An Overview of the NCI SBIR Program Michael Weingarten Director, NCI SBIR Development Center April 14, 2008

Today’s Presentation Overview Eligibility Requirements Move to More Focused Solicitations New SBIR Bridge Award Submitting An Application SBIR Development Center

Overview

Charge from the NIH Director October 2006-- request from Dr. Zerhouni to Dr. Niederhuber for NCI to lead an effort to examine the $650 million NIH SBIR Program with a target of enhancing the program’s outcomes NCI’s Response December 2006-- Dr. Niederhuber presented recommendations to Dr. Zerhouni Presentations followed to the NIH Steering Committee and Institute Directors Dr. Zerhouni asked that all recommendations be implemented on a pilot basis at NCI with interested Institutes to work with NCI Since February 2007, NCI has been working to implement the recommendations 4

Recommendations Specific Recommendations Being Implemented Establish SBIR Development Centers Focus Solicitations on Commercially Viable Technologies Co-invest With the Private Sector to Bridge SBIR Projects Toward Commercialization 5

Why are SBIR and STTR Important? NIH’s primary resource for enabling commercialization of innovative high impact technologies, such as: Research tools Medical devices Therapeutics Provides incentive to academic investigators to translate technology (new company formation) One of the largest sources of early-stage life sciences financing 6

Reasons to Seek SBIR & STTR Funding Provides seed funding for innovative technology development projects Intellectual property rights are retained by the small business concern Not a loan – no repayment is required Doesn’t impact stock or shares in any way (no dilution of capital) Provides recognition, verification and visibility Can be a leveraging tool to attract other funding (VC, etc.)

SBIR & STTR: Three-Phase Program PHASE I – R41, R43 Feasibility Study $100K and 6-month (SBIR) * or 12-month (STTR) Award PHASE II – R42, R44 Full Research/R&D $750K and 2-year Award (SBIR & STTR) * Commercialization plan required PHASE III Commercialization Stage Use of non-SBIR/STTR Funds * Note: Actual funding levels may differ by topic.

A $100M Program at the NCI 2.5% 0.3% Program Descriptions Set Aside SBIR: Set-aside Program for Small Business Concerns to engage in Federal R&D with potential for commercialization STTR: Set-aside Program to facilitate Cooperative R&D between Small Business Concerns and U.S. Research Institutions with potential for commercialization 2.5% 0.3% A $100M Program at the NCI

Eligibility Requirements

SBIR Eligibility Requirements Small Business Concern Organized for-profit U.S. business 500 or fewer employees, including affiliates Must be: 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 Principal Investigator’s primary employment must be with the Small Business Concern at the time of award and for the duration of the project period

STTR Eligibility Requirements 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 Other non-profit research organization Federal R&D center Intellectual Property Agreement Allocation of IP rights and rights to carry out follow-on R&D and commercialization Principal Investigator’s primary employment may be with either the Small Business Concern or the research institution

Award always made to small business SBIR and STTR Programs (Critical Differences) SBIR Permits research institution partners (e.g., universities) Small business concern may outsource ~33% of Phase I activities and 50% of Phase II activities STTR Requires research institution partners (e.g., universities) 40% of the work should be conducted by the small business concern (for profit) and 30% by a U.S. research institution (non-profit) Award always made to small business

NCI SBIR Funding Opportunities

For more information visit: NIH Issues Multiple SBIR Solicitations SBIR/STTR Omnibus Grant Solicitation Release: January Receipt Dates: April 5, August 5, and December 5 SBIR Contract Solicitation (NIH, CDC) Release: August Receipt Date: Early November NIH Guide for Grants and Contracts Release: Weekly Receipt Dates: Various For more information visit: http://sbir.cancer.gov

NCI is Moving to More Focused Solicitations Goal is to improve success in commercialization by focusing on more directed research. Invest in the technology priorities of NCI that also have potential for commercialization Catalyze targeted technology development and draw private sector investment in areas such as drug development and assays that measure treatment response Significantly increase the use of SBIR contracts.

Examples of NCI SBIR Technology Priorities Nanotechnology Imaging and Sensing Platforms for Improved Diagnosis of Cancer To support the development of nano-enabled platforms for early detection and/or imaging, or for post-treatment monitoring to detect recurrence of disease Goal is to enhance clinical diagnosis of cancer Development of Anti-Cancer Agents To support the investigation of candidate therapeutic agents to establish the rationale for continued development to the point of filing an IND Compounds may be chosen from a list provided by NCI or by the small business Antibody Array for Cancer To support the development of high-throughput antibody arrays for the quantitative analysis of multiple biomarkers Goal is to provide new tools for the early detection and diagnosis of cancer Biopsy Instruments and Devices that Preserve Molecular Profiles in Tumors To support the development of devices with potential for stabilizing the molecular profile of cancerous lesions in visceral tissue sites during clinical biopsy procedures To improve the accuracy of biomarker assessment for diagnosis and prognosis

Request for Information We will be announcing a Request for Information in a couple of weeks. Opportunity to put forward your ideas on topics that the NCI should solicit. Please address why the topic is important in advancing the science in cancer research. Critical to also address the commercial potential and market opportunities in the area.

