BioLineRx Announces Regulatory Submission for Phase 2 Trial of BL-8040

BioLineRx Ltd. has announced that it has filed the regulatory submissions required to commence a Phase 2 trial for BL-8040, in combination with standard-of-care immunosuppressive therapy, as a novel treatment for two bone marrow failure conditions: hypoplastic myelodysplastic syndrome (hMDS) and aplastic anemia (AA). The trial is expected to commence shortly after receipt of regulatory approval, anticipated in the next few months.

The open-label Phase 2 trial will be conducted in collaboration with MD Anderson Cancer Center in Houston and is expected to enroll up to 25 patients. The study will examine BL-8040’s ability to improve bone marrow cellularity and peripheral blood counts in patients suffering from these bone marrow failure conditions.

Both hMDS and AA are characterized by a T cell-driven autoimmune attack on the bone marrow that results in depletion of hematopoietic precursors, leading to anemia and low white blood cell counts. In this regard, high CXCR4 expression on pathogenic T cells has been suggested to facilitate infiltration of the bone marrow. BL-8040, a CXCR4 antagonist, is expected to inhibit migration of pathogenic T cells to the bone marrow, thereby mitigating the severe depletion of hematopoietic stem and progenitor cells.

In addition, BL-8040 can directly affect the number of hematopoietic precursors. Preclinical studies in mice showed that multiple doses of BL-8040 led to a marked increase in the number of hematopoietic progenitor cells and hematopoietic stem cells in both the bone marrow and peripheral blood.

BL-8040 also promoted production of megakaryocytes in the bone marrow, leading to a prolonged increased platelet production. These direct effects of BL-8040, along with the exclusion of the pathogenic T cells from the bone marrow, may improve bone marrow cellularity and peripheral blood counts.

Dr. Kinneret Savitsky, CEO of BioLineRx, stated, “We are very pleased to have filed the necessary regulatory submissions for an additional Phase 2 trial of BL-8040, our unique oncology platform, in these novel and non-malignant indications. We are honored to collaborate yet again with the prestigious MD Anderson Cancer Center, expanding upon our current collaboration on the Phase 2 trial for treating relapsed/refractory acute myeloid leukemia (AML), as well as our planned Phase 2 trial for treating AML patients with the FLT3-ITD mutation. The hMDS/AA trial will assess BL-8040 in combination with standard of care immunosuppressive therapy, with interim results expected by the end of 2016. Both hMDS and AA represent significant unmet medical needs and we are very hopeful that BL-8040, as part of a novel treatment regimen, will significantly improve bone marrow cellularity and peripheral blood counts in patients suffering from these difficult bone marrow failure conditions.”