First-in-Kind Biodefense Drug Modeling

Project Summary: An established bio-pharmaceutical company was developing a medical countermeasure for a highly fatal bioterrorism agent. Rosa developed a pioneering methodology for modeling biodefense drugs that explicitly represented physiological components. The results enabled the company to confidently scale animal results to humans, and move ahead with informative proof-of-concept trials.

Methods: Rosa designed a PhysioPD-style model and performed simulations to determine appropriate human doses based on efficacy data from two animal species and human pharmacokinetic data, as required by the FDA. A wide range of data from the literature was explicitly incorporated into the model to enable confident scaling and defensible dose recommendations.

Results:

• The model accounted for critical factors never before addressed in published models or analyses of this type of biodefense capability.
• Effective doses were confidently and defensibly scaled from animals to humans due to the explicit representation of key physiologic relations in the model.
• Survival potential was understood by analyzing trade-offs among dose, dose regimen, and various adjuvant treatment strategies.
• The US FDA gave a favorable review of Rosa’s documentation of the model, simulations, and dose regimens, and enabled the company to swiftly move ahead with their subsequent proof-of-concept trials.