BioLineRx Announces Initiation Stem-Cell Mobilization for Autologous Transplantation Trial

BioLineRx announced initiation of the Phase 3 GENESIS clinical trial, in which BL-8040 will be compared to placebo, on top of granulocyte colony-stimulating factor (G-CSF), for the mobilization of hematopoietic stem cells (HSCs) used for autologous transplantation in multiple myeloma patients.

The GENESIS study is a Phase 3, randomized, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of BL-8040 and G-CSF, compared to placebo and G-CSF, for the mobilization of HSCs for autologous transplantation in multiple myeloma patients. The study will commence with a lead-in period for dose confirmation, which will include 10-30 patients, and progress to the placebo-controlled main part, which is designed to include 177 patients in more than 15 centers. Treatment will include 5-8 days of G-CSF, with a single dose of BL-8040 or placebo on day 4. Apheresis for stem cell collection will be performed on day 5. Further apheresis sessions may be conducted if needed in order to reach the benchmark of ≥ 6x106 mobilized HSCs.

The primary objective of the study is to demonstrate that BL-8040 on top of G-CSF is superior to G-CSF alone in the ability of mobilize ≥ 6x106 HSCs in up to 2 aphereses. Secondary objectives include time to engraftment of neutrophils and platelets and durability of engraftment, as well as other efficacy and safety parameters.

"The initiation of our first Phase 3 trial for BL-8040 is an important milestone in the robust development plan of our lead oncology platform," Philip Serlin, Chief Executive Officer of BioLineRx, said. "Treatment with BL-8040 as a single administration and up-to-two-day collection regimen for rapid mobilization of stem cells could represent a significant improvement over the current treatment, which requires up to four apheresis sessions. We look forward to top-line results from the study expected in 2020."

BL-8040 is a short peptide for the treatment of stem cell mobilization, acute myeloid leukemia and solid tumors. BL-8040 has demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, T, B and NK cells. It functions as a high-affinity antagonist for CXCR4, with long receptor occupancy. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells and immune-cells from the bone marrow, thereby sensitizing cancer cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis) and mobilizing immune-cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.