NINDS Renewal Awards of SBIR Phase II Grants (Phase IIB) for Pre-Clinical Research (R44 Clinical Trial Not Allowed)

Purpose NINDS is committed to advancing diagnostics and treatments for people burdened by neurological diseases, and the NINDS Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) programs have provided the small business community with critical seed funding to support the development of a wide variety of technologies and therapeutics for the diagnosis and treatment of neurological diseases. The SBIR/STTR Programs are structured in three phases. The main objective in SBIR/STTR Phase I is to establish the technical merit and feasibility of the proposed research and development (R&D) efforts, whereas in SBIR/STTR Phase II it is to continue the R&D efforts to advance the technology toward ultimate commercialization. At the conclusion of an SBIR/STTR Phase II, it is expected that the small business concern (SBC) will fully commercialize their product or technology using non-SBIR/STTR funds in Phase III. The development of medical biotechnology products is often impeded by a significant funding gap (known as the “Valley of Death”). To achieve commercialization, some projects initiated with SBIR or STTR funding require considerable grant-in-aid financing beyond the SBIR/STTR Phase II award to achieve follow-on investment or commercialization. In particular, the development of regulated products such as therapeutics and medical devices often requires several years and substantial capital investments, due in part to the high costs of clinical trials. In addition, NINDS recognizes that companies developing products that have small potential revenue streams or that target small patient populations face additional barriers to market entry that make them less attractive to investors and strategic partners at preclinical or early clinical stages of development. Many of these technologies require complex clinical trial designs because of small and geographically diverse patient populations. For the purposes of this FOA, NINDS has defined small markets as development of novel products that: Address a rare disease as defined in the Orphan Drug Act Amendment of 1984 as any disease or condition affecting fewer than 200,000 persons in the United States Qualify as a Humanitarian Use Device, defined as a medical device intended to benefit patients in the treatment or diagnosis of a disease or condition that affects or is manifested in fewer than 4,000 persons in the United States per year Target a young pediatric population defined as including neonates (0-28 days), infants (<2 years), and/or children (2-12 years of age), as indicated in the FDA Premarket Assessment of Pediatric Medical Devices. Are complex research tools. This is instrumentation comprising several distinct parts that must work together. Very often the goal of such projects is to deliver turnkey products for researchers. For example, high density electroencephalography instrumentation includes electrode arrays, amplifiers, data analytic and data visualization software, etc. Another example is non-invasive near infrared imaging instrumentation, which might include photon sources and detectors, timing devices for delivering photons, amplifiers, and software. Some high throughput assay systems may fall into this category. The goal of this FOA is to assist applicants in pursuing the next appropriate milestone(s) necessary to advance a product/technology that requires Federal regulatory approval or to bring a complex research tool to market. To achieve this goal, the FOA aims to facilitate the transition of previously funded SBIR or STTR Phase II projects to the commercialization stage by encouraging business relationships between NIH’s SBIR/STTR awardees and third-party investors and/or strategic partners. In particular, this FOA will give competitive preference and funding priority to applications deemed likely to result in a commercial product as indicated by an applicant's ability to secure partnerships within a broad range of potential third-parties. Specific Objectives Independent Third-Party Partners This FOA is specifically intended to encourage business relationships between applicant SBCs and third-party investors/strategic partners who can provide substantial financing to help accelerate the commercialization of promising new products and technologies initiated with NIH SBIR or STTR funding. In particular, applicants are expected to leverage their previous NIH SBIR or STTR support, as well as the opportunity to compete for additional NINDS funding under this FOA, to negotiate and attract third-party financing needed to advance a product or technology toward commercialization. The applicant’s ability to secure funds will provide a measure of commercial potential that is essential for the SBIR applications submitted to this FOA. This commercial potential will be strongly considered in making funding decisions. It is anticipated that many of the partnerships between applicant SBCs and third-party partners will involve a considerable level of project due diligence, thereby increasing the likelihood of commercial success for the