BL-8040, a 14-amino acid synthetic cyclic peptide, is a novel, best in class, high-affinity CXCR4 antagonist with long receptor occupancy (>48 hours) and extended CXCR4 inhibition. BL-8040 is developed for the treatment of solid tumors, hematological malignancies and indications requiring hematopoietic stem cell transplantation.

BL-8040 pipeline is based on the vital roles of CXCR4-CXCL12 (SDF-1) interactions in oncologic processes (growth, invasion, survival, angiogenesis and metastasis) in various cancer types and in the maintenance of hematopoietic stem cells, immune cells and hematopoietic cancer cells in their protective niche.


CXCL12 in bone marrow stromal cells retain leukemic cells in the protective bone marrow niche

BL-8040 blocks the interaction between CXCL12 and CXCR4 by binding to CXCR4

As a result, leukemic cells are mobilized from their protective niche into peripheral blood; in addition, CXCR4 inhibition can induce direct apoptosis of the leukemic cells

Mobilized leukemic cells detached from their niche are sensitized to chemotherapy as well as to direct and indirect BL-8040-mediated apoptosis

Acute Myeloid Leukemia (AML)

During 2016, BioLineRx completed and reported on a Phase 2a proof-of-concept trial for the treatment of relapsed or refractory acute myeloid leukemia (r/r AML) which was conducted on 42 patients at six world-leading cancer research centers in the U.S. and at five premier sites in Israel. The study included both a dose-escalation and a dose-expansion phase. At the annual meetings of SOHO and ASH in September and December 2016, respectively, BioLineRx presented detailed, positive safety and response rate data relating to the study. BioLineRx continues to monitor long-term survival data for patients in the dose expansion phase of the study.

BioLineRx is currently investigating BL-8040 as a consolidation treatment together with cytarabine (the current standard of care) for AML patients who have responded to standard induction treatment and are in complete remission and, in this regard, are running a significant Phase 2b trial in Germany, in collaboration with the German Study Alliance Leukemia Group. The Phase 2b trial is a double-blind, placebo-controlled, randomized, multi-center study aimed at assessing the efficacy of BL-8040 in addition to standard consolidation therapy in AML patients. Up to 194 patients will be enrolled in the trial. The primary endpoint of the study is to compare the relapse-free survival (RFS) time in AML subjects in their first remission during a minimum follow-up time of 18 months after randomization.
BioLineRx is considering carrying out an interim analysis on this study in the second half of 2018, with top-line results expected in 2020.