Managing SBIR at NCI 49

50. Managing NCI’s SBIR/STTR Portfolio Old Management Model • Awards were managed by 40-50 program staff from across the NCI who each spent a small percentage of their time on SBIR • Few of these program directors had significant industry experience or commercialization expertise New SBIR Development Center • 10-member management team exclusively focused on the administration of NCI’s SBIR/STTR portfolio • Center staffed by program directors with industry experience and a broad range of scientific expertise • Center collaborates with staff from across other NCI divisions to integrate the small business initiatives with the Institute’s priorities 50