Targeted Indication Selection Using Adaptive Design

A biotechnology sponsor developing a novel biologic therapy engaged Veristat to design and execute a Phase 2, multi-cohort oncology trial aimed at identifying tumor indications with the highest potential for success. With limited data across advanced and metastatic tumor types, the sponsor required an efficient approach to prioritize cohorts without the cost and delay of multiple independent studies. Veristat implemented a Simon 2-stage design, enabling each tumor-specific cohort to be independently assessed for objective response rates with predefined futility and continuation thresholds.

This design minimized patient exposure to non-responsive tumors while allowing rapid advancement of promising indications. Customized hypotheses and response benchmarks tailored to each tumor type ensured meaningful interpretation of early signals. To support real-time decision-making, Veristat established centralized cohort monitoring, interim analyses, and exploratory dose evaluations. By advancing only high-potential tumor types and eliminating unproductive cohorts, the trial safeguarded patient resources, optimized therapeutic exploration, and provided a robust foundation for future oncology development.