Evidence suggests that the CXC–chemokine receptor-4 (CXCR4) pathway plays a major role in cancer cell homing and metastasis and in shaping the tumor microenvironment (TME), and thus represents a potential target for cancer therapy. Under normal conditions the CXCR4 pathway regulates the hematopoietic stem cell niche in the bone marrow (BM)—a property that has led to the approval of the CXCR4 antagonist plerixafor together with G-CSF (AMD3100, Mozobil) for mobilization from the BM and collection of hematopoietic precursors from the blood for transplantation of myeloma and lymphoma patients.

Biokine leading product, BKT140/BL-8040: best-in-class CXCR4 antagonist is a bio-stable 14-residue cyclic peptide with high affinity (1- 2 nM, compare to 84nM of plerixafor) and a slow off-rate of the receptor. We have recently demonstrated in a Phase I study in healthy volunteers that single dose of the CXCR4 antagonist BL-8040 induces a rapid and robust mobilization and mega dose collection of human CD34+ stem and progenitor cells. A Phase 2 trial for BL-8040 as a novel approach for the mobilization and collection of BM stem cells from the peripheral blood circulation is currently conducted in collaboration with the Washington University School of Medicine, Division of Oncology and Hematology, St. Louis, MO, USA. Moreover BL8040 which as the ability to reshape the microenvironment of tumors is now in Phase IIb clinical trials for the treatment of Acute Myeloid Leukemia (AML), and is tested in multiple cancer indication together with immune check point inhibitors such as Pembrolizumab (Keytruda, Merck) and Atezolizumab (Tecentriq, Genentech/Roche).  BL8040 was licensed to BiolineRx on September 2012 and is being developed in the clinic together with BiolineRx Ltd. (NASDAQ:BLRX).