BL-8040, a CXCR4 antagonist, in combination with pembrolizumab and chemotherapy for pancreatic cancer: the COMBAT trial

Nat Med. 2020 Jun;26(6):878-885. doi: 10.1038/s41591-020-0880-x. Epub 2020 May 25.

Abstract

Programmed cell death 1 (PD-1) inhibitors have limited effect in pancreatic ductal adenocarcinoma (PDAC), underscoring the need to co-target alternative pathways. CXC chemokine receptor 4 (CXCR4) blockade promotes T cell tumor infiltration and is synergistic with anti-PD-1 therapy in PDAC mouse models. We conducted a phase IIa, open-label, two-cohort study to assess the safety, efficacy and immunobiological effects of the CXCR4 antagonist BL-8040 (motixafortide) with pembrolizumab and chemotherapy in metastatic PDAC (NCT02826486). The primary outcome was objective response rate (ORR). Secondary outcomes were overall survival (OS), disease control rate (DCR) and safety. In cohort 1, 37 patients with chemotherapy-resistant disease received BL-8040 and pembrolizumab. The DCR was 34.5% in the evaluable population (modified intention to treat, mITT; N = 29), including nine patients (31%) with stable disease and one patient (3.4%) with partial response. Median OS (mOS) was 3.3 months in the ITT population. Notably, in patients receiving study drugs as second-line therapy, the mOS was 7.5 months. BL-8040 increased CD8+ effector T cell tumor infiltration, decreased myeloid-derived suppressor cells (MDSCs) and further decreased circulating regulatory T cells. In cohort 2, 22 patients received BL-8040 and pembrolizumab with chemotherapy, with an ORR, DCR and median duration of response of 32%, 77% and 7.8 months, respectively. These data suggest that combined CXCR4 and PD-1 blockade may expand the benefit of chemotherapy in PDAC and warrants confirmation in subsequent randomized trials.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Aged, 80 and over
  • Antibodies, Monoclonal, Humanized / administration & dosage
  • Antineoplastic Agents, Immunological
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • CD8-Positive T-Lymphocytes / pathology
  • Carcinoma, Pancreatic Ductal / drug therapy*
  • Carcinoma, Pancreatic Ductal / pathology
  • Carcinoma, Pancreatic Ductal / secondary
  • Female
  • Fluorouracil / administration & dosage
  • Humans
  • Irinotecan / administration & dosage
  • Leucovorin / administration & dosage
  • Liver Neoplasms / drug therapy*
  • Liver Neoplasms / secondary
  • Lung Neoplasms / drug therapy*
  • Lung Neoplasms / secondary
  • Lymph Nodes / pathology
  • Lymphatic Metastasis
  • Lymphocytes, Tumor-Infiltrating / pathology
  • Male
  • Middle Aged
  • Myeloid-Derived Suppressor Cells / pathology
  • Pancreatic Neoplasms / drug therapy*
  • Pancreatic Neoplasms / pathology
  • Peptides / administration & dosage
  • Peritoneal Neoplasms / drug therapy*
  • Peritoneal Neoplasms / secondary
  • Receptors, CXCR4 / antagonists & inhibitors
  • Retroperitoneal Neoplasms / drug therapy*
  • Retroperitoneal Neoplasms / secondary
  • Survival Rate
  • T-Lymphocytes, Regulatory / pathology
  • Treatment Outcome

Substances

  • 4-fluorobenzoyl-TN-14003
  • Antibodies, Monoclonal, Humanized
  • Antineoplastic Agents, Immunological
  • CXCR4 protein, human
  • Peptides
  • Receptors, CXCR4
  • Irinotecan
  • pembrolizumab
  • Leucovorin
  • Fluorouracil

Associated data

  • ClinicalTrials.gov/NCT02826486