ATX-101 showed antitumor activity
PD, tumor
BLOOD, DISEASE, TUMOR
Fourteen (70%) of 20 patients belonging to the Efficacy Population had stable disease at the end of the dose escalation study. Twelve patients (60%) continued treatment in the long-term follow-up study. At treatment discontinuation, after a median time of 18 weeks [7–74], 5 of 12 patients (41.7%) had stable disease, and 7 patients (58.3%) had progressive disease (treatment duration is shown in Supplementary Fig. Of note, the above-mentioned disease stabilization was independent of the dose and was observed in 50%, 33.3%, 50% and 33.3% of patients belonging to the Efficacy Population in Cohorts 1–4, respectively.Due to the heterogenous patient population with different tumor entities characterized by differences in prognoses, prior treatment lines and life expectancies, no meaningful efficacy or efficacy-dose relationship evaluations were possible. So far, no clinical pharmacodynamic (PD) markers have been identified that help to describe the mode of action in patients and to support the clinical dose finding. Biopsies collected in an ongoing study will be analyzed in order to identify potential biomarkers. Blood samples have also been collected at all visits for all patients in this Phase 1 study for use in future analysis.