Sound preclinical efficacy in multiple xenograft models with dose-dependent activity and acceptable safety, but limited to in vivo animal studies without human data.
A prospective single-arm biomarker study in 31 HCC patients with supportive validation in external trial cohorts, identifying immune predictors of AtezoBev response via rigorous multimodal immunophenotyping, but limited by small primary cohort and surrogate endpoints.
A regulator-initiated market withdrawal based on confirmatory Phase Ib/III trial data showing a clinically significant safety signal (hematologic SPMs) that changes the risk–benefit profile of an approved agent.
Rigorous multi-level evidence (functional screens, large clinico-genomic cohort analysis with survival endpoints, and independent rapid autopsy validation) demonstrating SPEN inactivation as a biomarker associated with reduced ARPI benefit in metastatic prostate cancer.
Single-arm Phase 2 study in a small, heavily pretreated population showing no objective responses; negative result limits strength but rigorous conduct and clear null finding warrant reporting.
Prospective profiling study identifying spatial B-cell organization as a potential biomarker for ICI response in CLDN18.2-expressing gastric cancer, with validation across five cohorts including a phase 3 trial, but lacking randomized comparison or prospective outcome prediction.