SBIR R&D Contract Opportunities at NIH Xing-Jian Lou, PhD Program Director NCI SBIR Development Center Thursday October 23, 2014 NIH SBIR Conference

Today’s Presentation Why Does NIH Use SBIR Contracts? How Contract Topics Get Chosen Different between Contracts and Grants The Proposal, Review and Funding Process Contract Topic Example Questions

Why Does NIH Use SBIR Contracts? SBIR contracts allow an institute to direct companies to develop products in areas of high priority (usually narrow technical focus) Institute-run ad hoc peer review panels where there is balanced representation of business versus technical reviewers Program staff are very involved in funding decisions Deliverables negotiated by program staff in advance of award- once in the contract, these are “must do” items Quantitative milestones Program staff (project officer/contracting officer’s representative) are also very involved in management of funded projects

Administrative Supplements NIH Technical Assistance Programs NCI SBIR/STTR Award Budget Distribution Administrative Supplements NIH Technical Assistance Programs Contracts Grants

NCI scientific & technology NCI SBIR Targeted Contract Solicitations NCI scientific & technology priorities Areas of interest to the commercial sector, based on market opportunity Contract topics in NCI priority areas with strong potential for commercial success NCI SBIR contracts (thousands) % of total NCI SBIR $38,174 $37,406 $25,020 $26,102 $16,665 35% $12,387 33% 25% 25% 24% $7,757 17% 13% 13% 17% 8% 8% Fiscal Year

How Contract Topics Get Chosen? SPL Approval Submit the Topics TAG Review Publish the Contract Topics Development of SBIR Contract Topics Concept Development Trans-NCI Technology Advisory Group (TAG), with membership from all Divisions and offices whose missions overlap with SBIR TAG Committee reviews, discusses, and scores solicitation proposals (contracts, grants, and cooperative agreements) SBIR office prepares package for NCI Scientific Program Leaders Committee (Executive Committee) 10-25 NCI SBIR contract topics are approved for publication in Omnibus NIH SBIR Contract RFP published in August Feb Apr Jun Aug Jan Call for Contract topics

Different between Contracts and Grants Funding Solicitations for SBIR Grants Funding Solicitation for SBIR Contracts Scope of the proposal Investigator-defined within the mission of NIH Defined by the NIH (focused) Set-aside of funds for particular areas? NO YES Questions during solicitation period? May speak with any Program Officer MUST contact the contracting officer Receipt Dates 3 times/year for Omnibus Only ONCE per year Basis for Award Based on score during peer review If proposal scores well during peer review, must then negotiate to finalize deliverables with NIH Reporting One final report (Phase I); Annual reports (Phase II) Monthly or quarterly progress reports The decision point is whether the contract topic is a true fit for your project 7

NIH SBIR Contracts Request For Proposals (RFP) Both for NIH & CDC Issued once a year Released late summer Proposals usually due in early November Includes: Proposal preparation instructions Page Limits – All inclusive -50 for Phase I; 150 for Phase II Definitions Evaluation Criteria Institute Points of Contact Research Topics Additional Guidance (for example Human Subjects) Forms (with links)

Proposal and Funding Process (Phase I) Decide to Apply Build the application Submit the application Receive the Review Results Negotiation Receive the Award YES Must be received by 11/5/14, 4:30 pm ET NO Request a Debrief within 3 days NIH SBIR RFP published in August PO is barred from communicating with potential offerors Proposals due November NCI ad hoc technical review panels meet Jan-April (1- 55 proposals; end products are score 0-1,000, plus technical acceptability judgment, plus technical minutes document) PO recommends competitive range in memo to CO May-June Negotiations to address technical and budgetary weaknesses Self-certification of SBIR eligibility (company ownership structure) Award in September (1-10 contracts per topic)

Proposal and Funding Process (Phase II) Review Phase I final report (technical and commercial potential)- June, rolling basis Invitation Letter RFP goes out to company- Nov-Feb Phase II proposal (2 years, < $2M) due- Jan-April Technical review meeting (usually 1-3 proposals) Rest of process as for Phase I

Contract Review Criteria Phase I Technical Evaluation Criteria Weight 1. The soundness and technical merit of the proposed approach based on: a. Clear measureable goals (milestones) that have a reasonable chance of meeting the topic objective in Phase I; b. The approach is innovative and not routine, c. Offeror’s ability to implement technical approach 40% 2. The qualifications of the proposed PDs/PIs 20% 3. The potential of the proposed research for technological innovation. 15% 4. The potential of the proposed research for commercial application. The commercial potential of a proposal will be assessed using the following criteria: a. Whether the outcome of the proposed research activity will likely lead to a marketable product or process. b. The offeror’s discussion of the potential barriers to entry and the competitive market landscape as well as method to overcome. 5. The adequacy and suitability of the facilities and research environment 10%

