Phase 2, open-label, 1:1 randomized controlled trial exploring the efficacy of EMD 1201081 in combination with cetuximab in second-line cetuximab-naïve patients with recurrent or metastatic squamous cell carcinoma of the head and neck (R/M SCCHN).

Aim: to determine whether EMD 1201081, a TLR9 agonist, added to cetuximab had antitumor activity in second-line recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) Methods: this was a phase 2, open-label, randomized trial of EMD 1201081 0.32 mg/kg subcutaneously weekly plus cetuximab (combination) vs cetuximab monotherapy (control) in cetuximab-naive patients with R/M SCCHN who progressed on 1 cytotoxic regimen. Crossover to combination was permitted after progression Results: objective response rate in both arms was 5.7 % (95 % CI 1.2–15.7 %) by independent assessment. Disease control was 37.7 % for patients on combination (24.8–52.1 %) and 43.4 % on control (29.8–57.7 %). Neither independent nor investigator assessments showed significant differences between study arms. Median progression-free survival was 1.5 months (1.3–2.6) for patients on combination, and 1.9 months (1.5–2.9) on control.