Formulation Development From Preclinical To First-In-Human

By Jim Huang, PhD, Founder & CEO, Ascendia Pharmaceuticals

There is only a 16% probability of being approved for compounds entering the Phase 1 stage. The average R&D budget for each new medicine is $1.2 billion, with more recent studies estimating the costs to be even higher.

When a compound enters preclinical development for a GLP tox study from the drug discovery stage, we face a question of how to develop a tox and clinical formulation that ensures the success of IND and first dose in human. According to Lipper, et al, poor biopharmaceutical properties of compounds is attributed to 39% of the failure of the new drug program under development.1 A compound with poor biopharmaceutical properties or improper formulation design could lead to a delay in the project or even program termination. The key considerations for successful tox and Phase 1 formulation development consist of the following:

• Pre-formulation of drug candidate
• Biopharmaceuticals evaluation
• Analytical method development
• Formulation development
• cGMP manufacturing for clinical trials