BioLineRx announced the filing of regulatory submissions required to commence a randomized, controlled Phase 3 registrational trial of BL-8040 for the mobilization of hematopoietic stem cells for autologous transplantation in patients with multiple myeloma. The trial, named GENESIS, is expected to commence by the end of 2017, following receipt of regulatory approvals.

The Phase 3 GENESIS trial is aimed at evaluating the safety, tolerability and efficacy of the combination treatment of BL-8040 and granulocyte colony-stimulating factor (G-CSF), as compared to the control arm of placebo and G-CSF. The trial will be conducted in two parts: The first part, designed to validate the optimal dosing of BL-8040, is a lead-in, open-label, multi-center study that will include 10-30 patients, in order to assess the efficacy and safety of treatment with BL-8040 and G-CSF. This part will be followed by a randomized, placebo-controlled, multi-center study in approximately 180 patients. The primary endpoint will be the proportion of subjects mobilizing ≥6.0 x 106 CD34+ cells/kg with up to 2 apheresis sessions in preparation for autologous transplantation after a single administration of BL-8040 and G-CSF, as compared to placebo and G-CSF.

"We are excited to move forward with BL-8040 into a Phase 3 registration study. We have previously reported positive results supporting BL-8040 as a one-day dosing and up-to-two-day collection regimen for rapid mobilization of stem cells," Philip Serlin, Chief Executive Officer of BioLineRx, said. "This represents a significant improvement over the current treatment, which requires four-to-eight daily injections of G-CSF and one-to-four apheresis sessions. We therefore hope that this Phase 3 trial will further support these results and help improve the standard of care for multiple myeloma patients."

BL-8040 is a short peptide for the treatment of acute myeloid leukemia, solid tumors, and stem cell mobilization. It functions as a high-affinity antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. 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 from the bone marrow, thereby sensitizing these cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). In addition, BL-8040 has also demonstrated robust stem-cell mobilization, including the mobilization of colony-forming cells, T, B and NK cells. BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

High-dose chemotherapy followed by stem cell transplantation has become an established treatment modality for a variety of hematologic malignancies, including multiple myeloma, as well as various forms of lymphoma and leukemia. Stem cells are mobilized from the bone marrow using granulocyte colony-stimulating factor (G-CSF), harvested from the peripheral blood by apheresis, and infused to the patient after chemotherapy. This type of treatment often replaces the use of traditional bone marrow transplantation, because the stem cells are easier to collect and the treatment allows for a quicker recovery time and fewer complications.