Blueprint Neurotherapeutics Network (BPN): Small Molecule Drug Discovery and Development for Disorders of the Nervous System (U44 Clinical Trial Optional)

The National Institutes of Health (NIH), through multiple participating Institutes and Centers (ICs), has reissued the Blueprint Neurotherapeutics Network (BPN) funding opportunity to support small molecule drug discovery and development for nervous system disorders. Administered under the U44 SBIR Cooperative Agreement Phase II mechanism, this grant enables neuroscience investigators to collaborate with NIH-supported consultants and contract research organizations (CROs). These collaborations offer access to specialized services such as medicinal chemistry, pharmacokinetics, toxicology, formulation development, GMP-compliant synthesis, and Phase I clinical testing. The program allows projects to enter at either the Discovery or Development stage depending on the maturity of the compound and data available. In the Discovery phase, researchers aim to optimize promising small molecule hits using medicinal chemistry to enhance drug-like properties and understand structure-activity and structure-property relationships. Projects in this stage undergo a U44 Phase I period focused on assay validation and feasibility studies before transitioning into a more resource-intensive Phase II for SAR development and candidate selection. For Development stage entries, projects must have already selected a clinical candidate with sufficient pharmacological and safety data, and the focus shifts to IND-enabling studies and Phase I clinical trials. Eligibility is inclusive of small businesses with the infrastructure or partnerships necessary to carry out disease- or target-specific assays, models, and bioactivity tests. Participants may choose to conduct activities within their institutions or leverage BPN resources. Each awarded project forms a customized Lead Development Team (LDT) co-chaired by the PI and an NIH consultant, which oversees project strategy, milestone development, and execution. Applications must align with the mission of one of the participating NIH ICs, including the National Institute on Aging (NIA), National Institute on Alcohol Abuse and Alcoholism (NIAAA), National Eye Institute (NEI), and others. Specific aims of interest include treatments for Alzheimer’s disease, alcohol use disorders, and vision disorders. The application process requires a Letter of Intent (LOI) 30 days prior to each deadline and submissions are accepted biannually with the next due date on January 15, 2025. Applications must be submitted through one of the NIH-approved systems: ASSIST, institutional S2S, or Grants.gov Workspace. Review and award decisions follow the NIH standard peer review and council processes. Funded projects will undergo continuous oversight with milestone-based progression, and final approval for Phase II funding depends on successful completion of Phase I requirements. This funding opportunity remains open through August 19, 2026.