More Information on NCI SBIR & STTR Website http://sbir.cancer.gov

New SBIR Bridge Award

Phase II SBIR and Commercialization Success Significant resources are required for getting through the FDA approval process This funding gap is known as the “Valley of Death” Today, many awardees complete the SBIR Phase II award without advancing the technology far enough to attract private investment

New SBIR Bridge Award Strategy: Accelerate projects to commercialization by: Filling the funding gaps that currently exist Sharing in the investment risk and incentivizing private investors to fund earlier stage projects Expectation that the SBIR company will raise raise matching funds from the investment community or industry partners Opportunity to leverage external resources Leverage private investor’s due diligence process Modeled after NSF’s “Phase IIB Option” program In FY 06, NSF invested $18M in this program and raised $58M in matching funds from the investment community

Example: How the Bridge Award Would Apply in the Area of Drug Development Target Identification & Validation Preclinical Development (Lead Development, Animal Studies, File IND) Safety Review Clinical Trials NDA Review Commercialization Phase I & Phase II SBIR Private Investment The “Valley of Death” is the problem

Example: How the Bridge Award Would Apply in the Area of Drug Development Target Identification & Validation Preclinical Development (Lead Development, Animal Studies, File IND) Safety Review Clinical Trials NDA Review Commercialization Phase I & Phase II SBIR SBIR Bridge Award Private Investment SBIR Bridge Award addresses the problem by bridging the “Valley of Death” SBIR Bridge Award allows NIH to share investment risk by incentivizing investors or strategic partners to evaluate projects and commit funds much earlier

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

Submitting an Application

Keys to a Strong Application Significant, innovative, and focused science Significant product and/or commercial potential A product-focused application is more likely to have support of business reviewers A project with sound financial projections is more likely to attract a partner Translational research/clinical applications projects should involve the appropriate collaborators Oncologists Pathologists Statisticians

Know NIH Review Criteria Does the study address an important problem and have commercial potential? Significance Are design and methods well-developed and appropriate? Are problem areas addressed? Approach Are there novel concepts or approaches? Are the aims original and innovative? Innovation Is the investigator appropriately trained and capable of managing the project? Investigator Does the scientific environment contribute to the probability of success? Is the environment unique? Environment Is the company’s business strategy one that has a high potential for success? Commercialization

Preliminary Data Solicitation states “Preliminary data are not required” Reviewers like to see preliminary data Preliminary data should support your proposal and the feasibility of the project Preliminary data may consist of your own publications and unpublished data from your laboratory

Electronic Submission Process (Grants Only) The PHS398 grant application form is being phased out and replaced with the SF424 Research and Related (R&R) application NIH has transitioned from paper submission of SBIR/STTR grant applications to electronic submission Company and company official must be registered in Grants.gov PI and company official must be registered in the eRA Commons http://era.nih.gov/ElectronicReceipt

Application and Award Process Solicitation Topics NCI describes topics in solicitation Proposal Submission About 6-9 months Small business concerns prepare short (usually 25-page) proposals Unsolicited proposals are not accepted Evaluation NCI evaluates proposals based on technical merit, applicant qualifications, and commercial potential/societal benefit Award NCI makes awards

Changes in Program Management

NCI Creating an SBIR Development Center to Optimally Manage the Program Today, SBIR program management is generally dispersed across NIH, with few full-time SBIR program managers For example, at NCI, SBIR awards are managed by ~40 people who each spend only a fraction of their time managing small business awards Few NIH SBIR program managers have significant industry or commercialization experience 33

NCI Creating an SBIR Development Center to Optimally Manage the Program Development Center Goals Assemble the scientific and business expertise needed to optimally manage the SBIR program at the NCI These will be full time dedicated SBIR management teams Integrate all SBIR initiatives with NCI’s program priorities Foster collaborations with other ICs at NIH which share common technology needs Enhance the return on investment for the SBIR program for the benefit of the cancer community and the public health in general 34

New NCI SBIR Development Center Center will offer a menu of services: Assess commercial potential of NIH technology priorities Write solicitation topics/post-solicitation activities Market program to attract the best companies Evaluate commercialization potential of proposals Provide awardee management & support More active monitoring of awards Program managers will have expertise and networks to mentor emerging SBIR companies in commercialization strategy and process 35

Employment Opportunities Positions to be announced soon looking for expertise in: Biomarkers and Diagnostic Assays Cancer Imaging Radiation Therapy Cancer Prevention Cancer Biology Cancer Control and Population Sciences To receive job announcement information, go to sbir.cancer.gov and click on “Sign up for Updates”

NCI SBIR Development Center NCI SBIR & STTR Programs http://sbir.cancer.gov Michael Weingarten Director NCI SBIR Development Center Phone: 301-496-4413 weingartenm@mail.nih.gov Andrew Kurtz, Ph.D. Program Manager NCI SBIR & STTR Programs Phone: 301-594-6846 kurtza@mail.nih.gov