Principles, Assumptions, and Processes Established to Designate Biologics Assets as a Fast to FIH (First-in-Human) Program and the Associated Core Concepts for the Utilization of low and High-Risk Activities, Timelines and Functional Level Expectations

Abstract

Biologics development represents a substantial advancement in the pharmaceutical industry because of their promise and huge success in the oncology, immunoscience, and cardiovascular disease areas. Prior to entering the marketed product development phase, each biopharmaceutical needs to go through series of stages that will allow or disallow the biologics asset to become a commercialized product. Each of those phases includes development planning and designing of studies to test relevant hypotheses to support the drug label if approved. The current thesis will focus on the principles, assumptions, and processes that are established to designate an asset (biologics) as a targeted first-in-human program. First-in-human studies are included under phase 1 trials, where initial human exposure is initiated to the investigational new drug (IND). Phase 1 is critical since it affirms if a compound’s mechanisms of action in humans and its development can result in a potentially new drug entity. Subsequently, step by step initiatives and processes from the perspective of different functional groups within the pharma will be revised to outline the staged procedures, methods, critical, and noncritical paths taken when a molecule is nominated as a clinical candidate. Overall alignment of deliverables will be presented between the different functional areas that partake in the first-in-human development. Strategic changes to the biologics development process, cell line development with multiple candidate sequences, initial platform fit assessment for a process, analytical and formulation will be acknowledged. Platform strategy for drug substance production, as well as, drug product composition will be outlined along with boilerplates for analytical method development to fit or not fit the platform approach. The functional groups that will be reviewed will be; Discovery, Cell line development, Drug Substance process development, Formulation development, Toxicology, Quality, Drug Substance manufacturing, Drug Product manufacturing, Stability and regulatory.