Funding available through this RFA supports the ongoing research and development of innovative products, services, and infrastructure with significant potential impact on patient care. Generally, at this stage, the company has identified and characterized a lead compound; demonstrated efficacy in multiple translationally relevant animal models; completed pilot/dose ranging toxicology studies; determined the feasibility of a scalable, GMP compliant manufacturing process, including release assays; and identified a prototype formulation suitable for further development. The applicant is typically within one year from filing an IND/IDE or already in Phase 1.

With appropriate justification, companies may use CPRIT funds to support the following:

Studies that establish preclinical proof of concept (safety and efficacy)
CMC/manufacturing development
GLP safety studies to support INDs
Phase 1 in humans to establish safety and a recommended dose for phase 2
Phase 2 studies to determine safety and efficacy in initial targeted patient population