Clinical activity of a htert (vx-001) cancer vaccine as post-chemotherapy maintenance immunotherapy in patients with stage IV non-small cell lung cancer: final results of a randomised phase 2 clinical trial
on behalf of the Vx-001-201 trial team, Cesare Gridelli, Tudor–Eliade Ciuleanu, Manuel Dómine, Aleksandra Szczęsna, Isabel Bover, Manuel Cobo, Nikolaos Kentepozidis, Paul Zarogoulidis, Charalabos Kalofonos, Andrzej Kazarnowisz, Magdalena Korozan, Ramon de las Penas, Margarita Majem, Antonio Chella, Frank Griesinger, Evangelos Bournakis, Parvis Sadjadian, Αthanasios Kotsakis, Thierry Chinet, K. Syrigos, Pierpaolo Correale, Catherine Gallou, Jeanne- Menez Jamet, Eleni‐Kyriaki Vetsika, Kostas Kosmatopoulos, Vassilis Georgoulias
Abstract
BACKGROUND: CD8 + cytotoxic T cells; this immune response is associated with improved overall survival (OS) in patients with advanced/metastatic non-small cell lung cancer (NSCLC). METHODS: A randomised, double blind, phase 2b trial, in HLA-A*201-positive patients with metastatic, TERT-expressing NSCLC, who did not progress after first-line platinum-based chemotherapy were randomised to receive either Vx-001 or placebo. The primary endpoint of the trial was OS. RESULTS: Two hundred and twenty-one patients were randomised and 190 (101 and 89 patients in the placebo and the Vx-001 arm, respectively) were analysed for efficacy. There was not treatment-related toxicity >grade 2. The study did not meet its primary endpoint (median OS 11.3 and 14.3 months for the placebo and the Vx-001, respectively; p = 0.86) whereas the median Time to Treatment Failure (TTF) was 3.5 and 3.6 months, respectively. Disease control for >6months was observed in 30 (33.7%) and 26 (25.7%) patients treated with Vx-001 and placebo, respectively. There was no documented objective CR or PR. Long lasting TERT-specific immune response was observed in 29.2% of vaccinated patients who experienced a significantly longer OS compared to non-responders (21.3 and 13.4 months, respectively; p = 0.004). CONCLUSION: immune response but failed to meet its primary endpoint. Subsequent studies have to be focused on the identification and treatment of subgroups of patients able to mount an effective immunological response to Vx-001. CLINICAL TRIAL REGISTRATION: NCT01935154.