Efficacy
DISEASE
After enrollment of 27 and 25 patients in cohort 1 and cohort 2, respectively, considering that the primary study endpoint had been met for both cohorts, (cohort 1 with 18 responders and cohort 2 with 10 responders). By intention-to-treat analysis, of the 52 patients in the original cohort (i.e., before the protocol amendment), in cohort 1, objective responses were achieved in 18 (67%; 95% CI, 47.7–85.7) of 27 patients, and 22 patients had disease control (82% [65.8–97.1]) in the full analysis set. In cohort 2, objective responses were achieved in 40.0% (95% CI, 19.4–60.6) of 25 patients and 18 patients had disease control (72.0% [53.1–90.9]) in the full analysis set (Supplementary Table After the protocol amendment and inclusion of additional patients, the median time of follow-up at the time of data analysis (the data cutoff was 31 October 2022) was 23.3 months (IQR 22.4–24.2 months) for cohort 1 and 18.5 months (17.3–19.7 months) for cohort 2. In cohort 1, 3 (7.5%) of 40 patients had a confirmed complete response, 23 (57.5%) had a confirmed partial response, 6 (15.0%) had stable disease, and 4 (10.0%) had progressing disease in the full analysis set. In total, objective responses were achieved in 26 (65%; 95% CI, 49.6–80.4) of 40 patients, and 32 patients had disease control (80% [67.0–93.0]) in the full analysis set (Fig. 