Background: The objectives of the dose-escalation part of this study (NCT04008797) included safety/tolerability, pharmacokinetics, biomarkers, and preliminary efficacy of E7386 (a novel oral anticancer agent modulating Wnt/β-catenin signaling) plus lenvatinib in patients with HCC or other solid tumors. We present results from the HCC subpart.
Methods: In cycle 0, E7386 was administered orally in escalating doses QD or BID for 5 or 6 consecutive days. From cycle 1, E7386 QD or BID, plus daily oral lenvatinib (<60 kg: 8 mg; ≥60 kg: 12 mg), were administered in 28-day cycles. TEAEs were graded using CTCAE v5.0. Prophylactic antiemetics were not allowed during DLT evaluation but were permitted after nausea/vomiting. Tumor response was assessed by investigators using mRECIST.
Results: By data cutoff (9 December 2022), 25 patients with HCC were treated with E7386 doses ranging from 10-80mg QD and 60-120 mg BID. Among the E7386 120 mg BID cohort (n=3), grade 3 maculopapular rash (1 patient) and grade 5 acute kidney injury (1 patient) DLTs were observed. No other DLTs were observed. The most common TEAEs across cohorts were nausea (76.0%), vomiting (60.0%), constipation (52.0%), palmar-plantar erythrodysesthesia syndrome (48.0%), diarrhea (44.0%), and proteinuria (40.0%). The most common grade ≥3 TEAEs were proteinuria (20.0%) and aspartate aminotransferase level increased (8.0%). Nausea and vomiting were well controlled by a 5HT3 antagonist. Among treated patients, 9 (36.0%) partial responses (PRs) were observed, including 3 PRs in 10 patients previously treated with lenvatinib. Cmax and AUC for E7386 increased with increasing E7386 dose.
Conclusions: E7386 100 mg BID plus lenvatinib (8/12 mg QD per bodyweight) was deemed the recommended phase 2 dose in HCC, was tolerable and manageable with antiemetics, and showed promising activity, including in patients pretreated with lenvatinib.