funded projects. Third-party partners include, but are not limited to, another company, a venture capital firm, an “angel” investor, a foundation, a university, a research institution, a state or local government, or any combination of the above. In light of these goals, the NINDS strongly encourages applicants to establish business relationships with investors and/or strategic partners that have appropriate prior experience in the commercialization of emerging biomedical technologies. NINDS expects companies working in small markets (as defined above) to secure independent third-party funding equal to or greater than one-third of the NINDS funds being requested throughout the project period. For all other projects, it is expected that the level of this independent third-party funding will be equal to or greater than the NINDS funds being requested throughout the Phase IIB Bridge Award project period. Scientific/Technical Scope This FOA is specifically designed to provide additional support for products/technologies that require ultimate approval by a Federal regulatory agency or complex research tools. Such products include, but are not limited to: Therapeutics or diagnostics that require FDA approval, including medical implants, drugs, biologics, biomarkers, and new treatment or diagnostic tools. Complex research tools. Clinical research tools: Such tools would include those that are developed for clinical research use that do not require any federal regulatory approval, but still require extensive development in order to demonstrate validity in a diverse population. The technical and commercial objectives described in the SBIR Phase IIB application must represent an extension of the development efforts that were pursued in a previously funded NIH SBIR/STTR Phase II grant or contract. Applicants are encouraged to read the NINDS Notice NOT-NS-11-023 on Improving the Quality of NINDS-Supported Preclinical and Clinical Research through Rigorous Study Design and Transparent Reporting. Renewal applications of SBIR Phase II awards (i.e. SBIR Phase IIB) to conduct clinical trials are not included in this FOA and should be submitted to NINDS Renewal Awards of SBIR Phase II Grants (Phase IIB) for Clinical Trials and Clinical Research (R44 Clinical Trial Optional). A clinical trial, as defined by the NIH, is a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of those interventions on health-related biomedical or behavioral outcomes. Please read Notice NOT-OD-15-015 for more details. An objective of the SBIR and STTR programs is to foster and encourage participation in innovation and entrepreneurship by socially and economically disadvantaged persons and women-owned small businesses. This PAR encourages applications from diverse teams of investigators, including team members that are underrepresented in the biomedical, behavioral, or clinical research workforce (see data athttp://www.nsf.gov/statistics/showpub.cfm?TopID=2&SubID=27and the most recent report on Women, Minorities, and Persons with Disabilities in Science and Engineering). Such individuals include those from underrepresented racial and ethnic groups, those with disabilities, and those from disadvantaged backgrounds. Applicants should take note of the following: Applicants are strongly encouraged to leverage existing NINDS research resources for their studies whenever possible. Such resources may include biospecimens from NINDS Human Biospecimen and Data Repository (BioSEND) or informatics system. The NINDS BioSEND repository receives, processes, stores, and distributes biospecimen resources from NINDS funded studies that can be shared by the neuroscience research community, and currently banks a variety of biospecimens including DNA, plasma, serum, RNA, CSF, and saliva. The NINDS Human Cell and Data Repository provides 1) disease-relevant stem cell lines for biomarker discovery, and/or 2) the capacity to bank blood for the creation of new cell lines relevant to their disease of interest. Leveraging the resources and support from neurological disorder advocacy groups, private research foundations, academic institutions, other government agencies and the NIH Intramural program are also encouraged. Finally, applicants are encouraged to leverage the resources of ongoing clinical trials supported through other Federal or private funds. Applications proposing to collect biospecimens are strongly recommended to use the BioSEND protocols and procedures, and all specimens collected and banked with BioSEND must come from individuals who have consented to banking and sharing broadly with academia and industry. Note that costs for collection are NOT included as a component of the NINDS Biomarkers Repository award. Therefore, most costs for the biospecimen banking are borne by the grantees utilizing this resource (see NOT-NS-15-046). Applicants planning projects in which biospecimens will be collected are strongly advised to consult the BioSEND website for more information about samples banked at the repository. In addition, applicants are advised to consult with BioSEND staff to obtain a quote for biospecimen banking costs (email:biosend@iu.edu).