BioLineRx Readies Phase 3 Myeloma Trial to Mobilize Stem Cells for Transplant

BioLineRx has launched a Phase 3 trial to assess if adding BL-8040 to standard treatment will improve the outflux of blood stem cells from bone marrow before a stem cell transplant in multiple myeloma.

“I am very excited to test the role of BL-8040, a novel CXCR4 inhibitor with G-CSF for the mobilization of peripheral blood stem cells from patients undergoing autologous transplant for multiple myeloma,” John F. DiPersio, MD, chief of the Division of Oncology at the Washington University School of Medicine, said in a press release.

“I am hopeful that this will provide another approach to the optimal hematopoietic stem cell collection in this challenging group of patients,” said DiPersio, who also is lead investigator of the study.

The company announced in August 2017 its intention to advance the treatment into Phase 3. After obtaining regulatory approval, the study has been set up, but is not yet recruiting participants.

Blood stem cell transplants are a mainstay of myeloma treatment, in which a patient’s own bone marrow stem cells are used. Currently, a compound called granulocyte colony-stimulating factor (G-CSF) is used to lure stem cells from the bone marrow, allowing physicians to harvest them from the blood.

BioLine believes its compound, BL-8040, can make this process more efficient.

The Phase 3 trial, called GENESIS (NCT03246529), will compare a combination of BL-8040 and G-CSF to a placebo and G-CSF. Researchers will measure if they can harvest more stem cells with the combination treatment, compared to G-CSF alone.

Patients will receive only one dose of BL-8040, after which researchers will gather stem cells on two occasions, by a procedure called apheresis.

“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,” said Philip Serlin, BioLineRx’s CEO.

BL-8040 acts by blocking a factor called CXCR4, which is needed to retain stem cells within the bone marrow. Inhibiting this factor induces mobilization of stem cells from the bone marrow into the blood.

In addition to allowing physicians to harvest stem cells from the blood, the treatment is thought to have an anticancer effect by sensitizing tumor cells to treatment. BL-8040 also may directly kill cancer cells and mobilize immune cells to fight the tumor.

BioLineRx expects data from the trial to be available in 2020.