Vx-001 is composed of two separate peptides: the native cryptic peptide ARG-Vx001 (TERT572) and its optimized variant TYR-Vx001 (TERT572Y). Vx-001 has been tested in in vivo experiments in mice, in vitro experiments on human lymphocytes, and a large exploratory Phase I/II clinical trial. Vx-001 vaccination of humanized mice demonstrated protection against tumor growth in vivo (J Clin Invest.  2004 113:425-33). Furthermore, Vx-001 induces anti-tumor immune responses by human lymphocytes in vitro. Vx-001 completed a Phase I/II trial with 33 patients with NSCLC extended to 83 patients with different cancers (mainly breast, prostate and pancreatic cancers) (Oncology .2006;70:306-14, Journal of Clinical Oncology , 2007 19, 2729-34, ASCO 2008 abstract 3030, AACR 2008 abstract 2541, Ann. Oncol., 2012, 23: 442-9, Cancer Immunol. Immunother., 2012, 61: 157-68). The majority of patients (70%) entered the study with progressive disease. Moreover, more than 70% of patients had metastatic disease.

The major conclusions of this Phase I/II trial are the following:

  1. Vx-001 was safe and well tolerated: only Grade I/II vaccine-related toxicity was observed in vaccinated patients (local skin reaction).
  2. Vx-001 was strongly immunogenic. hTERT specific Immune responses were observed in 44% of patients

Vx-001 has completed a Phase IIb trial in NSCLC

Vx-001 completed a randomized, placebo controlled, double blind Phase IIb clinical trial in HLA-A*0201 positive patients with TERT expressing NSCLC (stage IV and distant recurrent stage I-III) and controlled disease after first line chemotherapy (NCT01935154) (Clinical Lung Cancer 2013, 14 : 461-5). The primary endpoint is overall survival. Two hundred twenty one patients have been randomized out of 1400 patients screened in more than 70 sites in eight European countries. Results of the trial showed that Vx-001 drastically and very significantly prolonged overall survival (20.7 months for Vx-001 vs 7.9 months for placebo ; p=0.0007) and time to treatment failure (5.6  months for Vx-001 vs 3.1 months for placebo ; p=0.006) in never and light smoker patients with non-immunogenic tumors. They also strongly suggested that Vx-001 can turn tumor initially resistant to immune checkpoint inhibitors sensitive.