Lead Optimization and Preclinical Development of Therapeutic Candidates for Diseases of Interest to the NIDDK (R43/R44) | PA 17 130

Opportunity Information: Apply for PA 17 130

The funding opportunity titled "Lead Optimization and Preclinical Development of Therapeutic Candidates for Diseases of Interest to the NIDDK (R43/R44)" (Funding Opportunity Number PA-17-130) is an NIH Small Business Innovation Research (SBIR) grant program designed to help small businesses move promising therapeutic concepts toward a stage where they are ready for formal FDA submission. Its central focus is on supporting the practical, experiment-driven work needed to turn an early lead into a well-characterized development candidate and to complete the key preclinical package required to enter human testing, specifically for diseases that fall within the mission area of the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). In other words, it is aimed at therapies relevant to conditions in areas such as diabetes, endocrinology, metabolism, digestive diseases, nutrition-related disorders, and kidney and urologic diseases, with an emphasis on advancing tangible product development rather than basic discovery research.

This FOA uses the SBIR phased structure, with Phase I (R43) supporting early, enabling steps that set up or de-risk a development program, and Phase II (R44) supporting more substantial lead optimization and preclinical development of the selected candidate(s). Phase I is positioned for projects that are still at the point of refining a lead or assembling the initial evidence package needed to justify full preclinical development. That can include activities like improving potency or selectivity, evaluating preliminary pharmacology, addressing early safety red flags, confirming mechanism of action, optimizing formulation feasibility, or establishing key assays and models that will be used to judge candidate performance. Phase II is meant for projects that are beyond the exploratory stage and ready to execute the more complete development plan, such as systematic lead optimization, IND-enabling studies, and the types of integrated pharmacology, toxicology, and manufacturing readiness activities that regulators typically expect before first-in-human studies.

A major expectation of the program is that Phase II (and where applicable Phase IIB) projects are sufficiently advanced so that an Investigational New Drug (IND) application or an Investigational Device Exemption (IDE) application can be submitted to the FDA by the end of the SBIR Phase II or IIB project period. That requirement strongly shapes what NIH is looking for in an application: the proposed work should not be open-ended science, but a development plan that is aligned with regulatory reality, addresses known development risks, and produces the specific data and documentation needed for FDA engagement. Even though the FOA is described broadly as supporting "new therapies," the practical benchmark NIH highlights is readiness for an IND or IDE submission, which implies the project should be building toward defined deliverables such as a nominated development candidate, validated preclinical efficacy evidence, an acceptable safety profile in required studies, and a credible plan for chemistry, manufacturing, and controls (CMC) or device design controls as appropriate.

Applications under this FOA are expected to be milestone-driven. The announcement explicitly calls for defined Milestones and Timelines that show how the project will move forward, along with clear Go/No Go decision points. This is important because it signals that reviewers and program staff will be evaluating not only scientific merit, but also whether the applicant has a realistic development strategy with measurable endpoints and decision criteria. In practice, that means applicants are expected to lay out what success looks like at each stage (for example, hitting a target exposure level, achieving a minimum efficacy threshold in a relevant model, meeting a safety margin, demonstrating manufacturability, or satisfying a stability target), when those results will be generated, and what happens if those benchmarks are not met. This milestone structure is intended to keep the work focused on de-risking and advancing a product candidate, and to ensure the project is managed like a development program rather than a purely academic research effort.

Eligibility is centered on small businesses, consistent with the SBIR program rules. The FOA also makes clear boundaries around foreign involvement: non-domestic (non-U.S.) entities, meaning foreign institutions, are not eligible to apply, and non-domestic components of U.S. organizations are also not eligible to apply. However, the FOA notes that "foreign components," as defined in the NIH Grants Policy Statement, may be allowed. Practically, that means the applicant organization must be a U.S. small business, and while certain foreign activities might be permitted under NIH policy in limited, well-justified circumstances, the core applicant and project leadership must remain within the eligible U.S. small business framework and comply with NIH rules governing any foreign components.

From the source information provided, this is a discretionary grant opportunity administered by the National Institutes of Health, categorized under Food and Nutrition and Health, and associated with CFDA number 93.847. The original closing date listed is 2020-01-05, and the opportunity was created on 2017-01-24. Although an explicit award ceiling and expected number of awards are not provided in the source excerpt, the key takeaway is the program structure and readiness expectations: this FOA is meant to fund small businesses working on NIDDK-relevant therapeutics that are ready to execute a disciplined lead optimization and preclinical development plan and that can credibly reach IND/IDE submission readiness by the end of Phase II or IIB, supported by clearly articulated milestones, timelines, and objective decision points.

• The National Institutes of Health in the food and nutrition, health sector is offering a public funding opportunity titled "Lead Optimization and Preclinical Development of Therapeutic Candidates for Diseases of Interest to the NIDDK (R43/R44)" and is now available to receive applicants.
• Interested and eligible applicants and submit their applications by referencing the CFDA number(s): 93.847.
• This funding opportunity was created on 2017-01-24.
• Applicants must submit their applications by 2020-01-05. (Agency may still review applications by suitable applicants for the remaining/unused allocated funding in 2026.)
• Eligible applicants include: Small businesses.

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