Dose escalation study of ODM-203, a selective dual FGFR/VEGFR inhibitor, in patients with advanced solid tumours

2576Background: Inhibitors of fibroblast growth factor receptors (FGFR) are being developed for treatment of solid tumours with FGFR genetic alterations. ODM-203 is a small molecule with balanced inhibitory effects on both FGFR 1-4 and VEGFR 1-3 subtypes. We present the results of the first-in-man study. Methods: ODM-203 was evaluated in a 3+3 dose escalation design to identify the maximum tolerated dose (MTD), dosed daily in a 4 week cycle. PK profiles were taken on days 1 and 8, and safety labs, ECG, vital signs and adverse events were recorded on days 1, 4, 8, 15, 22, 29, week (wk) 6 and wk 8. Plasma markers of FGFR and VEGFR inhibition were measured at baseline, day 8 and day 29 and tumour response by RECIST criteria every 8 weeks. Patients continued ODM-203 treatment until disease progression or dose limiting toxicity (DLT). Results: 24 patients (median age 55, range 28-80 years) with advanced solid tumours received ODM-203 in doses between 50-800mg once/day. The longest treatment period so far is 40...