Abstract

Metastatic basal cell carcinoma (mBCC) is a rare condition with no effective second-line treatment options. Cemiplimab is an immune checkpoint inhibitor that blocks binding of programmed cell death-1 (PD-1) to its ligands, PD-L1 and PD-L2. Here, we present the final analysis of cemiplimab in patients with mBCC after first-line hedgehog pathway inhibitors (HHIs) (NCT03132636).In this open-label, single-arm, phase II study, adults with mBCC and Eastern Cooperative Oncology Group performance status =1, post-HHI treatment, received cemiplimab 350 mg intravenously every 3 weeks for =93 weeks or until disease progression or unacceptable toxicity. The primary endpoint was objective response rate (ORR) by independent central review (ICR). Duration of response (DOR) was a key secondary endpoint. Other secondary endpoints included ORR per investigator assessment (INV), progression-free survival (PFS), overall survival (OS), complete response (CR) rate, safety and tolerability.Fifty-four patients were enrolled (70% male, median age 64 years [interquartile range (IQR) 57.0-73.0]). Median duration of follow-up was 8 months (IQR 4-21). ORR per ICR was 22% (95% confidence interval [CI] 12-36), with two CRs and 10 partial responses. Among responders, median time to response per ICR was 3 months (IQR 2-7). Estimated median DOR per ICR was not reached (95% CI 10 months-not evaluable [NE]). Disease control rate was 63% (95% CI 49-76) per ICR and 70% (95% CI 56-82) per INV. Median PFS per ICR was 10 months (95% CI 4-16); median OS was 50 months (95% CI 28-NE). The most common treatment-emergent adverse events were fatigue (23 [43%]) and diarrhoea (20 [37%]). There were no treatment-related deaths.Cemiplimab demonstrated clinically meaningful antitumour activity, including durable responses, and an acceptable safety profile in patients with mBCC who had disease progression on or intolerance to HHI therapy.

View details for DOI 10.1016/j.annonc.2023.10.123

View details for PubMedID 38072158