Adding BioLineRx‘s BL-8040 to standard treatment greatly improves the mobilization of blood stem cells to be collected for stem cell transplants in patients with multiple myeloma, according to interim data of a Phase 3 study.
High doses of chemotherapy followed by autologous stem cell transplant — a procedure that transplants a patient’s own blood stem cells — has been the standard treatment for multiple myeloma for two decades.
Currently, a compound called granulocyte colony-stimulating factor (G-CSF) is used to force blood stem cells to move from the bone marrow, where they are produced, to the bloodstream, where they can be collected using a process called apheresis.
In apheresis, a machine draws blood from the patient, separates the different types of blood cells, collects only the stem cells, then returns the remaining blood cells back to the patient.
However, mobilization of blood stem cells using G-CSF is a slow procedure, usually requiring four to eight daily injections and one to four apheresis sessions.
Previous studies have shown that one dose of BioLineRx’s compound BL-8040 with up to two apheresis sessions can rapidly mobilize and collect as many stem cells as the standard treatment with G-CSF, representing a significant improvement.
BL-8040 acts by blocking CXCR4 — a molecule involved in the retention of stem cells within the bone marrow — inducing the mobilization of stem cells from the bone marrow into the bloodstream.
The randomized, double-blind, placebo-controlled, and multi-center Phase 3 study, called GENESIS (NCT03246529), is evaluating whether the combination of BL-8040 and G-CSF induces a stronger mobilization of stem cells into the blood in myeloma patients than G-CSF alone.
The study’s primary goal is to assess the proportion of myeloma patients receiving a single dose of BL-8040 in combination with the standard regimen of G-CSF who mobilized enough stem cells for transplant in up to two apheresis sessions, compared with patients receiving a placebo and G-CSF.
Its first part aimed to assess the safety of the treatment, how the body processed it, and the effectiveness of BL-8040 combined with G-CSF in up to 30 patients before moving to the randomized, placebo-controlled second part. The data would be reviewed by a Data Monitoring Committee (DMC) after 10, 20, and 30 patients.
Results of the first 11 patients enrolled showed that the combination of BL-8040 and G-CSF is safe and tolerable. Nine reached the target number of collected stem cells with only one dose of BL-8040 in up to two apheresis sessions. Seven of those patients reached it with a single apheresis session.
“A single BL-8040 dose combined with G-CSF is not only safe and tolerable, but … may reduce the number of required apheresis sessions to a single session in the majority of patients,” John F. DiPersio, the study’s lead investigator and chief of the Division of Oncology at the Washington University School of Medicine, said in a press release.
“Based on the robust data received from the first 11 patients, the DMC issued a positive recommendation to stop the lead-in part of the study and move immediately to the randomized placebo-controlled part of the study,” said Philip Serlin, BioLineRx’s CEO.
The company next plans to enroll 177 myeloma patients who will be randomized to receive either BL-8040 or a placebo in addition to G-CSF treatment. Information for those interested in enrolling is available here. Results are expected in 2020.