Fig. 1: Clinical outcome data from the PEMDAC trial at data cutoff in December 2019.

a Waterfall plot showing maximum change in sum of target lesion diameter from baseline to data cutoff. One patient did not have a response assessment after baseline and is not included in the figure. Dotted lines represent thresholds for progressive disease (+20%) and partial response (−30%), respectively, according to RECIST v1.1 criteria. b Spider plot showing change in the sum of target lesion diameter over time for all patients with at least one follow-up scan. c Swimmer’s plot showing time on treatment, time to best response, and duration of response in all patients who received at least one dose of study drug. In panels (a,b), n = 28 and in (c) n = 29 patients are shown, respectively. d Kaplan–Meier analysis showing overall survival (OS) of all patients. e Kaplan–Meier analysis showing progression-free survival (PFS) of all patients except one. f Circulating tumor DNA levels in patients with UM. Plasma from twenty-five patients was analyzed for the presence of mutated reads in either GNA11 (Q209L) or GNAQ (Q209L/P). Variant allele frequencies (VAF) in percent compared with reads with wildtype alleles are plotted. g Kaplan–Meier analysis comparing OS between patients with high or low (relative to median) levels of total detected ctDNA copies, n = 14 and n = 15 patients, respectively. h Kaplan–Meier analysis comparing OS between patients with lactate dehydrogenase (LDH) baseline greater or lower than the upper limit of normal (ULN), n = 17 and n = 12 patients, respectively. In (g, h), p-values for survival associations were calculated using log-rank tests. No adjustments for multiple comparisons were made. All statistical tests were two-sided. Source data are provided as a Source Data file.