Phase II-III Trial for a Novel Asthma Drug

Project Summary: An emerging, publicly-held, pharmaceutical company was undertaking Phase II-III trials to test a treatment for severe asthma episodes. Rosa created a model that included the actions of the drug as well as its interactions with coadministered drugs. Rosa’s simulations fundamentally changed the company’s views about the appropriate dose to use for Phase IIb trials.

Methods: Rosa created a PK/PD model of the key interactions among trial compounds and simulated a coadministered standard of care to evaluate both efficacy and safety of the drug.

Results:

• A proposed Phase IIa trial was averted because the model showed it would not be informative.
• A planned Phase IIb trial was modified with more informative doses and regimens based on model simulation results, and by moving subjects from active to placebo arms.
• A novel relationship was identified between a common analyte and response variability – a relation not previously reported in the literature.
• Safe doses were identified with an adaptive protocol specifically designed for this project.