Contract Review Criteria For Fast-Track applications: Phase II Technical Evaluation Criteria Weight 1. The soundness and technical merit of the proposed approach based on: a. Clear measureable goals (milestones) that have a reasonable chance of meeting the topic objective in Phase II; b. The approach is innovative and not routine, c. Offeror’s ability to implement technical approach 30% 2. The potential of the proposed research for commercial application, as documented in the Commercialization Plan and evidenced by: a. Offeror’s record of successfully commercializing prior SBIR/STTR or other research projects b. Commitments of additional investment during Ph I & Ph III from private sector or other non-SBIR funding sources c. Other indicators of commercial potential for the proposed research 3. The qualifications of the proposed PDs/PIs, supporting staff and consultants. 25% 4. The adequacy and suitability of the facilities and research environment 15%

NCI Management of Funded SBIR Contracts Individual Program Staff First deliverable is kickoff presentation to COR by contractor- tied to an invoice (first payment of contractor) Communication with contractor unlimited Quarterly progress reports tied to invoices which COR must approve (electronic) Annual report required for Phase II (end of year 1) Commercialization plan required at end of Phase I project period and then again within Phase II proposal Final report required for both Phase I and Phase IIs Mentoring as time allows Investor Forums Phase II contract can be followed with Phase IIB Bridge grant funding

Example: Development of Novel Therapeutic Agents That Target Cancer Stem Cells (Fast-Track proposals will be accepted.) Budget (total costs): PhI: $300,000 for 9 months; PhII: $2,000,000 for 2 years Number of anticipated awards: 2-3 Project Goals: Proposals under this topic should be involved the development of novel therapeutic agents that target CSCs. These small molecules or biologics should be designed to target CSCs, CSC-related biomarkers, or CSC pathways that affect fundamental processes associated with carcinogenesis, tumor progression, maintenance, recurrence or metastasis. Particular emphasis is placed on agents that target CSC self-renewal, regeneration, or differentiation processes. Phase I Activities & Deliverables: Demonstrate in vitro efficacy for the agent(s) that targets CSCs. Validate the effect of the agent(s) on CSCs. The offerors are required to provide evidence confirming that the agent(s) specifically targets CSCs (e.g., measurement showing reduced quantity, viability, or frequency of CSCs). Conduct structure-activity relationship (SAR) studies, medicinal chemistry, and/or lead antibody optimization (as appropriate). Perform animal toxicology and pharmacology studies as appropriate for the agent(s) selected for development. Develop a detailed experimental plan (to be pursued under a future SBIR Phase II award) necessary for filing an IND or an exploratory IND. This topic is looking for small molecule or biologics based drug discovery that targets cancer stem cells. To apply for this topic, offerors should: Have at least one validated target. The target can be, but is not limited to: a marker, a pathway, a set of markers or pathways, or any other molecular targets that are specifically associated with CSCs in the cancer of choice. Provide data or cite literature to support that the target is tightly associated with CSCs. Have ownership of, or license for, at least one lead agent (e.g., compound or antibody) with preliminary data showing that the agent hits the identified target. Have experience with a well-validated assay for CSCs. Describe what is known about the mechanism by which the agent acts on CSCs. Phase II Activities and Expected Deliverables: Complete IND-enabling experiments and assessments according to the plan developed in Phase I (e.g., demonstration of desired function and favorable biochemical and biophysical properties, PK/PD studies, safety assessment, preclinical efficacy, GMP manufacturing, and commercial assessment). The plan should be re-evaluated and refined as appropriate. Develop and execute an appropriate regulatory strategy. If warranted, provide sufficient data to file an IND or an exploratory IND for the candidate therapeutic agents (i.e., oncologic indications for CSCs). Demonstrate the ability to produce a sufficient amount of clinical grade materials suitable for an early clinical trial. 14

Advancing Cancer Research Medical Devices & Radiation Therapy FY15 NCI Contract Funding Topics - Due Date: November 5, 2014 Therapeutics 336 Development of Novel Therapeutic Agents that Target Cancer Stem Cells In Vitro Diagnostics 337 Cell-Free Nucleic Acid-Based Assay Development for Cancer Diagnosis Advancing Cancer Research 334 Vacutubes to Preserve the Viability of Circulating Tumor Cells 335 Development of Advanced Culture Systems for Expansion of Cancer Stem Cells Medical Devices & Radiation Therapy 338 Predictive Biomarkers of Adverse Reactions to Radiation Treatment 339 Systemic Targeted Radionuclide Therapy For Cancer Treatment Health IT 340 Validation of Mobile Technologies for Clinical Assessment, Monitoring, and Intervention 15 15

Should I apply for a contract or a grant? Do you specifically fit the goals and deliverables of the contract? Would this project be a focus of the company? Remember, you cannot submit the same project to both mechanisms Either Way, Use NIH Reporter http://projectreporter.nih.gov/reporter Search previous funded grants and contracts Search for potential collaborators How does your project stack up?

Refer to Contact Us Page for Individual Contacts Ask Away… http://sbir.cancer.gov Email: ncisbir@mail.nih.gov Refer to Contact Us Page for Individual Contacts Xing-Jian, Ph.D. Program Director NCI SBIR Development Center