Dr Kenny on Phase 2 Data With Neoadjuvant Cemiplimab in BCC of the Head and Neck

“The imaging alone isn't always adequate to capture the significance of the disease in some of these patients.”

Hannah Kenny, MD, a fourth-year resident in the Otolaryngology program at Sidney Kimmel Medical College, Thomas Jefferson University, and Joseph M. Curry, MD, a clinician at Jefferson Health, discussed findings from a phase 2 study (NCT05929664) evaluating outcomes with neoadjuvant cemiplimab-rwlc (Libtayo) in patients with locally advanced basal cell carcinoma (BCC) of the head and neck, highlighting how pathologic tumor response tracked with radiographic assessment.

The trial was designed around two primary end points, objective response rate (ORR) and disease control rate (DCR), both of which were met, Kenny said. The ORR was 60.1% (95% CI, 42.1%-76.1%), and the DCR was 91.4% (95% CI, 76.9%-98.2%), reflecting a large proportion of patients (n = 35) who achieved either a complete or partial response or stable disease relative to the prespecified thresholds for success.

Beyond the topline end points, Kenny noted that correlative analyses, though still preliminary, demonstrated a relationship between RECIST-defined radiographic response and pathologic tumor response. Investigators incorporated clinical caliper measurements alongside standard radiographic assessment to better characterize this relationship. Approximately 40% of patients who achieved a response per RECIST criteria also had a major or complete pathologic response, compared with 0% of patients classified as non-responders by RECIST criteria. The magnitude of that difference extended to average pathologic tumor response as well: responders had a mean pathologic tumor response of 70% vs 10% among non-responders, Kenny stated.

Curry expanded on why this correlative work mattered for this specific population, noting that basal cell carcinomas are notably heterogeneous—some lesions are poorly defined or difficult to visualize on imaging despite covering substantial surface area, while others are small but located in high-risk, anatomically sensitive sites. The study specifically enrolled patients with high-risk tumors in these critical locations to evaluate treatment benefit. Although imaging was obtained for every patient, Curry emphasized that it could not always be relied upon alone to accurately capture response, which is why RECIST caliper measurements were used in parallel to track and validate treatment effect.