Aims: Lenvatinib showed noninferiority in overall survival (OS) compared with sorafenib for first-line treatment of patients with uHCC in a phase 3 trial (REFLECT). Pembrolizumab has shown promising activity in HCC. We report preliminary results from a phase 1b trial of lenvatinib+pembrolizumab in uHCC (NCT03006926).
Methods: In this open-label, multicenter study, patients with uHCC, BCLC stage B or C, Child-Pugh class A, and ECOG PS ≤1 received lenvatinib (body weight ≥60 kg: 12 mg/day; <60 kg: 8 mg/day) daily and 200 mg pembrolizumab intravenously Q3W. Dose-limiting toxicities (DLTs) were assessed during Cycle 1 in patients ineligible for other therapies (3+3 design; Part 1). Once tolerability was confirmed, patients with no prior systemic therapy for uHCC were enrolled (Part 2). The primary endpoint was safety. Secondary endpoints included objective response rate using modified RECIST. Tumor assessments were confirmed ≥4 weeks after initial response.
Results: As of December 1, 2017, 18 patients had received lenvatinib+pembrolizumab (Part 1: n=6; Part 2: n=12). Patients had BCLC stage B (n=6) or C (n=12), Child-Pugh scores of 5 (n=14) or 6 (n=4); 4 patients (22%) had received prior sorafenib. No DLTs were reported in Part 1. All 18 patients remained on study. TEAEs occurred in 17 patients (94%); the most common TEAEs were decreased appetite and hypertension (56% each). No new safety signals were identified. Efficacy outcomes are shown below (table). At data cutoff, tumor reduction was observed in all evaluable patients except one.
Conclusion: Lenvatinib+pembrolizumab was well tolerated with encouraging anti-tumor activity in patients with uHCC.
Outcome |
Part 1 (n=6) |
Part 2 (n=7) |
Total (n=13) |
Best Overall Response, n (%) |
|||
Partial response* |
4 (67) |
2 (29) |
6 (46) |
Stable disease |
2 (33) |
4 (57) |
6 (46) |
Progressive disease |
0 (0) |
0 (0) |
0 (0) |
Not evaluable |
0 (0) |
1 (14) |
1 (8) |
*3 Partial responses confirmed.